BioCryst at Goldman Sachs Conference: Strategic Growth and Profitability

BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) participated in the Goldman Sachs 46th Annual Global Healthcare Conference on Monday, 09 June 2025. The company, led by CEO John Stonehouse, presented a robust strategic overview highlighting its commercial successes, notably with its flagship product Orlodayo, and its plans for growth. While BioCryst is accelerating towards profitability, it also faces challenges in pipeline development and market expansion.

Key Takeaways

• BioCryst is on track to achieve net income and cash flow positivity for the full year.
• Orlodayo’s success is driven by its efficacy and convenient oral administration.
• The company aims for a 20% annual revenue growth and a 5% increase in expenses.
• BioCryst is developing new treatments for Netherton syndrome and DME.
• Strategic goals include reaching $1 billion in Orlodayo revenue and expanding the drug pipeline.

Financial Results

• BioCryst has accelerated its path to profitability, expecting net income and cash flow positivity for the year.
• Orlodayo is the primary revenue driver, with expectations of a compound annual growth rate of 20% from 2025 to 2027.
• Expenses are projected to increase by 5% annually within the same period.
• The company projects having $600 million in cash by the end of 2027.
• In April, BioCryst paid down $75 million of Pharmakon debt, saving an estimated $23 million in interest over the loan’s lifetime.
• Revenue guidance for 2025 is set between $580 million and $600 million.
• The paid therapy rate for Orlodayo patients stands at 84%, with a goal of reaching 85%.

Operational Updates

• Orlodayo continues to see strong demand, attributed to increased confidence among physicians and patients.
• The company is focused on transitioning patients from free drug programs to paid therapy.
• A pediatric NDA for Orlodayo has been submitted, with a PDUFA date set for September 12.
• No significant commercial expansion is needed for the pediatric launch.
• BioCryst is developing BCX17725 for Netherton syndrome and Avorostat for DME, with patient data expected by the end of the year.

Future Outlook

• BioCryst aims to achieve $1 billion in Orlodayo revenue and secure approval for a second product, potentially BCX17725.
• The company is exploring external corporate development opportunities to complement its existing pipeline.
• Management is focused on maintaining a continuous engine for discovering and developing new drugs.

Q&A Highlights

• BioCryst utilizes a sole-source specialty pharmacy to gather insights about patients, aiding in market research and decision-making.
• The company is addressing friction points that prevent patients from switching to Orlodayo.
• BioCryst plans to generate data to address skepticism surrounding its pipeline programs.
• Management is confident in the potential of BCX17725 to alter the course of Netherton syndrome.

BioCryst Pharmaceuticals Inc. remains focused on executing its commercial program and delivering on its strategic goals. For more details, refer to the full transcript below.

Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference:

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Welcome. Thrilled to have you all here at the Goldman Healthcare Conference, for 2025. We are early in the conference. It’s my great pleasure to be here with BioCryst, with John Stonehouse, president president and CEO of BioCryst. I’m Ryan Fisk, managing director in the health care investment banking group at Goldman Sachs.

Thank you for for joining us today.

John Stonehouse, President and CEO, BioCryst: How are Thanks for having us. I’m doing great.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Well well, wonderful. Why don’t why don’t we jump in? Like to spend some time on Orlodayo, which has been doing very well commercially in recent quarters. Would also like to make sure that we spend some time on the pipeline today and just talk about some of the some of the programs that are gonna produce data this year.

But maybe to set the stage, can you share a high level overview of how you think about BioCryst’s strategy, particularly as you approach rare diseases and how that thus far has translated into commercial success for BioCryst?

John Stonehouse, President and CEO, BioCryst: Yeah. Great. Before I get started, I’ll be making forward looking statements. They have risks. You can find our risk factors on our website.

So, we chose rare disease, and it started that we believed if you could bring oral drugs to patients with rare disease, you had a real disruptor because vast, vast majority of any therapies were injectable. But we’ve evolved that, and and we’re actually starting our first protein therapeutic program with BCX seventeen seventy five in Netherton. So it’s gone beyond just oral and and a program injectable in the back of the eye. But, really, what what we’re trying to do is build a rare disease company with the engine of Orlodayo as the revenue source and cash source, and and it’s a disruptor. And we we could talk more about why, but, it’s definitely a disruptor in the HAE market.

Followed by, we believe there’s another Orlodayo that comes out of our discovery group, and we’ve got two programs that are gonna be starting to generate data by the end of this year. So we’re excited about that as evidence that there’s another Orlodeyo in there. And then third, financially, we are in a fantastic spot, and we we had laid out a couple of years ago our path to profitability, and we accelerated by year. So this year, we we will be, net income and cash flow positive for the full year. And so that just puts us in a different position.

We’re capital markets independent. We wanna pay down our debt and clean up our balance sheet, and and we started that in April, and so we’re really excited about where the company is.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yep. Okay. Alright. Well, that’s that’s a great setup, and let’s let’s dive into each of those subcategories as we get into this. So starting with ORLIDEAU, as you said, it’s done remarkably well in a competitive HAE space.

What do you really think of as the core drivers behind the strong launch? And what’s continuing to fuel growth today? Yeah.

John Stonehouse, President and CEO, BioCryst: I think, one, you gotta have a good drug. And I think when we first put out the pivotal data, some people questioned whether it was an effective drug and could be competitive or not. But the key, if I can leave investors with one thing that’s really important to remember, it’s this drug when it works in some HAE patients, the majority of HAE patients, it works as well as any other drug, as well as injectables. And we’ve been able to show that in real world evidence, data, with real world evidence data and studies that we’ve been running since launch. And that has allowed us to start to have conversations with physicians about switching.

But what you gotta remember is also is patients, because they’re controlled now in this marketplace, they don’t see their doctor much more than once or maybe twice a year. And you can bet that the first time the doctor brings it up, they’re not gonna say, oh, yeah. Yeah. I wanna switch. It’s usually if I’m controlled, why would I bother?

And so chipping away at that and getting them to understand that if they’re one of the, you know, majority of people that try Orlodeyo and are controlled, you could be controlled on a once daily capsule to manage your disease, which is a game changer for patients.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. That’s interesting. And so what what specifically do you hear from those patients once they have switched to to Orlodan?

John Stonehouse, President and CEO, BioCryst: Yeah. Life changing from you know, we’ve gotten to know patients really well. And, you know, one of the coolest things we hear is they forget they’re sick. Right? When you when you stick yourself with a needle, when you see your medicine in the refrigerator, it’s a reminder that you have a disease that you need therapy for.

When you’re taking a capsule once a day, it’s like your daily vitamin. And and stress is a trigger for HAE attacks. And so it kinda all builds together where you’re less stressed. You don’t feel like you’ve got the disease anymore, and they’re doing things that they never thought they would be able to do, is wonderful to hear.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. That’s great. So maybe let’s flip the lens to the physician prescriber side of it. You know, you you’ve shared very interesting market research on patient preference, orals, orals versus injectables.

What what do you hear from the physicians, and what what do you think is driving increased confidence in prescriptions?

John Stonehouse, President and CEO, BioCryst: Yeah. And I I think that increased confidence is the key to what we’re seeing in the marketplace. It it really comes down to when they try it and they have success with the drug, they start using it more. And and and I think there was this perception, oh, it doesn’t work as well. People will trade efficacy for convenience.

No way. The table stakes for patients and physicians is it’s got you gotta have control of the disease first. And if you can have that and be on a really convenient one’s daily oral, it’s great. So physicians that, you know, maybe the first time they tried it, they had a patient that it didn’t work in, and so they kind of backed off and didn’t wanna switch. And when we started putting out the real world evidence, they gave it another shot and they started having success with the drug and then started using more of it.

And that’s why they say forward looking that they see more and more of their patients will go will be trying Orlodayo. Mhmm.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yep. Got it. Okay. And then an important metric that you’ve always forecasted is the idea of patients moving from free drug to paid.

That transition has been accelerated relative to how you’ve guided the street, you know, over the course of the last, you know, quarters quarters and years. What tactics have really helped accelerate that?

John Stonehouse, President and CEO, BioCryst: Yeah. Well, maybe what I’ll do is explain what we’re shooting for. So in in this path to a billion dollars, a key element and driver of that value is getting about eighty five percent of the patients on Orlodayo on paid therapy. And honestly, when we first introduced the drug, the idea was to get patients onto drug fast, and so we put them on free drug quickly. What that led to is some payers saying, oh, they’re on free drug.

You know, we’ll make it a little bit more difficult. And we ended up with, you know, people that were on longer term free drug that had insurance and should have been paid. So we built an apparatus around patient support and helping offices with the process of getting through the insurance review and making sure that they had all the tools and information that they needed to have the best shot at getting the insurance to cover it. And I think the other piece that’s really helpful with the plans is this real world evidence again. They see that the drug works and their confidence in Orlodayo has gone up, so their willingness to pay has improved as well.

But really, this this kind of basket of services we call empowered patient services has played a big role in in the improvement. That being said, you know, we’re we’re at 84% right now. We had a huge jump this in the first quarter of this year, largely for Medicare. A couple of years ago, the charities had run out of, money to help with copay assistance for patients on Medicare, and a senior can’t afford a big co pay for a rare disease drug. And so we ended having I think we’re in the high to mid eighties in terms of paid rate, snapped to, like, high forties.

And in the tail end of this year, I think we were at 56%, and in the, in April, we’re up to 89% with Medicare. So that we’re one of the few that says the inflation reduction act has actually been really helpful because that $2,000 co pay has made it more affordable and and allowed charities to spread the money out further.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Mhmm. Got it. Okay. That’s great color. Thank you for that.

You recently submitted a pediatric NDA. How do you think about the opportunity in younger patients? Is that gonna be a meaningful market for you all?

John Stonehouse, President and CEO, BioCryst: Yeah. Unmet need wise, I think it’s huge. Right? You think about a three or four year old and and giving an injection every two weeks. It just the treatment might be worse than the actual disease, honestly.

And so this idea of, sprinkling these mini tabs either in a glass of water that they can drink down or some soft food that they can eat it with is just game changer. And and we’ve heard that from patients and the community really being supportive. And the the enrollment my experience in my thirty plus years of being in in involved in clinical trials is when enrollment goes faster than expected, you’ve got something that patients want, and that’s exactly what happened here. So size wise, it’s not huge. It’s about five hundred patients in The US in terms of potential population, of which about two hundred are are treated that we think are available.

But we also think that patients are sicker than previously thought through the data we’ve collected in our study. And so we think having a more convenient therapy that isn’t as challenging as injectable may the universe of who gets treated on prophylaxis earlier and longer, number one. And then there’s a halo effect that we’ve seen with adolescents where the child has success with Orlodayo. This is a hereditary disease. The mother or father likely has the disease, and they’re on TaxiRO, for example.

And they’re like, wow. I see my kid doing really well. Maybe I should ask my doctor about it as well. And we think there’ll be a halo effect. So we’re not ready to predict what it’ll do.

It’s not in the forecast. It’s not in the billion dollars. But, I think once we get about twelve months ish under our belt, we’ll have a better idea of what the potential is here.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. Got it. Okay. Great.

And so maybe you you’ve been you’ve been very forthright in your public commentary about what the path to $1,000,000,000 is over the next four to five years. Maybe just thinking about that lens of a five year lens, what do you see as the most important levers to expand Orlodea’s market share over that period? You know, what what are you learning about HAE market dynamics that inform that view?

John Stonehouse, President and CEO, BioCryst: Yeah. I I I think we said it, I don’t know, it was a couple of years ago that we actually laid out the path, and we’re ahead of schedule on almost everything that we we said we needed to achieve to get there. So what are the the components? An average of 200 new patients, net new patients per year. We’ve been above that for the last well, since launch, we’ve been significantly above that.

And so the demand, surprisingly, fifth year into launch, continues to be really strong. And I think it’s this increased confidence we talked about before both with doctors and and patients. And so keeping that going, you know, another year, a year after that, you know, it has to be an average of 200. And so, you know, as you get closer to 2028, ’20 ’20 ’9, we think that it will slow down, but we’re well ahead of schedule on that front. The second piece that we talked about is paid.

We didn’t think we’d get there until, you know, 2029, ’20 ’20 ’8, and we’re there now basically at 84%. And I think we can do better than 85%, so that’s upside. And then the last piece is price increases. And and, you know, we said very minimal price increases to to add to that value, and we’ve been doing that on an annual basis and, again, a a little bit higher than the the minimal increase. But, yeah, it’s it’s it’s all ahead of schedule.

So our confidence in that you know, this year, we’re gonna be somewhere between five eight or five ninety and six hundred. And so our or I’m sorry. Five eighty and six hundred. And so, you know, we’re well on our way to a billion dollars at peak.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. Absolutely. Okay. Alright.

Cool. You’ve been very fulsome in the market research that you’ve shared, you know, with in public statements, which is interesting just to watch the evolution year over year on consistent views. Could you expand a little bit on that, of just how you think about key insights that, I suppose, you observe in the market research and how it’s led to, you know, some of the performance in recent quarters?

John Stonehouse, President and CEO, BioCryst: Yeah. It it it’s core to what we do. We we chose a model where we use a sole source specialty pharmacy. And as a result of that, we get a lot of really rich data and insights about patients, both those that have success on Orlodayo and those that don’t. And and it just makes us smarter about what’s going on in the marketplace.

And one of the things you use market research for is friction points. Like, what what’s gonna prevent somebody from making a decision to switch? And, you know, we get at our goal is we wanna get at the truth. You know, a lot of times in companies I’ve been in the past, the senior leader says, you know, find me the data that makes this, you know, believable. Here, we’re like, tell us what’s true, and we’ll follow that.

And so it it it’s been really helpful. And I I think the best place that, we’ve used this is we put out our market research, on competitors and and ourselves and patient share over a ten year period. And, you know, we showed you the robustness. You know, a 75, docs, a hundred patients, over 50 payers. We get preference shares.

We then put it into a Monte Carlo simulation that simulates, you know, patients only see their doctor once a year, and it spits out an answer. And the answer is the early disruptors are the ones that are gonna be market leaders, you know, come 2033, and that’s TaxiRO and Orlodayo. And it’s because it’s really sticky. Right? You if if you’re controlled on a drug that’s once a day and that control is similar to anything else you could go on, what’s gonna be the the benefit of switching to something else?

There’s nothing. And so losing patients to other drugs when you have patients controlled on Orlodayo is just not something that we see in the data.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. Interesting. And maybe with that concept of picking up at the idea of Tektayo, you had mentioned you’ve referenced on a couple times in this conversation, the difficulty in getting patients to switch. So with the comparison to TAKHZYRO or C1 inhibitors or just any injectable prophylactic treatment for HAE beyond the oral, talk a little bit more, please, about the benefits of Orlodayo, which is ultimately getting these patients to to switch off the injectable.

John Stonehouse, President and CEO, BioCryst: Yeah. It it I mean, it all comes the biggest obstacle we get, Ryan, is, you know, my patient’s controls. Why on earth would I mess that up? And and if you think about it from the patient perspective too, they if they’re on TaxiRO or Heygarda and they had a tax before and now they’re controlled on a prophylaxis, they’re scared to switch from something like that. So what we’ve done, and it probably sound like a broken record, but our our, real world evidence has really given physicians, you know, large patient sample sizes.

Right? 450 patients in, you know, types one and two HAE and 350 patients in normal c one and shows that the reduction in attacks matches injectables. And and so that’s opened the door for people to say, hey. Maybe we should maybe I should consider trying this. And then when they have a great experience with it, they wanna try it in more patients.

And and so that’s been that’s been the big win is generating that data, sharing it with physicians, and and then, you know, chipping away at, hey. Now it’s time to try. And I you know, back to the pediatric, there’s some we still have about twenty percent of our, top, potential prescribers, about five to six not prescribed Orlodayo yet, but they have pediatric patients. And we’re gonna go into those offices and say, come on now. There is no reason that you wouldn’t switch a kid that’s on injectable prophylaxis to Orlodea.

And if they have great success, they may consider using it for their adult patients as well.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. Great. Let’s transition. As as mentioned, we wanna spend time on Orlodea.

We wanna spend time on the pipeline. We wanna spend time on the financial profile of the company. Let’s transition a little bit to the pipeline assets. Maybe one more word on the pediatric opportunity for Orlodea. Remind us, please, the time line so that so NDA has submitted a time line for potential approval.

John Stonehouse, President and CEO, BioCryst: Yes. PDUFA is September 12. So, you know, we’re working with the agency now as they go through the review and responding to questions, but, we’re hopeful that there could be a launch yet this year.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yep. And is there is there substantive expansion of the commercial footprint needed for None.

John Stonehouse, President and CEO, BioCryst: None, which is wonderful. So So the Salesforce size is is where it needs to be, and so, you know, it’s a highly profitable segment. Yep.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yep. Okay. Alright. So turn turning to the pipeline. So you’ve got two new programs coming into focus, BCX seventeen thousand seven twenty five and Avorostat.

So let’s about let’s talk about these two. So so firstly, can you walk us through the rationale behind pursuing Netherton syndrome with with the $17,007.25 asset? What’s the unmet need? How does your k l five inhibitor uniquely address it?

John Stonehouse, President and CEO, BioCryst: Yeah. So it’s a horrible disease. It’s basically a that leads to a missing protein that you lose the kind of break that you need for normal skin turnover. And so you have this just rapid skin turnover because you don’t have the break. And it it can, at birth, be, you know, lethal.

Right? You know, think of how important skin is to protecting against infection, hydration, you know, a bunch of other things. And so, it can be, there’s a decent mortality rate with with young children, but you can have it for lifelong. And it’s one of these diseases as we’ve gotten to know patients better that’s, you know, there’s nothing to treat them. They use lotions and creams to keep their skin moist.

They cover themselves up. They you know, sometimes they wear wigs because it affects your hair as well, and there’s hair loss. And and it’s just it’s very sad, honestly. And and these patients haven’t had a whole lot of hope. And so to bring something you know, what we hope to bring is something that will restore that break that we thought that I talked about in terms of our KLK five inhibitor, b six seventeen seven two five, and restore normal skin turnover.

So the idea is, I mean, these patients could potentially have, you know, normal skin again. And, you know, and and this is a disease that affects you from head to toe. And so it’s not like you can spot treat and things like that. You need something that works systemically.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. Okay. And so what what are the next clinical catalysts here? What

John Stonehouse, President and CEO, BioCryst: It’s pretty interesting. So we’re in a phase one study. It has its normal healthy volunteer, sad, mad portion that were well along the way. And and now we’ve chosen a dose to to start to study patients, Notherkin syndrome patients. Because the key to this program and the biggest risk in the program is does the drug get to the skin?

And then when it gets to the skin, does it act the way we hope it does, which is restore the normal turnover and bind to the site. And so you can only do that patients and do the skin biopsies and the like. So we’re gonna get a bunch of really interesting biomarker data in addition to clinical data of looking at the skin. And we hope to have a handful of patients in this what we call part three of the phase one study, by the end of the year. And and, know, if it has the kind of effect we hope it will, we hope to start to see some level of activity and get some sense of dose.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Mhmm. Yep. Yep. Understood. I guess if you think about market size, you know, unmet need, I mean, these are these patients, as you said, you know, have there there’s no standard of care now.

They’re using they’re using lotions. What is the patient population, the prevalence in The US and globally?

John Stonehouse, President and CEO, BioCryst: Yeah. So there’s no diagnostic code. Mhmm. The way we kind of got an estimate of the population is there is one clinical effect called bamboo hair where you actually have to look at a strand of hair under a microscope, and it looks like bamboo. And we found about sixteen hundred patients in The US.

But if there’s no diagnostic code, there’s no treatment, there’s really what can a doctor do for a patient? And and so we believe this is like a lot of other rare diseases, maybe even like HAE back, you know, ten, fifteen years ago where the folks at Viral Thar Viral Pharma thought there were two thousand patients, and now there’s eleven thousand. Right? And and when you start to have therapies, you know, in a population in a dermatology practice that has a group of ichthyosis patients, and you do a genetic test and you screen for it, and now you have a drug, we suspect that could be two, three, four times the size of the 16 hundred. And so we’re really excited to get the data and then to start to see if we can get a better handle on what the true population is.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yep. Yeah. Understood. Yeah. It’s very interesting.

Okay. So transitioning to the eye. So you were developing a Vorostat for DME. Talk a little bit, please, about, you know, the use of a plasma, calacrine inhibitor. You know?

Is it well suited for for the space? How do you think about potential treatment in the eye? Yeah.

John Stonehouse, President and CEO, BioCryst: I mean, investor might say, you said you guys are a rare disease company. What the heck are you doing in an eye disease? But the answer is, opportunistically, we think it’s worth studying this drug because DME, while VEGF inhibitors work really well in a lot of people, there’s still forty ish percent that continue to progress and and have decay in their visual acuity. And and so why is that? Well, there’s some pretty deep scientific evidence that says there’s an alternative pathway other than VEGF, and that’s plasma calacrine.

And so similar to what you see with HAE where contact activation ultimately leads to the swelling, you get the same thing with diabetic patients in the back of the eye and and this release of fluid that causes the swelling in the back of the eye and effect on on the retina. And so, there have been a number of Kallikrein inhibitors that have failed, and and so, we believe it’s because you didn’t get a potent drug in the right space for a long enough period of time. And so we’ve taken the characteristics of oralstat that we studied in HAE where it wasn’t a really good oral drug. It was poorly soluble and poorly permeable, and we now deliver it through a device from Clearside suprachoroidal, device in the back of the eye, and it acts like a depot, and it it just sits there. And we’ve we’ve seen, in animals, nine times EC 90 drug concentrations at six months.

And so we’re really excited to see, with a small study, a phase one SAD study in patients, because you don’t want to inject healthy volunteers, what this drug could do in terms of the swelling of the eye, and and even visual acuity over time, in a single dose. Right? You give one dose, and if you see an effect, you know, duration between three and six months, and you see, you know, VEGF like effect, you’ve got a real drug. And and it’s a small study. If we’re successful, it was a great investment.

If it was unsuccessful, it was a small investment that, you know, was worth the risk. Mhmm.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. Absolutely. So an efficient use of capital. Mhmm.

Remind us, please, on exact timelines on on when we’re gonna follow-up.

John Stonehouse, President and CEO, BioCryst: So we hope to get this thing started sometime between summer and the early part of the fall, and we hope to start to get some patient data by the end of the year where, again, like in the Netherton program, we have some sense of activity and some sense of dose.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Mhmm. Yep. Yep. Understood. Okay.

Interesting. And then as you think about the market, like you said, it’s a it’s a large it’s a large market. It’s a crowded market. The biological rationale and just the differentiated approach relative to VEGF treatments is all well reasoned, how do you forecast the potential placement of a vorostat in the ultimate commercial treatment paradigm? How do you think about that differentiation?

John Stonehouse, President and CEO, BioCryst: Yeah. I I think the simplest way to say it is it’s a huge market. And if there’s a you know, close to fifty percent of patients continue to have visual acuity decay, then if we can bring something forward that addresses that population, it might even be used first line then. Why would you go to something else that you’re not a % sure is gonna work, or you gotta switch from one VEGF to the next? And so I I don’t know until we see the data what the application’s gonna be and what kind of efficacy we will see, but, it could be huge.

And one of the things that’s really interesting is from a commercial perspective, the number of retinal specialists in The United States is about the size of the HAE prescriber population. So from a commercial footprint perspective, it’s not too much different than a rare disease. The clinical trials are much more expensive and bigger, but but the commercial effort is not.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Mhmm. Yep. Yep. Understood. Okay.

That’s great. Thank you for that. Let’s talk about the financial side of the business. So you are a commercial stage rare disease company who has demonstrated strong financial discipline. Revenue is growing, profitable, growing profitability.

So maybe to get into that, so you’ve you’ve said that you expect to be profitable in 2025 now. That was previously 2026. The guidance has been brought up. Tell us about what’s driving that shift and then that balance, and it gets into comments that you just made around DME as well, how you are balancing profitability with pipeline investment.

John Stonehouse, President and CEO, BioCryst: Yeah. So what’s driving the acceleration is Orlodea, plain and simple. The the expenses are going up slightly because of the revenue going up, but Orlodea revenue related expenses like distributor, incentive comp, and and things like that. But it’s growing so fast that we were able to pull forward the profitability by a year. And and we put out guidance that basically shows, twenty five, twenty six, twenty seven, the compound annual growth rate of revenue around 20% and expense around 5%.

And what that leads to is in the end of twenty seven about 600,000,000 in cash. So we wanna not only be profitable, but we wanna give you a sense of the magnitude of that profit. And we have, Pharmacon debt that’s now down to $249,000,000 left to be paid off. And our goal is to get that thing paid down, and not wait for the bullet payment that’s due in April But with that 600,000,000, we can easily pay that loan off and still have a good chunk of cash on the balance sheet.

Mhmm.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. Absolutely. So so with that Pharmacon debt, so it was in April, you paid down 75,000,000, which is beneficial from an interest expense profile perspective, as you said, a capital structure perspective. The the the view is to pay that down over time.

Any any sense of how you’re going to approach that in the coming

John Stonehouse, President and CEO, BioCryst: quarters and years? I’m not gonna guide to it, Ryan, but, you know, the goal is pay as much, if not all of it, down before the bullet payments do as as we can based on the revenue that we have. I I mean, with the $75,000,000 payment, I think it was, an estimate of close to $23,000,000 in interest savings over the life of the loan. I you know, I’d like to do that one or two or three more times to not have to pay the interest. And and so, it’ll be a balance of how much cash do we have on the balance sheet, you know, what as you said, what do we need to do to invest?

Those compound annual growth rates that I shared with you, that has pivotal study costs for both of oralstat and BCX seventeen seven two five for Netherton. So it’s it’s not insignificant investment, but the the revenue far outstrips the expense growth rate, and so you end up building more and more cash. And so, you know, one of the questions is once you pay off the debt, then what? And, you know, my belief is we’re gonna look to go on offense in terms of, you know, looking for things that could, you know, from the outside that could complement our own pipeline in Orlodayo. And, you know, I won’t get into any specifics, but, you know, there’s a number of things that we could take a look at.

There’s a lot of stuff that we don’t wanna take on, you know, somebody else’s risk, whether commercial or or clinical. But, we wanna be opportunistic, and we wanna be in a financial position where we can do that and not have to use our shares. Mhmm.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. That’s that’s very that’s very interesting. So the com the company moving moving to, to an offensive posture from a corporate development perspective as you as profitability continues to grow, cash accrues and the debt profile is continues to be addressed. Okay.

Maybe with that in mind, the concept of bringing it all together, Orlodea performance, pipeline programs that are gonna produce data over the course of 2025, the balance sheet being addressed, and and we’re actively seeing you do that in the comments that you made, John, just now about potential for corporate development and the what’s next are very interesting as something to think about as the next phase. Let’s let’s talk about that next phase. So just the idea of who BioCryst will be, you know, three to five years from now in the next phase of evolution. What does that look like from from your perspective?

John Stonehouse, President and CEO, BioCryst: Yeah. The the in in my mind’s eye, it’s becoming very clear, as evidenced by our performance. So a billion dollars in Orlodayo revenue, looks very achievable from our perspective. And as I said before, on all the parameters that lead to that, we’re ahead of schedule. So that that’s one piece of it.

And then, you know, in a phase three program and on our way to filing and getting approval on a second product, and I’d love it to be BCX seventeen seven two five in Netherton’s disease, and we find out that the market, oh, no. It’s not 1,600. It’s, you know, 4,000. And, you know, we’re the only drug available for it, and we alter the course of you know, and restore normal skin turnover. And then third, you know, we’ve got the capital to be able to look at external things as well and certainly fund there’s more in the pipeline as well.

There’s a bunch of complement programs that are still way too early to talk about, but this kind of continuous engine that brings out another Orlodeyo and another Orlodeyo onto the marketplace for for patients in need and financially supports the company. If you look at companies today that have a product on the market, a promising product in the clinic or the next product, and it’s sourced by their own engine or, you know, they’re really good at BD, you know, the size of those companies are, you know, $10,000,000,000, right, in terms of market cap. So, I don’t see why BioCryst ultimately can’t get to that spot with the ingredients that we have to get there.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yeah. Yeah. Absolutely. Absolutely. So the share price has done well over various periods.

You know, over the last year, the share price has done very well as a result of the strong results from Orlodeaux and just kind of the broader picture of of the company pipeline financial profile. Are there areas where you think The Street may be underappreciating the story, whether it’s commercial durability, pipeline, optionality, anything else?

John Stonehouse, President and CEO, BioCryst: Yeah. I I I think there’s a number of things that as we execute, we can start to have a counterargument. One might be, hey. You had some programs that you had to stop, like our factor d program, and so why are you confident that you have another one? Well, you know, you know what the attrition rate in our industry is, Ryan.

I think we did a good job of, making sure that when we saw there was no commercial viability for our programs, we shut We didn’t spend good money after bad. And so while it’s painful to stop any program, I think making that decision at the right time is really important, and I and I think we did that. But, you know, our our confidence that the risk profile of these two pipeline products is very different than our factor D inhibitors. You know, a protein therapeutic, the off target toxicity is just very different than a small molecule for factor D.

So so I could see people being skeptical of it, but as we generate data, the skepticism, I think, is gonna decrease. I think there’s some people saying the competition, you know, they don’t believe our market research. I would argue it has the least bias of anything I’ve seen out there in terms of predicting the future, but there’s three competitors coming to the market this year. There’s gonna be more coming in in in future years. As we continue to steadily execute on the commercial program, that argument’s gonna disappear.

Mhmm. And so the only way I know you know, you said the share price is doing well. It’s not doing well enough in in my opinion, and I would like to see it do better. But the way that we’re addressing that is by executing. Yep.

And, you know, saying what we’re gonna do and then executing and doing it. And I think we’re well on our way.

Ryan Fisk, Managing Director, Healthcare Investment Banking, Goldman Sachs: Yep. Absolutely. Well, John, I think that’s the time we have. Congrats on recent progress. Looking forward to seeing what the

John Stonehouse, President and CEO, BioCryst: rest of 2025 brings for you all. Yeah. Thanks for inviting us. Sure. Take care.

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