Good day and welcome to the BioCryst Pharmaceuticals conference call. All participants will be in a 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 then one on a touchtone phone. To withdraw your questions, please press star then two. Please note this event is being recorded. I would now like to turn the conference over to Nick Wilder with BioCryst. Please go ahead.
Good morning and welcome to BioCryst's conference call to discuss its proposed acquisition of Astria Therapeutics.
Participating with me today are CEO Jon.
Stonehouse, President and Chief Commercial Officer Charlie Gayer, 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 conference call will contain forward-looking statements, including statements related to the proposed.
Transaction, including financial estimates and statements.
To the expected timing, completion, and effects of the transaction. 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 slides.
Two and three of the presentation.
I'd now like to turn the call.
Thanks, Nick. We are very excited to announce the acquisition of Astria Therapeutics today. This strategic move will be the source of the next product to market for BioCryst Pharmaceuticals that supports our strategy in several ways. Let me start by reminding you of the growth pillars of our strategy so you understand where the deal fits. The first pillar is we have a highly profitable, growing product in ORLADEYO that is on its way to $1 billion at peak around the end of the decade and intellectual property protection out to 2040. ORLADEYO is the source of double-digit revenue growth for the next few years before we bring other products to market. The other two pillars are intended to answer what comes after ORLADEYO. The sources for this are discovery engine and business development.
In both cases, we're focused on rare disease assets as we built a highly effective commercial engine and supporting infrastructure to drive ORLADEYO growth. This will be leveraged to support and drive growth and profit for future rare disease products. The next products to fulfill this strategy are from our discovery team. We are currently developing BCX17725, a promising early-stage compound to address patients living with Netherton Syndrome. Today, with the announcement of the acquisition of Astria Therapeutics, we are adding a late-stage compound for patients with hereditary angioedema (HAE), Nevenibart, to our pipeline. We believe Nevenibart is a perfect fit next product for our company. Charlie Gayer and Babar Ghias will explain why. With that, I'll pass the call to Charlie.
Thanks, John. We have watched Astria with great interest for several years because we knew they were developing something special. This is a very exciting day for us. Beyond what this transaction means for BioCryst, we believe it will mean even more for patients. BioCryst started engaging with the HAE community over a decade ago when we began our first oral therapy development program. What we learned then, and have continued to learn over the many years, is how important it is for patients to have treatment options that meet their needs. With ORLADEYO, it was the promise of a safe and effective targeted oral therapy to prevent attacks. We are grateful to the patient community because we would not have succeeded without their support and encouragement. ORLADEYO continues to grow.
As expected, we had another strong quarter for demand in Q3, in line with what we have seen over the past two years with no impact from new competition. This tells us what we already know from our deep market insight work. Patients only switch therapy if another option satisfies an unmet need in a meaningful way. We believe Nevenibart is a potentially transformative therapy that will meet the needs of many patients, particularly those who are already on injectable prophylaxis therapy. That's about 5,000 HAE patients in the United States alone. The potential for Nevenibart to be transformative is especially true for patients on Takhzyro, the current market leader. Takhzyro is a very effective drug and Takhzyro and ORLADEYO have established kallikrein inhibition as the standard for HAE prophylaxis.
Roughly 70% to 80% of patients on Takhzyro require injections every two weeks, based on real world data presented by the manufacturer. A sizable proportion of patients are willing to treat that way despite a high rate of injection pain because the treatment controls their attacks. If they could control their attacks with similar efficacy with an injection that doesn't hurt and do it two to four times per year instead of 24 or even 12 times per year, that would be significant. Low dosing frequency is the main remaining unmet need identified by HAE patients and their physicians and they see every three month dosing as the tipping point that would motivate a switch. That is the potential of Nevenibart. The opportunity to bring this potentially best in class product to patients is incredibly exciting for us.
We believe our team at BioCryst is second to none in terms of commercial execution in the rare disease and HAE space. Our team has deep HAE experience. We have the ability to use real-time customer data. We have the ability to market directly to thousands of HAE patients and HAE treaters. This experience and expertise will allow us to accelerate the launch curve for Nevenibart and help the greatest number of patients. Even as ORLADEYO reaches and maintains peak revenue of $1 billion with the two products together, we believe our HAE portfolio could drive double-digit annual revenue growth that will reach at least $1.8 billion by 2033. I'll turn it over to Babar to describe this transaction in more detail and how it will be transformative for BioCryst.
Thanks, Charlie. On our last earnings call, right after we announced the sale of our European business, we emphasized the role of BD as one of our key growth pillars, which Jon described earlier. We shared that this growth could come from both HAE and non-HAE opportunities, as our plan is to be a rare disease consolidator. We said that the first deal needs to make very clear strategic sense, and while finding the right opportunity can take some time, I am thrilled that our first transaction checks all the strategic boxes and timing could not be more perfect. ORLADEYO continues to deliver strong performance, and with a steady-state cost structure, every dollar of growth is now contributing significantly to our operating profitability and cash flow generation.
With the addition of Nevenibart to our portfolio upon closing of the transaction, we will be poised to deliver double-digit growth upon potential approval and, more impressively, on a significantly larger revenue base. We expect very minimal incremental SG&A investment required to commercialize Nevenibart, as we have put in place already one of the best commercial engines in the rare disease space. That low investment combined with accelerated revenue is expected to supercharge our earnings growth well into the next decade. Let me comment on the nearer-term financial impact of the acquisition. We have shared with you that with the closing of the sale of our European business, our cost structure will be simplified and our operating profitability on our standalone basis is expected to significantly improve. Even after absorbing the R&D development spend of Nevenibart, we will remain highly profitable on a non-GAAP basis and cash flow positive.
We expect the first full year of revenue after Nevenibart's anticipated launch will be significantly accretive to operating profit. We will guide to the relevant combined numbers post-closing of the transaction, which is expected in Q1 2026. We will be financing this acquisition with a mix of BioCryst's equity issued to Astria shareholders to enable them to participate in the upside here and the remainder with cash. With the sale of our European business, we have retired the remaining Pharmakon debt of approximately $200 million. To finance this new acquisition, we have entered into a strategic financing partnership with Blackstone at a highly attractive cost of capital, with access to up to $400 million of cash for this transaction. Due to our continued growth behind ORLADEYO and improving cash flow profile, we anticipate to achieve over $1 billion of cash on hand by 2029.
Our plan is to continue our strategy of becoming a rare disease consolidator and the partnership with Blackstone provides us with further access to capital. This acquisition timing is perfect as we are hitting so many critical milestones for the business, most importantly, I'm excited about what this means for the patients. We are uniquely positioned to continue the growth of ORLADEYO and accelerate the vision of Nevenibart meeting the needs of patients with multiple therapeutic options. We will now open the call for Q and A.
We will now begin the question and answer session. To ask a question, you may press star 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 momentarily to assemble our roster. The first question today comes from Jessica Fye with JP Morgan. Please go ahead, Jessica. Your line is open, you may ask your question.
Operator, you can go to the next question.
The next question comes from Stacy Ku with TD Cowen. Please go ahead. Hi, congratulations on this announcement and thanks so much for taking your questions. First, just curious because we've been getting some investor feedback and questions. There is some precedence with Shire's acquisition of Dyax. Just comment, maybe high level, your level of confidence, what you all are putting together, maybe the differences between oral and injectable and the market space and let's say separation there as we think about the Q1 2026 closing. That's the first question. When it comes to the competitive landscape, can you just maybe talk about what work the team did when it comes to both public and private companies in the hereditary angioedema (HAE) prophylaxis space and how you landed on Nevenibart? That's the second, especially as we think about maybe some of the private companies that are coming along.
The last question is maybe on the Nevenibart development, do you expect early 2027? We understand this is still early days, but just given your relationships and assuming Q1 2026 closing, do you expect early 2027 will be conservative timing for results? Thank you so much.
Let me take the first one, then Charlie, you can take the next two. The first one regarding precedent in the space of companies having more than one product like Takeda now, there's more than one, CSL as well. There's definitely tons of precedent and there's tons of competitors in the space. We think, and Charlie said it in his prepared remarks, these complement each other really well. There's plenty of patients that still seek injectables that we think are an opportunity for us.
Charlie. Thanks, Jon.
Yeah.
What I'll add to the oral versus injectable as well is that patients tend to have a very clear preference based on where they are. Brand new patients to prophylaxis really prefer oral. Patients who are already doing well on an injectable would prefer to have an injectable that even better meets their needs, specifically with dosing. That's where we think that this makes a lot of sense as far as the landscape for competition. As I mentioned in my prepared remarks, the real tipping point for patients on injectable therapy today that would make them switch is every three month dosage or better. We think that Nevenibart with a known profile with that potential for three to even six month dosing, and the fact that they're off to a good start with their clinical program, gives this product a first mover advantage.
As we've looked at other products, we think that Nevenibart is likely to be the first one to launch with that profile. Sorry, Stacy, I forgot your.
Yeah, the last one was top line data in early 2020.
We think that again, Astria's got a very strong team, they're doing all the right things. Early 2027 data will be great. Of course, when we're able to complete this transaction, we have a lot of experience as well and we'll do whatever we can to keep that timeline or improve it if we can.
Stacy, the last thing that I'd say on your second question is this is a sticky market, as you very well know. That first mover advantage, if you've got patients that are well controlled with really convenient dosing, they're not going to switch to something that has similar dosing.
Okay, wonderful. Congratulations. We've obviously done a lot of work in the HAE space and also really like Astria. Thanks.
Great, thank you.
The next question comes from Laura Chico with Wedbush Securities. Please go ahead.
Good morning.
Thanks very much for the question and congrats on the deal. I'm not sure who to direct the question to, Jon or Babar, but could you talk a little bit more about the downstream revenue opportunity for Nevenibart and kind of how you're building your assumptions there and the market opportunity and just kind of the overall peak assumptions. Secondly, could you share any color on whether this was a competitive process? Thank you.
I think on the revenue as we have shared with you, ORLADEYO continues its trend to be a billion dollar product. Naturally, we have a very high level of confidence on the Nevenibart product. At this point we're not sharing any projections. What I can share with you very confidently is beyond Nevenibart starts to hit our profile right when the growth for ORLADEYO comes to a steady state. That enables us to basically build onto that double digit growth and well into the next decade. I think we are very confident that with the machinery that we have put in place, we will absolutely deliver on that double digit growth.
On the second question around competitive process, the proxy will go out from Astria Therapeutics and you'll know the details of the process.
Thanks very much, guys. Congrats.
Thank you.
The next question comes from Steven Seedhouse with Cantor. Please go ahead.
Good morning. Thanks for taking the question. Congratulations on the proposed deal. Two questions. One is, I guess I'm interested or maybe even surprised that you're not planning to develop the OX40 antibody that you're getting from Astria here as well. Is that because it doesn't fit in the sort of orphan disease strategic focus, or is there something else to say there? Also, would ask you to sort of expand your comments on just appetite for additional deals after this. Where does this leave you in terms of what you're thinking about in terms of being acquisitive going forward?
Yeah, so I'll take the first one, Babar will take the second on OX40. You know, we think it's a really interesting product but it's not rare disease and it's better in the hands of somebody that's in that area. That's the reason. That's the rationale. You're correct, Robert.
Yeah. I think we've done a very transformative major acquisition. Our first plan is to make sure that we make the priority and integrate this acquisition well. Naturally speaking, ORLADEYO will continue to put points on the board. As we said, while we anticipate that by 2029 we'll have a $1 billion cash balance, we are not going to sit on that. Our plan, once we've integrated, once we've made sure Nevenibart is on its way to potential enrollment success as it continues to do, we will be looking at other opportunities as well.
Yeah, I think, you know, listen, I've been in BioCryst for almost 19 years. This team just showed you that they have an appetite for this and will continue to do this.
The next question comes from Brian Abrahams with RBC Capital Markets. Please go ahead.
Hey guys, good morning. Thanks for taking my questions and congrats on the deal. Just commercially speaking, what are some of the different considerations to take into account for marketing a long-acting injectable in the space? It sounds like there's going to be limited additional SG&A spend, but I'm just curious how that might adjust and where you might be making the commercial investments. Then just secondarily, it sounds like most patients are on Takhzyro every two weeks and every three-month dosing could be a tipping point. HAE also can be a sticky market. I guess I'm curious what your market research tells you could be the potential conversion of patients from current to future long-acting injectables and maybe the types of patients who would switch. Thanks, Charlie.
You want to take those?
Sure, Brian. As far as the different considerations, clearly as we've laid out, patients have strong preferences one way or another based on where they are in the market. That'll be a big part of our marketing campaign. I think another really important part is getting patients access to therapy. We've built what we think is also a second to none program with helping patients with patient services and market access. We've done a great job with ORLADEYO and we would expect to do a great job with Nevenibart given our services and the profile of the product. As far as additional investments we have, we continue to believe we have the sales force that is the right size out there. We've got a great marketing team. There'll be incremental investments as we continue to grow with ORLADEYO on the patient services and market access side.
There really don't need to be huge investments. One thing we do also very well is develop real world evidence around our product and we would expect to do that in the future with Nevenibart. It's been an important part of our ORLADEYO story. It would be an important part of Nevenibart as well in the future.
I mean, here's a simple exercise for those of you that cover Astria as well. You take our model with the growth of SG&A that you have planned, and you take the revenue from Nevenibart, and you take the development cost for Nevenibart, and that's it. Run that model. Thanks.
Yep.
The next question comes from Jonathan Wolleben with Citizens. Please go ahead.
Hey, good morning guys, and congrats on the transaction. Wondering if you could talk a little bit about the confidence you have around Astria's early data set being relatively small and open label, and what you anticipate to see in a phase three profile. If you think these impressive efficacy rates will stand up, or if this is going to be the longer duration driving adoption, or if it's going to be a competitive profile. Just if you could talk a little bit about what you want to see in a target product profile coming out of a phase three trial.
Yeah, thanks, John. We're very impressed with the early data from Astria. If you recall going back to Takhzyro in the early days, Takhzyro also showed very impressive early data in a phase 1b study. There's a real analog to what Astria shows. The difference being, of course, nevenibart has the three to six month dosing and the greater than 90% attack reduction across both doses is very impressive to us. Where we think the need for efficacy in the market is largely met across the board for some patients, if they don't get efficacy on one product, they have other options, which is great for patients and they move. The market isn't looking for more efficacy. What it's looking for is less burdensome dosing. That's what nevenibart has the potential to provide with every three to six month dosing.
What we've seen in our market research, again, is that that three month profile with very low to no injection site pain is really something that gets patients' attention and we think is a tipping point to what will make them switch.
Charlie, the projections you guys gave out today, is that assuming just that every three months works or that the six month works as well, or is that an upside scenario to what you guys are thinking about?
No, it's really a blend of both. Three months alone is the really significant thing. Months is just icing on the cake. Got it.
All right, thanks, Charlie.
The next question comes from Gina Wang with Barclays. Please go ahead. Thank you for taking my questions. Also, congrats on the deal. Maybe two questions regarding. One is what will be additional clinical trial cost before the data readout in early 2027. The second, when I look at the Phase 3 trial design, they do have a 3COH and there was a placebo. Total trial size is relatively small. Maybe if you can share a little bit thoughts on the trial design. Is that any cohort positive or proved three cohorts versus placebo to be positive and that will claim positive outcomes. If you can share a little bit more color on the alpha or clinical trial design and maybe some assumption behind it.
Babar, you want to take the first one on the costs, and Charlie, you take the trial design.
Yeah. Gina, thanks for the question. We have not, we will not be naturally giving guidance as to the projections of the spend right now because the transaction is still pending approvals. Once the transaction closes, we will be back and revise the operating guidance for 2026 when we are ready to do so. Having said that, as I said before as well, with the divestiture of the European business, our standalone business is going to deliver strong operating profit growth. Even when we add the development spend, when we will guide you at the right time, you'll see our profitability will remain quite strong. Maybe I'll hand it to Charlie for the second question.
Sure.
We think that the Astria Therapeutics team's been really smart in how they've designed this trial, given the precedence in the hereditary angioedema (HAE) space, but also given the newness of every three to six month dosing. Based on the likely efficacy of this product, we think that all three cohorts are likely to show statistical significance. The key is just getting them all enrolled, and we think that the Astria Therapeutics team's off to a really good start on this.
Thank you. As a reminder, if you would like to join the question queue, please press star then one. The next question comes from Maury Raycroft with Jefferies. Please go ahead.
Hi, good morning. Congrats on the update and thanks for taking my question. Maybe just a follow up.
How you're thinking about the $1.8 billion assumptions there.
In the past you showed a Monte.
Carlo simulation with some projections for Nevenibart.
Apart, and it seems higher today. What's changed since then for your projections, and how do you view?
The future breakdown in market share for.
Both ORLADEYO and Nevenibart?
Yeah. Just to clarify, we are not giving projections on Nevenibart as we highlighted, that is based on a Wall Street consensus average number. As you have said repeatedly, we feel very confident that ORLADEYO is on its path to a billion dollar product. With respect to the projections, yes, you're right. We do a highly, highly comprehensive market survey in terms of all the products and things. I think it's important to note what it does not tell you is the execution risk for some of these products. That's where I think people underappreciate how difficult it is to commercialize a rare disease product, particularly in a space where there are many incumbents. We have repeatedly shown that. We continue to show that.
That is why I think we feel so confident in terms of hitting that double digit revenue growth on a going forward basis when ORLADEYO starts to become a steady state product.
I'd just add to Babar's point about execution, if this deal goes through, we have the better part of three years to prepare for the execution and our team is ready for that.
Yeah, honestly, I would argue that there's no better team to launch this drug than BioCryst.
Right.
With the experience that we've had with ORLADEYO.
The next question comes from Serge Belanger with Needham and Co. Please go ahead.
Hi, good morning everyone.
This is John. I'm for Serge today. Congrats on the acquisition. I just wanted to double click on the commercial dynamics between ORLADEYO and Nevenibart. That is, if Nevenibart is approved, would you expect any potential pressure to ORLADEYO considering the improvements in dosing and the levels of efficacy that Nevenibart could provide, even considering the stickiness between the market segments. If you could provide any color on the IP landscape for Nevenibart right now, that'd be great. Thanks.
Sure.
Thanks, John. On the first question, with any pressure between the two, what we've shown repeatedly and described repeatedly is that when patients start ORLADEYO, 60% of them make it to a year because they're doing really well, and very few of them drop off after that. We expect that dynamic to continue. We think the great opportunity potentially with Nevenibart is those other 40% may choose to move over to an every three or six month dosing with Nevenibart. We don't expect pressure on ORLADEYO. We expect that the two products can offer patients what they need in an oral or in an injectable setting. As far as the IP landscape, we're very comfortable with their IP out.
To 2042, and that was a key component in the diligence process.
The next question comes from Jessica Fye with JP Morgan. Please go ahead. Hey, guys, good morning. Thanks for taking the question. Can you speak to your confidence that the FTC will be comfortable with this transaction? Thank you.
Yeah, we have no reason to believe that we won't be successful with the regulators, and there are tons of competitors in the market currently, and there are more coming. Very confident. Sorry we lost you there, by the way.
Thank you. This concludes our question and answer session. I would like to turn the conference back over for any closing remarks.
Let me wrap up with this. We've been saying for a while that BD is a key component to our strategy. As Babar said earlier, we've also been saying that the first deal needs to make a lot of sense. Good fit, high probability of success, right timing in terms of gap filler with our pipeline, and then driving revenue growth into the next decade. Today, I believe we've announced a deal that checks all those boxes. As always, thank you for your interest in BioCryst. We look forward to continuing to keep you updated. Have a great day.
The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.