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Good day. Welcome to the BioCryst 1st quarter 2026 earnings conference call. All participants will be in listen-only mode. Should you need assistance, please signal a conference specialist by pressing the Star key followed by 0. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press Star and then 1 on a touch-tone phone. To withdraw your question, please press Star, then 2. Please note this event is being recorded. I would now like to turn the conference over to Nick Wilder. Please go ahead.

Good morning, and welcome to BioCryst's first-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 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.

Thanks, Nick. 2026 is off to a very good start for BioCryst, marked by strong execution on our commercial and development programs, the smooth integration of Astria Therapeutics, disciplined management of our finances. ORLADEYO net revenue of $148.3 million for the quarter was right in line with our expectations. Monthly new patient prescriptions have tracked slightly ahead of 2025 averages. The pediatric launch, pediatric indication launch is just beginning, but early signals confirm the need for oral prophylaxis for kids with HAE. We have received prescriptions for each of the 4 product strengths of ORLADEYO pellets, which demonstrates that even younger children need prophylaxis. We are committed to bringing oral prophy to the many kids who need it.

We have also recently discovered a manufacturing issue that will delay the first product fulfillment. Our team is working closely with our manufacturing partner to identify the root cause. We expect to have more information about the product readiness later this quarter. We do not expect this delay to affect our revenue guidance for 2026. We look forward to bringing ORLADEYO to the kids who need it as soon as possible. The pediatric indication is exciting. It does not distract our team from driving new patient demand for ORLADEYO capsules. We are pleased that new prescription demand for ages 12 and up remains consistent with our quarterly averages since launch, despite new competition. Physicians and patients continue to trust ORLADEYO as an effective and proven option for HAE prophylaxis.

Turning to our pipeline development, progress on the pivotal ALPHA-ORBIT trial for navenibart has exceeded our expectations. Enrollment will be completed by the end of next month, and at approximately 145 enrolled patients, ALPHA-ORBIT will be the largest pivotal trial ever for HAE prophylaxis. The speed of enrollment is a testament to our team's execution as well as the attractiveness of navenibart's profile. Our Phase I study for BCX17725 in Netherton syndrome is also hitting its stride. Investigator enthusiasm has been strong, patients have started dosing, and we are on track to have proof of concept data by the end of the year in this high-need rare disease. I am thrilled that Dr. Sandeep Menon has joined us as Chief R&D Officer last month. We're making great progress in our pipeline, and adding Sandeep makes us stronger.

He has a track record of disciplined and successful drug development, and he shares our vision of building great value through serving rare disease patients. I'll turn it over to Sandeep to introduce himself.

Thank you, Charlie. I'm very excited to be joining BioCryst at this important inflection point in the company's evolution. The company is in a unique position of strength, especially from our strong performance of ORLADEYO. It's not just the commercial excellence that drew me to BioCryst, but the commitment to rigorous science to improve patient lives. I see a tremendous opportunity to build on the momentum that ORLADEYO has delivered as we guide the next wave of programs through the clinic. navenibart, our injectable plasma kallikrein inhibitor for HAE prophylaxis, will complete enrollment in the ongoing pivotal phase III ALPHA-ORBIT study by the end of June this year. This keeps us on track to submit a regulatory filing in the U.S. by the end of next year. navenibart has generated compelling clinical evidence so far.

In the long-term, open-label ALPHA-SOLAR study, it recently demonstrated 92% and 90% mean attack reductions in the 3-month and the 6-month dosing regimens respectively. We now have a great opportunity to bring potentially best-in-class injectable to HAE patients that will complement our best-in-class oral ORLADEYO. Moving on to our next program, BCX17725, which is a KLK5 inhibitor, a potentially first-in-class therapy for Netherton syndrome, a very severe skin condition with no targeted or approved therapies for patients. I'm happy to share that we have started dosing patients in Part 4 of the ongoing phase I study. Part 4 will enroll up to 12 patients with 3 months of dosing. We expect to report this data by end of this year. Beyond BCX17725, we will continue to be disciplined in what we pursue, both internally and externally, focusing on rare diseases which play to our strengths.

I'm excited to have the opportunity to shape the next chapter of BioCryst's growth. We have a proven track record in HAE, exciting assets progressing in the clinic, and now a clear strategy for further expanding our presence in rare disease. I look forward to updating you on our continued progress over the course of the year. I will now transition to my colleague, Babar Ghias.

Thanks, Sandeep. In my remarks today, I will be referring to non-GAAP figures which are adjusted for revenues and expenses related to our European ORLADEYO business, stock-based comp, and expenses related to the acquisition of Astria Therapeutics. This acquisition was classified as an asset acquisition for accounting purposes. As a result, we recorded an in-process R&D charge of $698 million in Q1, as well as some other transaction-specific charges. I encourage you to review our press release for additional details on these adjustments. We believe that non-GAAP figures provide a clearer view of the strength of our business on a forward-looking basis. We encourage you to focus on these metrics in your analysis.

With that said, we are off to a strong start in 2026, with first quarter results reflecting continued momentum in ORLADEYO, further demonstrating the product's durability in an HAE market where there are increasing options for patients. During the quarter, not only did we deliver strong top-line growth, we also posted a strong non-GAAP operating profit. We are pleased with the consistency of our profitability trend that is adding to our strong balance sheet position. Additionally, we have been very pleased with the overall integration of Astria Therapeutics into BioCryst family. This process has been running ahead of our expectations. This demonstrates that our team can, in the future, not only do transformational BD, but also integrate seamlessly. To shed more light on our financial figures, our non-GAAP Q1 2026 total revenue increased approximately 17% year-over-year.

Other revenues include contribution from RAPIVAB and licensing revenues. I would draw your attention to the ORLADEYO revenues of $148 million, which increased 21% year-on-year, excluding the European divestiture impact. New prescription demand is strong, and we continue to see growth in prescribers even in the sixth year of launch. We remain very excited about the future growth prospects for ORLADEYO as more prescribers get experience with the pediatric formulation and the commercial team continues to reinforce and expand the value proposition of ORLADEYO. In Q1, our non-GAAP operating profit came in at $54 million, an increase of 25% year-on-year, further demonstrating our ability to convert strong top-line growth to bottom-line growth. R&D costs increased in Q1 2026 versus the prior year, as you would expect, since we are now consolidating the costs related to navenibart.

To refresh from the last call, we anticipate that 2026 R&D costs will increase over 2025 as we complete the ongoing phase III trial and BLA-enabling CMC activities for navenibart. With the addition of Sandeep to our team, we will continue to refine a focused and disciplined R&D strategy to enhance our portfolio. To that end, we will continue to evaluate internal programs that do not make viable business sense. As part of that evaluation during Q1, we decided to discontinue the development of avoralstat in DME. Our sales and marketing expenses for the quarter were $37 million on a non-GAAP basis, down slightly from Q1 2025. We are very pleased that our sales and marketing structure is at steady state. While there may be some smaller increases in variable expenses, the current team is well sized to support continued ORLADEYO growth.

We anticipate sales and marketing expenses supporting our HAE franchise, ORLADEYO, and navenibart upon potential approval, as a whole will be very stable and poised to deliver substantial returns. Our G&A expense increased slightly by $1.8 million over Q1 2025 on a non-GAAP basis. This increase was primarily driven due to incremental overhead with the closing of Astria. We will continue to monitor our G&A over the course of 2026 and explore further areas of efficiencies. Driven by our strong operational results, we ended the first quarter with substantial liquidity of approximately $261 million in cash and investments, despite funding part of the Astria purchase price from our balance sheet. During the quarter, we also closed on a senior credit facility of $400 million to fund the remaining cash portion of Astria acquisition, along with approximately 37 million BioCryst shares issued to Astria shareholders.

Our cost of capital decreased compared to last year due to our strong financial position, and you will notice the benefit year-over-year in our interest expense. Earlier this week, we finalized a license agreement with Neopharmed Gentili to commercialize navenibart in Europe. We are very pleased to be collaborating with NG on this transaction, a team that carries a unified vision to benefit people living with HAE and a commercial team that we carefully developed over many years before transitioning it to NG as part of our European divestiture last year. As part of this deal, we will receive $70 million in cash upfront and up to $275 million in future regulatory and sales milestones, along with tiered royalties on net sales ranging from 18%-30% overall.

This will meaningfully strengthen our balance sheet in the near to medium term, naturally, the downstream economics will be very attractive to our overall financial profile. On a pro forma basis, including the net proceeds from the license agreement, our liquidity position stands at total of $331 million as of March 31, 2026. This attractive position allows us to evaluate a wide range of capital allocation strategies to maximize value to our shareholders. Moving on quickly to guidance. As Charlie mentioned, we are maintaining expectations for full year 2026 ORLADEYO revenues to be between $625 million and $645 million. We expect full year 2026 non-GAAP OPEX to be between $450 million and $470 million. We continue to execute well, as the year progresses, we will guide the street of any changes.

In closing, we have entered 2026 with strong momentum and are pleased to see strong trends in early Q2. Our goal is to keep driving top and bottom-line growth, advancing our pipeline through both organic innovation and selective discipline BD to accelerate our impact in the rare disease space. We are delighted to be sharing these positive business updates with you all today. Operator, we are now ready for the questions.

Thank you. We will now begin the question-and-answer session. Your first question today will come from Jessica Fye of JPMorgan. Please go ahead.

Hey, this is Abdullah for Jess. Just two questions from us. What are your expectations for the Duk Orterband XR's pivotal readout, and what profile do you expect? What are your latest trends on paid rate in each para segment? Thank you.

Sure. For DAWNZERA, as we've said before, when we do a lot of work to try to predict the future. We have forecasting models that have proven to be really accurate, launch to date, really predicted the growth of ORLADEYO. We always give our future competitors their best profile. What we assume is DAWNZERA will have very good efficacy when they report out in Q3, and that's built into our future forecast. We still believe that ORLADEYO is on a path and will reach $1 billion in peak sales. As far as the trends in the paid rate, this year, we're still in the blizzard season, where we go through all the reauthorization.

Every year, it moves a little bit into the future as patients go through this process. We're still in that process. We'll comment more at the end of Q2. We're having really good success converting patients from long-term free product to paid product. We're progressing as we expected.

Thank you.

Your next question will come from Tazeen Ahmad of Bank of America. Please go ahead.

Hi, guys. This is Jeremiah on for Tazeen. Thanks for taking our question. Maybe a couple of questions from us. First on the navenibart out-licensing deal. Can you maybe just help us understand how much of that, you know, $225 million milestone pools is tied to regulatory versus commercial thresholds? Just where you expect that royalty rate to settle at scale. Maybe a question on pipeline, especially considering, you know, the deprioritization of avoralstat. Just wondering, we haven't heard many updates about the complement pipeline in some time. Just wondering what are the next key decision points for bringing those programs forward. Thanks.

Babar Ghias, you wanna take the royalty question? I'll cover the pipeline.

I think, at this point in time, we're not necessarily breaking down the milestones and think, you know, we're very excited that the upfront is a very sizable upfront for an asset at this stage and naturally in sort of in line with some of the past licensing deals at this stage. You know, as you can see that we feel very, very confident of that healthy royalty rate because as I mentioned, it's a team that we actually developed and curated over many years. We're very confident that that team will do a great job, and we'll realize the maximum benefit of that royalty rates.

You can imagine that, a typical deal on the back end in terms of, things, but something that we feel really confident to realize over the course of time.

As far as the pipeline, you know, number 1, what we're focused on right now is the two programs that we have in clinical development, navenibart, in late stage, and then we're very excited about the potential for 1775, given the need in the Netherton syndrome population. With Sandeep on board, we will be making future updates on our pipeline and our rare disease strategy, and we look forward to doing that as soon as we can.

Great. Thanks.

Your next question today will come from Laura Chico with Wedbush Securities. Please go ahead.

Good morning. Thanks very much for taking the question. I guess back to ORLADEYO for one. Charlie, I'm not sure if you can share any feedback here about the impact of some competing launches right now. I guess I'm thinking about DAWNZERA. What are the impacts you're seeing on ORLADEYO retention? Has this changed your call strategy with physicians at all? Just a follow-up, with respect to the manufacturing issue. I think I missed it, but any impacts or could you just expand a little bit more with implications on the pediatric launch? Thanks very much.

Sure. Yeah, Laura, as far as the competing launches, what we're seeing is what we expected, and this kind of goes back again to the forecasting we do. The recent launches, as you know, have been injectables. What we see is they're competing mainly with the existing market leader, TAKHZYRO. It's not affecting ORLADEYO. As Babar Ghias and I both mentioned, you know, our demand is as strong as it's ever been, and we're really pleased. Not surprised, but really pleased to see that. That's why we have that continued confidence about the future growth of ORLADEYO and heading to the $1 billion in peak sales. Our, you know, our retention rates, you asked about that, are also in line with, you know, past expectations.

Patients who start on ORLADEYO, 60% of them get to a year and then very slow, or very sticky after that. Very strong retention after a year. On the peds manufacturing, as we said, we're not changing our guidance. We'll have more information on the issue and timing hopefully later this quarter. We're not changing our guidance because the early demand has been very strong. You may recall from past calls, in our forecast for this year, we were relatively conservative about the peds opportunity because we didn't know how quickly it would kick in. We're very bullish on the long term, and we're on track for this year.

Thanks very much.

The next question will come from Brian Abrahams of RBC Capital Markets. Please go ahead.

Hi. Thanks so much for taking our questions. Two from me. I guess first on the navenibart phase III, it sounds like enrollment's gone really well there. I'm wondering if you could talk about any learnings there, just from the enrollment conduct, just in terms of the appetite for long-acting injectables, the types of patients who could go on this therapy commercially, and the potential size of that opportunity. Then just secondarily following up on the manufacturing issue, can you just give us a sense of what would need to be done to resolve this? Might this require any sort of FDA re-inspection? Is there any overlap at all with this facility with regards to ORLADEYO for adults or navenibart?

Could there be upside to your guidance if this resolves more quickly than expected, just given that you're seeing good launch demand already? I know pediatric revenue was not really included all that much in your guidance. Thanks.

Sure thing. Thanks, Brian. Yeah, first of all, you know, navenibart, we're really excited about the pace of the enrollment. As I said in my remarks, I think it is a reflection of the appetite that both patients and physicians have for an every 3 or 6 months dosing. You know, this is why we wanted to acquire this product in the first place, because we saw that, we heard that from customers. Getting to 3 and 6-month dosing is really meaningful and transformative. We think there's a potential to switch a lot of patients to this. As we've described before, there's 2 segments in this market. There are those patients who want oral therapy and do well on it, and then there's those who are on injectable therapy and are very comfortable with that.

The profile of navenibart is something that's really understandable to both physicians and patients, and I think that's, you know, again, reflected in the trial enrollment. We're excited about that future launch. As far as the manufacturing issue with ORLADEYO pellets, first of all, it's a different plant. It's a different manufacturer than capsules, so it has nothing to do with capsules. It's not an FDA issue at all. It's not a safety issue. It's just a batch problem in the specifications. We're digging in to get to the root cause so that we can, you know, manufacture consistent batches going forward. We'll have an update on that timing when we have it. We do not expect this to be a long-term problem.

As far as, you know, the demand for the pediatric indication, so far it has been very strong. If we get this issue, we get the product out there in the relatively near future, we're right on track for this year and certainly very much on track for our long-term predictions.

Thanks.

The next question today will come from Steven Seedhouse of Cantor. Please go ahead.

Yeah, great. Good morning. Thanks so much for taking the question. Wanted to see if you'd be willing to frame your expectations for the phase III navenibart data. You know, obviously most phase IIs, including this one, in HAE smaller than a phase III would be, but the attack rate reduction was pretty profound in that phase II. Are you expecting like a greater than 90% attack rate reduction? Are you expecting that in both the every 3 month and the every 6 months arms? Is that what's framing, you know, pretty aggressive guidance? The $1.8 billion in 2033 sort of implies an even better launch than ORLADEYO. I was hoping you could just frame what you're expecting from the data. Thanks.

We are expecting really good results, whether it's 90%, a little over 90%, a little less. I don't think that really matters so much. What matters is the attack rate that patients get down to. In the open label study, as you've seen, patients in both doses are getting down to a mean attack rate of just 0.16 per month. That's less than 2 attacks per year. Those attacks tend to be more mild than other attacks. That is functionally attack free for patients. To be able to get that from 3 or 6-month dosing, we do expect there to be comparable efficacy between the two. Because remember, the 6-month dosing is twice the dose of 3 months.

That phase II ALPHA-SOLAR data is very important, really gives us confidence for the future.

The next question will come from Stacy Ku of TD Cowen. Please go ahead.

Hey there. Thanks so much for taking our questions, and welcome to Sandeep. First, just as we think about ORLADEYO, noticed that you had talked about the continued growth in prescribers. Just curious if you could further elaborate there. That's the first question. A quick follow-up on the pediatric product fulfillment. Sounds like you need a quarter or 2 to kind of get more information or is it to resolve the fulfillment issue? Just help us understand if the base case assumption for the potential delay is potentially within the year. That's 2.

Three, as you started to dose Netherton patients, just, maybe talk a little bit more about what you're learning about the patient population, diagnosis awareness, potential size of the market, and remind us your go and no-go decision on efficacy. Thanks so much.

Great. Thanks, Stacy. I'll see if I can get all these. In the growth of prescribers, you've heard us talking before in 2025, around 60 new prescribers per month, give or take. We're in that same range in Q1. You know, as Babar Ghias said, in year six, to still be adding prescribers, to still be getting the prescriptions at the same rate is really a reflection of this product profile and how our teams launched it. As far as the pedes pellet timing, you know, as I said, we'll know more this quarter. Our expectation is we'll get this resolved pretty quickly. You know, we don't know the root cause. We haven't identified all the root cause yet.

It'll take a little bit of time, but based on what we see right now, we're on track for the year. As far as Netherton patients, we've done some work in the epi of this disease because that's really key. We know it's a very serious disease. We've met patients. We know how much they suffer from this. The real key is, you know, what's the size of the market? What is the overall opportunity? We've a high level of confidence that the U.S. market is 3,000 plus patients at this point. Those patients need to be correctly diagnosed. They need to be identified. Having a drug in the market is always a catalyst in rare disease for patient identification.

The, you know, the keys for efficacy, no drug in this space has to be perfect. What we'll wanna see is that, you know, the patients are getting better in their overall scales of measuring Netherton syndrome. Ichthyosis scales measured by the physicians and the patients. We'll be looking at itch. We'll be looking at, you know, does the drug continue to get to the epidermis the way that we've seen in healthy volunteers? A range of things that'll help us design a pivotal program that we hope can start next year.

Thank you so much.

Our next question today will come from Maury Raycroft of Jefferies. Please go ahead.

Hi, good morning. Congrats on the progress, thanks for taking my questions. Maybe just a quick follow-up on Netherton. Can you just talk more about how much data we should expect by year-end in that data update?

Yeah. I think, Maury, you know, we'll have up to 12 patients. That's what we're shooting for, and we wanna release the data that we have all together because, you know, it's a rare disease. That's not a huge number of patients, but we think it'll give us the data we need to say, "Do we have a drug here?" The package will come from that, plus the 1-3 patients that we will have from the part 3 of the study, as well as the healthy volunteer data. We'll, you know, as the data comes in, we'll figure out what the package is at the end of the year, but it'll be based on those roughly 12 patients.

Got it. Okay. Just for digging into Prophy competition a bit more, are you hearing about more patients considering switching or switching from ORLADEYO? Even if you're not seeing an impact yet, are you getting more pressure or headwinds in capturing new patients or switches from TAKHZYRO? I guess it seems like ORLADEYO is resilient with these new launches. Are you doing anything differently?

Yeah. We're really not doing anything differently. You know, we don't need to add to our sales force. We don't need to change our customers. We're already calling on the list of HAE prescribers and expanding that list, as we've talked about. We see most of the switching, most of the market impact hitting TAKHZYRO, not ORLADEYO. Of course, patients switch from ORLADEYO all the time. 60% of them get to a year, 40% don't, and they switch to other products. What we're seeing more is those patients, when they do switch, they're more likely to switch to one of the new products, which makes a lot of sense. The impact overall on ORLADEYO has been very minor.

Got it. Okay. Thanks for taking my questions.

The next question will come from Jonathan Wolleben of Citizens. Please go ahead.

Hi. This is Catherine on for John. Thank you for the color on the ORLADEYO launch. Just wondering, if you could provide a little bit more commentary on kind of ex U.S. sales beyond Europe, what you guys are seeing there and kind of plans for any expansion and then your thoughts about same for navenibart. Thank you.

Sure. Thanks, Catherine. With our sale of the European business, Europe had been the large majority of our ex-U.S. sales. Ex-U.S. is still an important contributor towards our peak, but we are the vast majority, well over 90% of our revenue now comes from the U.S., and we expect it to be that way even as the brand grows towards $1 billion. We are, though, committed. We wanna get our drugs to patients around the world because HAE is a global disease, and there are patients who need it. From a revenue perspective, the great majority will be the U.S. I'm sorry, you had a question on navenibart?

Do you think for navenibart you're gonna kind of see the same trends if?

Yeah.

European markets in the majority of ex-U.S.?

We would expect it to be largely the same as Babar Ghias was describing. You know, our partners at Neopharmed Gentili are equally excited about this drug and having both ORLADEYO and navenibart in the portfolio. There's an important place for both of these products in all the different markets. We're excited about that opportunity.

Thank you so much.

The next question will come from Gavin Clark-Gartner of Evercore ISI. Please go ahead.

Hi, this is Yesha on for Gavin. We were just wondering if on the pediatric patient side, are you able to proactively work through prior auth and reimbursement while resolving the manufacturing? Kind of also wondering how quickly you could convert the demand to paid drug when that's resolved? Thank you.

Yeah, thanks for the question. Absolutely. We've been doing that already, and we'll continue to do it. The ORLADEYO pellets slot right into our existing contracts for ORLADEYO capsules, so it's really not a separate market access initiative. Our patient services team will help patients and healthcare providers work through that process. You know, that could create patients moving more quickly to paid therapy once we do have the product supply.

At this time, we will conclude our question and answer session. I'd like to turn the conference back over to Charlie Gayer for any closing remarks.

Thank you. Again, as Babar Ghias and Sandeep Menon and I have described today, 2026 is off to a really strong start for BioCryst. I'm very happy with the team we have in place here, the way that we're executing on our commercial and development programs. I believe that the best is yet to come for BioCryst. Thank you for your interest and have a great day, everyone.

The conference is now concluded. We thank you for attending today's presentation, and you may now disconnect your lines.