BioCryst at Bank of America Conference: Strategic Growth Insights

On Wednesday, 14 May 2025, BioCryst Pharmaceuticals (NASDAQ:BCRX) presented at the Bank of America 2025 Healthcare Conference, highlighting its strategic growth initiatives. The company reported strong financial performance and outlined its future plans, focusing on both opportunities and challenges in the market.

Key Takeaways

• BioCryst reported a 51% year-over-year growth in Q1 2024, driven by the successful launch of Orlodeyo.
• The company aims for $800 million in U.S. sales and $1 billion globally by 2029.
• Pipeline programs for Netherton Syndrome and Diabetic Macular Edema are progressing, with data expected by year-end.
• BioCryst is navigating potential competition and regulatory changes with confidence.

Financial Results

• BioCryst achieved a 51% year-over-year growth in Q1 2024, attributed to strong demand for Orlodeyo.
• The paid rate increased by 10 percentage points to 84%, with Medicare reaching 89%.
• The company targets at least $800 million in U.S. sales, with a global sales target of $1 billion by 2029.
• A net price increase of approximately 3.5% has been implemented over the past few years.

Operational Updates

• New patient starts in the U.S. matched 2021 levels, with an average net addition of 200 patients per year needed to reach sales targets.
• Real-world evidence indicates a median attack rate of about four attacks per year for Orlodeyo patients.
• Retention rate at one year is approximately 60%, comparable to other prophylactic therapies.

Future Outlook

• BioCryst is focused on sustainable growth in revenue and profit.
• The company is open to partnerships for its DME program if larger firms can expedite development.
• The Netherton Syndrome program could validate BioCryst’s discovery engine, with potential accelerated paths being considered.

Pipeline Updates

BCX1775 (Netherton Syndrome):

• Phase 1 study underway, with initial data expected by year-end.
• Aims to restore normal skin turnover, with a market size of approximately 1,600 U.S. patients.

Avorostat (Diabetic Macular Edema):

• Phase 1 study to begin in Q3 2025, with data expected by year-end.
• Focus on assessing drug impact on retina thickness and durability.

Q&A Highlights

• The executive order on the most favored nation status is not expected to impact Orlodeyo significantly.
• Tariffs are mitigated by a low cost of goods and a redundant supply chain.
• FDA interactions are proceeding normally, with a PDUFA date received for pediatric granules.

For more detailed insights, please refer to the full transcript of the conference call.

Full transcript - Bank of America 2025 Healthcare Conference:

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Welcome back to the Bank of America Healthcare Conference. I’m Tazeen Ahmad. I’m one of the senior SMID biotech analysts at the firm. It’s my pleasure to have our next presenting company with us, BioCryst. Sitting up on stage with me is John Stonehouse, who is, of course, CEO of the company, and next to him is Charlie Geier, who is chief commercial officer.

Gentlemen, good morning still, and welcome to the conference.

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

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: So I’ve been starting off these conversations with macro questions, which, again, is quite unusual in my space, but, you know, the times call for it. So I’ll probably ask ask you from the most recent updates, and we can go from there. On Monday, the president made an executive order focusing on most favored nation status. Details are still coming in. It might be hard to give a concrete answer, but can you, to the best of your ability, talk about what the impact of this could mean to BioCryst in particular?

John Stonehouse, CEO, BioCryst: Yeah. And Charlie and I are gonna make some forward looking statements, they have risks, and you can find our risk factors in our filings on our website. Yeah. It’s a hard one, Tazeen, because it’s short on detail, and I think people are still trying to figure out exactly what does it mean. Looks like it could be more part b in Medicare than d, and we’re in d, so that could be a difference.

But it’s you know, we’re we’re gonna continue to execute our plan and, you know, we got our ear to the ground on this, and we’ll see how things unfold. But right now, I can’t really make a whole lot of forward looking statements on that.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Sure. And then similar question on tariffs.

John Stonehouse, CEO, BioCryst: I’ll let Charlie answer that.

Charlie Geier, Chief Commercial Officer, BioCryst: Tariffs, we we feel like we’re in a a good position. First of all, the cost of goods for Orlodea was is low. Second, we have redundant redundant supply chain. So we make API in Europe as well in The US. And then we also have, finished goods made in The US.

And so we have the ability to to serve different markets. And importantly, all of our IP is in The US as well.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: And in terms of interactions with FDA, let’s say over the last eight weeks, if you’ve had any, have they been any different from what they’ve been in the past in terms of type of interaction, people that you’re interacting with, types of questions being asked, etcetera?

John Stonehouse, CEO, BioCryst: Yeah. I don’t know if you saw, but we got our PDUFA date this morning. Yes. So our file was accepted for the pediatric granules, which is really exciting. That was on time.

So that’s a good sign. And we have an open IND for BCX1775 that, you know, kind of went on the normal timeline that you would expect. So, so far so good. I can’t predict the future, but so far so good.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Okay. So let’s move on to specific questions to BioChris. So this Orlodea launch, it keeps getting better. And I think probably some people have been surprised by that. We’ve always viewed this as a pretty steady Eddie ramp.

You’ve talked about your confidence in this market, in your product. Can you talk to us about some of the more meaningful dynamics that have been contributing to the results most recently, your 1Q results, which allowed you to increase your sales guide.

John Stonehouse, CEO, BioCryst: Yeah, and I think before Charlie, Charlie will answer that, but I think one thing that’s really important is when you go into a rare disease with seven other drugs ahead of you, it’s a market that you’ve probably never seen before. And one of the things that I’ll give Charlie and his team, I mean, a ton of credit for in the execution is just really understanding the data, making big moves early on that helped us gather data that was incredibly helpful, and then making these tweaks and adjustments along the way to adjust to what we see. And that has led to this beautiful steady growth of the product.

Charlie Geier, Chief Commercial Officer, BioCryst: So as you know, Tazeen, we had a great first quarter. It was 51% year over year growth. What I would say the most important baseline of that is really strong demand. So we had last year, we had as many new patient starts in The U. S.

As we did back in 2021, the first year of the launch. And then Q1 of this year was actually just a little bit better than the best quarter of last year. So that’s really important. If we didn’t have the demand, the next part wouldn’t matter as much, but the demand is really solid. Then the other big thing that happened in Q1 is we made a big jump forward in our paid rate.

And so something that we thought we wouldn’t get to till the end of twenty twenty seven, We jumped 10 percentage points all the way up to eighty four percent of our patients being paid all through, you know, in the first four months of this year. And that was two big components. One was the IRA is helping make Borlodayo and other drugs more affordable for patients. And so patients who previously couldn’t afford their co pays and were on free product now can afford it. And so in our Medicare segment, the paid rate is up to eighty nine percent.

And then the other thing is with commercial payers, we keep making progress. John mentioned the evidence. We’ve developed a lot of real world evidence that is convincing payers that this drug isn’t just a pill, it’s a very effective preventative therapy. And so those, all of that combined to make just a really great Q1 and sets us up on a strong path for this year and the next several years.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Yeah, so we’ve always talked about stickiness of the market. So John, you talked about entering a sticky market, but now you’re part of the sticky market. What do you think

John Stonehouse, CEO, BioCryst: is

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: particularly particular about Orlodea that you think will help you keep the patients that you have now as well as continue to add on even potential new competition coming?

Charlie Geier, Chief Commercial Officer, BioCryst: I think Tazeen, the key is of course patients love the idea of an oral therapy, but in HAE they will not accept a drug that doesn’t work as well. And so getting efficacy is is primary, and what patients have discovered is they can do really well and do do really well on Orlodayo. If they don’t, that’s fine. What we’ve learned is no drug works perfectly for every patient. They try it and they move on.

But the patients who stay on, they stay on because it’s convenient, but also because they have a very low rate of attacks relative to what they might have experienced in at some point in their past. And so that makes it sticky. That is what they’re looking for is just to take a pill and almost forget that I have HAE. And that’s why we think it’s those patients will be so sticky because what could any new therapy offer them that is better than that? They’ve already found what they’re they’re looking for.

John Stonehouse, CEO, BioCryst: Yeah. And I I think one other thing in the marketplace, and we saw this from the start and we still hear it today, the number one objection from a doctor about switching to Orlodeyo is if it ain’t broke, don’t fix it. And so if you’re on Orlodeyo and you’re controlled, as Charlie said, there is nothing more that you can do for that patient, right?

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: So

John Stonehouse, CEO, BioCryst: that’s where the stickiness comes from.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: What is the definition of controlled? Because sometimes different people, different doctors actually have different ways of looking at But what is the most standard way of thinking about control? Does that mean you don’t have occasional breakthrough attacks?

Charlie Geier, Chief Commercial Officer, BioCryst: Well, that would what everyone would love to be is completely attack free. Right. That’s an admirable goal. What I think physicians really know and patients very much understand is that is almost unrealistic for most patients. And so control means rare breakthrough attacks, and when you have an attack, that you’re able to deal with it more easily with a with a rescue medicine.

And so what our long term, and our real world evidence is showing is, the median attack rate for patients on Orlodea was getting down to about four attacks per year on a rate. So that is great control. We have a decent percentage who are effectively attack free. Some have a little bit more. It’s all relative to what the patient expects based on their past history.

And we’ve looked at other therapies and what they’ve reported on drugs, really good drugs like TaxiRO, Heygarda. The same types of patterns are seen where some people are attack free, but most people do have an occasional breakthrough attack. So that’s expected. But as long as they can deal with it quickly with a rescue medication, they’re comfortable with that.

John Stonehouse, CEO, BioCryst: And Shirley, you might want to talk about the data that we gathered through claims where the persistence of other Yeah. Prophy therapies bears that out.

Charlie Geier, Chief Commercial Officer, BioCryst: Yeah. I think this this kind of shows, and also what I said earlier, that no drug is perfect for absolutely everybody. So we did work identifying patients starting Orlodayo, starting TaxiRO, and starting Hagarda, and followed them through their first year of therapy. And what it showed is there is no difference between those three products. They all end up with about a sixty percent retention rate at one year.

And I think that just that just shows patients need to find what works for them. Mhmm. And we at the moment have the most differentiated product in the market.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: So John, you’ve talked about being able to achieve at least $800,000,000 in sales, but at the time that you’re at now, it seems like it’s not that far away. So how are you thinking about it now in terms of where this could peak?

John Stonehouse, CEO, BioCryst: Yeah. I mean, our confidence in The US in hitting that 800 number is really high. And it’s because when we first put it out, we gave you the steps of things that we needed to hit, and we’re ahead of plan on all of them. And I’ll let Charlie describe them. Yeah.

Charlie Geier, Chief Commercial Officer, BioCryst: There are three big components, and and what I said in the first answer is really key. The number one is the demand. And so as long as we average, and this was from 2024 through 2028, average a net of 200 patient additions per year, we would that’s the biggest piece to getting to 800,000,000. We were well ahead of that in 2024. The first quarter shows we’re we’re on that same strong pace.

The next piece was getting to an 85% paid rate, and we expected that would take three years. We we basically were there at the April with 84%. And then the final piece is is modest price increases. And we’ve been able to increase price by 5% the last each of the last couple of years. We’ve net about three and a half.

So we’re we’re ahead of that pace, as as well. So we feel really good about peak of 800 at least 800,000,000 in The US and then global sales of a billion in 2029.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Okay. Can I just ask you how you expect to be impacted from launches of new on demand therapies in the near term? What percent of your patients do you think would opt for something that’s fast acting if they need it rather than be on a prophy,

Charlie Geier, Chief Commercial Officer, BioCryst: if not? Yeah, think there’s maybe a couple parts to your question, which is will what we expect to launch very soon is an oral on demand therapy. And we know that that’s something that patients would really like. Yeah. We’re paranoid about this, so we’ve done a lot of market research to figure out what will they actually do, what will physicians do, and we see very little, idea that the patients would go backwards and say, I’m just gonna treat my therapy as attacks come because that puts a lot of burden on them to be ready.

As as easy it is to take a pill, you have to constantly be ready. They’d rather prevent and then have a pill for the occasional breakthrough. And to be able to do that with a fully oral regimen of oral every day to prevent attacks and then occasionally oral to for breakthrough attacks is something that we think would be very attractive.

John Stonehouse, CEO, BioCryst: I think there’s another piece too, which is what is the characteristic of an attack if you’re treating it on demand without prophy versus a breakthrough attack if you’re on prophy? And what we’ve seen and what other companies that have prophylactic medicine is that the attack is shorter in duration, less likely to grab for rescue medicine, you know, fewer body parts. And so who wants to go through that? Right? And then the other piece is that you need two things to have an attack, low C1 and a trigger, And stress is the number one trigger.

Who wants to have a patient that’s worrying about, Oh my god, if I have an attack today or You know, a lot of times that will trigger an attack. And so it’s just way better to prevent. The guidelines suggest that everybody should have the opportunity to have a prophylactic therapy. And so this idea that we’re going to go backwards and treat more on demand. I think it, you know, like Charlie said, no drug is perfect.

There are breakthrough attacks. There will always be some people that are on on demand only. And so there’s a place for these medicines, but, prophylactic is here to stay.

Charlie Geier, Chief Commercial Officer, BioCryst: To John’s point, we’ve put a big study that we did with Orlodayo patients. We put this data out publicly. Eighty percent of them report less severe attacks on Orlodayo, and only five percent say their attacks are are are the other way or or worse. So people really do do better when they’re controlled with Orlodayo, and I think this would be true controlled on any prophy therapy.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Okay. So I did want to have a good amount of time to talk about your pipeline because there’s a couple of exciting things upcoming later this year. So maybe John, can you give us an overview of those?

John Stonehouse, CEO, BioCryst: Yeah. So we’re back in the clinic, which is great with two programs, BCX17-seven twenty five, which is a fusion protein, our first biologic that we’re moving into the clinic for Netherton Syndrome patients. And this is a horrible disease where you have uncontrollable KLK activity that causes uncontrollable skin shedding. And the more we learn about it, sadder we get about having patients not having anything to really treat the underlying cause of their disease. And so we’re really excited to be in the clinic and moving that forward.

And then the second one is Avorostat, which you remember back in the day was our first generation kallikrein inhibitor, poorly soluble, poorly permeable. So it wasn’t a great oral drug. But in DME, we believe that given with a suprachoroidal injection could be really interesting for patients that have DME and are continuing to progress even when they’re on VEGF therapy.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Okay, so let’s go into a little bit more detail about Neverton. So can you talk about mechanistically why you’re excited about this particular approach?

John Stonehouse, CEO, BioCryst: Yeah. So the disease, you know, in a simple layman’s explanation is faulty gene, missing protein, overactive enzyme, KLK5. And what it does is it starts to gobble up skin cells and you shed uncontrollably. And so like I said, it’s a horrible disease. So what we’re able to do is replace the protein and control the KLK5.

So what we’re shooting for is restoring normal turnover of skin, which would be, you know, just unbelievable for these patients.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: And so in this first stop that you’re looking at, just tell us how we should be thinking about what the data could show us.

John Stonehouse, CEO, BioCryst: Yeah. I’m going to get to a slide, I believe. Yeah. Here we go. So this is a slide 13.

Don’t know if it’s 13 in the deck, but it was recently presented in the our earnings deck, and it’s the phase one healthy volunteer and patient study in Nothersen. And so we’re in parts one and two right now Mhmm. Which is your standard healthy volunteer SADMAD study. And what we’re looking for in that portion of the study is PK and getting a sense of dose and also safety and tolerability. Because of normal skin turnover and having the protein, not missing the protein,

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: you

John Stonehouse, CEO, BioCryst: can’t get great measurements in skin. You know, we are doing biopsies, but it, you know, it’s going to be harder to get a sense of this drug getting there or not. So what we’re really excited about, and this is where the IND in The US open up to treat patients in part three is really exciting. This is a four weeks of treatment. So three doses weeks one, or starting day two weeks, and then four weeks.

And then we follow them for another four weeks, and we’re probably going to get a handful of patient data out of the study because it’s an ultra rare disease. But what we’re looking for, we’re starting at six milligrams dose, is to see what six milligrams a dose does in these patients. And if we have to go up, we will go up in in that in that study. And we’re you know, really what we’re looking for is do we see the drug getting to So we’ll do biopsies there.

It will tell us.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Okay.

John Stonehouse, CEO, BioCryst: What effect is it having on KLK5? And then are there any clinical symptoms? And we don’t know if in a four week treatment study if we’ll see that or not. We don’t know if six milligrams is the right dose, but we think we’ll start to get a sense of dose and activity in that study. And then the next part, which we also expect to start this year, we don’t know how much data we’ll have, is part four where we have twelve weeks of treatment, a longer duration where we expect that we should start to see some real clinical benefit.

And the combination of all of this, if it does what we think it will do, will give us something, I think, really exciting to take to the regulators in a disease where there’s nothing to treat these patients.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Yeah. So let’s do a deeper dive into a couple of things that you just mentioned. So is there a minimal threshold of impact on KLK5 that you need to see?

John Stonehouse, CEO, BioCryst: Yeah. You know, without going into too much, we want to restore normal skin turnover. So it’s got to be inhibition that is similar to what you and I have. And so that means you’re not going to be flaking. You’re not going to have the redness.

You’re not going to have the itching. And so that’s what we’re shooting for. So this isn’t some little incremental, you know, patient reported, physician reported. We’re looking for restoration of normal skin turnover. And Tazeen, I think part of

Charlie Geier, Chief Commercial Officer, BioCryst: the answer to this, we don’t know if there’s a minimal threshold, but we’ll find out with the dosing schedule. And the fact that for these patients, their skin turns over on about every two weeks, that’s why these short studies with the replacing essentially, the replacing the missing you know, the the controlling protein or controlling enzyme is it’s it’s plausible that we could actually discover what that minimum dose is Okay. In these short studies.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Even, like, when you get to the twelve weeks, that would be you think sufficient to get answers to- might

John Stonehouse, CEO, BioCryst: find it out in the four week study, Okay.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Yeah. You’ve listed some other markers there outside of KLK5, which I’m not familiar with, but can you talk us through the importance of those?

John Stonehouse, CEO, BioCryst: Yeah. Mean, there’s, just in general, are patient reported outcomes and physician reported outcomes of how much of the body do you see an improvement, you know, how much of the area do you see an improvement? Yeah. Those kinds of standard ichthyosis atopic dermatitis type measurements we’ll be using, and those probably will be the endpoints that’ll be used And again, if we achieve, you know, the restoration of normal skin turnover, they should be dramatic differences from what patients are experiencing.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Right. So it seems to me like you’ll be able to and again, we don’t know how many doses are going to be in Part IV, but you’d be able to know pretty early when we’re, you know, is this going to work or is this not going to work So I would say, you know, the investment that you’re making in this is not something that’s multi year

John Stonehouse, CEO, BioCryst: Yes. And, you know, it’s probably one handful of patients in part three and maybe two in part four. It’s an investment that either the drug gets to the skin or it doesn’t, it’s having the intended effect. Because remember, BCX seventeen seven two five is a million times more potent than the natural ligand, and it has a high affinity for the site. And so, you know, we think that that tends well to or or gives us the confidence that we’re gonna have a drug.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: How many patients are there again for another 10?

Charlie Geier, Chief Commercial Officer, BioCryst: Yeah. The market. We’ve there is no ICD 10 code. So this is a critical thing to figure out. We’ve gotten a good start on it because there’s a unique feature that many patients have called bamboo hair, where their hair literally looks like bamboo under a microscope.

And we’ve done US claims data analysis and found about 1,600 patients that have a mention of bamboo hair. Then what we know from talking to patients is many of them, because there’s no therapy out there, they kind of retreat from care, and they’re not asking their doctors anymore. So what our belief is, if there’s a targeted therapy out there on the market, like we’ve seen in other rare diseases like HAE, that the market could grow. We have people who launched, Synrise on our team back in the day, back in 02/2009. They used to think there were two thousand to 3,000 HAE patients in The US.

Now we know that there’s over 10,000. And so a similar kind of growth could happen in Netherlands, and we’re doing more work to gain more confidence around that kind of hidden undiagnosed population size.

John Stonehouse, CEO, BioCryst: We got a really good question today from an investor about what what role does this drug play for BioCrystin for an investor? And and the answer is sustainable growth both in revenue and profit about the time this comes to market, and will be at, we think, a pretty good trajectory. Orlodea will be starting to hit its peak and flattening out. Profitability spreading another product across the infrastructure and investment that we’ve made improves profitability. And then validation of our discovery engine.

Know, how many companies do you know that actually do this twice? Right? And so we have a high degree of confidence for that. So that’s the role that we play.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Sure. So if everything that you’re looking for in this study goes in the direction that you would hope, I guess, first of all, are we going to see the twelve week results this calendar year or would that flow into next year?

John Stonehouse, CEO, BioCryst: Yeah, I think that’s gonna flow into next year. We may start enrolling part four this year that we may have some early data out of part four, but I think part three is where you’re, you know, like you said, a handful of patients

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: treated for four That part three is when again?

John Stonehouse, CEO, BioCryst: We’re going to start it towards the end of this summer and have data by the end of year.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: By the end the

John Stonehouse, CEO, BioCryst: year for Yeah.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Okay. So what would be the next steps if this turns out to be what you want to do?

John Stonehouse, CEO, BioCryst: It all depends on what we see. But let’s assume that part four gives us a real indication of clinical improvement that we’re talking about. And we have a dose. We’re going to go to the regulators. There’s nothing to treat these patients.

And, you know, I can’t predict with certainty that we’ll be able to go right into a pivotal study, but, you know, we’re gonna make an argument for that and see what happens. And I think the other piece that will turn on at that point is what Charlie was talking about. How big is this market? Can we start to, you know, get a program where we’re getting more dermatologists that have big ichthyosis patient populations to start doing the genetic testing and to start helping, you know, with advocacy for patients and the like and really start to find the patient. That that would be very exciting and that we think that could happen as early as sometime next year.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: So for something like this, do you think that it’s worth trying to pursue an accelerated path Or just based on what you said, you’ll be able to know like in a short amount of time and even in a bigger study if it’s going to work or if it’s not going to work. So does make sense to even think about, and I’m jumping way ahead, of whether or not it makes sense to try to pursue an accelerated.

John Stonehouse, CEO, BioCryst: Yeah, I think that’s everything’s on the table, again, just based on what we see in terms of the results of these early studies. So, you know, because, you know, and I think the agency in general is pretty amenable to accelerated pathways if there’s nothing, and you have a big treatment effect. So I don’t think that’s off the table.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Okay, great. So we’ll look forward to that. And then I did also want to talk about DME. Sure. So I think some people are intrigued by it because the DME market is seemingly pretty saturated with the big players, the VEGFs.

Can you just talk to us in particular why DME might be more amenable relative to some of the, I guess, let’s say wet AMD to an alternative treatment form?

John Stonehouse, CEO, BioCryst: Yeah. I think part of it is, while VEGF inhibitors are really good therapies, in some patients they’re not good enough is number one. And number two, the biology of the disease is such that plasma kallikrein likely plays a role, in patients. And there’s a slide, it’s number 15, in this deck, but it’s addressing unmet need in diabetic macular edema, and it’s pulling data that one shows that, you know, upwards of forty percent of patients on VEGF inhibitors continue to have a decrease in their visual acuity. So it’s just not working.

And then the other part of the slide is a graph that shows from a study that was run -in the vitreous, do you see VEGF or plasma kallikrein in the vitreous of DME patients? And you see it in almost every patient that they took samples of with plasma kallikrein, and in some, no VEGF. So, you know, and this makes sense, right? While it’s diabetic driven, it’s contact activation just like we see in HAE where plasma kallikrein rein plays a role in leaky vessels that causes swelling that causes the enlargement of the retina and ultimately decrease in visual acuity and blindness in patients. So

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: what type of data would you think is encouraging and worthy of moving forward?

John Stonehouse, CEO, BioCryst: Yeah, let me say first that, you know, we’re not the first to try this and there have been failures with plasma kallikrein inhibitors in the past. And so what we’re trying to do is do a study where we get a sense of is our drug getting to the right spot, staying there long enough, and having an effect on the disease. And so really the thickness of the retina will be the first. We’re going to look at visual acuity and some of these other things, but what I love about this study is in a simple sad study where we’re not looking at healthy volunteers, we’re actually looking at patients with DME, we can give a single injection and see what happens to the thickness of the retina over time. And what is the durability of that single injection?

And so we’re starting again, we’re going to start that study sometime towards the tail end of this summer in the third quarter. And, you know, we believe we’re going to get some data.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: How many patients are there?

John Stonehouse, CEO, BioCryst: Yeah, the design was in our recent deck as well. And I believe we’ve got it here. Avoralstat phase one patients with DME. And so there’ll be a low dose, mid dose and high dose three patients per cohort. So you know, again, it’s a small number of patients, but we’re going to get some sense of what’s happening in these patients in terms of the thickness of their retina.

And we should have some data by the end of this year. So back to your point about a, you know, reasonable investment to get an answer. If we start to show some sort of activity, let’s say we don’t find the dose yet, we have to go higher, we don’t have the duration that we want, we’ll keep going. Sure. And pushing the dose until we find a dose that, you know, we feel is right to control over a long period of time.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Mechanistically, would you expect to see a different result in newly diagnosed patients versus those that have already been using VEGFs?

Charlie Geier, Chief Commercial Officer, BioCryst: I think that is a question it’s gonna be depends. In this study, we’re gonna be studying both newly diagnosed patients as well as some who have tried VEGF. As as Don Fong, our our chief medical officer, will always say, patients can fail on a drug for a number of different reasons. And so that’s the it depends part of it. But if it works in newly treated patients, as well as some who have experience with VEGF, we’re looking for that range of experience.

John Stonehouse, CEO, BioCryst: Yeah. I think back to that slide I talked about of looking at the vitreous and, you know, plasma Kallikrein versus VEGF, that you can see in these samples that some patients, you know, have a pretty large VEGF component and some have a smaller Kallikrein component. We may enroll some of those. We don’t know what the effect is going be in those So in a small study, what we’re looking for is are there signs of activity of shrinking the swelling of the retina? And what’s the durability of a single injection?

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: I mean, is all potentially really exciting and could be transformative for BioCrest. Would this be, the size of this opportunity, something that BioCryst would feel comfortable taking on on its own? Or do you think that this would benefit from large companies that have done big ophthalmology launches before?

John Stonehouse, CEO, BioCryst: Yeah, I’ll say yes and yes. Right now, we feel and one of the things that made it attractive, it’s not rare, right? And these trials are big. Doctor. Yeah.

We can talk about, you know, the investment in these trials, but we can afford it based on the compound annual growth rate that we’ve shown in the past and our path to profitability. But the commercial footprint is very similar to HAE treaters. And you know, because there’s these retinal centers that you know, are about the size of the population of treaters of HAE. You know, we have a 40 ish person Salesforce in The US. We could do that.

And so, but I could also, you know, and I say yes and yes, I could also see companies being very interested in this program. And I’ve always said if somebody can do it bigger, faster, better than us, we’re open to that.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Yeah. I mean, just given the dynamics of, you know, certain drugs coming off of Yeah. Protection and and all of that, I was curious as to how you’re I mean, are you getting inbound interest for from people wanting to learn more?

John Stonehouse, CEO, BioCryst: I think there’s a bit of cynicism about kallikrein inhibitors, just to be honest, you know, with the failures that we’ve seen in the clinic before. And so people want to see, is this And I think if we show that, there could be real interest.

Tazeen Ahmad, Senior SMID Biotech Analyst, Bank of America: Okay, cool. With that, we’re out of time. So thanks so much for joining us on stage for the last thirty minutes, and thanks everybody for listening. We’ll stay in touch, John, and we’ll talk soon.

John Stonehouse, CEO, BioCryst: Yeah, great. Thanks Tazeen. Thanks Tazeen.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.