BioCryst (BCRX) Q2 2026 Earnings Call Transcript

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DATE

Wednesday, Aug. 5, 2026 at 8:30 a.m. ET

CALL PARTICIPANTS

• President and Chief Executive Officer - Charlie Gayer
• Chief R&D Officer - Dr. Sandeep Menon
• Chief Financial Officer - Babar Ghias

TAKEAWAYS

• Total Revenue -- $218.3 million, representing a 45% increase on a comparable basis excluding the divested European business and driven by licensing revenue.
• ORLADEYO Net Revenue -- $158.2 million, reflecting 10% comparable year-over-year growth driven by new patient demand and a higher paid rate.
• 2026 Total Revenue Guidance -- $690 million to $715 million, an increase from the previous range of $635 million to $660 million following the recognition of new licensing revenue.
• 2026 ORLADEYO Revenue Guidance -- $625 million to $645 million, maintained despite the timing impact of the pediatric formulation launch.
• Non-GAAP Operating Profit -- $113.2 million, compared to $60.9 million in the second quarter of the prior year.
• GAAP Operating Profit -- $98.5 million, an increase from $29.8 million in the second quarter of 2025.
• Cash and Investments -- $354.0 million as of June 30, 2026, including cash, cash equivalents, and restricted cash.
• Licensing Revenue Recognition -- $55.7 million recognized in the quarter from an upfront payment related to the navenibart European license agreement with Neopharmed Gentili.
• 2026 Non-GAAP Operating Expense Guidance -- $420 million to $440 million, reduced from the initial range of $450 million to $470 million due to the planned closure of internal research facilities.
• ORLADEYO Paid Rate -- 84% at the end of the second quarter, an improvement from 83% one year ago.
• Pediatric Prescription Demand -- 47 prescriptions for ORLADEYO oral pellets received through July 31, 2026, which exceeded management's initial full-year expectations.
• Pediatric Prior Authorization -- over 50% of initial pediatric prescriptions have completed the prior authorization process with a high approval rate.
• Net Income -- $78.4 million, or $0.30 per diluted share, for the three months ended June 30, 2026.
• Non-GAAP R&D Expense -- $46.5 million, an increase of 37% year over year primarily due to Phase 3 activities for navenibart.
• Non-GAAP Sales and Marketing Expense -- $33.9 million, representing a 2% increase on a comparable basis excluding former European operations.
• Non-GAAP G&A Expense -- $20.8 million, a 2% decrease on a comparable basis after adjusting for divestiture-related costs.
• Uninsured Patient Coverage -- 4% of total patients receiving product for free, representing a segment of the 16% non-paid patient base.
• Physician Prescriber Base -- approximately 60 new physicians per month prescribing ORLADEYO, which is consistent with historical trends.
• Total ORLADEYO Patient Reach -- 3,500 patients who have tried the therapy since its launch, with approximately 1,600 currently on treatment.
• Clinical Milestone Timing -- Q3 2027 for top-line data from the navenibart ALPHA-ORBIT pivotal study following the completion of enrollment in June.
• Netherton Syndrome Program -- up to 12 patients being enrolled in Part 4 of the BCX17725 Phase 1 trial, with proof-of-concept data expected by the end of 2026.
• Pricing and Reimbursement Dynamics -- 9% gross price increase for ORLADEYO, contributing approximately 4.5% to net revenue growth after accounting for reimbursement impacts.
• Research Facility Consolidation -- planned closure of the Birmingham research facility by the end of 2026 as the company shifts toward an external innovation model.
• Pharmacy Network Transition -- migration to CareMed as the sole-source specialty pharmacy partner for both adult and pediatric patients beginning in August 2026.

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RISKS

• CEO Gayer noted that while overall retention is stable, the company loses patients to new injectable competitors, stating, "we tend to lose them to the newer products as opposed to the older products."
• Gayer also acknowledged potential operational risks during the specialty pharmacy migration, stating, "Anytime you do a switch like this, there can be bumps," in reference to the transition to CareMed.

SUMMARY

Management at BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported a strategic transition toward an external innovation model following the decision to discontinue internal research programs and close its Birmingham facility by the end of 2026. The company commenced the commercial launch of its pediatric oral pellet formulation for hereditary angioedema while maintaining full-year revenue targets for its established capsule therapy. Financial results included the recognition of significant licensing revenue from European partnerships and a focus on maintaining positive cash flow and operating profitability. Leadership indicated that current capital reserves and operational efficiency provide the necessary flexibility to fund pipeline development and potential strategic acquisitions without requiring additional external financing.

• CFO Ghias stated that the company is in a position to "finance BD and R&D, deliver profitable growth with excess cash flow, remain flexible to consider delevering the balance sheet and/or buybacks."
• Chief R&D Officer Menon noted that the navenibart pivotal study will remain blinded for 12 months, as agreed with the FDA, to reach "one year of safety and efficacy data" across the six-month and 12-month dosing regimens.
• CEO Gayer attributed the early success of the pediatric launch to established payer relationships, stating, "the pediatric pellets slot right into our contracts that we already have," avoiding the need for new formulary negotiations.
• Management reported that market research in the second quarter indicated preference for and satisfaction with ORLADEYO increased for both patients and physicians compared to one year prior.
• The company transitioned its commercial pharmacy operations to CareMed to scale for future growth and leverage their experience in managing long-term prophylaxis programs for rare diseases.
• CEO Gayer observed that current injectable competition in the HAE market is primarily impacting other injectable products, such as TAKHZYRO, rather than oral therapy market share.

INDUSTRY GLOSSARY

• Hereditary Angioedema (HAE): A rare genetic condition characterized by severe, unpredictable swelling in various parts of the body.
• Plasma Kallikrein Inhibitor: A type of drug that blocks the activity of plasma kallikrein to prevent the overproduction of bradykinin, the peptide responsible for swelling in HAE.
• KLK5 (Kallikrein-5): A protease enzyme involved in skin barrier function; its dysregulation is a primary driver of Netherton syndrome.
• Netherton Syndrome: A rare and severe genetic skin disorder characterized by chronic skin inflammation, structural hair defects, and life-threatening infections.
• Prophylaxis: Treatment administered or action taken to prevent the occurrence or recurrence of a disease or its symptoms.
• Proof of Concept (POC): A clinical milestone where a drug demonstrates a desired biological effect in a specific patient population, indicating potential efficacy.

Full Conference Call Transcript

Operator: Hello and welcome to the BioCryst Second Quarter 2026 Earnings Call. [Operator Instructions] Please be advised that today's conference is being recorded. It is now my pleasure to introduce Nick Wilder.

Nick Wilder: Good morning and 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. 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 2 of the presentation. In addition, today's conference call includes non-GAAP financial measures. 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.

Charles Gayer: Thanks, Nick. During the second quarter, we continued to execute effectively on the commercial and development fronts. We completed the integration of Astria Therapeutics and 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 and Babar will describe. Sandeep 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 and 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. Despite the manufacturing delay, we have already received 47 prescriptions for ORLADEYO pellets through July 31, which is well ahead of the total we expected for all of 2026. Prior authorizations are complete for about half of these patients and so far, the approval rate has been strong.

We have a lot to learn about ongoing prescription rates, rates of paid therapy and real world outcomes for kids taking ORLADEYO, but we are thrilled to be delivering this much-needed therapy. Our market research in Q2 showed that preference for and satisfaction with ORLADEYO were up for both patients and physicians compared to 1 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.

On the payer side, the heavy part of reauthorization season is now behind us and we continue to make incremental improvements with the paid rate, which ended the quarter at 84% compared to 83% 1 year ago. Finally, we are pleased to partner with CareMed as our new sole-source pharmacy for ORLADEYO shipments to patients. The sole-source model has been a cornerstone of our strategy since launch and 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 to describe our pipeline developments.

Sandeep Menon: Thank you, Charlie. 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. 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 6 months dosing, we will keep the study blinded to reach 1 year of safety and efficacy data as agreed by the FDA, putting us in a unique position to evaluate efficacy at 6 and 12 months.

I'd like to also address our June decision to discontinue internal discovery programs and close our research unit in Birmingham. 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. This decision has no impact on our clinical programs including BCX17725, which remains on track for early clinical data in patients from Part 4 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. 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.

Babar Ghias: Thanks, Sandeep. 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 in the way. 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. 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. 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. New patient demand remains strong. And as we build on our understanding of the peak 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. And now with the reauthorization season largely complete, we achieved a slight increase in our paid rate over last year, but 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 and that is a true reflection of the benefit of ORLADEYO for HAE patients.

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 3-month and the 6-month period remained relatively flat compared to prior periods. 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. 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. 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 $450 million to $470 million to $420 million to $440 million.

The financial discipline continues to positively impact our balance sheet. We ended the quarter with just over $350 million in cash, cash equivalents and investments. And 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 2 very important catalysts, one of which adds to our growth profile in the near term. So from a BD perspective, we don't feel any need to unnecessarily stress our balance sheet.

As Sandeep 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. But 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 delevering 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 to $715 million. 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 to $645 million. On non-GAAP operating costs, we are maintaining the range at $420 million to $440 million that we announced on June 29. 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.

And while the new BioCryst, I would say, is still in an evolutionary phase, one thing that has remained constant is our strong operating performance record throughout. For that, I'm 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: [Operator Instructions] Your first question comes from the line of Laura Chico with Wedbush.

Laura Chico: Just 2 for 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 or anybody else want to comment, any anecdotal evidence with respect to ORLADEYO persistence in the period? I guess are you seeing more pressure from competitors? And then just one on the Netherton program. I think you mentioned enrollment is proceeding. Have you -- are you approaching the completement of recruitment? And just kind of curious if you could elaborate on any challenges or learnings that you've made so far?

Charles Gayer: Great. Laura, I'll take the first question. Sandeep can address the second one on Netherton. Yes, obviously we have new competitors out there, new injectables. And what we're 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 mentioned in his statement. So 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. And our overall view on ORLADEYO growth, we're very positive about. Sandeep, do you want to address the Netherton?

Sandeep Menon: Yes. Thank you for the question. So we are on track for completing our POC data that will be coming end of this year. And what are we seeing? As you have seen in our healthy volunteer data, the safety has been well tolerated and the drug distribution that has been part of the epidermis where all the action is. So 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.

Charles Gayer: And Laura, just as far as enrollment in the trial, yes, 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 will inform next steps for the program.

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

Gavin Clark-Gartner: Just following up on the Netherton side. I guess what are your expectations for how a placebo arm could potentially perform? Like 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. So I guess I'm just curious how you're piecing together this and contextualizing what a good result is on the efficacy side.

Sandeep Menon: Yes. 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. And 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. So this is the KLK5, which is what we are targeting. So for us when we look at these patients, they have no approved therapies. So any relief for improving signs and symptoms for Netherton would be a big advance based on our discussions with the patients and the KOLs. So 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. And so obviously the placebo is -- there will be some placebo response, but then there is the disease itself has its own waxing and waning. It's a very variable disease in general.

Operator: Our next question comes from the line of Stacy Ku with TD Cowen.

Stacy Ku: We have a couple. So 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 you expect with the pediatric launch? I understand your comments on the injectable kind of share with new patients, but just help us understand kind of how ORLADEYO would continue to progress in a steady fashion. That's one. And then two, maybe talk about the new specialty pharmacy setup. 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 2 quarters? So that's the second question. And then just a very quick follow-up on Netherton. Given that you are in Part 4, any early comments you're willing to share on Part 3?

Charles Gayer: All right. On the clinician additions, you might recall from the past, we had talked about approximately 60 physicians per month prescribing ORLADEYO. We were a little bit above that so 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. So that's a good early sign. Like I said, there's a lot to learn about peds 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. But in the process, we also did look at experience bringing in prophy 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 and 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.

And our guidance, as Babar said, is still the same $625 million to $645 million for the year. And I don't even need to pass this over to Sandeep on sharing anything on Part 3. No, it's too few patients, too short a time. We're really pleased with how Part 4 is enrolling and we look forward to having data at the end of the year.

Operator: And our next question comes from the line of Timur Ivannikov with Cantor.

Timur Ivannikov: This is Timur Ivannikov on for Steve. So my 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. And you mentioned increased paid rate. So what does this imply about volume growth year-over-year in the quarter?

Charles Gayer: Sure, Timur. It's basically half and half. So you might recall that on the 9% price increase, we previously said we net about 4.5% and then obviously we improved the paid rate a bit. So the overall was about half volume, half kind of price and reimbursement.

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

Unknown Analyst: This is [ Sylvia ] on for Jess Fye. Two 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 prophy businesses does this look like? And 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?

Babar Ghias: Yes. So 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 will put stress. We are very laser-focused on delivering on our catalysts. So it could be the clinical mid-stage assets and with an emphasis on clinical assets. That's where we think we can do a real impact. So that's from a BD perspective how we're thinking about the world.

And 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. 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. And when we are ready to provide that guidance, we are anticipating that the costs will continue to go down because we will be past the Birmingham facility closure. We will be past like closing out internal programs. So looking ahead, it's basically Netherton and navenibart expenses.

And as you can imagine that navenibart expenses will be coming down as well as that program reaches its completion. So 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. So that is a key imperative for us to remain profitable and cash flow positive.

Operator: Our next question comes from the line of Serge Belanger with Needham.

Serge Belanger: First one regarding the pediatric launch. Charlie, 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? And then I guess second question, just looking at Slide 6 where you highlight 1,600 patients are currently on therapy, 3,500 other ones have tried the product since launch. So I assume there's another 3,000 to 4,000 patients that have yet to try the ORLADEYO at this point.

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

Charles Gayer: Yes. As far as the peds, 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 and so it's a little too early to call, but certainly the early signals are very positive. 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.

So it's going to really be a split between commercial and Medicaid, but the early signs there are good as well. And the pediatric pellets slot right into our contracts that we already have. So 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. So the pattern on that really hasn't changed and pediatrics can only further help that.

Operator: And our next question comes from the line of Brian Abrahams with RBC Capital Markets.

Brian Abrahams: Two for 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 like 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? And then 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.

Charles Gayer: Sure, Brian. Let's do the navenibart and I'll take the first part and you take the second part, Sandeep. Just on the upsizing, no, it had nothing to do with anything about powering. It was purely about patient and investigator interest. And 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. And so I think it's a reflection of the team as well as the profile of the product. Do you want to just talk about the overall powering?

Sandeep Menon: Yes. So 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. So that actually is a very, very positive sign for all of us in terms of the uptake of how much value the patient -- the physicians are adding to this kind of a modality and the dosing regimen.

Charles Gayer: And then 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. And 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. And so 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?

And that's going to take us some quarters to really see that in the real world. But in the clinical trial in Apex P, we saw that long-term patient retention at 1 and 2 years was above what we saw in our adult trial in Apex 2. So we'll see if that plays out in the real world.

Operator: Our next question comes from the line of Maury Raycroft with Jefferies.

Maurice Raycroft: Congrats on the quarter. For the new patient prescription momentum being maintained in 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?

Charles Gayer: Sure. Yes, the 12 and up population for ORLADEYO capsules, demand was very consistent with what we've seen over the last couple of years. So that was what we expected, but it was also great to see that continued consistent strong demand. And then 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.

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

Charles Gayer: Yes, that does only include the adult population because we didn't have the peds -- the pellets out there yet in Q2. And just a reminder, the paid rate also includes patients who are completely uninsured so that's about 4% out of that 16% that are getting free product. So we feel like we're making great progress and continuing to chip away. Our long-term goal is 85%. But I always say that if we get to 85%, our goal will then go up and we feel good about it.

Typically, and 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. And so the typical pattern is 1 or 2 percentage is lost over the second half of the year and then we gain it back in the reauthorization season next year. So 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.

Operator: Our next question comes from the line of Jon Wolleben with Citizens Bank.

Jonathan Wolleben: 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 if those were 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?

Charles Gayer: Yes. Again I mean, Jon, 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. And so 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 because there should be more like 1,200 kids diagnosed in this age population. And so we're going to be watching for signs 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: Do you expect to continue giving us pediatric prescription data going forward?

Charles Gayer: 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 is the demand directionally going in pediatrics. But we just wanted to give -- and you hit the point, it's close to 10% of that 500 number. And so we thought it was significant to talk about it at this point, but we probably won't give the numerical number going forward.

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

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