Blueprint Medicines Corporation

If you would like to ask a question during this time, simply press star, followed by the number one on your telephone keypad. If you would like to withdraw your question, please press star, followed by two. Please plan to limit yourself to one question. Thank you. Cassie Sato, you may begin your conference.

Thank you, Nadia. Good morning, everyone, and welcome to Blueprint Medicine's fourth quarter 2024 Financial and Operating Results Conference call. This morning, we issued a press release which outlines the topics we plan to discuss today. You can access the press release as well as the slides we will be reviewing today by going to the investor section of our website at www.blueprintmedicines.com. Joining me today are Kate Haviland, Chief Executive Officer, Felina Lee, Chief Commercial Officer, Becker Hughes, Chief Medical Officer, and Mike Lanceville, Chief Financial Officer, Fuad Namuni, President, Research and Development, and Christy Rossi, Chief Operating Officer, are also on the line and available for Q&A. Before we begin, I'd like to remind you that some of the statements made during the call today are forward-looking statements, as outlined on slide three, and are subject to a number of risks and uncertainties. These may cause our actual results to differ materially, including those described in our reports filed with the SEC. You are cautioned not to place any undue reliance on these forward-looking statements, and Blueprint disclaims any obligation to update such statements. With that, I'll hand the call over to Kate.

Thank you, Cassie, and good morning, everyone. At Blueprint Medicines, we are driving growth and innovation with operational excellence. Our aspiration is to fundamentally shift the way allergic and inflammatory diseases are treated by targeting the mast cell. This begins with Avakit, our breakthrough medicine for patients with systemic mastocytosis. In 2024, we delivered $479 million in product revenue. representing impressive annual growth of 135%. Avakit's deep and sustained clinical impact and very strong safety profile have transformed the treatment paradigm in ISM. Today, we are pleased to announce our revenue guidance for 2025. We expect Avakit to achieve $680 to $710 million in revenue this year, placing us firmly on the path to realize $2 billion in revenue by 2030. With a 45% growth rate at the midpoint, our expected 2025 revenue continues to position Avakit among the most successful rare disease launches to date. And the SM market is bigger than we thought and is growing faster than we expected, as we talked about at the JP Morgan conference in January. We now project that Blueprint's SM franchise will reach a peak value of $4 billion with Avakit as our anchor. We've gotten a lot of questions on what makes us confident to increase the peak SM franchise opportunity to $4 billion at this point. Our confidence is based on a few key factors. First, only a fraction of currently diagnosed SM patients today are being treated with Avakin. We estimate that we have penetrated less than 10% of the existing pool of diagnosed SM patients, giving us tremendous headroom for growth. Second, This pool of diagnosed SM patients is growing, and it is growing faster than we expected. We have seen consistent double-digit growth over the last five years, and as of today, we can see more than 25,000 diagnosed SM patients in U.S. claims data. Our team has done a tremendous job increasing awareness and the clinical suspicion of SM among providers. as well as establishing the commercial testing infrastructure needed to enable this growing number of providers who are catalyzed to pursue an SM diagnosis. And importantly, we expect this growth in diagnosed SM patients to continue for years to come, based on our view that the true prevalence of SM is much larger than we originally thought. This larger estimated prevalence is informed by both new epidemiology data published in 2024 and our own real-world experience. The new epidemiology data suggests a two-fold increase in the number of SM patients relative to prior estimates, bringing the number of potential SM patients in the U.S. up from approximately 30,000 patients to 60,000 patients. In our real-world experience, we have seen SM patients being diagnosed from a broader range of providers than we expected at this point in the launch, most notably from medical dermatologists and gastroenterologists. As both med derms and GIs start to actively look for ISM patients, they are finding them in their practice. Many of these healthcare providers are motivated to manage these SM patients directly and build their expertise in SM. And this leads me to the last factor, the critical mass of experienced hematology oncology and allergy immunology providers that we have established. This foundation of Avakit experience providers will be an engine for growth this year And we have a significant opportunity to continue to increase the depth of prescribing amongst this group while we also grow the breadth of new prescribers. In 2025, we are increasing our investments in education and market building initiatives, as well as in the expansion of our commercial and medical field infrastructure in order to drive multidimensional growth as we make progress towards our goal of $2 billion in advocate revenue by 2030. Felina is going to spend more time and go into more depth on our planned investments and the factors that inform our 2025 guidance later on the call. Beyond driving advocate revenue growth, we have clear areas of focus to create value this year. This includes building a durable SM franchise with L-anestinib that will enable us to drive growth throughout the next decade. L-anestinib is our next generation KIT D816V inhibitor. With our phase three trial of LNS-Nib now underway, we are driving innovation in the treatment of SM by moving beyond symptom control to disease modification. We are prospectively measuring the impact of LNS-Nib on significant ISM disease morbidities in the Harbor Registration Study. And we are confident we will be able to clinically differentiate LNS-Nib and deliver improved outcomes for ISM patients, putting Blueprint in the driver's seat of innovation in SM for years to come. This year, we are also initiating a range of proof of concept studies to further define the broad potential of Blue808, our next blockbuster opportunity. The healthy volunteer data we presented last month showed that Blue808 has a wide therapeutic index and impressive pharmacodynamic impact. Blue808's clinical profile positions it as the best in class and likely the first in class oral kit inhibitor across a range of mast cell mediated allergic and inflammatory diseases that impact many thousands of patients. Later on the call, Becker will talk more about our next steps as we stand up our proof of concept studies, which we expect to begin generating data later this year. And last but not least, we are advancing our portfolio of earlier stage programs that will be the core value drivers of tomorrow for Blueprint. Our 2025 capital allocation strategy is aligned with our plan for value creation. We are prioritizing investments that will enable us to drive significant near-term growth while solidifying us as leaders in developing novel mast cell therapies. Importantly, we are leveraging our clinical development and commercial infrastructure, as well as our deep scientific expertise, to drive growth and innovation while at the same time maintaining a financial discipline that only allows our best programs to advance. You can see the impact of our approach as evidenced by the significant reduction in cash burn we achieved in 2024. Mike will talk more about our investment plans and the strength of our financial profile at the end of the call. Altogether, we have an industry-leading expertise in targeting mast cell biology. We have a unique portfolio of novel mast cell targeting commercial and investigational programs. And we have a track record of operational excellence which placed us in the strongest position we have ever been in as a company, as we've set up 2025 to be another great year. With that, I'll turn it over to Felina to dive deeper into Avakit Performance. Felina?