Blueprint Medicines Corporation (NASDAQ:BPMC) Q1 2025 Earnings Call Transcript

Blueprint Medicines Corporation (NASDAQ:BPMC) Q1 2025 Earnings Call Transcript May 1, 2025

Blueprint Medicines Corporation misses on earnings expectations. Reported EPS is $-0.74 EPS, expectations were $-0.42.

Operator: Good morning. My name is Angela and I’ll be your conference operator today. At this time, I would like to welcome everyone to the Blueprint Medicines 1Q 2025 Earnings Release and Conference Call. All lines have been placed on mute to prevent any background noise. After the speakers’ remarks, there will be a question-and-answer section. [Operator Instructions] Thank you. Jenna Cohen, you may begin your conference.

Jenna Cohen: Thank you, Angela. Good morning everyone and welcome to Blueprint Medicine’s first quarter 2025 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 that we’ll 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; Philina Lee, Chief Commercial Officer; Becker Hewes, Chief Medical Officer; and Mike Landsittel, Chief Financial Officer. Fouad Namouni, President of Research & 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 3 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’re cautioned not to place any undue reliance on these forward-looking statements and Blueprint disclaims any obligation to update such statements. I’ll now hand the call over to Kate.

Kate Haviland: Thank you, Jenna and good morning everyone. We at Blueprint Medicine, strive to be a standout top tier biotech company with a core focus on innovation, commercial excellence, and maintaining a durable financial profile that enables disciplined global investment across our portfolio. Following strong performance in 2024, we’ve continued our executionable momentum in 2025. This quarter we achieved 61% year-over-year AYVAKIT revenue growth as we continue to capture the substantial and growing multi-billion dollar commercial opportunity and systemic mastocytosis, which we expect will drive our global revenue growth well into the next decade. With every additional quarter of performance AYVAKIT progresses on the path to our goal of $2 billion in revenue by 2030.

The underlying fundamental demand driven by growth and patients on therapy is the critical determinant of long-term revenue potential and we are pleased that our results in Q1 reflect continued strength in this metric as we expected. This strong fundamental growth coupled with significant favorability in our free versus paid good mix is leading us to raise our revenue guidance for the year. Philina will go into this more specifically in a moment. We also advanced our industry-leading pipeline of mass cell-directed investigational medicines. Our two prioritized pipeline programs elenestinib and BLU-808 have the potential to drive significant upside value for Blueprint as they progress in the clinic this year and beyond. With BLU-808, our wild-type KIT inhibitor, our goal is to raise the bar on what a treatment for a wide range of allergic and inflammatory diseases can offer by considering the full patient experience, efficacy, tolerability, and the burden associated with administration.

BLU-808’s early clinical profile supports our differentiated approach to development allowing us to explore a range of doses as well as dose regimens. As we announced this morning, BLU-808 has achieved key milestones with the initiation of two proof-of-concept studies, one in allergic rhinoconjunctivitis and one in chronic urticaria. We hope to be able to share some early data this year from these studies which Becker will talk more about later on the call. Elenestinib, our next-generation KIT D816V inhibitor, has the potential best-in-disease profile that builds on our years of experience in SM. Given the strong receptivity of AYVAKIT by both physicians and patients, we know it is not enough to incrementally innovate with a follow-on medicine that will enter the market years after AYVAKIT.

With the pivotal Harbor study up and running, we have a plan to deliver substantially differentiated and meaningful innovation with elenestinib that goes beyond symptomatic control to measures of organ healing and disease modification. Blueprint’s durable commercial growth pro profile, strong cash position, and disciplined capital allocation strategy afford us the ability to focus on executing our business to plan despite the broader market volatility. With our intellectual property being domiciled in the US and our low cost of goods, we are insulated from potential biopharmaceutical tariffs and do not anticipate any material impact on our business. In terms of the staffing changes at the FDA, we do not have any major regulatory filings planned over the next 12 months, and we have not experienced an impact in our routine engagements with the agency to date.

We will, of course, continue to monitor the rapidly evolving external environment and work to mitigate any changes that do impact our business. Our results today are possible, thanks to the impressive team of people we have at Blueprint Medicines. And I thank them for their thoughtful and urgent commitment to the people whose lives we set out to improve every day. With that, I will turn it to Philina to discuss the quarter’s commercial performance in more depth.

Philina Lee: Thanks, Kate. In the first quarter, we achieved $149.4 million in AYVAKIT global net product revenues with $129.4 million in the US and $20 million ex US Our strong commercial execution drove revenue growth in what we expected to be a challenging quarter due to the typical Q1 financial headwinds inherent in our industry. Let’s walk through the headwinds and tailwinds. The short-term financial headwinds we’ve discussed previously played out as we expected, including typical first quarter insurance dynamics impacting gross to net and the impact of fewer ordering days. With our growing base of business, these factors can have a meaningful impact on a quarter. As Kate mentioned, fundamental demand growth driven by a growing number of patients on therapy is a critical determinant of long-term revenue potential.

This is a function of more patients starting and staying on therapy. We’re pleased that Q1 met our expectations as we saw growth in new patient starts, both in the US and in our international business. Discontinuation rates remained low, and we continue to see encouraging trends towards multiyear duration of therapy in both advanced SM and ISM. A key variable we were watching this quarter was our mix of free versus commercial goods as we managed through the Q1 reauthorization process and monitored the impact of the Part D redesign and foundation funding availability. This played out more favorably than we expected with our free goods rate now well below 10%. As a result of these favorable dynamics, today, we are raising our guidance range to $700 million to $720 million for the year.

We are exactly where we want to be at this point in the year, and we are well positioned to drive growth through the rest of 2025 and beyond. We have a clear view of the SM opportunity in front of us as we move further down the path towards $2 billion in AYVAKIT revenue by 2030 and a $4 billion peak SM franchise opportunity. SM diagnoses are increasing globally and with approximately 25,000 diagnosed SM patients in the US, we know we’re in the early innings of market penetration. Roughly 20% penetration of currently diagnosed patients in the US alone gets you to $2 billion in annual revenue. We expect to grow new patient starts through the end of the year and beyond. Of course, it all starts with the right medicine, AYVAKIT, an effective therapy with a broad label that addresses the root cause of disease and is also very well tolerated, enabling patients to start and stay on therapy over the long term.

Becker will discuss the growing body of evidence supporting AYVAKIT’s long-term benefit in SM. In addition to AYVAKIT’s compelling clinical profile, we know that what drives prescribing in this market is the powerful combination of an aware and ever-expanding educated prescriber base and an activated patient base, both recognizing the benefit and urgency to treat the root cause of disease with AYVAKIT now. Let’s start with prescribers. Since launch, we’ve been focused on increasing the breadth of our prescriber base through education and awareness. Driving awareness among allergy has been a key focus and is critical to long-term growth as the providers managing the highest volume of SM patients are predominantly allergists. A first experience with AYVAKIT predictably leads to broader use in more patients.

So a growing foundation of experienced providers, prescribers directly drives growth in the number of patients starting AYVAKIT over time, increasing market penetration. Since ISM approval, our prescriber base in the US has tripled with adoption split across academic and community sites care. Against the backdrop of a growing number of diagnosed patients this creates an engine to drive growth in the number of patients prescribed AYVAKIT. What really catalyzes deepening of use over time are positive perceptions and experiences with AYVAKIT. Since ISM launched, we have conducted market research with over 700 providers. Initial favorable perceptions of AYVAKIT from the PIONEER study results have grown even more positive with firsthand clinical experience and now with longer term data.

Across all specialties even dermatology and gastroenterology where we haven’t yet started to deploy promotional effort. The vast majority of providers view AYVAKIT’s profile as favorable. Since launch, we’ve been targeting HEMOX and allergists. HEMOX treat less than one-third of our ISM patients. We’ve talked about the importance of allergy for the long-term opportunity and by expanding our call point to include derms and GIs, we will meaningfully increase the addressable patient pool. Across these specialties, roughly 70% of the providers managing the highest volume of SM patients are allergists, derms and GIs. This is why we’re investing in expanding our field team to increase our reach and frequency where it matters most. Our field force expansion is designed to capitalize on where the majority of SM patients are treated.

We’ve just hired new team members who are on boarding and training. They bring a diverse set of experiences with an emphasis on allergy, dermatology and rare disease. They’ll be in the field by the second half of this year, and I look forward to the growth what they’ll catalyze in 2026 and beyond. AYVAKIT’s positive reception among providers is matched by growing enthusiasm in the patient community. SM patients are highly engaged and active in their care decisions and we’re starting to see the clear impact of marketing efforts launched last year, including new direct-to-consumer and peer-to-peer programs. Providers report that significantly more of their ISM patients are asking about AYVAKIT compared to last year. And once patients are on treatment, they have a very positive experience with over 95% of patients saying they’re highly satisfied with AYVAKIT as a treatment for their SM.

The SM market is highly promotionally sensitive with the top two drivers being the efforts of our experienced sales team who are outperforming industry comps in promotional effectiveness scores and our direct to patient marketing efforts. Our growing base of experienced prescribers an increasingly engaged and activated patient base and the compelling profile of AYVAKIT put us in a great place to drive growth in 2025. But we’re not just focused on this year. We are on the path to driving continued growth towards $2 billion in revenue by 2030 and are committed to continue to grow this market well into the next decade. We plan to drive that growth by taking the same powerful combination of aware and educated prescribers with activated patients and amplifying it with a strategic investment in our field.

We are confident in our multifaceted approach to develop and capture this market. I’ll now hand the call over to Becker to share clinical updates across our portfolio.

Becker Hewes: Thanks Philina. First, I want to talk about AYVAKIT’s long-term data in systemic mastocytosis that we presented at QAD AI [ph] and that Philina just referenced. What was remarkable in PIONEER is that the safety profile of AYVAKIT was superior to best supportive care at six months, a rare occurrence for an efficacious treatment. Now after three full years of treatment, we see the same safety profile. This is extraordinary in my experience. Generally the safety profile becomes more complex every time. But for AYVAKIT, the frequency and severity of treatment-related adverse events have been consistent and remain low throughout three years, and importantly only 3% of patients discontinued due to treatment-related adverse events over this extended time frame.

The 25 milligram dose of AYVAKIT provides an exceptional benefit risk profile for the vast majority of patients. In our real world post-marketing experience, more than 90% of patients who start at 25 milligram stay at that dose. The patients are highly compliant, satisfied and our discontinuation rates are very low. However, we know that there is a spectrum of disease severity in systemic mastocytosis and a one dose fits all approach is not ppropriate in this disease. The PIONEER study included patients with very high disease burden, we’ve enabled more severe patients to dose escalate to 50 milligrams in part three of the study. We recently presented data showing that these patients have had a safety profile consistent with that we’ve seen at 25 milligrams.

Efficacy continues to improve for PIONEER patients as indicated by durable responses measured by TSF and quality of life measurements through three years. Among the patient subsets and PIONEER, who titrated their dose to 50 milligrams, 93% of patients saw improved or stable TSF benefit at 50 milligrams without any trade-off in safety. We’re expanding our commercial and medical reach to an even larger target physician audience of allergists, dermatologists and gastroenterologists with multiple years of consistent data demonstrating that AYVAKIT is a safe and effective treatment in hand. This helps drive comfort in trying a new therapy and prescribing AYVAKIT. Finally, we continue to advance the science of systemic mastocytosis in the HARBOR study of elenestinib.

Our study design explores disease-modifying measures of clinical benefit, notably the impact on bone health and recurrent anaphylaxis. These have been enthusiastically received by investigators and they report also from patients interested in the study. Turning now to our wild-type KIT inhibitor, BLU-808. At QUAD AI this year, we presented data from the healthy volunteer study. And with this promising data in hand, we’re moving into four proof-of-concept studies this year. We know that a strong safety profile is critical in the field of allergic disease. And with BLU-808, we have a wide therapeutic index to work within. We’re building in a range of flexible dose strategies across these studies, including consistent dosing, induce and maintain and titrate to effect, which we know is in line with how allergists treat.

We’ve initiated our first two proof-of-concept studies, one in allergic rhinoconjunctivitis and one in chronic urticaria. We will be initially testing doses between 1 and 6 milligrams. This covers the IC90 well and will allow us to examine a spectrum of biologic impact that ranges from calming or stopping mast cells from degranulating to killing them entirely. This will allow us to determine the impact on symptoms so that we can achieve the right balance of safety and efficacy for chronic treatment. I’ll start with chronic urticaria, where the role of mast cells as the key driver of disease is already well established. In our proof-of-concept study, we’re using this indication to explore various dosing regimens for BLU-808. Our initial 12-week Phase 2a study will include two key parts: an open-label study of patients with chronic inducible urticaria or CIndU, and a randomized double-blind, placebo-controlled study of patients with chronic spontaneous urticaria.

Both parts will include multiple doses and dosing regimens. Our goal is for BLU-808 to become an attractive oral option that achieves that balance of tolerability and efficacy and differentiates us from the rest of the field. Now let me turn to the Phase 2a study in allergic rhinoconjunctivitis. We see this as a way to demonstrate the activity of BLU-808 in the respiratory tract. This is a 28-day placebo-controlled challenge study where patients are exposed to allergen, then treated with BLU-808 or placebo and then re-exposed to compare their before and after treatment reaction. Similar to chronic urticaria, in this study, we will assess multiple doses for safety, PK and clinical efficacy. We expect to have some early data by the end of this year in the CIndU cohort.

In the second half of the year, we plan to initiate studies in allergic asthma and in MCAS. We discussed the role of the mast cells in allergic asthma at our webinar last year and similarly plan to share Blueprint’s approach to MCAS at our webinar on June 4 with Dr. Matt Giannetti, the leading expert in mast cell diseases at the Brigham Women’s Hospital here in Boston. Results from these proof-of-concept studies will help guide BLU-808 development plans as we work towards improving the lives of patients with mast cell-driven diseases. Now I’ll turn the call over to Mike.

Mike Landsittel: Thanks Becker. Earlier this morning we reported detailed financial results in our press release and for today’s call I’ll touch on a few highlights. In the first quarter we achieved total revenues of $149.4 million from net product sales of AYVAKIT. Based on the positive fundamentals that Philina discussed, as well as the additional insight we gained around free goods in Q1 we are raising our AYVAKIT net product revenue guidance to $700 million to $720 million for the year. Consistently strong fundamentals and continued growth in new patient starts in all markets will continue to drive performance through the remainder of the year. For international business, the timing and outcome of ongoing pricing and reimbursement negotiations are another consideration.

Turning to operating expenses, we observed an incremental quarter-over-quarter increase in R&D expenses related to elenestinib and BLU-808 clinical studies. SG&A expenses were flat in the first quarter relative to the prior quarter and we anticipate that we will see continued modest increases in both R&D and SG&A expenses, as we invest in our priority pipeline programs as well as increase investment in our sales and marketing efforts for AYVAKIT as Philina spoke to in detail earlier on the call. We continue to expect that our operating cash burn will decline significantly on an annual basis. To reinforce what Kate said earlier in the call, we are in an incredible position of strength today particularly when you consider the macroeconomic environment.

We have strong and consistent top line revenue growth driven by global sales of AYVAKIT. The ability to continue to invest in innovation to drive future growth and a strong and durable cash position of $900 million. Our commercial and financial profile stands out among biotech as a positive differentiator and now more than ever before we are in a position of incredible strength to drive sustained growth for the long-term. With that I’ll turn the call back over to the operator for questions operator.

Q&A Session

Operator: Thank you. [Operator Instructions] Your first question comes form Marc Frahm with TD Cowen. Your line is open.

Marc Frahm : Hey, thanks for taking my questions. First on the commercial side, can you frame the type of rebound and kind of growth or acceleration of growth you might see in Q2 given the fact that the free grip the free goods is already so low which maybe limit some of the kind of typical Q1 to Q2 rebounds that we see that we see across the industry. And then on the R&D side for BLU-808 just talk to the trategy on the dose selections that you that you put into those are the two doses supposed to really kind of test that intermediate range with the titration or are you trying to maybe with the top dose really show the full power of BLU-808 and inability to get close to it not matching the antibodies?

Kate Haviland : Yes. Thanks Mark for that question. Maybe Christie, if you want to talk a little bit about how we’ve considered kind of the fundamental growth and the drivers of demand and as we think through the year and then maybe to please add color Becker, do you want to you can add into the question on that side.

Becker Hewes: Sure. Hey, Mark. So if we think about the year we obviously updated guidance and so if I think about the remaining three quarters I would certainly think about the guide and that guide does imply growth which is really what we expected when we set guidance at the beginning of the year as well. We always knew that Q1 was going to be challenging for all of the reasons that Selena said, but what we expected to see was strong underlying growth in terms of those drivers of the longer-term potential for AYVAKIT, which includes patients starting persistence duration et cetera. And so and that’s exactly. What we’ve seen that’s played out in line with our expectations. And so if you kind of play that forward through the year we would expect to see growth in the remaining quarters.

The one wrinkle we saw in Q1 round sort of a shorter quarter from an ordering perspective I believe if I’m not mistaken it’s made up actually in Q3 interestingly enough, but we definitely expect to see nice steady growth as we go through the remainder of the year.

Kate Haviland : Let’s say in our results this year really our big card flip in Q1 was around how we’re going to see free goods play through and I know do you want to talk a little bit more about that.

Philina Lee: Yes, as we entered the year I think one of the biggest variables that we had that informed our guidance range was that we had a large book of business, i.e. a really large number of patients and we didn’t know to what extent these patients would be able to transition and access paid versus free goods. Over the course of Q1, like this is a big variable that has been tremendously de-risked and that has been one of the key drivers for our guidance update, together with the expected growth in the underlying fundamentals i.e. growth in patients on therapy as Christie just said. So I think at this point, thinking about the free goods trends, looking for the rest of the year, certainly the range has narrowed. We have a better sense of the range to expect, we’re below 10%, we don’t expect this to be able to really be able to go appreciably down further and really over the rest of the year, we’ll need to be watching as new Medicare patients come on therapy their ability to access commercial versus free drug.

But I would just take it back to the trajectory that we’re seeing in Q1, we firmly believe puts us on pace for our guidance, as well as marching towards that 2 billion by 2030. Becker, do you want to talk about this?

Becker Hewes: Sure, let me talk about the dose range of 808 and how we selected this and what our strategy is here. Just a reminder, that we do have all three regimens one, which is consistent dosing the other which is an induce and maintain which will look at speed to symptom resolution and then our ability with a lower dose to maintain those that symptom resolution and then one where we will allow titration to the effect. And I think it’s important to remember that in many diseases including chronic urticaria, there are a multitude of symptoms. And really driving the relief of the patient at the right dose is what we’re exploring and we’re doing it in a way that the antibodies really can’t do. So we’re going to better understand the biology of the disease and the response with respect to the symptomatology as we use these different dosing strategies.

And the intent of the study is to really set things up for the next phase of studies, in chronic urticaria and in other indications, where we get the optimized dosing for each of these indications. As we said earlier, the one to six mg gives us a range that covers the IC 90 well and we all mass cells don’t die at once. And so, we’ll look at different levels of killing in different patients to better understand killing dose and a dose that’s designed to calm down the mast cells. With respect to comparisons again, these studies are designed to help us understand how to use the drug and then to understand the full efficacy in the next wave of studies we will bring that optimized dosing into a more definitive study.

Operator: Thank you. The next question comes from Laura Prendergast with Raymond James. Your line is open.

Laura Prendergast: Hey guys, congrats on the progress of this quarter. Just need to reiterate that question, what exactly have you guys baked into the guidance, any expected seasonality, EU contribution, any specific tailwinds or headwinds on new starts that you expect? And then just if you guys could comment on part two Harbor data, when should we expect that and is any LNS data priced into the two billion by 2030 is that pricing in LNS and being on the market? Thank you.

Kate Haviland: Thanks, Laura. Just a reminder everybody let’s TRY to keep it to one question, just given the number of people we have in the queue, but we’ll try to handle some other things offline if we can. Maybe Christian you or Christi excuse me will you take the guidance question and then Becker, we can talk about part two harbor. I can just clarify right away, there’s nothing from an LNSN perspective in the 2 billion by 2030 that’s completely driven by AYVAKIT and we expect AYVAKIT to grow beyond that to be — so just so I can take that one. Christy, do you want to talk about guidance?

Christy Rossi: Sure. So, the factors that we think about with guidance are very much consistent with how we framed it at the beginning of the year, actually how we talked about it last year, right? So the two biggest determinants in my mind of AYVAKIT getting to $2 billion by 2030 and the $4 billion dollar opportunity we see across the SM franchise over time is really treating more patients, right? So having patients start on therapy and having them continue to stay on therapy. So those are the two big drivers and we expect to see growth in new patients starts as they go through the year based on our expanding breadth and depth of prescribing that we’re seeing in the US and increasingly activated patient base et cetera. This is obviously still in the context of our disease market you’re going to see fluctuations there, but the overall trend line is clear and positive.

We expect to see strength and duration of therapy as patients are staying on therapy for — we think 3+ years potentially multiple years here and so, those are two of the most important variables as we go over time. Then there’s a number of other things that actually may have more impact in the short term and so we think about gross to net free goods rates et cetera. And then of course, our international business is coming along. So those continue to be the most important variable. As Philina said, we had a major, I would say carb flip in the first quarter by understanding how a very large base of patients who are now on therapy as they went through the re-verification process understanding how that free commercial mix was going to play out we knew, it was going to be important we knew that was going to be a big swing as we thought about top line revenue and so we’re really pleased to see.

How that played out in the first quarter and also pleased to see continued strength along the other variables that we’ve mentioned that really again portend that longer term — term potential. So those are still going to be the things that we’ll continue to watch. There’s ranges of outcomes on all of those on all those variables, but pleased to see that we were able to raise the guidance off of Q1 performance, in terms of quarter-to-quarter performance. We know our business. You talked about things like seasonality et cetera. We’ve we’re now far enough into this launch where I think we have a good sense of how these factors will play out in terms of impacting quarter to quarter revenue. We’ve baked that into the guide and in fact it baked it in when we started the year and expected Q1 to kind of play out as it did.

Kate Haviland: Becker, do you want to talk a little bit about Harbor. Obviously, we’re just getting this up and up and running now.

Becker Hewes: Yeah. So we’re just starting this study. We have a highly motivated group of investigators and I’m hearing patients as well who are very interested in the study as we see the evolution of the systemic mastocytosis in the world and we see how this study unfolds we’ll be able to keep you all more well informed about when we might see top one data but I think it’s premature to speculate at this point.

Operator: Thank you. Your next question comes from the line of Michael Schmidt with Guggenheim. Your line is open.

Unidentified Analyst: And this is Paul on for Michael. Thanks for taking our question. On AYVAKIT for the ex-US, it looks like it was sort of flat quarter-on-quarter. Can you just comment on what your overall expectations are for the international market drivers this year, including when ISM reimbursement beyond Germany could start to kick in. And then maybe a quick follow-up on those escalations that you mentioned in ISM patients, your quad AI data suggested around 25% escalated to 50 mg. What’s your current visibility into what percentage of commercial patients have booked 50 for ISM? Thank you.

Kate Haviland: Yeah. Thanks for the question and we’ll start Christy you take international and then Philina you talk a little bit about what we’re seeing in dosing. One thing I should say is the international teams have just done a tremendous job and their year-over-year performance more than doubled from Q1 last year. So we’re really excited about where we are. But you want to talk about the Q4 to Q1 dynamic. Christy?

Christy Rossi: Yeah, absolutely. So right as Kate said, the international team has been doing very well. We’ve talked about the fact that if we think about this contributions to the top line. Last year, we expected it to be sort of in that 10% to 15% range. This year, on a much bigger base, we expect it to also be within the 10% to 15% range, right? So the international team continues to perform, and we’re seeing nice growth there. It’s important to remember that Germany is the only ex-US market with ISM reimbursement at this point. We do expect some others to come online through the year, but we’ll really start to see ISM growth across a number of markets as we head even into next year and beyond. The business in Germany is doing quite well.

We’re continuing to see trends that look very similar to the US, which has been really encouraging to see. So, nice growth in patients being treated, very similar trends in terms of uptake. Fluctuations can happen quarter-to-quarter. There were some things between Q4 and Q1, for example, like timing of distributor orders, which can be lumpy, a little bit of FX at the beginning of the year. We’re still talking about a relatively small revenue base relative to the US, right? So these little factors can obviously have a role when we look at Q-on-Q. And we’ve seen that before, if you go back and look at international performance over the last year or so. But the bigger picture is that business is doing well. German launch is doing really well, and we’re looking ahead and looking forward to having some other ISM launches come online.

Philina Lee: And to the question on dosing, the 25 mg benefit risk profile continues to serve the vast majority of patients very well. It’s under 10% of patients that we’re seeing who may dose escalate to 50 mg over time. And this is occurring against the backdrop of the profile of the 25 mg holding strong and improving with continued long-term efficacy and safety outcomes. We also see this reflected in just the really positive sentiment for AYVAKIT reflected in both our provider and patient satisfaction.

Kate Haviland: And just one thing to add to that is that even what Becker showed you from the — both the safety and efficacy of the 50 mg is that what we see is that, with AYVAKIT at 25 milligrams or 50 milligrams patients don’t have to weigh a trade-off between efficacy and safety. And they can experience a very low treatment burden that really allows them to just get back to their lives and kind of with a medicine that really empowers them to live and do the things they want to do. So both the 25 and 50 have really come through in a very nice way for these patients.

Becker Hewes: And the other thing to remember is that, the PIONEER population was a highly advanced patient population. Enrolled in the middle of a pandemic first study out there with a really effective therapy and so, it’s probably not indicative of what will be seen in the real world.

Operator: Thank you. Your next question comes from the line of Michael Yee with Jefferies. Your line is open.

Michael Yee: Thanks. Good morning. Congrats on the continued growth. I’m thinking about growth for the rest of the year. I know you’ve gotten a bunch of questions about headwinds and tailwinds and different dynamics. Can you help give some color as to perhaps the trajectory or shape of the curve in Q2, Q3, Q4? Is it sort of consistent year-over-year growth each quarter? Are there different things that impact those quarters and as part of that perhaps even OUS, given that it appeared to be more flattish and just wondering if that’s a factor as well into any of these quarters for the rest of the year. Thank you.

Kate Haviland: Thanks, Michael. So I think what you’re asking is, how to think about kind of the remainder of the year. Obviously, we’re really focused here on how we’re exiting the year, because that is what really drives us to that that laser focus on that two billion by 2030, but understand that you guys want to kind of get a sense of how we’re thinking about the quarters. I don’t know, Mike, if you want to provide a little bit color on how do you think about that?

Mike Landsittel: Yeah. I’ll start. I mean, I think, you know what’s most important is to put this in the frame of what our updated overall annual guidance is and using that like that’s the benchmark that we’re guiding too. And as we’ve talked about before, like each quarter can have unique variables in the US and International, I think the one thing that Christy mentioned that we did want to point to, if you think about like the growth especially over like Q2, Q3 it’s like we’ve talked about we’ve missed an order day in Q1, right? That actually gets made up in Q3 in the calendar it’s and just for context like our biggest customers tends to order on the same day each week. We had one less of those in Q1, it gets made up in Q3.

So right there, you’re going to see a differential dynamic between Q2 and Q3, where you’d say there’s probably a little bit more shifted into the Q3 period, but fundamentally, I just I point to consistent underlying patient growth and that’s been as expected and we continue to drive towards that as we as we go for the updated guidance range.

Operator: Thank you. Your next question comes from the line of Colleen Kusy with Baird. Your line is open.

Colleen Kusy: Great. Good morning. Thanks for taking our questions and congrats on all the progress. One quick one from us for the growth this year, can you talk to how much of that is driven by the newer specialties that you’re going after in terms and GI’s versus the allergist and hem-onc that your first target prescribers?

Kate Haviland: Yeah. Thanks Colleen. Philina, do want to talk about how you’re thinking about the prescriber mix this year versus next year I guess. Yeah.

Philina Lee: Yeah. Thanks for the question. So I think as Christy started alluding to we expect growth we expect to drive increased treatment rates and growth in the number of patients on therapy. We have a strong engine in place to do this already with what we’ve established across our target specialties and an increasingly activated patient base. So I would say a primary driver for growth over the course of this year is really that activated prescriber base and our ability to both grow and expand that as well as deepen it overtime. Our chart shows we understand very well the dynamics of how a first patient starts and then a positive experience leads to growth and deepening overtime. Secondly, we see more and more patients coming in and asking about AYVAKIT showing that our direct-to-patient efforts are really working.

And we expect this to also capitalize growth over the course of this year. As we shift into the newer specialties, we think this will enable us to further amplify these efforts. There’s really an untapped opportunity of additional SM patients, who are being seen by derms and gastros. I think what we’ve learned from this market is hem-onc alone are only about a third of the opportunities so we know it’s important to expand. Our objectives with derms and gastros are really the ability to move these patients towards treatment whether or not they’re literally the prescribers at the outset, but they can also refer to our very strong and growing prescriber base. And we expect this to further catalyze growth really in 2026 and beyond.

Kate Haviland: And just for clarity, Philina, we really do expect our primary prescriber base to be that growing base of allergists, our stable base kind of the hem/onc group, which sees the minority or the smaller group of patients. And then the growth into GIs and derms will really be 2026.

Operator: Thank you. Your next question comes from the line of Derek Archila with Wells Fargo. Your line is open.

Derek Archila: Hey, good morning and thanks for taking the questions. I just want to understand how durable a sub-10% free drug rate is. And I wonder if you could characterize how the patient adds have trended in April relative to 1Q. Thanks.

Kate Haviland: So Derek, we don’t really talk about kind of the ongoing quarter, but we can certainly talk about our view of the durability of the free patient goods rate. And just to note, it’s actually well below 10% at this point. So Philina, do you want to talk a little bit about how you’re thinking about that for the rest of the year, the free goods rate?

Philina Lee: Yes. I think what we’ve — what we’re really emphasizing is we’ve derisked — we’ve majorly derisked the variable of free goods by just moving this big base of patients, this large number of patients have been able to access commercial therapy in Q1, and that’s been due to the availability of foundation funding, the ability of patients to navigate the new smoothing process. And so we also feel like with this type of floor, well, there’s probably not room for this to further improve, but we expect that rate to remain relatively durable over the course of the year. Now of course, what we’ll be watching is as new Medicare patients come on, how able will they be to access commercial therapy. And that depends on factors like how long will foundation funding continue to be available as well.

But I would say, it’s the derisking of the patients who have already moved, which have led to a favorable — more favorable than expected upside. That’s one of the key factors in our updated guidance range. But really, it’s the underlying fundamentals that we expect continued growth in patient starts and patients on therapy that we expect to grow throughout the course of the year.

Operator: Thank you. Your next question comes from the line of Salveen Richter with Goldman Sachs. Your line is open.

Unidentified Analyst: Hey, good morning. This is Mark on for Salveen. Thanks very much for taking my question. You guys mentioned you may show 808 data from the POC studies in CIndU this year. In your view, what is the bar for 808 in CIndU and also in allergic rhinoconjunctivitis? And how do you think this will compare to the antibodies here?

Becker Hewes: Yes. I just in terms of the cadence of the data, we’re going to need to see how the enrollment goes CIndU is a relatively rare form of the disease, and we’ll update to you all as we know more about how the enrollment is going. And as I stated earlier, in terms of the bar, I think they’re going to have to stay tuned to — over time to really understand the full efficacy of 808 in these diseases because what we’re really trying to do is learn the optimization of the regimen and which symptoms matter most to the patients and how quickly we can resolve these with various dosing regimens of 808.

Kate Haviland: I think for the allergic ARC study, again, we’re watching enrollment there. The study is up and running. And again, we hope to have some data by the end of the year, and we’ll kind of keep you guys posted on both of those as we continue to execute those programs.

Operator: Thank you. Your next question comes from the line of Brian Cheng with JPMorgan. Your line is open.

Brian Cheng: Hey, guys. Thanks for taking our question. Can you elaborate a little bit more on the drivers behind the flat growth ex US this quarter? Is the flat growth there, driven partly by the negotiated price? Thank you.

Kate Haviland: Thanks, Brian. I think as Christy was mentioning, the underlying fundamental growth in terms of growing patient starts and keeping patients on therapy was exactly where we expected it internationally. As Christy had mentioned, we do get some lumpiness in terms of our distributor markets in particular, and there was some pull forward ordering in those markets in Q4 that really just kind of influence the dynamics between Q4 and Q1. I think what’s most important is that the international growth year-over-year nearly doubled. And as Christy mentioned, we’re just in one — we’re just in Germany right now in ISM, and we’re going to have other of the — more of the larger markets coming online this year. And so it’s flat from a revenue perspective, it’s certainly not flat growth from an underlying fundamental demand perspective.

Operator: Thank you. The next question comes from the line of Reni Benjamin with Citizens. Your line is open.

Reni Benjamin: Hey. Thanks guys for taking the questions, and congratulations on the quarter and the raise of guidance. I’d love to just learn a little bit more about the metrics you guys used to gauge the success and failure of the DTC advertising strategy. Those tend to be quite costly and I’m curious as to how you evaluate that. And I think Philina you mentioned that there’ll be promotional efforts that you’re going to employ the dermatologists and gastroenterologists. Wanted to see how big that physician pool was and whether this would increase the total pool of diagnosed patients or that 25,000 that you mentioned already is taking into account the dermatologists and gastroenterologists.

Kate Haviland: Yes. Thanks Reni. I mean all DTC is not the same, and we certainly have a more targeted approach here but Philina, do you want to talk a little bit about what we do from a DTC perspective. And then also just kind of the universe of additional specialties we’re looking to target.

Philina Lee: Yes, absolutely. I would say that, our direct to patient efforts are really focused on two things. The first is increasing the awareness of AYVAKIT as a new treatment option and the second is really creating opportunities for patients to hear from the very positive experiences of other patients who have benefited from AYVAKIT. And so we use a lot of metrics. I think it really comes down to the growth in awareness in AYVAKIT non-users. Obviously, we’ve seen the number of users increase but continue to drive the awareness of AYVAKIT using our direct to consumer ads. We are, I think importantly, executing on these initiatives in a highly targeted way for this rare disease market. And some of the — I think most resounding method metrics we’ve seen is more patients going into offices asking about AYVAKIT as well as the growth that we’re seeing in the patients who are starting.

To your question about dermatology and gastroenterology, I would say it’s a bit of both things that you mentioned. The first is, there’s already an untapped opportunity of already diagnosed SM patients being treated by these additional specialties. And secondly, we would actually expect us to grow the treatment rates over time which can lead to some of that longer term sustained growth.

Operator: Thank you. Your next question comes from the line of Ami Fadia with Needham. Your line is open.

Ami Fadia: Hi. Good morning. Thanks for taking my question. If you think about the sort of different buckets of physicians the germ and gastros, the allergists and the hemats [ph]. Can you give us a sense of what’s the mix of the patients that are being treated by each specialty and maybe give us a sense of your penetration in terms of region frequency, where you are today and then how you see that evolve with this expansion into the germs and gastros data this year? Thank you.

Kate Haviland: Philina, do you want to talk a little bit more about SM — any specialties?

Philina Lee: Yes. So I’d say, there are a number of SM patients being treated across all of these specialties today. It’s our conviction the market opportunity that’s really triggering us, I think to invest in expanding the field force which enables us to increase reach and frequency across the allergists and hemats where we’ve targeted primarily to-date as well as expand into the germs and GIs. I would say there’s incredible headroom across all of these specialties to continue the growth. Most of the prescribing to-date has of course, been in allergist and hemats. One of the things we’ve been really pleased to see is that the growth of breadth of prescribing has actually been faster into allergy and we know this is really important to capture that long-term opportunity.

And again, for germs and GIs we are just getting started and we know that they are both treating an already diagnosed number of SM patients but there are also patients coming in who can increase that diagnosis rate. They’re presenting with cutaneous mastocytosis in the dermatology offices, or things like IBS with some other signal of systemic involvement in the GI office.

Kate Haviland: Maybe one thing I’ll just add is that, as we think about AYVAKIT, it really is the opportunity that the long-term safety data that really lowers the bar as people think about the patients who are most — who could really benefit from the treatment. And so we all use these, kind of, measures of TSS score and we try to classify patients as moderate, mild or severe all these things that’s really a regulatory tool. And what we see out in clinical care is it’s about a patient and whether or not they are well controlled, and whether or not they can do the things they want to do, go to work, participate in family events and what we know is that there’s a lot of patients who cannot, across all of these specialties and that’s really the clinical context in a commercial setting that is very different than a context that we need for a regulatory approval.

And so I think the fact that AYVAKIT is so well-tolerated, we have three years now plus data, really makes us believe this is the right moment to be continuing to expand in allergy and moving into GI and derms who we know will have — will be very positively receive that clinical long-term profile.

Operator: Thank you. Your next question comes from the line of Peter Lawson with Barclays. Your line is open.

Unidentified Analyst: Hey, good morning. It’s Alex [ph] on Peter. Thanks for taking the question. Just a quick one on the new field force you’ve hired, could you quantify that relative to the most recent or I guess existing sales force? Thank you.

Kate Haviland: So you want to talk about the size increase.

Philina Lee: Yeah. So this is an incremental field force expansion that will enable us to both increase our reach and frequency on the current prescriber base, as well as to expand to these other specialties. But importantly we’re able to do this in an incredibly targeted way, leveraging the strength of our analytics to know where patients are engaging most frequently across these specialties. I think the key piece underlying all of this is like most of this market we can see resides outside of hematology, and so this is an important lever for us to continue to drive growth in allergy, as well as moving into other specialties.

Operator: Thank you. Your next question comes from the line of David Dai with UBS. Your line is open.

David Dai: Great. Thanks for taking my questions. I just wanted to drill down on the a prescriber Base, last you mentioned that as you’re explaining to the dermatologist and the GI prescribers, How should we think about the compliance rate of these patients given that these patients could have moderate symptoms.

Kate Haviland: Yeah. David, that was a little bit too what I was mentioning is that it’s — the idea of kind of milder symptoms is not really the pull-through here. In a clinical care setting, it’s patients who do not have enough control over whatever their symptoms may be to be able to do the things that they want to do in life. And so really in the commercial context that is the context. It’s around — is a patient well-controlled or not, and so the rubric of patients being more severe versus less severe across specialties is really not relevant for the commercial setting like it is for a clinical development setting.

Philina Lee: The only thing I would add to that is that our commercial compliance across the board has been exceptionally high, which I think just speaks to the profile AYVAKIT once the patients makes the decision to start on therapy, we see patients doing really well. They’re staying on therapy, they’re highly compliant and they’re sticky.

Operator: Thank you. Your next question comes from the line of Judah Frommer with Morgan Stanley. Your line is open.

Judah Frommer: Hi. Thanks for taking the question. Just to follow-up on the urticaria indication for 808, I’d be curious how you’re viewing unmet need [indiscernible] and CSU, whether the Dupixent approval in CSU affects that and whether risk benefit profile could be viewed differently by derms versus allergists. Thanks.

Becker Hewes: Hi. Yeah. So just first of all, I think that in most of these diseases, having a small molecule daily oral solution is really what patients are looking for. And so we were pleased to see that Dupixent had the activity that it did, really helping everyone understand that this is a chronic inflammatory disease, but we still believe that addressing the mast cell directly, which is the driver of the disease is the right way to approach it and that a small molecule will be the preferred solution.

Operator: Thank you. Your next question comes from the line of Sudan Loganathan with Stephens. Your line is open.

Sudan Loganathan: Hi. Good morning. Thank you for taking my question, and congrats again on the sales results for AYVAKIT. My question is regards on the progress with elenestinib, BLU-808 and [indiscernible], your first protein degrader programs, how is your OpEx spend breakdown between the commercial and development programs prioritized? Is the profitability and free cash flow in the cards potentially this year? Or could we expect any of those earnings from AYVAKIT to be reinvested into the pipeline?

Kate Haviland: Yeah. So Mike, do you want to take that?

Mike Landsittel: Yeah, this is Mike. Yeah. So I mean, as we’ve talked previously, like capital allocation and prioritization has been one of our key priorities really over the last couple of years to make sure that we are investing in what we see are the greatest opportunities to drive top line growth, both now with AYVAKIT and in the future with pipeline. And so we’ve been really disciplined on both being able to make sure that we’re investing appropriately in AYVAKIT, but also targeting within our pipeline where we think we have the greatest potential to drive growth. So it’s not so much like we’re expecting to see modest increases, as I mentioned, in both SG&A spend and R&D spend because we’ll be investing in both areas to drive, again, both near-term and long-term growth.

And I think specifically, we don’t typically break down like within the pipeline where that program spend is going. But clearly, elenestinib and 808 are going to be the top priorities in the near term to drive that.

Kate Haviland: And one thing I’d just add is that I think we have a really good track record here at Blueprint as — in terms of our business development strategy. And I think we are very committed to maintaining a durable financial profile, making sure we’re sustainable and able to kind of invest in the highest prioritized drivers of growth. And if there is — if there are programs that either we believe we would benefit from a partnership in terms of execution and/or maybe not our highest strategic priority, we have a track record of having out-licensed those. And for instance, just recently, we put a program in the hands of IDRx, and we just received $80 million at the beginning of this year due to that transaction. And that was a program that we chose not to move forward ourselves, but certainly need — was beneficial to move forward in someone else’s hands.

So we will continue to look at using business development to enable us to, first and foremost, meet our corporate strategic interest — and then secondly, to make sure we’re maintaining that really sustainable financial profile.

Operator: Thank you. That’s all the questions we have time for today. Kate Haviland, I will turn the call back over to you.

Kate Haviland: So thank you, everybody. We are off to a very strong start here in 2025 with AYVAKIT firmly on the path to realizing its multibillion-dollar peak opportunity. We’re advancing our pipeline. We have the assets in place. We have the strategy in place to really achieve our goal of fundamentally shifting the way allergic inflammatory diseases are treated by targeting the mast cell. So we thank you all for your continued support of Blueprint Medicines, and we invite you to continue to follow our progress throughout this year.

Operator: Thank you. This concludes today’s conference call. You may now disconnect.