バイオクリスト2026年第3四半期決算説明会：オルラデオの成長、ガイダンス引き上げ

要点

• 2026年第3四半期のORLADEYOの純売上高は1億7,480万ドルに達し、その95%以上が米国で計上されました。欧州での売上高を除く売上高は前年同期比20%増となりました。
• 当四半期には、CareMedによる初期在庫積み増しから1,000万ドル台後半の寄与が含まれています。経営陣はこれを一括性・一時的な効果と位置付けており、今後の売上高は患者の需要により連動した形で推移すると見込んでいます。
• Non-GAAP営業利益は前年同期比70%増の8,600万ドルとなり、Non-GAAP純利益は前年同期の2倍以上の7,100万ドルに達しました。現金及び現金同等物は合計3億7,600万ドルとなりました。
• BioCrystは、ライセンス収入の実績増加および海外からのRAPIVABの受注増を反映し、2026年通期の総売上高見通しを7億1,500万ドル〜7億3,000万ドルに上方修正しました。ORLADEYOの売上高見通しは6億2,500万ドル〜6億4,500万ドルで維持されました。
• 2026年通期の営業費用見通しは4億1,500万ドル〜4億2,500万ドルへと絞り込まれました。構造改革策が完全に実施されれば、年間で約3,000万ドル〜3,500万ドルのコスト削減効果が見込まれています。
• 経営陣によると、Navenibartのピボタル試験は予定通り進行しており、2027年第3四半期に12ヶ月時点のトップラインデータが得られる見通しです。ネザートン症候群を対象としたBCX17725の初期データは2026年末までに得られる見込みです。

主要財務データ

事業および業績の動向

ORLADEYOの処方需要は引き続き堅調です。経営陣は、新規患者への処方が2026年を通じて見られた高い水準を維持しており、治療を受けている患者基盤も当四半期中に順調な伸びを示したと述べました。

BioCrystは8月、2〜11歳児向けのORLADEYOペレット剤を発売しました。経営陣は初期需要が予想を上回っていると説明しており、対象年齢全域にわたる普及に加え、予防投与や急性期治療のみからの切り替え患者と未治療患者（トリートメントナイーブ患者）がバランスよく含まれているとしています。同社は小児向け市場の機会が、以前議論されていた診断・治療済みの500名の患者プールを超える可能性があると考えていますが、定常的な需要を評価するにはさらに数四半期分のデータが必要であるとしています。

同社はまた、承認済みの12歳以上対象の150ミリグラムペレット製剤の発売も計画しています。この製剤は、カプセルの飲み込みが困難な患者や、ペレットを好む患者を対象としています。

BioCrystは、ORLADEYOの流通を単独のスペシャリティ調剤薬局パートナーであるCareMedへと移行しました。経営陣によると、初期データでは新規患者への初回出荷までの時間が改善しています。有償処方率は第3四半期末時点で約82%〜83%となり、同社は長期的な定常目標である85%に到達、あるいはそれを上回る機会があると見ています。

パイプラインに関して、Navenibartのピボタル試験の被験者登録は6月に完了しました。BioCrystは、2027年第3四半期の12ヶ月時点のトップラインデータを待つ間、生物学的製剤承認申請（BLA）ファイルの作成と販売開始計画を進めています。

ネザートン症候群を対象としたBCX17725の研究ではパート4の登録が完了しました。経営陣は2026年12月に有効性、安全性および耐容性を網羅するデータが公表されると見込んでいます。同社は社内調査や主要意見指導者（KOL）との協議に基づき、主要評価項目であるIASIスコアの20%〜25%の改善が臨床的に意味のある結果となり得ると指摘しつつも、規制当局との協議は保留中であると述べています。

業績予想（ガイダンス）

経営陣は、既存事業が売上成長を維持し、2026年よりも低い営業コスト基盤で2027年に入ると見込んでいます。正式な2027年ガイダンスは後日提供されます。また同社は、構造改革策の通期効果が実現するにつれ、2027年の営業費用がさらに減少すると予想しています。

RAPIVABは戦略的なコア資産とはみなされていません。経営陣は、利益率が低く主にパススルー（仲介的）ビジネスであると説明しており、売上高寄与の増加が利益に与える影響は限定的であるとしています。

リスクおよび注視すべきポイント

• CareMedへの移行に伴い、一時的な業務上の複雑性が生じています。経営陣は完全な適用の経験が6〜8週間にとどまっているため、ORLADEYOのガイダンスに対して保守的な姿勢を維持しています。
• 1,000万ドル台後半の在庫寄与は一回限りの事象でした。今後の四半期売上高は患者の需要をより正確に反映することになりますが、短期的にはわずかな在庫変動が発生する可能性があります。
• 小児向け需要の立ち上がりは当初の予想を上回っていますが、発売から数ヶ月しか経過しておらず、安定した定常需要はまだ確立されていません。
• Navenibartの長期的な商用化の機会は、ピボタル試験のデータ、承認、および他の注射用予防療法を使用している患者が切り替えるかどうかに依存します。
• BCX17725の開発推進は、有効性、安全性、および耐容性データの全体性と一貫性に依存し、その後に規制当局との協議が行われます。

アナリストQ&Aの要点

CareMedの在庫影響：経営陣は、第3四半期の在庫積み増しが毎四半期継続する寄与ではなく、一回限りの利益であったことを確認しました。CareMedは、通常の7〜21日分の範囲の低い水準近くで在庫を維持すると見込まれています。

ORLADEYOの競合的地位：BioCrystは、自社の市場調査において、deucrictibantのプロファイルをその後の第III相試験結果よりもやや優れているとモデル化したと述べました。シミュレーションでは、deucrictibantはORLADEYOの既存の患者基盤を侵食することなくシェアを獲得できる可能性が示されました。経営陣は、将来的に経口競合薬が市場に参入した後もORLADEYOの成長が続くと見込んでいます。

患者の定着率とコントロール：ORLADEYOの治療を開始した患者の約60%が12ヶ月時点でも治療を継続しています。経営陣は、発作回数の中央値が月あたり約3分の1回であることを示すリアルワールドエビデンスを引き合いに出し、これらの患者の症状は概ね十分にコントロールされていると述べました。

Navenibartの投与頻度：経営陣は3ヶ月に1回の投与頻度を画期的なものと捉えており、3ヶ月と6ヶ月の両方の投与選択肢を提供することが製品のプロファイルを強化できると考えています。BioCrystは、臨床および規制面の結果次第ではあるものの、第II相試験で観察された持続性に基づき、両方の投与頻度が添付文書（ラベル）に含まれると見込んでいます。

外部イノベーション戦略：BioCrystは、明確な生物学的根拠を保有し、既存の商業化能力と親和性の高い、主に第I相または第II相の初期臨床段階の資産を優先しています。同社の科学諮問委員会は、社内の科学チームおよび事業開発チームとともに、可能性のある機会を積極的に評価検討しています。

決算発表トランスクリプト全文

決算説明会の完全なトランスクリプト

経営陣による説明

Operator

Good day, and thank you for standing by. Welcome to the BioCryst Third Quarter 2026 Earnings Call. [Operator Instructions] Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Nick Wilder with BioCryst. Nick, go ahead.

Nick Wilder

Good morning, and welcome to BioCryst's Third Quarter 2026 Corporate Update and Financial Results Conference Call. Participating with me today are President and CEO, Charlie Gayer; Chief Financial Officer, Babar Ghias; and Chief R&D Officer, Dr. Sandeep Menon. 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 non-GAAP financial measures against the most directly comparable GAAP financial measure, please refer to the earnings press release available on our Investor Relations website.

I'd now like to turn the call over to Charlie.

Charles Gayer

Thanks, Nick. The third quarter was significant for BioCryst as we continue to deliver strong execution during a time of strategic evolution. On the HAE front, new patient prescriptions for ORLADEYO continued at the same high level that we have seen all year. We again added new prescribers at a rate above the average of the past several years, and the total base of treated patients grew at a healthy rate in the quarter. We were thrilled to launch ORLADEYO pellets to kids in August and early signs of performance continue to be promising. Children of all ages across the 2 to 11 years range have started on ORLADEYO pellets and the mix of kids switching from other prophy and acute only as well as those who were treatment-naive has been balanced. This suggests the pediatric market may grow beyond this 500 patients that we have previously described.

We are also looking forward to launching the recently approved 150-milligram ORLADEYO pellets at the upcoming American College of Allergy, Asthma and Immunology Meeting this month. This new formulation for ages 12 years and up provides an option for kids who started on pellets for adolescents and adults who have difficulty swallowing a capsule or for anyone who simply prefers the pellet formulation. We are looking forward to generating real-world evidence to track the progress and patterns of use for all patients treated with ORLADEYO pellets.

During the third quarter, we also implemented the transition to CareMed as our sole-source pharmacy partner for shipments to patients. We switched to CareMed because the volume of patients on ORLADEYO has grown to the point where operational excellence at the pharmacy level can have a significant impact on the growth in treated patients and the shipment in paid product. We are working closely with CareMed to integrate best practices within our Empower patient services program and are encouraged by the early signs we are seeing in the partnership. We believe CareMed can scale with us as our portfolio of HAE prophy offerings continues to grow.

Turning to our pipeline. As we have previously announced, the pivotal trial for Navenibart fully enrolled in June, and we remain on track to announce top line data through 12 months of therapy in the third quarter of next year. Our team is working diligently on the BLA file as well as launch planning for when we add this potentially transformative product to our HAE portfolio. Continuing on the theme of strong execution, we have enrolled Part 4 of our trial of BCX17725 for Netherton syndrome patients and are on track to have early clinical data by the end of this year. There are no treatments approved for this devastating skin condition that we believe includes at least 3,000 patients in the United States alone.

Once the data reads out, we will review the data in totality, which includes key efficacy endpoints like IASI, the Ichthyosis Area Severity score as well as safety and tolerability to inform potential next steps in the program.

Finally, we are excited about our strategic shift toward building our pipeline through external innovation. We recently announced a Scientific Advisory Board of leading biotech industry experts who will partner with us to deliver on our strategy. These esteemed advisers with a successful track record did not join us just for the honor of being on the Board. They joined us because they believe that BioCryst's profitability, discipline and strong execution position the company for success in our rare disease strategy.

With that, I'll turn it over to Babar to describe our increasingly strong financial position.

Babar Ghias

Thanks, Charlie. We are pleased to share the results of a very strong third quarter, which included not only the important accomplishments just described, but also continued financial excellence. The intentional proactive steps we have taken throughout the year to position BioCryst for durable growth and increasing profitability continue to be reflected in our financial performance.

In my remarks today, I will refer to certain non-GAAP financial measures, which adjust for revenues and expenses associated with our former European ORLADEYO business, stock-based comp, Birmingham site closure and organizational restructuring costs and expenses associated with the Astria acquisition. Additional information regarding these adjustments is included in today's press release. We believe these non-GAAP measures provide investors with a useful view of the underlying performance of the ongoing business.

Now turning first to revenue. ORLADEYO net revenue was $174.8 million for the quarter with more than 95% coming from the U.S. business. On a comparable basis, excluding European revenue, ORLADEYO revenue increased 20% year-over-year. ORLADEYO revenues benefited this quarter from the CareMed transition. One of the benefits of switching to a larger specialty pharmacy is that they maintain a level of inventory at multiple sites and broader set of working operation hours that facilitate timely fulfillment to efficiently respond to patient demand. The inventory they hold typically ranges from 1 to 3 weeks of product. As part of the transition to this model, CareMed built an inventory level towards the lower end of this range during the third quarter, resulting in a revenue contribution in the upper teens of millions of dollars in the quarter.

Since we expect their inventory level to remain in this lower end, revenue should more closely reflect underlying patient demand going forward. While a transition of this nature naturally creates some temporary operational complexity as systems and processes are implemented, we are very pleased with the progress to-date. Although it is still early, we are already seeing an improvement in days to first shipment for new patients, an important measure of the patient experience. Despite the transition, even when removing the impact of the inventory build, ORLADEYO revenue, excluding Europe, grew year-over-year. This is evidence of the continued favorable patient dynamics across the approved population. Throughout this process, our priority has remained the same, maintaining a high-quality patient experience and uninterrupted access to ORLADEYO.

Before turning to operating expenses, let me briefly provide some context around the GAAP to non-GAAP adjustments this quarter. These adjustments primarily relate to the closure of our Birmingham facility and associated organizational restructuring activities that remain on track to be substantially completed by year-end. Of the approximately $19 million in restructuring-related expenses in the quarter, only $10 million are real cash costs. Noncash charges primarily reflected accounting adjustments to assets that no longer will have economic use following the closure. The most important takeaway today is the structural benefit to our cost base. The actions we have taken are expected to generate approximately $30 million to $35 million of annualized savings once fully realized with the potential for additional savings as certain programs are wound down.

As we look towards 2027, the business as it exists today is positioned for continued revenue growth alongside a lower operating cost base than in 2026. We will provide formal 2027 guidance at the appropriate time, but the combination should support continued expansion in profitability.

Turning now over to operating expenses. Non-GAAP operating expenses were slightly lower year-over-year, consistent with the financial discipline we have been emphasizing. We are actively investing in the Phase III development program for Navenibart, which is naturally an important driver of R&D spending. At the same time, we are taking a disciplined data-driven approach to capital allocation across the rest of the pipeline. BCX17725 is a good example. Our approach has been to generate early clinical data in patients first and let that data prospectively inform the appropriate level of future investment. We remain on track to share those data before year-end. Sales and marketing expenses were modestly higher year-over-year, driven primarily by the transition-related fees associated with the Specialty Pharmacy arrangement, and G&A expense has remained essentially flat. This operating leverage from the trend is increasingly visible in our results.

Non-GAAP operating profit for the quarter was $86 million, an increase of 70% year-over-year, while non-GAAP net income of $71 million more than doubled from the prior year period. As profitability continues to grow, we also expect to benefit from our substantial net operating loss carryforwards, which should help limit cash taxes over the medium term, subject to applicable tax rules and limitation. That creates the potential for strong growth, not only in earnings, but also in cash generation. Our liquidity position remains strong with cash and equivalents of $376 million.

As we have discussed, we have made a deliberate pivot towards external innovation, but we are going to remain disciplined in how we build the pipeline. For us, the priority is not adding programs simply to increase the size of the pipeline. It is identifying opportunities where we believe BioCryst can create meaningful value for patients and shareholders. At the same time, we will always continue to evaluate opportunities to strengthen the balance sheet and return capital while maintaining the financial flexibility to invest in compelling innovation.

Turning finally to our 2026 financial guidance. When we established our ORLADEYO revenue guidance, we did not assume a benefit from the initial specialty pharmacy inventory build. Given that we still remain relatively early in the CareMed transition implementation, we are not changing ORLADEYO revenue guidance at this time. We are encouraged by the progress we are seeing, but we also recognize that we are transitioning 6 years of launch experience, systems and operating knowledge from one partner to another on an accelerated time line. Importantly, we view this as a finite transition and not a long-term operating dynamic. Underlying patient demand and trends remain strong, and we are excited about the opportunity to expand the Pediatric launch while also offering the newly approved oral pellet formulation for patients with HAE 12 years and older, providing a continuum of oral treatment options.

On the other hand, we are increasing our total revenue guidance to $715 million to $730 million, reflecting higher realized licensing revenue and international RAPIVAB orders from our partner. To emphasize, RAPIVAB is not a strategic core asset for us and is mostly a pass-through business with low margins. On expenses, we are again tightening our full year operating expense guidance now to $415 million to $425 million for 2026. When we realize the full year benefit of the actions we have taken in 2026, we expect operating expenses in 2027 to decline further while the underlying business continues to grow.

To close, this was a very strong third quarter and strong first 9-months of 2026. We have 1 quarter remaining, and our focus is on finishing the year well and entering 2027 from a position of considerable strength with growing revenue and increasingly efficient cost structure, expanding profitability, strong liquidity and important pipeline catalysts ahead, we believe we have the opportunity to make 2027 a transformational year for BioCryst. The entire organization is focused on executing against the opportunity, and I am very grateful to our colleagues across BioCryst for everything they have accomplished this year.

With that, operator, we are now ready to take questions.

Operator

[Operator Instructions] Our first question comes from the line of Gavin Clark-Gartner with Evercore ISI.

質疑応答

Gavin Clark-Gartner

Great to see the progress. I guess, first, just to clarify on the inventory. So there was upper teens contribution from the CareMed inventory build. How should we expect that to trend next quarter? Like will there be a reversal of a similar amount? Is there some level of inventory build still to happen here? Like what should we think about next quarter?

Babar Ghias

Yes. Thanks for the question, Gavin. So I think your intuition is correct. It's a onetime inventory build that they have to [indiscernible] for dispensing. And our expectation is that, as I mentioned in the call, that typically, they hold 7 to 31 days of -- 7 to 21 days of inventory. And in this quarter, we were on the lower side of that range, and we expect that to remain in the lower side. So you're not going to expect a onetime benefit or, I guess, a recurring benefit of inventory every quarter from this point onwards. There may be in the nearer term, in another quarter, there may be small fluctuations. But as it gets smoothed out, it should reflect patient demand. So to summarize, it was really a onetime benefit of this order of magnitude, and that should be the expectation.

Gavin Clark-Gartner

Okay. Great. That's helpful. And just a quickly quick follow-up. What was the pediatric prescriptions that you saw this quarter?

Charles Gayer

We're not going to get into the specific numbers, Gavin, but it continued to be strong. We announced at the end -- I think it was at the end of July, we had 47. That was well ahead of our expectations for the year. We continue ahead of our expectations. The pediatric revenue we generate is still going to be a very small part of this year's revenue, but it's a good sign for the future. And like I mentioned, we're looking forward to starting to collect real-world evidence in this population. So I think we'll have more to report out in the future as a little more time passes.

Operator

Our next question comes from the line of Laura Chico at Wedbush.

Laura Chico

I guess I wanted to ask one more strategic. Could you expand on your commentary around the 2033 and beyond outlook for the HAE franchise? If I'm looking at the slides correctly, it looks like you're describing it as a range from about $1.4 billion to $2.0 billion in 2033. I think before you talked about this as a $1.8-plus billion opportunity. So I guess help us understand some of the dynamics there that go into the lower and upper bounds. What needs to go right? And I guess, has anything changed to shift your outlook?

Charles Gayer

Thanks, Laura. Yes, the range that we provide is actually based on sell-side analyst views of the portfolio. And we put it out there because directionally, we agree with the two products with the most established oral at that point, the market-leading oral prophy and then what we think will be really a transformative product with every 3- and 6-month dosing with navenibart, it really allows us to hit both big segments of the market, those who prefer oral prophy, those who prefer injectable prophy.

And so the things that need to go right for that to happen, obviously, is continued growth of ORLADEYO, which we expect and all of our forward-looking research metrics tell us that, that will continue. And then a strong launch that we're preparing for, for Navenibart. And what we see in our research with doctors and patients is that, that 3- and 6-month dosing with high efficacy and with a convenient administration is something that gets their attention and will encourage patients to switch. So the real key is going to be how many of the patients who are on other injectable prophies want to take advantage of this longer-term dosing and switch. That will be the key driver of kind of the pace of our revenue growth and kind of the ultimate peak of where we get with Navenibart.

Operator

Our next question comes from the line of Steve Seedhouse at Cantor.

Steven Seedhouse

Sort of related to the long-term revenue projections, but specifically as it pertains to ORLADEYO and just the stickiness of that, have you guys done any updated market research post the Deucrictibant Prophylaxis Phase III result? And do you have a sense if that was above, below or sort of in line with expectations of what you've been modeling really for the past several years?

Charles Gayer

Thanks, Steve. We have not done specific research since the data came out. However, what we did do is our annual large exercise that we do every -- in the middle of every year, where we talk to 175 HAE treaters, 100 patients, 50 payers and run it through a big conjoint exercise and market simulation model. What we put in there for deucrictibant, recall that we always put all of our current and future competition in, with their best case profile. And so the profile that we put in for deucrictibant was actually slightly better than the actual -- what they proved out in the Phase III. So comparable but slightly better.

And it showed that, yes, that product will gain share, as you would expect, but what it's not going to do is take away the base of ORLADEYO patients. And so we continue to see ORLADEYO growing even in the face of future competition in a couple of years from deucrictibant.

Operator

Our next question comes from the line of Stacy Ku at TD Cowen.

Stacy Ku

We have a couple of clarifications. So first, maybe can you talk about your -- given your inventory commentary, I really appreciate all the detailed thoughts there, your level of confidence in the high end of ORLADEYO guidance that you provided for the year. This is just a follow-up on inventory. And then second, on the pediatric launch, is it -- would you characterize it more as steady additions? Or are you seeing a bit of a bolus type of dynamic for the pediatric prescriptions?

As we look to next year and consensus, I know you won't comment, but does that include what you're seeing for the pediatric launch? Or would you look at that as upside? And then third clarification. When we speak to clinicians, they tend to have their patients track very detailed data on their attacks for payer reimbursement access. So as you're looking at the oral competitive landscape in the next couple of years, do you have a good sense of the percentage of patients that are well controlled on ORLADEYO, so having really competitive attack rate reductions as folks try to understand the stickiness of the drug?

Charles Gayer

Great. Thanks, Stacy. Maybe I'll take -- go through the second question with all those pieces, and then we'll come back and Babar can talk about the inventory.

So on the pediatrics, so number one, its -- what we're seeing is kind of steady, strong additions. So there was, of course, a bolus at first, particularly because of the supply delay that we had. So there was a bolus to start. Those patients have been transitioned to therapy. And then we're seeing continued strong demand that is ahead of what our original expectations are. It's still only been a few months. So we'll have to see how long this continues, but the early signs are very promising. As we kind of look to the future of ORLADEYO and when we give guidance for 2027, pediatrics will be absolutely a part of all of that. And then when we have additional data that, as I mentioned, real-world evidence, when we generate other evidence that we think is interesting and helpful, we may comment on other pieces of how pediatrics breaks out of the whole bit.

And then as far as the patients who are well-controlled on ORLADEYO, you've heard us talk before about when patients start, 60% of them roughly get to 12 months of therapy. And then those patients are largely the very well controlled. And so those are the ones who are getting down to a median attack rate. We've put out real-world evidence on this where they're getting down to a median attack rate of about 1/3 of an attack per month and patients generally are reporting significant reductions in attack rates regardless of where they're coming from, whether they switch from other prophy or not previously on-prophy. And so we feel like we're holding on to the patients who are doing well. They're getting market competitive efficacy. So then Babar, do you want to talk about the...

Babar Ghias

Yes. So thanks, Stacy, for the question. Allow me to explain a little bit more perspective on the pharmacy. So look, at the end of the day, the progress of any product should be on patient demand and not inventory dynamics. And as we -- as Charlie mentioned in his remarks that the performance of a pharmacy can actually create a meaningful impact on the progress of these products. And when we made that switch, we were getting to a point of scaling the product that we needed to move with a larger partner who can support the vision for ORLADEYO and potentially Navenibart when it gets approved. So when we think about basically keeping the guidance as we -- at the start of the year, we had a different pharmacy partner. We had a different operating model. It was not necessarily incorporating the dynamics of the inventory.

As I said, what we don't want to do is artificially change the guidance because fundamentally, it should be about patient demand. So we are still in early days of that. So we didn't feel the need that at this point in time, we need to change the guidance for ORLADEYO just because we have a onetime inventory dynamic. And as I said, that once we get past this quarter, as you can appreciate, it's only been 6 weeks that we've actually been like fully with CareMed because the implementation started somewhere in the August time frame. So call it, 6 weeks to 8 weeks. We don't even have a full quarter under the belt. So as we get past through that, it should really reflect the patient demand. So that's why we were being a little bit careful and being conservative in terms of changing guidance at this point in time just because of an inventory dynamic.

Operator

Our next question comes from the line of Tazeen Ahmad with Bank of America.

Jeremiah Lorentz

This is Jeremiah on for Tazeen. I wanted to ask a question on the Netherton readout that's expected around year-end. I'm just wondering what's the benchmark or what level of data you guys are looking for in order to move that program forward? And I guess, depending on if the study [is a hit], how quickly could you move to pivotal stages?

Charles Gayer

Sandeep, do you want to take that?

Sandeep Menon

Yes, I'll take that. Thank you for the question. As you know, Netherton syndrome is a devastating skin disease and that has got multiple other layers of disease manifestation, including immunity and inflammation. We are getting ready for our data readout in December. What we will be presenting is the primary endpoint and the key secondary endpoints along with obviously the safety and tolerability of that. And what good looks like at this point, these patients don't have any treatment options. And given the severity of the disease, any improvement, which is clinically meaningful is still better than what patients don't have at this point.

So we have talked to the patients and the relevant KOLs and also in discussions very soon with the FDA to come up with a benchmark basically. But from our perspective, as long as we are able to get 20% to 25% in the primary IAZ score, I think it should be a pretty good clinically meaningful endpoint based on all the research that we have done internally, talking to the KOLs and obviously, the regulatory discussions are pending as well. So I think we'll put it out there for now. And once the data reads out, we'll share more.

Charles Gayer

Obviously, within that, we'll be looking for kind of consistency of effect as well. And so we'll look at this in a number of different ways. Do you want to just talk about -- what do we think next will be with the [indiscernible].

Sandeep Menon

Yes. The next -- basically, we will be reflecting on the totality of the data. And at this point, we want to do what is best for the program and for the patients. So we are not going to rush into anything that is a pivotal right away. Depending on what the data is, we'll talk to the regulators and digest the data because this is going to be the first-in-class, first therapy ever coming for Netherton. We want to be very clear in terms of where we want to take this. But we will be swift in decision-making, and we will move fast to be -- at the same time, we will be very scientifically rigorous and thoughtful in how we take the clinical development forward.

Operator

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

Maurice Raycroft

I'll shift gears and maybe just ask one on BD. I guess with the new Scientific Advisory Board, can you talk more about their mandate and what your latest thinking is related to BD and the type of assets you're looking to in-license?

Sandeep Menon

Yes. So from our -- thank you for the question, Maurice. So our Scientific Advisory Board, as you can see, it comes with a high pedigree and a very, very successful track record, including with Dr. Tony Manning, who is chairing the SAB. So it is a working Scientific Advisory Board, like Charlie mentioned. It is not that people are coming in to get some luxurious seat on the Board here. They are working with us, as we are -- at least a few times a week. We are rolling our sleeves, looking at multiple assets. As we have shared before, it's going to be early clinical assets, Phase I, Phase II. Mostly we'll go with a very, very clear biology.

We are not going to take chances on things that are far-fetched in terms of an inflection point. And we will ensure that this is sticking to our core strengths because we have a commercial fabric that we are going to take advantage of. So that is what we are going to play. And you will see in the next few months as we have more information, we'll be ready to share. But the punchline here is the SAB is very, very seasoned. They have been there, done that, and we are working together as a team to get to a place where we have a pipeline that is going to be rich and valuable to our stakeholders.

Charles Gayer

And Maury, just to add to that, too, we've, of course, built a very strong internal team for both scientific evaluation and business development. So the internal team is generating the ideas. And then when we get excited about something, the SAB helps us really wrestle it to the ground, -- this is something that we want to do. So we think it's a great partnership. Early days, it's working very well, and we think that this is going to be very supportive to our strategy.

Maurice Raycroft

Got it. That's helpful. And maybe just a quick follow-up on Netherton. As you guys are thinking about next steps with the program. In the press release, you mentioned that expenses were lower related to the program in third quarter. I guess any specific reasons there? Is it just because the study is enrolled and it's kind of at a status quo? Or how should we think about that?

Babar Ghias

Yes. And I think I will point it back to the financial discipline that we have been instituting across the organization, Maury, and basically taking an evidence-based approach. So I'll give you an example, like historically, we may have been a little bit aggressive in our sense that like we need to do CMC campaign before we've actually seen results. I think what I was basically emphasizing earlier was we're taking a very evidence-based and rigorous scientific approach that do we first have a product? Do we have the available data that allows us to unlock the next step of development.

So I think across the organization, we've been instituting that discipline. And in Netherton like I said, that was a perfect example where we didn't feel that we need to spend dollars at risk on things before we actually can unlock the data. So that was basically the primary reason why you see a decline in expense. And I think that is going to be a theme, right? That is how we're going to go about and develop on this innovation aspect that we're talking about BD, but also being very committed to delivering strong profitability going forward.

Sandeep Menon

And Maury, just to add what Barba nicely summarized, we will ensure that we are still very swift. It's not that we are going to just make it so sequential that we will delay the program. And that's not what it is. Basically, we will get some early data and we'll take the right bets that is going to give us scientific and development success.

Operator

The next question comes from the line of Jessica Fye with JPMorgan.

Unknown Analyst

This is Sylvia on for Jeff Fye. To confirm with the prior model pre-CareMed, was there any inventory sitting elsewhere that needs to be worked down that maybe leads to the hesitation to update the ORLADEYO guide?

And as a follow-up, and I appreciate the conservatism so soon after the specialty pharmacy shift. But if the high teens inventory benefit was not contemplated in the fundamental ORLADEYO guidance you laid out and are maintaining, would it be wrong for investors to expect full year ORLADEYO revenue landing in the $650 million to $655 million range? Which is just the midpoint of the fundamental guidance plus $15 million to $20 million.

Charles Gayer

Thanks, Sylvia. First of all, from inventory from the prior pharmacy, the billing model was different there. It was -- there was a flash sale for every pack that went out. So there basically was no inventory. And then with CareMed, it's a little bit more traditional pharmacy model where they buy some inventory. And as Babar described, it's at a relatively low level, about a week's worth of inventory. And now, from the guidance perspective, with everything we see at this point and with the changes that as we're saying, we still haven't worked through completely, we see the range of $625 million to $645 million is still the right guidance for the year.

Operator

Our next question comes from the line of Serge Belanger.

John Todaro

This is John on for Serge today. Just a couple on ORLADEYO. First, curious if you could provide an update on the paid Rx rate exiting the third quarter? And if your outlook has changed on where this could reach considering the efficiencies yielded from the CareMed partnership?

And then second, on the pediatric side of things, you highlighted the launch is progressing ahead of expectations. Just curious how you view the overall opportunity now versus the original 500 patient pool?

Charles Gayer

Thanks, John. As far as the paid rate, just recall, what typically happens in the second half of the year is, our paid rate typically peaks in the second quarter after going through all the reauthorizations in the first part of the year. And then in the second half of the year, it starts to gradually tick down mainly because the rate is driven more by new patients coming on and new patients don't get to the 80-plus percent paid rate that we have in the overall population as quickly. So we're seeing that same dynamic right in line with our expectations. I think we ended the quarter somewhere around 82%, 83% paid.

To your question around CareMed, we do think there -- with their capabilities working with our team, it gives us the opportunity to meet and potentially exceed our long-term goal of getting to 85% paid rate as kind of a steady state. So we think we're on track for that.

I'll note that with pediatrics, too, we're seeing excellent early paid rates. Pediatrics, as you would expect, is basically a commercial plus Medicaid business, so a little bit different dynamic than our overall population. But the early signs are we're getting these kids at a high rate to paid and I expect that to continue. And then as far as the overall opportunity for paed, yes, we're cautiously optimistic that it could end up being bigger than we originally expected. It certainly is turning out to be that this year. I think we need a couple of quarters -- in addition, at least to see what the steady state is in terms of demand. There's still physicians out there who are still learning about this indication and haven't acted yet. And so I think there's likely a lot more opportunity than the 500 diagnosed and treated patients that we've previously described.

Operator

Our next question comes from the line of Jon Wolleben at Citizens.

Catherine Okoukoni

This is Catherine on for Jon. I have a quick clarification question about the number of patient adds. I know you said that the patient adds have been kind of tracking along the average -- from like quarter-by-quarter. I just wanted to provide a little bit more color on how that's tracking and how -- what your expectations are for fourth quarter based on your revenue guidance?

Charles Gayer

Thanks, Catherine. Yes, what we described is that new patient prescriptions coming in has been very much in line. And then the net patient adds in the third quarter were quite healthy. So we expect [ particularly ] with our transition to CareMed with the continued demand with the strong demand in paeds that we would expect another good quarter in the fourth quarter and that this trend will largely continue into 2027.

Operator

Our next question comes from the line of Brian Abrahams at RBC Capital Markets.

Brian Abrahams

I'm curious, are you seeing any effects on ORLADEYO use patterns from the recent launch of oral on-demand or other long-acting injectable therapies for prophylaxis? And then I guess, secondarily, curious what your market research tells you about the potential opportunity differences if Navenibart can demonstrate every 6-month dosing versus every 3 months?

Charles Gayer

Thanks, Brian. We've said before, and at this point, it's been over a year since those -- since the oral acute came on to the market and then also the 2 new prophies that came on last summer. We're really not seeing any impact from the oral acute patients. Some ORLADEYO patients are using it. We're certainly not seeing any negative trends nor would I say it's anything particularly positive. It just hasn't had that much of an impact on our business. The oral -- or sorry, the other longer-acting injectables -- what we're really seeing there is that when a patient ORLADEYO doesn't work out for them, instead of moving preferentially to Takhzyro, more of those patients are going either to Andembry or Dawnzera, that's what you would expect.

And then with Navenibart, what we're seeing, again, in all the research that we've done, 3 months is -- is really a transformative -- every 3-month dosing is really transformative. And then having the ability to have 3-months and 6-months as an option makes the whole package even better. And so we expect to get both doses in our label based on -- certainly based on the Phase II data that we've seen with both doses having very durable reduction in attacks. And so to have that option, we don't know yet which dosage form would be -- which frequency would be most used. I think that's something we'll learn more about. But to have both really makes Navenibart a very strong option for patients and physicians, very really, really intriguing to them.

Operator

This concludes the question-and-answer session. I would now like to turn it back to Charlie Gayer for closing remarks.

Charles Gayer

Thank you. So as we've described here, another really strong quarter for BioCryst as we've gone through a year of transformation, both in leadership, in strategy, in how we run the business. And I'd also like to thank everybody, all our colleagues at BioCryst who've worked so hard during this year of transition. And I think we've set ourselves up for a really great 2027 and beyond. So thank you, everyone.

Operator

Thank you for your participation in today's conference. This does conclude the program. You may now disconnect.