Good noon and welcome to the Jefferies 2024 Global Healthcare Conference. It is my pleasure to now introduce Kate Haviland, CEO of Blueprint Medicines Corporation. Just a reminder that this will be a 25-minute presentation. Thank you very much for that introduction, and I want to thank Phil Ross and the entire Jefferies Global Healthcare Banking team for inviting us to participate this year at the conference. As I was just stated, I'm Kate Haviland. I joined Blueprint Medicines about nine years ago, and I was named CEO in 2022. Very pleased to be here representing the tremendous work in progress across the entire team. I have two of my colleagues in the audience, Fouad Namouni, our President of Research and Development, and Cassie Saitow, who is part of our Investor Relations team. A nd as we can stand here today. W e have very much built Blueprint Medicines into a global, fully integrated biopharmaceutical company where we've now brought two medicines from our labs to approval and have many more life-changing medicines in our pipeline that we're developing in the fields of allergy, immunology, and hematology-oncology. So, before I begin, I'd just like to remind everyone that I will be making forward-looking statements and just direct you to our SEC filings for any additional information about that. So, I really like this slide as it depicts kind of on one page the tremendous progress we have made across all aspects of our business in 2024. First is the strong top-line revenue growth that we have driven with the launch of AYVAKIT and Indolent Systemic Mastocytosis. Our impressive year-to-date results are providing what is just an incredibly strong foundation as we think about driving long-term shareholder value into 2025 and beyond. We are now at a $500 million run rate, our first full year into launch, placing AYVAKIT firmly on the path to realizing its greater than $2 billion peak potential that we have been talking about. This also positions AYVAKIT and the launch of ISM to be one of the most successful rare disease launches to date. Second, we have made significant progress advancing our prioritized pipeline of programs. And our focus in our pipeline is really on mast cell-mediated diseases where we know that mast cells play a role in the core biology across a number of different patient types who suffer from type 2 inflammation. This is also a place where we can leverage both our deep expertise in KIT biology and mast cell biology, as well as our medical and commercial infrastructure. And then third, we have been achieving both of these things while maintaining financial discipline. Our strong and durable financial position is a result of that prioritized capital allocation and really puts us in a unique position for sustainable growth over time. So, let's turn now and talk to why we are focused in the area of mast cell-driven diseases. So, Blueprint Medicines was founded with a premise that we could find a solution for patients with systemic mastocytosis. So we have over a decade of research and development underpinning our successful launch of AYVAKIT in systemic mastocytosis. And we've established a clear leadership position now in mast cell-driven diseases. We have a very deep understanding of the mast cell biology. We have a global clinical development infrastructure and expertise in this space, regulatory experience, and now we have a strong commercial infrastructure and execution. We are building a portfolio of therapies that you see here on this slide as you move from AYVAKIT on the left into Elenestinib and BLU-808 of therapies around this differentiated expertise and knowledge in mast cell-driven diseases. Building on the leadership we have in AYVAKIT, we have our next-generation KIT D816V inhibitor, which is called elenestinib. This is the same mechanism as AYVAKIT, but we're designing a strategic development plan to expand our SM-branded franchise and our leadership well into the next decade. And then on the right-hand side of the slide, you see BLU-808. This is our wild-type KIT inhibitor that is poised to significantly grow our footprint in mast cell diseases with a true kind of pipeline-in-a-pill opportunity. And that is often something that is a terminology that we use in this industry. And there are not many occasions where you're able to realize that. And we really believe we have a unique and exceptional opportunity here with BLU-808. We'll talk more about that today. Together, this portfolio of both selective mutated and now wild-type KIT inhibitors targeting the mast cell is designed and developed on the premise that KIT inhibition in particular requires titratable and tunable dosing to find that right risk-benefit profile for any kind of given patient in any disease state or even amongst a disease like SM that is a spectrum of disease. Let's start with AYVAKIT. AYVAKIT is the cornerstone of our mast cell-driven portfolio. And as I said, the launch of AYVAKIT is off just to a tremendous start. And it's really what we think is one of the most exciting, rare disease launches happening today. And that is because of AYVAKIT's multidimensional value proposition, which you see here. AYVAKIT is a disease-modifying therapy designed to address the source of the disease. We have demonstrated that it drives meaningful and deep sustained symptomatic relief for patients with ISM over years now in the PIONEER study with a consistent and well-tolerated safety profile with patients now on a median being treated two years on the last data set we presented in the middle in June of this year. We now have patients who've been out to four years in treatment on this study. This clinical profile has been exceptionally well received and launched through all of our key stakeholders, including healthcare providers broadly from hematology-oncology and allergy immunology, the payers, as well as patients. Treatment with AYVAKIT is transforming the treatment paradigm for ISM and is setting AYVAKIT up to be the durable market leader across the spectrum of both advanced SM and ISM for years to come. I'm tremendously proud of the impact our team has been having and on the success of this launch today. So, let's take a look at that from a financial perspective. So, a few weeks ago, we just reported our Q3 earnings results. And what we saw was another strong quarter of AYVAKIT growth, which is adding additional momentum to our already impressive year-to-date results and providing an incredibly strong position as we think about launching into 2025 and beyond. AYVAKIT's impressive sales trajectory has really given us the confidence to significantly raise guidance over the course of this year. And we've now both been able to raise and tighten the range of guidance. And we believe we'll be ending the year somewhere between $475 million and $480 million in revenue. Being on a $500 million run rate in our first full year of ISM launch places us squarely on that path to realize the $2+ billion potential of AYVAKIT. Very exciting place to be. Now, the way to capture this blockbuster opportunity is to continue to grow in a strong and steady way that base of patients who are being treated. And what we've seen to date, although it's early days, is that patients start AYVAKIT and they stay on AYVAKIT. So, as we continue to see the strong and steady growth in patients on therapy and this positive trend for multi-year duration of therapy, we also want to look at what's driving that. And an important part of that is both the breadth and depth of prescribers. So this is a graph that we show on our earnings call this year. So you can see the evolution. The green graph on the left is really just demonstrating the breadth and depth of prescribing and part of our distribution channel where we have the most access to valid data, which is a specialty pharmacy channel, and this is really only for the top decile of treaters who have the largest volume of SM patients in their practices. A nd what you can see here is that the first positive experience with AYVAKIT leads to repeat prescribing amongst this top 400 physicians by SM volume. B ut that's only part of the story, so both you have new prescribers still having their first patient come on, and then as that experience matures, you see that more and more people put additional patients on, so this kind of kinetics is something we would like to continue to see and we believe we will see throughout next year. But as I said, that's only part of the story. As we are engaging with several thousand providers beyond this top 400, adoption is growing across the entire range of providers that we engage with. And as the experience with AYVAKIT grows, we are seeing that the new prescribing from hematology and allergy will also continue, and that that has been very evenly split, actually, between academic and community settings, which is a very strong sign. As often in rare disease launches, you see adoption of a new mechanism of action, a new medicine to be concentrated in the academic setting. And seeing the amount of demand coming out of the community is something that we've been very encouraged by this early in launch. So again, these are the types of dynamics that we expect to see and that we hope to see in a strong launch. They really do plant the seeds for the future growth of the product. We've also started to see our first examples of additional specialties who are caring for these patients and starting to get comfortable to prescribe AYVAKIT. That is including medical dermatology, which is something we talked a lot about on our Q3 call. That's another opportunity as we think about how do we further grow this market is how do we then tap into the additional medical specialties where patients may be cared for and diagnosed with SM and also where patients may be, again, erroneously diagnosed with another disease where the symptomatology may mimic that disease, such as something like IBS. For us, most importantly, we're very much focused on AYVAKIT's peak opportunity and the value we are driving over the long term. As we think about the SM market overall, we see significant headroom for continued growth with multiple levers. First is the growing number of diagnosed patients that we see in claims data in the U.S. We will update that data as we have now become accustomed to doing at the beginning of 2025, a few short weeks from now. We've continued to see nice growth in the number of diagnosed patients. We've also seen that the prevalence estimates from epidemiology studies have been updated this year with new studies that have been done with the newer diagnostic technology and with physicians who have a greater clinical suspicion of mast cell-driven diseases, including systemic mastocytosis. What that suggests is that the number of potential patients with SM is perhaps double what we had originally expected from the historical epidemiology work that had been done. And then what we also see is that prescribers and healthcare providers start to really broaden their lens on what type of patient could benefit from AYVAKIT treatment as they get more clinical experience. Our label in the U.S. is for any adult with ISM. A nd so it really enables physicians and their patient to have that conversation about whether or not bringing in a new therapy that is specifically designed to target the source of this disease is appropriate for that patient, even if they have one really burdensome symptom that makes them unable to go to work or changes their ability to kind of live their lives. And so, broadening that perspective on who an AYVAKIT-eligible patient is, we've already started to see, and we expect that that will also increase over time as people get more and more clinical experience. Lastly, but very importantly, is our global expansion. Right now, the only global market where ISM is currently commercially available is in Germany. In 2025, we expect, as we move through pricing and reimbursement procedures and a number of other markets, that they will be coming online as well. We also have distributor relationships outside of the footprint in which Blueprint, where we are bringing the medicines directly to patients in places like Eastern Europe, Israel, and in Canada. Now moving on from AYVAKIT, let's go on to elenestinib. As we build this broad portfolio of therapies around our differentiated knowledge and infrastructure in mast cell diseases, we are bringing forward our next-generation KIT-D816V inhibitor called elenestinib. The goal of elenestinib is to expand our branded SM franchise leadership well into the next decade. We plan to do this through a strategic and differentiated development approach in our registration-enabling HARBOR Part 2 study, which is on track to initiate by the end of this year. Let's take a look at how we're planning to do that with that study. With the development and approval of AYVAKIT in ISM, we really pioneered a new treatment paradigm that's enabled patients to regain control of their lives by managing the symptoms of their disease and decreasing those symptoms in a very durable and deep way and improving their quality of life. This has been a step change for these patients who had no approved therapies in ISM before AYVAKIT. Today, what we're doing is applying those learnings as we expand development across our mast cell disease portfolio. And with elenestinib, our development strategy is to build on the strength of our AYVAKIT experience and to extend the long-term value of the SM franchise by generating data that adds to the evidentiary set that we are modifying the disease. So we believe that we can set a higher bar for ISM treatments in the future by shifting towards measuring a broader clinical impact, which we've now learned to do through the course of AYVAKIT's development and the pioneer study that goes beyond symptoms and quality of life and that will enable physicians and patients to have a very differentiated value proposition showing how treating with a mutated KIT inhibitor can impact things like bone health, anaphylaxis, the risk of progression. All things that we know based on our current experience are very important to patients and their healthcare providers. So, we have designed HARBOR Part 2 not to be a repeat of the PIONEER study, but rather to push the innovation forward in how we think about optimally treating patients with SM. The three key pillars of this study are first leveraging the primary endpoint that we established as the first endpoint ever to enable the approval of a medicine for ISM, and that is the ISM-SAF and the TSS score. In addition, we're looking at the broad symptom impact, as I just mentioned, and quality of life, prospectively looking at including bone health, anaphylaxis frequency and severity, plus a number of other outcome measures and biomarkers as we kick off the registration-enabling HARBOR Part 2 study. Importantly, we are also going to be assessing two different elenestinib doses in this study. There's one thing we know is that as SM is a spectrum of disease, you need multiple dose strengths to be able to optimally treat patients who sit along that spectrum. That has been an incredible strength of our AYVAKIT launch, where our labeled dose for ISM is 25 mg. Our labeled dose for advanced SM is 200 mg, and we have a 100 mg strength and a 50 mg strength available. S o there's four strengths available to enable both a physician to tailor the treatment to the patient who's sitting in front of them based on the burden of their disease and other measures as they monitor their patient's progression. We believe that this approach will allow us to offer an innovative option with even more comprehensive clinical evidentiary set of benefit of treating patients with a KIT D816V inhibitor in the future. Again, as I said, we're on track to initiate this study by the end of the year, and we will provide more information about it as we get into 2025. So, in our last few minutes today, I want to spend some time on BLU-808, which we at Blueprint are very excited about. This program, again, leverages our deep expertise in mast cell biology as we work to broaden our impact by addressing the significant medical needs across thousands of patients with mast cell-mediated diseases. So importantly, BLU-808 is our wild-type KIT inhibitor. So again, AYVAKIT and elenestinib are addressing the mutated form of KIT, and BLU-808 is designed to address the wild-type form of KIT. We designed BLU-808 to really raise the bar on what a treatment for diseases like chronic urticaria and other inflammatory diseases can offer by taking into account the full patient experience, that is, efficacy, tolerability, as well as the burden associated with administration. We see the opportunity for BLU-808 to really be twofold, which is to command a large share of significantly established markets, but also to be able to drive further growth by expanding the treated populations in those markets. On this slide, you see we've presented this previously, the profile of BLU-808. I think things that are important to note, first and foremost, is the sub-nanomolar potency and the selectivity if you look at the S- score. Indeed, BLU-808 is one of the most selective molecules that we at Blueprint Medicines have designed to date, which is incredibly important when you're modulating a wild-type KIT target and you're thinking about how to find that balance between significantly ameliorating patients' symptoms while also having a very tolerable profile where they can stay on medicine for a long time. At the bottom of the slide, what you see is that we have excellent selectivity across the kinome, including key off-targets that are closely related to KIT, such as PDGFR, FLT3, CSF1R. We believe this remarkable potency and selectivity could enable a wide and differentiated therapeutic index for BLU-808 to allow us to achieve that goal of having a tunable oral medicine. In preclinical studies, we've demonstrated that we can tune BLU-808 activity, and we've shown some of that, and we have some videos on our website where we can block the degranulation of mast cells or dose higher to go all the way to depleting the mast cells based on that differential exposure. This tunability can really provide the basis to optimize the risk-benefit across a number of disease stages, which I say this is our reason to believe that we can achieve a true pipeline-in-a-pill opportunity for BLU-808. So, we believe BLU-808 has the ability to be the first and potentially best-in-class oral KIT inhibitor to address this broad array of diseases of type 2 inflammation and mast cell-mediated diseases. We are currently undertaking a healthy volunteer study for BLU-808. This is a very standard single and multiple dose ascending study, and it's designed to assess early safety, pharmacokinetics, pharmacodynamics. We're looking at a range of markers there, and what we have guided to is we anticipate data early next year. The most important thing we'll be looking for in that data set is the dose-dependent impact across each of these domains so that we really can reveal the breadth of BLU-808's therapeutic window and the potential to have this customizable and tunable dosing. So, what would we like to see? We want to see predictable dose-dependent range of exposure and target coverage. We'd like to see pharmacodynamic outcomes that include serum tryptase but go beyond that respond in a dose-dependent way, and safety, including the ability to see the tunability of both of the on-target adverse events that you would expect to see. This is what we'll be looking for early next year. Last week, we hosted a webinar where we spent more time saying, "okay, if we are able to achieve that in the healthy volunteer data, then what will be the next steps for BLU-808?" And so, our goal is to efficiently execute an early clinical development strategy that is able to define BLU-808's broad footprint, but also retaining that disciplined approach to investment. So, we aim to do this by serially de-risking BLU-808 with key data generated from early proof of concept studies. So, assuming positive phase I data, one of the first places we'll be looking to kick off our proof-of-concept study will be in chronic spontaneous urticaria, chronic inducible urticaria. There's already been tremendous work done in that space that validates that targeting KIT is the way to go to really ameliorate symptoms for those patients. And then we'll move on and look at in parallel other respiratory diseases like allergic rhinitis, allergic asthma, where early development pathways, including the use of challenge studies, are clear. We're also looking at doing a proof of concept in mast cell activation syndrome or MCAS. One of the things that's interesting about MCAS, and we showed this data at the webinar last week, is that when patients with MCAS are screened with an ultra-high sensitivity test that is not currently commercially available for the D816V mutation, about 20% of those patients actually had the D816V mutation. And so are truly more applicable for AYVAKIT treatment than a wild-type KIT inhibitor. And then MCAS patients really have no approved therapy now as well. So, we will be looking as we prosecute the MCAS program. We'll be seeing patients who will be identified for a mutated KIT treatment, and then those who could then go and get treated with wild-type KIT. So, it really shows the strength of the synergy across the portfolio and how we see the interconnection between both AYVAKIT, elenestinib, as well as BLU-808. BLU-808's potential indications represent really the largest opportunity set we at Blueprint have had a chance to work in. And we're really excited for that healthy volunteer data, which will set that strong foundation to move into these proof of concepts early next year. So in summary, we are wrapping up what has been just a tremendous year of growth in 2024 at Blueprint. First and foremost, with our launch of AYVAKIT and ISM, where we're exiting the year at a $500 million run rate, we've made tremendous progress advancing our prioritized pipeline of programs, with our focus really being in the mast cell-driven disorders with high medical need and large patient populations. And we've done this while also maintaining a very strong and durable financial position. So, as we look to 2025 and beyond, we'll be focused on maintaining this executional excellence and investing in our business responsibly to drive very compelling growth next year and throughout the rest of this decade. So with that, I think actually we're at time. So I don't think we have time for any questions, but I appreciate you all being here today. And please do follow up with us if you do have questions.