BioCryst Pharmaceuticals Inc Morgan Stanley 24th Annual Global Healthcare Conference

Okay. Good morning, everyone. I am Jason Russell from the Morgan Stanley banking team. It is a pleasure to welcome Charlie Gayer here from BioCryst. Newly appointed CEO of BioCryst. I think he took the job on January 1 of this year. We are excited to have BioCryst here. It is obviously very topical given some of the recent news, and I think we will get into that. Charlie, thanks for being here.

Thanks, Jason. Maybe just to set the stage for a minute.

As I mentioned, you started as the CEO a long time with BioCryst, but started as CEO at the beginning of this year. It kind of feels like BioCryst is entering a new chapter. Obviously, ORLADEYO, which is the underpinning of the business, is a scaled commercial franchise generating operating profit. You took the step to bring in another asset with navenibart just a bit ago, which adds a second modality in HAE. You have another data point coming towards the end of this year with BCX17725, and you also just took moves on your own perspective on how you are going to drive the pipeline forward from here. Maybe with all of that, set the stage. Let us think about your vision.

How has the vision for BioCryst potentially evolved, and is that the result of the maturity of the business or other things on your mind? Maybe just set the stage for us here to start.

Sure. Absolutely. Before I start, I will be making, obviously, some forward-looking statements, and those statements have risks, which you can learn about at our website. For those of you who are not as familiar with BioCryst or who maybe were at some point in the past, BioCryst has been around for 40 years, but we are at a very different stage right now, so it is a different company. For most of that time, we did everything internally, in-house discovery, and we always had some good science, and that got us our drug, ORLADEYO. This year, we made a decision to pivot away from internal discovery because we have become a profitable company, and we think the best way to go forward is through external innovation to build on the success that we have with ORLADEYO, which we will talk about more, and to build a broader rare disease company.

As we look forward a few years, say to the end of 2030, four years from now, we see us with not just ORLADEYO and navenibart on the market, but probably a third product, well over $1 billion in revenue on a very stable cost basis, growing profitability significantly, which we can then plow back into building a broader pipeline through external innovation. We are really excited about where we are at, the strength of our business, and what we have got going in front of us.

Great. There is so much to unpack here, so maybe just to hit the evolution on the planned pipeline point first. You made a decision to shut down some longstanding capabilities around internal discovery you just mentioned. It sounds like you are not retreating from building the pipeline. What changed in your thinking to make that decision? You mentioned the profitability aspect. Which capabilities do you think are core to keeping at BioCryst that do make you differentiated?

The big reason we made that shift is despite our past success, we realized we can just do more by looking at the broader opportunities out there in rare disease when we just were not big enough to do enough internal programs. Balancing the cost, balancing the probability of success, we can do more externally. This year I have been able to build a really strong team. Dr. Sandeep Menon, who is here in the audience, joined as Chief R&D Officer earlier this year with this shared vision of building a pipeline through external innovation. We added David Jenkins as our Chief Scientific Officer, came over from Ipsen over the summer, and we are building other scientific leaders. We are not pivoting away from science at all.

The science we want now is to help us look at this broad set of opportunities out there and find things that we really believe in, limit our risk in what we acquire, and build a pipeline that way on our growing cash flow.

Great. Pull the thread. You've already taken a step. You acquired Astria, which brought in-house navenibart. I assume you would agree that that's a case study for how you want to think about building out the franchise going forward.

Astria was, I would say, a perfect example, not necessarily the example of what we'll do every time. To find something in a space where we know so well in HAE, we probably never will find something exactly like that again. But the benefit of Astria and navenibart, we've got a late-stage asset there, so we're well set on the late-stage side. And the next things that we want to do are balancing our pipeline going earlier, so kind of phase I, phase II assets to complement what we already have. And in the near term, I think that's going to be much more affordable for us as well.

Okay. Great. Let's get into ORLADEYO. It's a franchise that launched over 5 years ago. I believe 2025 was your strongest year as far as new patient adds. And so far this year, you've seen similar trends. It's kind of an unusual trajectory in some sense. And for rare disease, you often see these more hockey stick kind of launches. HAE is obviously a very unique market, a competitive market as well. I guess, what are the factors at a high level, and we'll pull the onion back here a bit, but what are the factors that have continued to drive demand, and allowed you to grow the franchise sitting here almost 6 years post-launch?

HAE is a unique space. I think it's an incredible rare disease success story. The fact that you've got, for a patient population of roughly 10,000 patients in the U.S., there have already been 11 branded products approved and I think another 5 or 6 in late-stage development, which is amazing. It comes down first to the product, so to have a differentiated product. ORLADEYO is the only oral prophylactic product in the market and has been for the last 6 years. It started there, but then it really comes down to the evidence around the product. With all the options that patients and physicians have, the number one thing that they want is to control HAE attacks, as you can imagine. So efficacy is super important. What we've been able to show over time is how well ORLADEYO works for most patients.

When patients start on ORLADEYO, 60% of them make it to a year, and that's because they're getting really good efficacy. So it's not a drug for everybody, but it's one that works really well for the majority of patients. The last piece is just how our team operates. We, from the very beginning, prior to launch, we knew we were the small guys coming up against some really big competitors. We put a lot of emphasis on the skill sets of the team, the data that we collect, and the need to generate long-term real-world evidence, which is what's shown to the market, shown to physicians, to patients, very importantly to payers, how well ORLADEYO has behaved in the real world, and that's been a big part of our continued growth and success.