Good day and welcome to the BioCRIST First 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 zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star and then one on a touch-tone phone. To withdraw your question, please press star and then two. 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 BioCris's first quarter 2026 Corporate Update and Financial Results conference call. Participating with me today are President and CEO, Charlie Geyer, Chief R&D Officer, Dr. Sandeep Menon, and Chief Financial Officer, Bob Ruggias. 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 two 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 Biocrist, marked by strong execution on our commercial and development programs, the smooth integration of astrotherapeutics and discipline management of our finances. Orlodeo net revenue of $148.3 million for the quarter was right in line with our expectations, and monthly new patient prescriptions have tracked slightly ahead of 2025 averages. The 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 four product strengths of Orlodeo 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, and 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, and we do not expect this delay to affect our revenue guidance for 2026. We look forward to bringing Orlodeo to the kids who need it as soon as possible. The pediatric indication is exciting, but it does not distract our team from driving new patient demand for Orlodeo 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 Orlodeo as an effective and proven option for HE prophylaxis. Turning to our pipeline development, progress on the pivotal alpha-orbit trial for Novenobart 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 HA prophylaxis. The speed of enrollment is a testament to our team's execution as well as the attractiveness of Novenobart's profile. Our Phase I study for BCX17725 and 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 BioCrist 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 Olideo. It's not just the commercial excellence that drew me to Biocrist, but the commitment to rigorous science to improve patient lives. I see a tremendous opportunity to build on the momentum that Oladeo has delivered as we guide the next wave of programs through the clinic. Navanibot, our injectable plasma calicarane 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. Navanibot 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 three-month and the six-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 or lirio. Moving on to our next program, BCX-17725, which is a KLK-5 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 1 Part 4 will enroll up to 12 patients with three months of dosing, and we expect to report this data by end of this year. Beyond BCX 17725, 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 Bioschrist'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, Babur Ghias.

Babur 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 holiday business, StockBaseCom, and expenses related to the acquisition of Astria. This acquisition was classified as an asset acquisition for accounting purposes, and 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, and 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 Olodeo, 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 Biocrist 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-on-year. Since other revenues include contribution from RappiWeb and licensing revenues, I would draw your attention to the Oladeo 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 Oladeo, as more prescribers get experience with the pediatric formulation and the commercial team continues to reinforce and expand the value proposition of Oladio. 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 Novenibard. To refresh from the last call, we anticipate that 2026 R&D costs will increase over 2025 as we complete the ongoing Phase 3 trial and BLA-enabling CMC activities for Novenibard. 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, and as part of that evaluation during Q1, we decided to discontinue the development of OralStat 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, and while there may be some smaller increases in variable expenses, the current team is well-sized to support continued holiday growth. We anticipate sales and marketing expenses supporting our HAE franchise, Oledeo, and Navinabad upon potential approval as a whole will be very stable and poised to deliver substantial returns. Our GNA 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 Pastria. We will continue to monitor our GNA over the course of 2026 and explore further areas. 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 a 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 BioPress 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-on-year in our interest expense. Earlier this week, we finalized the license agreement with Neofarmet Gentile to commercialize Novenevat 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 up front and up to 275 million in future regulatory and sales milestones along with tiered royalties on net sales ranging from 18 to 30 overall this will increasing meaningfully strengthen our balance sheet in the near to medium term and 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 a 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 Oledeo 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, and 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 a pipeline through both organic innovation and selective, disciplined BD to accelerate our impact in the rare disease space. We are delighted to be sharing these positive business updates with you all today. And, Operator, we are now ready for the questions.

Thank you. We will now begin the question and answer session. To ask a question, you may press star and then 1 on your touchtone phone. If you are using a speakerphone, please pick up your handset before pressing the keys. If at any time your question has been addressed and you would like to withdraw your question, please press star then 2. At this time, we will pause for a moment to assemble our roster. Your first question today will come from Jessica Fai of JPMorgan. Please go ahead.

Hey, this is Abdulan for Jess. Just two questions from us. What are your expectations for the Decorrect-to-Band XR's pivotal re-out, and what profile do you expect? And then what are your latest trends on paid rate in each pair segment?

For Decryctiband, 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, launched to date, really predicted the growth of Orladeo. We always give our future competitors their best profile. So what we assume is Decryctiband will have very good efficacy when they report out in Q3. And that's built into our future forecast, and we still believe that Orlodeo is on a path and will reach a billion dollars in peak sales. And 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, so we're still in that process. We'll comment more at the end of Q2. But we're having really good success converting patients from long-term free product to paid product. So we're progressing as we expected.

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

Hi, guys. This is Jeremiah on for Tazeen. Thanks for taking our question. Maybe a couple questions from us. Just first on the Nevada Bar out-licensing deal, can you maybe just help us understand how much of that, you know, $275 million milestone tool is touted to regulatory versus commercial thresholds and then just where you expect that royalty rate to settle at scale? And then maybe a question on pipeline, especially considering, you know, the deprioritization of OralStat. Just wondering, we haven't heard many updates about the complement pipeline in some time. I'm just wondering what are the next key decision points for bringing those programs forward.

Barbara, you want to take the royalty question? I'll cover the pipeline.

Yeah, so I think at this point in time, we're not necessarily breaking down the milestones and think, you know, we're very excited that Upfront is a very sizable upset for an asset at this stage and naturally in sort of in line with some of the process 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, so we're very confident that that team will do a great job and will realize the maximum benefit of that royalty rate. But 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.

And then as far as the pipeline, you know, number one, what we're focused on right now is the two programs that we have in clinical development, so Novenebart in late stage, and then we're very excited about the potential for 1775, given the need in the Nethertons 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.

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 Orlodeo 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 Don Zera. But what are the impacts you're seeing on Orlodeo retention? Has this changed your call strategy with physicians at all? And then 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.

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, And so what we see is they're competing mainly with the existing market leader, TaxIro. And so it's not affecting Orlodeo. And as Barbara and I both mentioned, our demand is as strong as it's ever been. And we're really pleased, not surprised, but really pleased to see that. And so that's why we have that continued confidence about the future growth of Orlodeo and heading to the billion dollars in peak sales. And, you know, our retention rates, you asked about that, are also in line with, you know, past expectations. Patients who start on Orlodeo, 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. But we're not changing our guidance because the early demand has been very strong. And 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. So 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 Abrams of RBC Capital Markets. Please go ahead.

Hi. Thanks so much for taking our questions. Two from me. I guess first on the Novenovart phase three, it sounds like enrollment's gone really well there. So I'm wondering if you could talk about any learnings there just in terms, 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. And 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 Orlodeo for adults or Nevenobard? And could there be upside to your guidance if this resolves more quickly than expected, just given that you're seeing good launch demand already, and I know pediatric revenue was not really included all that much

in your guidance. Thanks. Sure. Thanks, Brian. Yeah, first of all, we're, you know, Novenibart, we're really excited about the pace of the enrollment, and as I said in my remarks, I think it is a reflection of the appetite that both patients and physicians have for an every three- or six-month 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 three- and six-month dosing is really meaningful and transformative, and so we think there's a potential to switch a lot of patients to this. There's, as we've described before, there's two 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. And the profile of Novenobart is something that's really understandable to both physicians and patients. And I think that's, you know, again, reflected in the trial enrollment. So we're excited about that future launch. As far as the manufacturing issue with Orlodeo 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 just in the specifications. We're digging in to get to the root cause so that we can, you know, manufacture consistent batches going forward. So we'll have an update on that timing when we have it. But we do not expect this to be a long-term problem. And as far as, you know, the demand for the pediatric indication so far, it has been very strong. And so 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, Charlie.

The next question today will come from Steve Seedhouse of Cancer. Please go ahead.

Good morning. Thanks so much for taking the question. wanted to see if you'd be willing to frame your expectations for the phase three Nevenomar data you know obviously most phase twos including this one in HAE smaller than a phase three would be but the attack rate reduction was pretty profound in that phase two so are you expecting like a greater than 90% attack rate reduction are you expecting that in both the every three month and every six months arms and is that what's framing you know pretty pretty aggressive guidance, the $1.8 billion in 2033 sort of implies an even better launch than Orlodeo. So 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. And 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. So that's less than two attacks per year. And those attacks tend to be more mild than other attacks. So that is functionally attack-free for patients. And to be able to get that from three- or six-month dosing, we do expect there to be comparable efficacy between the two. Because remember, the six-month dosing is twice the dose of three months. And so that Phase II Alpha Solar data is very important, really gives us confidence for the future.

The next question will come from Stacey Koo of TD Kalman. Please go ahead.

Hey there. Thanks so much for taking your questions, and welcome to Sandeep. So first, just as we think about Orladeo, notice that you had talked about the continued growth in prescribers, so just curious if you could further elaborate there. That's the first question. And then a quick follow-up on the pediatric product fulfillment. It sounds like you need a quarter or two to kind of get more information, or is it to resolve this is not an issue? So just help us understand if the base case assumption for the potential delay is potentially within the year. That's two. And then 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, Stacey. 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. So, you know, as Barbara 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 peds pellet timing, as I said, we'll know more this quarter. Our expectation is we'll get this resolved pretty quickly, but we don't know the root cause. We haven't identified all the root cause yet, so it'll take a little bit of time, But based on what we see right now, we're on track for the year. And then 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. But the real key is, you know, what's the size of the market? What is the overall opportunity? And we've identified with 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. But having a drug in the market is always a catalyst in rare disease for patient identification. So, you know, the keys for efficacy, no drug in this space has to be perfect. What we'll want to see is that the patients are getting better in their overall, the overall scales of measuring Netherton syndrome. So 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. So a range of things that will help us design a pivotal program that we hope can start next year.

Thank you so much.

As a reminder, if you'd like to ask a question, please press star and then one. Our next question today will come from Mari Raycroft of Jefferies. Please go ahead.

Hi, good morning. Congrats on the progress and 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 and that data update?

Yeah, I think, Maury, we'll have up to 12 patients. That's what we're shooting for, and we want to 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 will give us the data we need to say, do we have a drug here? And so the package will come from that plus the one to three patients that we we'll have from the Part 3 of the study as well as the healthy volunteer data. So 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. And just for digging into prophy competition a bit more, are you hearing about more patients considering switching or switching from Orlodeo? and even if you're not seeing an impact yet, are you getting more pressure or headwinds in capturing new patients or switches from TexIro? I guess it seems like Orlodeo is resilient with these new launches. Are you doing anything differently?

Yeah, we're really not doing anything differently. 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 TaxIro, not Orlodeo. Of course, patients switch from Orlodeo all the time. 60% of them get to a year, 40% don't, and they switch to other products. And 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. But the impact overall on Orlodeo has been very minor.

Okay. Thanks for taking more questions.

The next question will come from John Wollobin of Citizens. Please go ahead.

Hi. This is Catherine on for John. Thank you for the color on the Orlodeo launch. I'm just wondering if you could provide a little bit more commentary on kind of ex-U.S. sales beyond Europe, which guys are seeing there and kind of plans for any expansion, and then your thoughts

about the same for Nevenibart. Thank you. Sure. Thanks, Catherine. With our sale of the European business, Europe had been the large majority of our ex-US sales. So ex-US 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 a billion dollars. We are, though, committed. We want to get our drugs to patients around the world because HA is a global disease, and there are patients who need it, but from a revenue perspective, the great majority will be the U.S. And I'm sorry, the question on, you had a question on Novenivart?

And you think for Nevenobart, you're going to kind of see the same trend if kind of European markets can be the majority of XUS?

We would expect it to be largely the same as Barbara was describing. Our partners at Neopharm Ed Gentile are equally excited about this drug and having both Orlodeo and Nevenobart in the portfolio. There's an important place for both of these products in all the different markets. And so, 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 Yasha on for Gavin.

We were just wondering if on the pediatric pellet side, are you able to proactively work through pair access and reimbursement while resolving the manufacturing? Kind of also wondering how quickly you could convert the demand to paid drug when that's Thank you.

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

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

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