BioCryst Pharmaceuticals Q2 2026 Earnings Report

BioCryst Pharmaceuticals EPS Results

• Actual EPS: $0.30
• Consensus EPS: $0.15
• Beat/Miss: Beat by +$0.15
• One Year Ago EPS: $0.15

BioCryst Pharmaceuticals Revenue Results

• Actual Revenue: $218.25 million
• Expected Revenue: $183.28 million
• Beat/Miss: Beat by +$34.97 million
• YoY Revenue Growth: +33.60%

BioCryst Pharmaceuticals Announcement Details

• Quarter: Q2 2026
• Date: 8/5/2026
• Time: Before Market Opens
• Conference Call Date: Wednesday, August 5, 2026
• Conference Call Time: 8:30AM ET

BioCryst Pharmaceuticals (NASDAQ:BCRX) is a clinical‐stage biotechnology company headquartered in Durham, North Carolina, that focuses on the discovery and development of novel, oral small‐molecule medicines for rare and serious diseases. Since its founding in 1986, the company has leveraged structure‐based drug design to advance a pipeline of targeted therapeutics designed to address underlying disease mechanisms rather than just treat symptoms.

The company’s first commercial product, Orladeyo (berotralstat), is an oral kallikrein inhibitor approved for the prophylactic treatment of hereditary angioedema (HAE) in both the United States and Europe. Orladeyo represents BioCryst’s entry into the specialty pharmaceutical market and offers an alternative to injectable prophylactic therapies, with the goal of reducing the frequency of HAE attacks and improving patient convenience.

In addition to its approved product, BioCryst is advancing a pipeline of investigational candidates, including BCX9930, an oral factor D inhibitor being studied for complement‐mediated diseases, and BCX10013, a novel kallikrein inhibitor under development for the treatment of acute HAE attacks. The company’s research and development efforts extend from preclinical stages through multiple phases of clinical trials, with a focus on expanding its small‐molecule platform to address additional rare and serious conditions.

Under the leadership of President and CEO Jon P. Stonehouse, BioCryst maintains a global orientation, collaborating with regulatory authorities and commercial partners in North America, Europe, and select international markets. The company’s headquarters in Durham support its research, development, and administrative functions, while strategic alliances and distribution agreements help facilitate patient access to its therapies worldwide.

BioCryst Pharmaceuticals Q2 2026 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: ORLADEYO revenue rose 10% year over year to $158.2 million on a comparable basis, with continued new-prescriber additions, stable demand and an improved paid rate of 84%.
• Positive Sentiment: The pediatric ORLADEYO pellets launch has begun strongly, with 47 prescriptions received by July 31—above the company’s full-year expectation—and early payer approvals and physician interest described as encouraging.
• Positive Sentiment: BioCryst completed enrollment in the pivotal ALPHA-ORBIT trial of long-acting injectable navenibart ahead of schedule; top-line data are expected in the third quarter of 2027.
• Positive Sentiment: The company raised 2026 total revenue guidance to $690 million-$715 million and lowered non-GAAP operating-cost guidance to $420 million-$440 million, supported by $113.2 million in second-quarter operating profit and more than $350 million in cash and investments.
• Negative Sentiment: BioCryst is shutting down its Birmingham research unit and discontinuing internal discovery programs, shifting to externally sourced assets; management expects lower costs but must establish a stronger pipeline through future business-development transactions.

Presentation

[Operator]

Hello, welcome to the BioCryst second quarter 2026 earnings call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question-and-answer session. To ask a question during the session, you will need to press star one one on your telephone. You will then hear an automated message advising your hand has been raised. To withdraw your question, please press star one one again. Please be advised that today's conference is being recorded. It is now my pleasure to introduce Nick Wilder.

[Nick Wilder, Manager of Investor Relations at BioCryst]

Good morning, welcome to BioCryst's second quarter 2026 corporate update and financial results conference call. Participating with me today are President and CEO, Charlie Gayer, Chief R&D Officer, Dr. Sandeep Menon, and Chief Financial Officer, Babar Ghias. A press release and slide presentation about today's news are available on our investor relations website. Today's call contains forward-looking statements, including statements regarding future results, unaudited and forward-looking financial information, as well as the company's future performance and/or achievements.

[Nick Wilder, Manager of Investor Relations at BioCryst]

These statements are subject to known and unknown risks and uncertainties, which may cause our actual results, performance, or achievements to be materially different from any future results or performance expressed or implied in this presentation. For additional information, including a detailed discussion of these risks, please refer to slide two of the presentation. In addition, today's conference call includes non-GAAP financial measures.

[Nick Wilder, Manager of Investor Relations at BioCryst]

For a reconciliation of these measures against the most directly comparable GAAP financial measure, please refer to the earnings press release available on our investor relations website. I'll now turn the call over to Charlie Gayer.

[Charlie Gayer, President and CEO at BioCryst]

Thanks, Nick Wilder. During the second quarter, we continued to execute effectively on the commercial and development fronts. We completed the integration of Astria Therapeutics, we further increased our cash generations through growing revenue and disciplined spending. That increase in cash flow, bolstered by our strategic decision to wind down internal drug discovery, will enable us to build a balanced pipeline of rare disease assets through external innovation, as Sandeep Menon and Babar Ghias will describe.

[Charlie Gayer, President and CEO at BioCryst]

Sandeep Menon will also provide more color on navenibart, where we completed enrollment of the largest blinded HAE trial ever and did it ahead of schedule. Our proof of concept trial for BCX17725 in Netherton syndrome is also enrolling well, we look forward to having data by the end of the year. This week, we also have exciting news for HAE families as ORLADEYO oral pellets started shipping to kids.

[Charlie Gayer, President and CEO at BioCryst]

Despite the manufacturing delay, we have already received 47 prescriptions for ORLADEYO pellets through July 31st, which is well ahead of the total we expected for all of 2026. Prior authorizations are complete for about half of these patients. So far, the approval rate has been strong. We have a lot to learn about ongoing prescription rates of paid therapy, and real-world outcomes for kids taking ORLADEYO. We are thrilled to be delivering this much-needed therapy.

[Charlie Gayer, President and CEO at BioCryst]

Our market research in Q2 showed that preference for and satisfaction with ORLADEYO were up for both patients and physicians compared to one year prior. This played out in market demand as new prescriptions for ORLADEYO capsules in Q2 were consistent with our history and expectations. We also activated new prescribers at a rate in line with historical trends.

[Charlie Gayer, President and CEO at BioCryst]

On the payer side, the heavy part of reauthorization season is now behind us. We continue to make incremental improvements to the paid rate, which ended the quarter at 84%, compared to 83% one year ago. Finally, we are pleased to partner with CareMed as our new sole source pharmacy for ORLADEYO shipments to patients.

[Charlie Gayer, President and CEO at BioCryst]

The sole source model has been a cornerstone of our strategy since launch. Because our HAE portfolio and the number of patients that we serve is growing, we chose CareMed for their ability to scale operations with our growth. All ORLADEYO pellets prescriptions for kids are shipping from CareMed, and the 12 and up population will transition over the course of this month. We look forward to serving these patients together. I'll turn the call over to Sandeep Menon to describe our pipeline developments.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

Thank you, Charlie Gayer. I'm pleased to share that in June, we completed enrollment of our pivotal study, ALPHA-ORBIT for navenibart, our investigational long-acting injectable plasma kallikrein inhibitor for HAE prophylaxis. We are excited about the potential for this product to transform the injectable segment of the market with its differentiated profile.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

We expect to announce the top-line data for this program in the third quarter of 2027. Because navenibart's extended dosing regimen includes every six months dosing, we will keep the study blinded to reach one year of safety and efficacy data as agreed by the FDA, putting us in a unique position to evaluate the efficacy at six and 12 months. I'd like to also address our June decision to discontinue internal discovery programs and close our research unit in Birmingham.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

Since I've joined BioCryst, we have completed a comprehensive review of our programs, capabilities, and strategic options to ensure we are positioned for future success. After a careful consideration, our team concluded that expanding our access to external innovation is the best way to allocate resources and accelerate the development of a stronger pipeline.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

This decision has no impact on our clinical programs, including BCX17725, which remains on track for early clinical data in patients from part IV of our phase Ib study by the end of this year. We remain excited about the potential to bring forward a treatment for patients with Netherton syndrome, a rare, severe skin condition with no approved therapies. We will continue to be active in our search for external assets.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

The decision to end internal research broadens our scope to build a balanced pipeline of differentiated assets across clinical stages in areas of high unmet need where we can leverage our scientific, clinical, and commercial strengths. I will now transition to my colleague, Babar Ghias.

[Babar Ghias, CFO at BioCryst]

Thanks, Sandeep Menon. Our second fiscal quarter reflected continued momentum across the business, steady growth in ORLADEYO, substantial progress across our clinical pipeline, as you heard, and very importantly, a key step forward in resolving the manufacturing issue to get our pediatric launch underway. It was also another quarter that reflected our financial strength, with strong operating profitability and a cash position that leaves our balance sheet in excellent shape to execute on our go-forward strategy.

[Babar Ghias, CFO at BioCryst]

In my remarks today, I will be referring to some non-GAAP figures, which are adjusted for revenues and expenses related to our former European ORLADEYO business, stock-based comp and expenses related to the acquisition of Astria. You can find additional details for adjustments and reconciliations in the press release. We believe the non-GAAP figures provide a better underlying view of our business on a forward-looking basis.

[Babar Ghias, CFO at BioCryst]

With that said, let me turn over to the financial results. Total revenue for the second quarter of 2026 increased 45% year-on-year on a comparable basis. That is excluding the European divestiture. I would draw your attention to ORLADEYO revenue of $158.2 million in the quarter, which grew 10% year-on-year on that same comparable basis.

[Babar Ghias, CFO at BioCryst]

New patient demand remains strong, and as we build on our understanding of the ped-market dynamics with real-world outcomes, we feel confident that it will continue to augment our growth. Market research results continue to deliver the same consistency of findings regarding the value of ORLADEYO, and we naturally see that play out in our day-to-day commercial execution. Still adding new prescribers, we see consistent long-term patient retention to past trends.

[Babar Ghias, CFO at BioCryst]

Now with the reauthorization season largely complete, we achieved a slight increase in our paid rate over last year, which is important and impressive at the same time because you may recall we had already made a substantial improvement on that metric last year. That is a true reflection of the benefit of ORLADEYO for HAE patients.

[Babar Ghias, CFO at BioCryst]

Our total revenue for the quarter also includes $55.7 million recognized in the quarter from the upfront payment of our navenibart European license agreement with Neopharmed Gentili. I'm also very pleased to share that we are seeing stable operating costs across our business. As you can see from our press release, our non-GAAP G&A and sales and marketing expense categories for both the three-month and the six-month period remained relatively flat compared to prior periods.

[Babar Ghias, CFO at BioCryst]

The increase in R&D is driven by ongoing phase III activities for navenibart, as you would expect. As a result of these trends, we posted a strong non-GAAP operating profit of $113.2 million for the quarter. Importantly, we have taken deliberate steps to further sharpen our cost structure going forward.

[Babar Ghias, CFO at BioCryst]

As we stated in our June news release, we are shifting our R&D strategy towards an external innovation model, discontinuing our internal discovery programs, along with the announced closure of our Birmingham facility by the end of this year. The result is a leaner, more focused operating model that is expected to generate meaningful cost savings as legacy R&D costs continue to come down.

[Babar Ghias, CFO at BioCryst]

This enabled us already to improve on our non-GAAP operating cost guidance range for the remainder of the year, which we brought down from the earlier range of $454 million-$470 million to $420 million-$440 million. The financial discipline continues to positively impact our balance sheet. We ended the quarter with just over $350 million in cash equivalents, and investments.

[Babar Ghias, CFO at BioCryst]

Importantly, we generated positive cash flow in the quarter, even setting aside the upfront proceeds from the license agreement. Simply put, our balance sheet has never been in a stronger position. On that front, let me address from a financial standpoint our strategy going forward. We have a business that continues to generate strong profitability and cash flow growth. We have two very important catalysts, one of which adds to our growth profile in the near term.

[Babar Ghias, CFO at BioCryst]

From a BD perspective, we don't feel any need to unnecessarily stress our balance sheet. As Sandeep Menon alluded earlier, with the shift to external innovation, it allows us to build a more balanced pipeline of clinical assets across all stages. These could be early to mid-stage clinical programs where we will have much more transaction structure flexibility.

[Babar Ghias, CFO at BioCryst]

Ultimately, the objective for us is not about the quantity, but rather the quality of the clinical programs that add to our overall growth profile. We are in a very unique position relative to other companies of our size in that we can finance BD and R&D, deliver profitable growth with excess cash flow, remain flexible to consider de-levering the balance sheet and/or buybacks. Moving on to financial guidance. Given closing of the navenibart EU license, we are raising our total revenue guidance to the range of $690 million-$715 million.

[Babar Ghias, CFO at BioCryst]

Despite the delay in the pediatric launch, we feel long-term ORLADEYO trends are intact, and hence maintaining our full-year 2026 ORLADEYO revenue guidance of $625 million-$645 million. On non-GAAP operating costs, we are maintaining the range at $420 million-$440 million that we announced on June 29th. Over the last 12 months, our business has gone through a number of critical transactions. Sale of Europe, followed by the Astria acquisition, a CEO transition, and now the recent Birmingham decision.

[Babar Ghias, CFO at BioCryst]

While the new BioCryst, I would say, is still in an evolutionary phase, one thing that has remained constant is a strong operating performance record throughout. For that, I am deeply grateful to our team for their unwavering focus on what matters most, advancing ORLADEYO and our pipeline to deliver critical life-changing medicines to patients. With that, operator, we are now ready for questions.

[Operator]

Certainly. As a reminder, to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. One moment, please. Your first question comes from the line of Laura Chico with Wedbush.

[Laura Chico, Analyst at Wedbush]

Hey, good morning. Thanks very much for taking the questions. Just two from me. One, it looks like a very solid ORLADEYO performance this quarter. I know one of your competitors had indicated their prophylactic product was seeing switches from a range of patients. Just curious maybe, Charlie Gayer or anybody else want to comment, any anecdotal evidence with respect to ORLADEYO persistence in the period, I guess?

[Laura Chico, Analyst at Wedbush]

Are you seeing more pressure from competitors? Then just one on the Netherton program. I think you mentioned enrollment is proceeding. Are you approaching the completement of recruitment? Just kind of curious if you could elaborate on any challenges or learnings that you've made so far? Thanks very much.

[Charlie Gayer, President and CEO at BioCryst]

Great. Thanks, Laura Chico. I will take the first question. Sandeep Menon can address the second one on Netherton. Obviously, we have new competitors out there, new injectables, and what we are seeing is what we expected from all of our market research is that the injectables are primarily affecting existing injectables, particularly TAKHZYRO. The overall retention for ORLADEYO is the same pattern as Babar Ghias mentioned in his statement.

[Charlie Gayer, President and CEO at BioCryst]

Of course, we lose some patients, and what now happens is we tend to lose them to the newer products as opposed to the older products, which is exactly what we expected. Our overall view on ORLADEYO growth, we are very positive about. Sandeep Menon, do you want to address the Nethertons?

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

Thank you for the question. We are on track for completing our POC data that will be coming end of this year. What are we seeing? As you have seen in our healthy volunteer data, the safety has been well tolerated, and the drug distribution has been a part of the epidermis where all the action is. At this point, that is the only thing we can comment on. We are still waiting for more data to come in terms of the holistic understanding of the PK/PD and the clinical efficacy endpoints.

[Charlie Gayer, President and CEO at BioCryst]

Laura Chico, just as far as enrollment in the trial, it's been going strong and we're confident that we'll have that up to 12 patients at the end of the year, and that'll inform next steps for the program.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

Yeah.

[Laura Chico, Analyst at Wedbush]

Perfect. Thanks, guys.

[Operator]

Thank you. Our next question comes from the line of Gavin Clark-Gartner with Evercore ISI.

[Gavin Clark-Gartner, Analyst at Evercore ISI]

Hey, guys. Thanks for taking the question. Just following up on the Netherton side. I guess, what are your expectations for how a placebo arm could potentially perform? We have some of the SPEVIGO data showing a bit of an improvement from baseline. We have the Daiichi data, which showed maybe a little bit of a decline on placebo. I guess I'm just curious how you're piecing together this and contextualizing what a good result is on the efficacy side. Thank you.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

Yeah, for us, the way we are thinking about BCX17725, it has the potential to be the first systemically administered and a targeted drug for Netherton syndrome. The rest of the modalities and other things that have been in use, they are not impacting the implicated target that is exactly needed for the Netherton syndrome.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

This is the KLK5, which is what we are targeting. For us, when we look at these patients, they have no approved therapies. Any relief for improving signs and symptoms for Nethertons would be a big advance based on our discussions with the patients and the KOLs. That's what we keep on learning, and we will have a better understanding of the totality of the data that will come at the end of the year.

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

Obviously, there will be some placebo response. There is the disease itself has its own waxing and waning. It's a very variable disease in general.

[Gavin Clark-Gartner, Analyst at Evercore ISI]

Sounds good. Thank you.

[Operator]

Thank you. Our next question comes from the line of Stacy Ku with TD Cowen.

[Stacy Ku, Analyst at TD Cowen]

Hey there. Thanks so much for taking our questions. We have a couple. First, as we think about ORLADEYO, maybe just can you talk through the clinician additions this quarter versus historical averages? What kind of halo effect might you expect with the pediatric launch? I understand your comments on the injectable kind of share with new patients. Just help understand kind of how ORLADEYO has continued to progress in a steady fashion. That's one.

[Stacy Ku, Analyst at TD Cowen]

Two, maybe talk about the new specialty pharmacy set up. Is there any expectation setting when it comes to the transition? Should it be really smooth? Just help us think about the cadence of the U.S. launch for ORLADEYO, maybe how the second half might look relative to the first two quarters. That's the second question. Just a very quick follow-up on Netherton.

[Stacy Ku, Analyst at TD Cowen]

Given that you are in part four, any early comments you're willing to share on part three? Thanks so much.

[Charlie Gayer, President and CEO at BioCryst]

All right. Thanks, Stacy Ku. On the clinician additions, you might recall from the past, we had talked about approximately 60 new physicians per month prescribing ORLADEYO. We were a little bit above that. ORLADEYO continues to gain in the market. From the halo of pediatrics, obviously it's still early, but of those 47 prescriptions, a number of them were actually prescribed by physicians who had not yet prescribed ORLADEYO. That's a good early sign.

[Charlie Gayer, President and CEO at BioCryst]

Like I said, there's a lot to learn about pediatrics now that we have product in the market, but we're off to a really good start. On the new SP transition to CareMed, the biggest decision was, as I mentioned, the ability to scale with us as we grow.

[Charlie Gayer, President and CEO at BioCryst]

In the process, we also did look at experience bringing in active programs, and CareMed has a lot of experience doing that, and we've kind of been impressed with their track record. Anytime you do a switch like this, there can be bumps. This whole switch is just starting this month. We don't expect it to affect ORLADEYO long term, but we'll have to just kind of see in the quarter. We think long term, this is going to be great for patients.

[Charlie Gayer, President and CEO at BioCryst]

Our guidance, as Babar said, is still the same, $625 million-$645 million for the year. I don't even need to pass this over to Sandeep Menon on the sharing anything on Part Three. No, it was too few patients, too short a time. We're really pleased with how Part Four is enrolling, and we look forward to having data at the end of the year.

[Stacy Ku, Analyst at TD Cowen]

Thank you so much.

[Operator]

Thank you. Our next question comes from the line of Timur Ivanov with Cantor.

[Timur Ivanov, Analyst at Cantor]

Thank you. This is Timur Ivanov on for Steven Seedhouse. Our question is for ORLADEYO. I was hoping you could provide some color on the volume growth in the quarter, because it sounds like you grew revenues 10% year-over-year, but there was also a 9% price increase. You mentioned increasing paid rate. What does this imply about volume growth year-over-year on the quarter? Thank you.

[Charlie Gayer, President and CEO at BioCryst]

Yeah, sure, Timur Ivanov. It's basically half and half. You might recall that on the 9% price increase, we previously said we net about four and a half of that, obviously we improved the paid rate a bit. The overall was about half volume, half kind of price and reimbursement.

[Timur Ivanov, Analyst at Cantor]

Great. Thank you.

[Operator]

Our next question comes from the line of Jess Fye with JPMorgan.

[Analyst at JPMorgan]

Hi, this is Sylvia on for Jess Fye. 2 questions from us. Can you elaborate on your business development priorities? Does this look more like bolt-on acquisitions to the clinical pipeline or partnerships, and what kind of profile businesses does this look like? The second question I have is, can you walk through the OpEx outlook for 2027 and beyond in the scenario where Netherton advances and in the scenario where it doesn't, just to help us understand the flex factors there? Thank you.

[Babar Ghias, CFO at BioCryst]

Yeah. I would say that, from a BD perspective, the objective is to build a really high-value pipeline, validated biology proof of principle. As I mentioned that naturally we are not going to stress our balance sheet, so we're not looking at large company acquisitions that would put stress. We are very laser-focused on delivering on our catalyst.

[Babar Ghias, CFO at BioCryst]

It could be like clinical mid-stage assets and with an emphasis on clinical assets. That's where we think we can do a real impact. That's from a BD perspective, how we're thinking about the world. Like I said, we are cash-generating, self-sufficient, so we don't necessarily looking at the large capital raises to fund our BD or R&D operations. From an OpEx standpoint, I think the outlook is really strong.

[Babar Ghias, CFO at BioCryst]

We haven't really given 2027 guidance, but as you may recall that when we lowered our guidance, that was at the middle of the year that we were making this decision. When we are ready to provide that guidance, we are anticipating that the cost will continue to go down because we will be past the Birmingham facility closure. We will be past closing our internal programs. Looking ahead, it's basically Netherton and navenibart expenses.

[Babar Ghias, CFO at BioCryst]

As you can imagine, the navenibart expenses will be coming down as well as that program reaches its completion. Going forward, the operating expense guidance, absent any new BD deals, will be really strong. Having said that, it is our objective that even as we bring BD assets in, BioCryst is committed to delivering profitable growth. That is a key imperative for us to remain profitable and cash flow positive.

[Operator]

Thank you. Our next question comes from the line of Serge Belanger with B. Riley.

[Serge Belanger, Analyst at B. Riley]

Hi, good morning. Thanks for taking my questions. First one regarding the pediatric launch. Charlie Gayer, I think you mentioned that the initial numbers were tracking ahead of your expectations. Just curious if that changes your outlook for that opportunity, and whether we should expect the paid Rx rates, which I think resides around 80% right now, to be similar on the pediatric side.

[Serge Belanger, Analyst at B. Riley]

I guess second question, just looking at slide six, where you highlight 1,600 patients are currently on therapy, 3,500 other ones have tried the product since launch. I assume there's another 3,000-4,000 patients that have yet to try the ORLADEYO at this point. You were able to capture these

[Serge Belanger, Analyst at B. Riley]

other patients via the current base of prescribers, you'll need to expand beyond the 1,500 unique prescribers you currently have?

[Charlie Gayer, President and CEO at BioCryst]

Yeah. Thanks, Serge Belanger. As far as the pediatrics, like I said, we're encouraged by the early demand since we just have product going out to patients this week and obviously that should probably make prescribers more comfortable too. We have to kind of see what the ongoing trend is. It's a little too early to call, but certainly the early signals are very positive.

[Charlie Gayer, President and CEO at BioCryst]

As far as the paid rate, we expect it to at least be in the range of what it's been for capsules. The payer mix will be a little bit different, obviously, because we're not going to have Medicare, it's going to really be a split between commercial and Medicaid. The early signs there are good as well, and the pediatric pellets slot right into our contracts that we already have.

[Charlie Gayer, President and CEO at BioCryst]

It's not like a new lift to get these things on formulary. As far as the ongoing opportunity, I think the fact that demand has continued to be as consistent and strong as it's been over the last couple of years, plus we're adding new prescribers, just shows that there's still plenty of opportunity out there, and we see that in our market research as well. We also see that new prescriptions are coming very strongly from existing prescribers as well as obviously these new prescribers that are opening up. The pattern on that really hasn't changed and pediatrics can only further help that.

[Operator]

Thank you. Our next question comes from the line of Brian Abrahams with RBC Capital Markets.

[Brian Abrahams, Analyst at RBC Capital Markets]

Hey, good morning. Thanks for taking my questions. Two from me. Maybe first on navenibart, just with the enrollment completion and upsizing in that study. Just wondering if, I guess the upsizing was based on just higher than expected interest or on any kind of observations on dropouts? The types of patients you're seeing there and sort of any implications for powering and should we assume this is almost entirely adults with just a few adolescents?

[Brian Abrahams, Analyst at RBC Capital Markets]

Secondly, it seems like with ORLADEYO, you're in a nice steady state with regards to patient retention. Just wondering if there may be any ways you can further improve on that just ahead of potential competitor launch. Anything that you might see in upcoming competitor oral prophy results that could adjust your strategy there, just in anticipation of potential additional competitive entrant? Thank you.

[Charlie Gayer, President and CEO at BioCryst]

Sure, Brian Abrahams. Let's do the navenibart and I'll take the first part, you take the second part, Sandeep Menon. Just on the upsizing note, it had nothing to do with anything about powering. It was purely about patient and investigator interest. As we mentioned, it was the largest blinded trial ever in HAE, yet it enrolled just as quickly as other recent trials that were smaller. So I think it's a reflection of the team, excuse me, as well as the profile of the product. Do you want to just talk about the overall powering?

[Sandeep Menon, Chief Research and Development Officer at BioCryst]

Yeah. The overall powering, we are already overpowered for the trial, so we don't have any concerns. In this case, it was just a function of more interest from the investigators. That actually is a very, very positive sign for all of us in terms of the uptake of how much value the physicians are adding to this kind of modality and the dosing regimen.

[Charlie Gayer, President and CEO at BioCryst]

As far as patient retention, I think one of the things we always look at is just our operations and how we're performing, and we think that there might be a few things there that we can further improve, but we're pleased with the overall rate of retention. It really comes down to what we said before, which is patients either do quite well on ORLADEYO and then they're very sticky or they don't and they move on.

[Charlie Gayer, President and CEO at BioCryst]

That's what our market research shows, that's what our real-world evidence shows, and we expect that to continue. One thing we will be looking for is in the pediatric indication, what is the retention there? That's going to take us some quarters to really see that in the real world.

[Charlie Gayer, President and CEO at BioCryst]

In the clinical trial in APEX-P, we saw that long-term patient retention at one and two years was above what we saw in our adult trial in APEX-2. We'll see if that plays out in the real world.

[Brian Abrahams, Analyst at RBC Capital Markets]

That's really helpful. Thanks so much.

[Operator]

Thank you. Our next question comes from the line of Maury Raycroft with Jefferies.

[Maury Raycroft, Analyst at Jefferies]

Hi, good morning. Congrats on the quarter and thanks for taking my questions. For the new patient prescription momentum being maintained at second quarter, is that only driven by the new pediatric patients or prescriptions, or can you provide more context on how we can quantify this and understand contribution from new adult patients versus pediatric patients?

[Charlie Gayer, President and CEO at BioCryst]

Sure. Yeah, the 12 and up population for ORLADEYO capsules demand was very consistent with what we've seen over the last couple of years. That was what we expected, but it was also great to see that continued consistent strong demand. The pediatrics off to a great start, that's just upside on top, and we'll see how that progresses over the next few quarters.

[Maury Raycroft, Analyst at Jefferies]

Got it. Okay. Just clarifying with the paid rate at 84%, does that only include the adult population? Wondering if you can comment on just how you expect the paid rate to continue increasing for third quarter and fourth quarter.

[Charlie Gayer, President and CEO at BioCryst]

Yeah, that does only include the adult population because we didn't have the pediatrics, the pellets out there yet in Q2. Just a reminder, the paid rate also includes patients who are completely uninsured, that's about 4% out of that 16% that are getting free product. We feel like we're making great progress and having continuing to chip away.

[Charlie Gayer, President and CEO at BioCryst]

Our long-term goal was 85%, I always say that if we get to 85%, our goal will then go up, and we feel good about it. Typically, I've said this before, in the second half of the year, the paid rate tends to drop off a little bit because the patient mix is driven more by new patients coming in who tend not to get to that 84% quite as quickly. For some of them, it takes longer.

[Charlie Gayer, President and CEO at BioCryst]

The typical pattern is 1% or 2% is lost over the second half of the year, then we gain it back in the reauthorization season next year. I think end of Q2 is a great point to measure progress, and that's why we pointed it out this year, where we picked up 1% versus last year. Next year, I would expect us to be at or above where we were at the end of Q2 this year.

[Maury Raycroft, Analyst at Jefferies]

Got it. That's helpful. Thanks for taking my questions.

[Operator]

Thank you. Our next question comes from the line of Jonathan Wolleben with Citizens JMP.

[Jonathan Wolleben, Analyst at Citizens JMP]

Hey, thanks for taking the question. Just talking about the pediatric opportunity, you've talked a little bit before about 500 diagnosed patients and with 10% already seeking prescriptions. Can you talk a little bit about that early dynamic of those where families coming in and asking about ORLADEYO, or are you guys getting out there in the community? How do you think about the ramp and then also the diagnosed population as you get out there and talk about it more?

[Charlie Gayer, President and CEO at BioCryst]

Yeah, again, Jonathan Wolleben, it's really exciting what we've seen. You pointed it out, it's close to 10% of that 500 number. What we are seeing in our market research is that physicians, A, are very enthusiastic about prescribing ORLADEYO pellets to kids, and B, the availability of an oral Prophy makes them think about prescribing to more of their pediatric population in general.

[Charlie Gayer, President and CEO at BioCryst]

The early signs are that we could not only get more of the existing Prophy patients onto oral Prophy, but also grow the market, expand the market. There should be more like 1,200 kids diagnosed in this age population. We're going to be watching for signs that that 500 grows. Early signals are positive, but we're going to need another many quarters to really see how this plays out.

[Jonathan Wolleben, Analyst at Citizens JMP]

Do you expect to continue giving us pediatric prescription data moving forward?

[Charlie Gayer, President and CEO at BioCryst]

No, I don't think we'll give you the exact number going forward. We'll give you the overall color on how is demand in general, and we'll probably say how's the demands directionally going in pediatrics. You hit the point, it's close to 10% of that 500 number. We thought it was significant to talk about it at this point, but we probably won't give the numerical number going forward.

[Jonathan Wolleben, Analyst at Citizens JMP]

Got it. Thanks, Charlie Gayer.

[Operator]

Thank you. I'm showing no further questions. Ladies and gentlemen, we would like to thank you for participating. This does conclude today's program, and you may now disconnect.

Participants

Analysts

• Nick Wilder, Manager of Investor Relations at BioCryst
• Charlie Gayer, President and CEO at BioCryst
• Sandeep Menon, Chief Research and Development Officer at BioCryst
• Babar Ghias, CFO at BioCryst
• Laura Chico, Analyst at Wedbush
• Gavin Clark-Gartner, Analyst at Evercore ISI
• Stacy Ku, Analyst at TD Cowen
• Timur Ivanov, Analyst at Cantor
• Analyst at JPMorgan
• Serge Belanger, Analyst at B. Riley
• Brian Abrahams, Analyst at RBC Capital Markets
• Maury Raycroft, Analyst at Jefferies
• Jonathan Wolleben, Analyst at Citizens JMP