BioCryst Pharmaceuticals, Inc.

Hello, and welcome to the BioCHRIST 3rd Quarter 2022 Earnings Conference Call. My name is Michelle, and I will be your operator for today's conference. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session. And during the question and answer session, if you have a question, please press 01 on your touchtone phone. As a reminder, the conference is being recorded. I will now turn the call over to Mr. John Blue with Biochrist. Sir, you may begin.

Thanks, Michelle. Good morning and welcome to Biochrist third quarter 2022 corporate update and financial results conference call. Today's press release and accompanying slides are available on our website. Participating with me today are CEO John Stonehouse, CFO Anthony Doyle, Chief Commercial Officer Charlie Geyer, and Chief R&D Officer Dr. Helen Thackeray. Following our remarks, we will answer your questions. Before we begin, please note that today's conference call will contain forward-looking statements, including those statements regarding future results, unaudited and forward-looking financial information, as well as the company's future performance and or achievement. These statements are subject to known and unknown risks and uncertainties, which may cause our actual results, performance, or achievement to be materially different from any future results or performance expressed or implied in this presentation. You should not place undue reliance on these forward-looking statements. For additional information, including a detailed discussion of our risk factors, please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website. I'd now like to turn the call over to John Stonehouse.

Thanks, John. We continue to see steady progress with the Orla Deo launch, and underlying growth trends remain strong. New patient starts are consistent with previous quarters, discontinuations appear to have stabilized, and we are investing more to keep this momentum going. Based on our expectations for continued growth in Q4, we plan to exit 2022 having more than doubled sales from the great start we had last year and will be a quarter of the way to our expected peak of $1 billion globally in just the second year of launch. We've shown you long-term data from our trials, and we will continue to show you real-world data that our drug works very well in many patients. The competitive landscape has changed, too, and is a reminder of how challenging it is to pursue a strategy of oral drugs and HAE. Right now, and perhaps for some time to come, Orladeo is the only direct-acting oral with once-daily dosing to prevent HAE attacks. With this unique offering and product profile, we believe this is a therapy every patient should have the opportunity to try to see if it works for them. Charlie will share more specifics on the Orlodeo quarter performance in a minute. Our strategy of going after challenging targets, specifically serine proteases, for patients suffering from rare diseases is rooted in our ability to discover potent, specific, and bioavailable molecules. In the case of HAE, we were able to do that, do what others haven't, develop an oral once-daily calocrine inhibitor. We believe this allows us to offer something unique to these patients, and we believe HAE is just the start. In addition, because these are difficult targets, we don't stop at the first molecule we discover. We work on improved next-generation compounds. We saw that with the Vorostat and Orladeo. We believe we've done it again with Factor D. We are fortunate to have two molecules again, and that puts us in a position to potentially have the first oral Factor D monotherapy molecule to market. and the next generation that perhaps will be the best. While the future of 9930 is still to be determined, our expectation is we will get an answer to our hypothesis on data from the first 15 or so patients treated for two to three months by the middle of next year. We have also been working on additional next-generation complement inhibitors, including BCX10013, a potential once-daily oral factor D inhibitor that we have advanced into the clinic. In Q1 of next year, we plan to show you data in humans that supports the potential of once-daily dosing. Our plan is to go into rare disease patients in dose-ranging proof-of-concept studies with 10.013 next year as well. But we won't stop there. The complement system is complex, and blocking more than one pathway allows us to go after more rare diseases and may be necessary to successfully treat some patients. So we also have a discovery effort. and we're making great progress on other targets in the complement system. If successful, we'll be able to go after other rare diseases and possibly combine molecules to make the best therapy in one capsule or tablet. We plan to show you more in an R&D day next year. So the goal, simply stated, is to be the global leader in complement. Simple to state, but very challenging to do. We believe this strategy and our discovery platform allows us to do things others may not be able to do, and we look forward to showcasing it to you next year. So what does this mean for you as a shareholder? It starts with the evidence that we can successfully discover, develop, and launch an oral drug directed at a challenging target that offers something unique to patients. The result we expect is Orlidale will fill an unmet need for thousands of HAE patients and will reach $1 billion in global peak sales. That is a major accomplishment that sets us apart to create meaningful value for patients and shareholders. And we plan to do it again and complement, but not with one molecule or one pathway. Imagine many molecules for multiple pathways making the best and unique therapies others may not. That value is many, many more patients and potentially multiples of Orlodeo peak sales. Now I'll turn the call over to Charlie to go over the Orladeo performance.