Blueprint Medicines Q1 2025 Earnings Call Transcript

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May 1, 2025
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Operator

Good morning. My name is Angela Lang, and I'll be your conference operator today. At this time, I would like to welcome everyone to the Blueprint Medicines' First Q twenty twenty five 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 Thank you. Jenna Cohen, you may begin your conference.

Jenna Cohen
Jenna Cohen
Vice President of Investor Relations and Global Business Communications at Blueprint Medicines

Thank you, Angela. Good morning, everyone, and welcome to Blueprint Medicines first quarter twenty twenty five 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 Investors section of our website at www.blueprintmedicines.com. Joining me today are Kate Havilland, Chief Executive Officer Celina Lee, Chief Commercial Officer Becker Hughes, Chief Medical Officer and Mike Lansidol, Chief Financial Officer.

Jenna Cohen
Jenna Cohen
Vice President of Investor Relations and Global Business Communications at Blueprint Medicines

Fuad Namuni, President of Research and Development and Kristi Rossi, Chief Operating Officer, are also on the line and available for Q and A.

Jenna Cohen
Jenna Cohen
Vice President of Investor Relations and Global Business Communications at Blueprint Medicines

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'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
Kate Haviland
President & CEO at Blueprint Medicines

Thank you, Jenna, and good morning, everyone. We at Blueprint Medicines 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 executional momentum in 2025. This quarter, we achieved 61% year over year AYVAKIT revenue growth as we continue to capture the substantial and growing multibillion dollar commercial opportunity in systemic mastocytosis, which we expect will drive our global revenue growth well into the next decade.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

With every additional quarter performance, AYVAKIT progresses on the path to our goal of $2,000,000,000 in revenue by 02/1930. The underlying fundamental demand driven by growth in 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. Selena will go into this more specifically in a moment. We also advanced our industry leading pipeline of mast cell directed investigational medicines.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Our two prioritized pipeline programs elenestadem and BLU-eight zero eight have the potential to drive significant upside value for BLUEPRINT as they progress in the clinic this year and beyond. With BLU-eight zero eight, 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-eight zero eight'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-eight zero eight 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. Elanestinib, 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 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 U.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

S. 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 twelve 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.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

With that, I will turn it to Felina to discuss the quarter's commercial performance in more depth.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

Thanks, Kate. In the first quarter, we achieved $149,400,000 in AYVAKIT global net product revenues with a hundred and 29,400,000.0 in The US and 20,000,000 ex US. Our strong commercial execution drove revenue growth in what we expected to be a challenging quarter due to the typical q one financial headwinds inherent in our industry. Let's walk through the headwinds and tailwinds.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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 U.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

S. 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 q one 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%.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

As a result of these favorable dynamics, today, we are raising our guidance range to 700 to $720,000,000 for the year. We are exactly where we wanna 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 $2,000,000,000 in AYVAKIT revenue by 2030 and a $4,000,000,000 peak SM franchise opportunity. SM diagnoses are increasing globally and with approximately twenty five thousand diagnosed SM patients in The US. We know we're in the early innings of market penetration.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

Roughly 20% penetration of currently diagnosed patients in The US alone gets you to $2,000,000,000 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.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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 to started to deploy promotional effort.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

The vast majority of providers view AYVAKIT's profile as favorable. Since launch, we've been targeting hemochs and allergists. Hemochs treat less than a third of all 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.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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 have just hired new team members who are onboarding and training. They bring a diverse set of experiences with an emphasis on allergy, dermatology, and rare disease.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

They'll be in the field by 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 ninety five percent of patients saying they're highly satisfied with AYVAKIT as a treatment for their SM.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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 and 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 $2,000,000,000 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.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Thanks, Felina. First, I want to talk about AYVAKIT's long term data in systemic mastocytosis that we presented at Quad AI and that Felina 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.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

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 remained low throughout three years. And importantly, only three percent of patients discontinued due to treatment related adverse events over this extended time frame. The twenty five 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 ninety percent of patients who start at twenty five milligrams stay at that dose.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

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 appropriate in this disease. The PIONEER study included patients with very high disease burden. We've enabled more severe patients to dose escalate to fifty 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 twenty five milligrams.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

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 in PIONEER who titrated their dose to fifty milligrams, ninety three percent of patients saw improved or stable TSF benefit at fifty 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.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

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-eight zero eight. 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.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

We know that a strong safety profile is critical in the field of allergic disease. And with BLU-eight zero eight, 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 rhinoconstruccivitis and one in chronic urticaria. We will be initially testing doses between one and six milligrams.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

This covers the I c ninety 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-eight zero eight. Our initial twelve week phase 2a study will include two key parts, an open label study of patients with chronic inducible urticaria or Syndu and a randomized double blind placebo controlled study of patients with chronic spontaneous urticaria.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Both parts will include multiple doses and dosing regimen. Our goal is for BLU-eight zero eight 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 rhinoconstrunctivitis. We see this as a way to demonstrate the activity of BLU-eight zero eight in the respiratory tract. This is a twenty eight day placebo controlled challenge study patients are exposed to allergen then treated with BLU-eight zero eight or placebo and then re exposed to compare their before and after treatment reaction.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

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 Sindhu 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 doctor Matt Giannetti, the leading expert in mast cell diseases at the Brigham and Women's Hospital here in Boston. Boston.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Results from these proof of concept studies will help guide BLU-eight zero eight 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
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

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 $149,400,000 from net product sales of AYVAKIT. Based on the positive fundamentals that Folina discussed as well as the additional insight we gained around free goods in Q1, we are raising our AYVAKIT net product revenue guidance to 700,000,000 to $720,000,000 for the year.

Mike Landsittel
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

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 our 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 and D expenses related to elanestinib and BLU-eight zero eight clinical studies. SG and 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 and D and SG and A expenses as we invest in our priority pipeline programs as well as increased investment in our sales and marketing efforts for AYVAKIT, as Felina spoke to in detail earlier on the call.

Mike Landsittel
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

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 $900,000,000 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?

Operator

Thank you. Your first question comes from Mark Fram with TD Cowen. Your line is open.

Marc Frahm
Marc Frahm
Biotechnology Equity Research Analyst at Cowen

Thanks for taking my questions. First on the commercial side, can maybe frame the type of rebound in, take growth or acceleration growth you might see in Q2, given the fact that the goods is already so low, which we can maybe limits some of the kind of typical Q1 to Q2 rebounds that we see across the industry. And then on the R and D side, for the eight zero eight, just talk to the strategy on the dose selections that you put into those. The two doses supposed to really kind of test that intermediate range with the titration or are you trying to, you know, maybe with the top dose really show the full power of eight zero eight and ability to get close close to it not matching the antibodies?

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Yeah. Thanks, Mark, for that question. Maybe, Christy, do wanna talk a little bit about how we've considered kind of the fundamental growth and and, the drivers of demand and as we think through the year? And then maybe, please add color. Becca, do you wanna you can add into the the question on the pipeline. Yeah.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Sure. Hey, Mark. So, you know, 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, you know, growth, which is really what we expected when we when we set guidance at the beginning of the year as well. We always knew that q one was going to be, challenging for all of the reasons that Selena said, but, you know, what we expected to see was, you know, strong underlying growth in terms of those drivers of the longer term potential for AYVAKIT which includes patient starting, persistence duration, etcetera.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

That's exactly what we've seen. That's played out in line with our expectations. So if you kind of play that forward through the year, we would expect to see growth in the remaining quarters. You know, the one wrinkle we saw in q one around sort of a shorter quarter from an ordering perspective, I believe, if I'm not mistaken, is made up actually q three interestingly enough. But, you know, we we definitely expect to see, you know, nice steady growth as we go through the remainder of the year.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Let's say in in our results this year, really, our big car flip in q one was around how we're gonna see free goods play through. And I know, Selena, do you wanna talk a little bit more about that?

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

Yeah. 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 q one, like, this is a big variable that has been tremendously derisked, and that has been one of the the key drivers for our guidance update together with the expected growth in the underlying fundamentals, I e, growth in patients on therapy as as Christy just said. So I think at this point, thinking about the free goods trends, looking for the rest of the year, certainly, you know, the the range has narrowed. We have a better sense of the the range to expect.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

You know, we're below 10%. We don't expect, this to be able to really be able to go appreciably down further. 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 you know the trajectory that we're seeing in q one we firmly believe puts us on pace for our guidance as well as marching towards that 2,000,000,000 by 02/1930. Becca do you want to talk about the MARC second question?

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Sure. Let me talk about the dose range of BLU-eight zero eight and how we selected this and what our strategy is here. Just a reminder that we do have all three regimens, one which 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.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

And I think it's important to remember that in many diseases including chronic urticaria, there are multitude of symptoms. And really driving the relief of the patient at the right dose is what And we're doing it in a way that the antibodies really can't do. So we're gonna 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 otocaria and in other indications where we get the optimized dosing for each of these indications.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

As we said earlier, the one to six milligrams gives us a range that covers the I c ninety well, and we all mast 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
Laura Prendergast
Vice President at Raymond James

Hey, guys. Congrats on the progress this quarter. Just maybe to reiterate that question, you know, what exactly have you guys baked into the guidance? You know, 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, you know, when should we expect that?

Laura Prendergast
Laura Prendergast
Vice President at Raymond James

And is any, l and s and if data priced into the 2,000,000,000 by 2030 sorry. The the 2,000,000,000 by 2030, is that pricing in l and s and is being on the market? Thank you.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Thanks, Laura. Just as a reminder, everybody, let's try to keep it to one question just given the number of of people we have in the queue, but, you know, we will try to handle some other things offline if we can.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Maybe, Kristen, you or Christy, 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 LNSNIP perspective in the 2,000,000,000 by 2030 that's completely driven by AYVAKIT. And we expect AYVAKIT to grow beyond that, to be honest.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

So so just so I can take that one. Christie, do you wanna talk about guidance?

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

Sure. So, you know, 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?

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

So, you know, the the two, you know, biggest determinants in my mind of AYVAKIT getting to 2,000,000,000 by by 2030 and the $4,000,000,000 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 those are the two, you know, big drivers, and, you know, we expect to see, you know, growth in new patient starts as we go through the year based on our expanding, breadth and depth of prescribing that we're seeing in The US, an increasingly activated patient base, etcetera. This is obviously, you know, still in the context of our disease market.

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

You're gonna see fluctuations there, but the overall trend line is is, you know, clear and and positive. We expect to see strength in in duration of therapy as patients are staying on therapy for, you know, we think three plus years potentially, multiple years here. And so, you know, those are two of the most important variables as we go over time. Then there's a number of other things that, you know, actually may have more impact in the short term. And so, you know, we we think about gross to net, free goods rates, you know, etcetera.

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

And then, of course, our international business coming along. So those are those continue to be the most important variables. You know, as Selena said, we had a major, I would say, flip in the first quarter by understanding how a very large base of patients who are now on therapy as they went through the reverification process, understanding how that free commercial mix is going to play out. We knew it was going to be important. Knew that was going to be a big swing as we thought about top line revenue. 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 mentioned that really again portend that longer term potential. So those are still gonna be the things that we'll continue to watch.

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

There's ranges of outcomes on all of those on all those variables, but, you know, pleased to see that we were able to to raise the guidance off of off of q one performance. In terms of quarter to quarter performance, we know our business. You talked about things like seasonality, etcetera. We've we've you know, we're now far enough into this launch where I think we have a good sense of, you know, how these factors will play out in terms of impacting quarter to quarter revenue. We've baked that into the guide, and in fact, we baked it in, you know, when we started the year and and expected q one to kind of play out as it did.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Becca, do you want to talk a little bit about Harbor? Obviously, we're just getting this up and up and running now.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

So Yeah. So we're we're just starting the study. We have a highly motivated group of investigators, and 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 line 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.

Paul Jeng
Vice President at Guggenheim Partners

Hey, this is Paul on for Mike Cola. Thanks for taking our question. Just on etiquette, so for the ex U. S, 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?

Paul Jeng
Vice President at Guggenheim Partners

And then maybe a quick follow-up on dose escalations that you mentioned in ISM patients, your quad AI data suggested around a quarter escalated to fifty milligrams. What's your current visibility into what percentage of commercial patients have moved 50 for ISAM? Thank you.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Yes. Thanks for the question. And we'll start, Christine, you take international and then, Puno, you talk a little bit about what we're seeing in dosing. One thing I should say is the international team has just done a tremendous job, and their year over year performance more than doubled from q one last year. So we're really excited about where we are.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

But do wanna talk about the q four to q one dynamic, Christine?

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

Absolutely.

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

So right. As as Kate said, you know, the international team has been doing very well. We've talked about the fact that, you know, if we think about just 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?

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

So, you know, the international international team continues to 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, you know, we'll really start to see ISM growth across a number of markets as we head even into next year and and beyond. You know, the the business in Germany is doing quite well.

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

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 of uptake. You know, fluctuations can happen quarter to quarter. There were some, you know, things between q '4 and q one, 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.

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

Right? So these these little factors can obviously, you know, have a role when we look at q on q.

Christina Rossi
Christina Rossi
Chief Operating Officer at Blueprint Medicines

And we've seen that before if you go back and look at international international performance over the last year or so. But, you know, the bigger picture is that business is doing well. German launch is doing really well, and we're, you know, looking ahead and looking forward to having some other ISM launches come online.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

And to the question on dosing, the twenty five mg benefit risk profile, continues to serve the vast majority of patients very well. It's under ten percent of patients that we're seeing who may dose escalate to fifty mg over time, and this is occurring against the backdrop of the profile of the twenty five 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
Kate Haviland
President & CEO at Blueprint Medicines

And just one thing to add to that is that even, you know, what Becker showed you from the the both the safety and efficacy of the fifty mgs is that what we see is that, you know, with AYVAKIT at twenty five milligrams or fifty 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, you know, with with a medicine that really empowers them to to live, and and do the things they wanna do.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

So both the twenty five and fifty have have really come through in a very nice way, for these patients.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

And the other thing to remember is that the PIONEER population was a highly advanced patient population, you know, in the middle of a pandemic, first study out there with a really effective therapy. And so it's probably not indicative of 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
Michael Yee
Managing Director at Jefferies Financial Group

Thanks. Good morning. Congrats on the continued growth. 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 q two, three, four?

Michael Yee
Michael Yee
Managing Director at Jefferies Financial Group

Is it sort of consistent year over year growth each quarter? Are there different things that, impact those quarters? And as part of that, 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
Kate Haviland
President & CEO at Blueprint Medicines

Thanks, Michael. Michael. So I think what you're asking is is how to think about kind of the remainder of the year. You know, obviously, we're really focused here on how we're exiting the year, because that is is what really drives us to that that laser focus on that 2,000,000,000 by 2030. But understand that you guys wanna kinda get a sense of how we're thinking about the quarters.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

I don't know, Mike, if you wanna provide a little bit color on on how you think about that.

Mike Landsittel
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

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 to. And as we've talked about before, like, each quarter can have unique variables in US and international. I think the one thing that Christy mentioned that we, you know, did wanna point to is you think about, like, the growth, especially over, like, q two, q '3, it's like we've we've talked about we've missed an order day in q one.

Mike Landsittel
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

Right? That actually gets made up in q three in the calendar. It's and just for for context, like, our biggest customers tend to order on the same day each week. We had one less of those in q one. It gets made up in q three.

Mike Landsittel
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

So right there, you're gonna see a differential dynamic between q two and q three where you'd say there's probably a little bit more, you know, shifted into the q three period. But, fundamentally, I just I point to, consistent underlying patient growth, and that's been as expected. And we continue to to drive towards that as we as we go for our updated guidance range.

Operator

Your next question comes from the line of Colleen Cousy with Baird.

Colleen Kusy
Senior Research Analyst at Robert W. Baird & Co

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 derms and GIs versus the allergists and hemonics that are your first target prescribers?

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

Colleen. Do you want to talk about how you're thinking about the prescriber mix this year versus next year? 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.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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 over time. You know, our chart shows, you know, we understand very well the dynamics of how a first patient starts and then a positive experience leads to growth and deepening over time. Secondly, you know, 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 catalyze, growth over the course of this year. As we shift into the newer specialties, we 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 germs and gastros.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

I think what we've learned from this market is, you know, hemox alone are only about a third of of the opportunity, so we know it's important to expand. Our objectives with germs and gastros are really the ability to move these patients towards treatment, whether or not they are 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, you know, really in in 2026 and, and beyond. So just for clarity, Flina, we really do expect our primary prescriber base to be that growing base of allergists, our stable base kind of of the hem onc group, which sees us 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
Derek Archila
Managing Director, Biotechnology Equity Research Analyst at Wells Fargo

Hey, good morning and thanks for taking the questions. I just want to understand how durable a sub-ten percent 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
Kate Haviland
President & CEO at Blueprint Medicines

So, Doug, we we don't really talk about kind of the ongoing quarter, but we can certainly talk about how our view of of the the, durability of the free patient goods rate. And and just to note, you know, it's actually well below 10% at this point. So, Selena, do you wanna talk a little bit about how you're thinking about that for the rest of year, the free goods rate?

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

Yeah. I think what we've what we what we're really emphasizing is, we've derisked free we've majorly derisked the variable of free goods by just moving this big base of patients.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

This large number of patients have been able to access commercial therapy in q one, 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, you know, with with this type of floor, you know, 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, you know, how long will foundation funding continue to be available as well. But I would say it's de risking of the patients who have already moved, which have led to a favorable more favorable than expected upside.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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.

Mark Aleynick
Mark Aleynick
Biotechnology Equity Research at Goldman Sachs

Hey, good morning. This is Mark on for Salveen. Thanks so much for taking our question. You guys mentioned you may show eight zero eight data from the POC studies in Sindhu this year. In your view, what is the bar for eight zero eight in Sindhu and also in allergic rhino conjunctivitis?

Mark Aleynick
Mark Aleynick
Biotechnology Equity Research at Goldman Sachs

And how do you think this will compare to the KIT antibodies here?

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Yeah. I I just, in terms of the cadence of the data, we're gonna need to see how the enrollment goes and do the relatively rare, form of the disease, and we'll update you all as we know more about how the enrollment's going. And as I stated earlier in terms of the bar, I think they're gonna have to stay tuned to over time to really understand the full efficacy of eight zero eight 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 eight zero eight.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

I think for the, the allergic, the ARC study, Again, you know, we're we're 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 we'll kinda keep you guys posted on both of those as we continue to execute those those programs.

Operator

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

Brian Cheng
Brian Cheng
Senior Biotech Analyst at J.P. Morgan

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
Kate Haviland
President & CEO at Blueprint Medicines

Thanks, Brian. I think as Christy was mentioning, the underlying fundamental growth in terms of, you know, growing patients on their growing patient starts and keeping patients on therapy was exactly where we expected it internationally. As Christy had mentioned, you know, we do get some lumpiness in terms of our dispute distributor markets in particular, and there were some pull forward ordering in those markets in q four that that really just kind of, you know, influence the dynamics between q four and q one. I think what's most important is that the international growth year over year nearly doubled. And as Christy mentioned, you know, we're we're just in one we're just in Germany right now in ISM, and we're gonna have other of the large more of the larger markets coming online this year.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

And so it's 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 Ren Benjamin with Citizens. Your line is open.

Reni Benjamin
Managing Director at Citizens JMP Securities, LLC

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, Selena, you mentioned that there'll be promotional efforts that you're going to employ to 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, you know, taking into account the dermatologists and gastroenterologists.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Yeah. Thanks, Ren. So, I mean, all all DTC is not the same, and we certainly are having more targeted approach here. But, Selena, do wanna talk a little bit about what we do from a DTC perspective? And then also just kind of the the universe of of additional per or specialties we're looking

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

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.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

I think it really comes down to the growth in awareness in AYVAKIT nonusers. Obviously, we've seen the number of users increase, but continue to drive the awareness of of AYVAKIT using our direct to consumer ads. We are, I think, importantly, executing on these initiatives in a highly, highly targeted way for this rare disease market. And some of the, I think, most resounding 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, 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 Neha. Your line is open.

Ami Fadia
Senior Analyst at Needham & Company

Hi, good morning. Thanks for taking my question. If you think about the sort of different buckets of physicians, the derm and gastros, the allergists and the hemoncs, 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 reach and frequency, where you are today, and then how you see that evolve with this expansion into the derms and gastros data this year? Thank you.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Jacqueline, do you want talk a little bit more about do you want the specialties?

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

Yeah. So I'd say, there are a number of SM patients being treated across all of these specialties today. It's our conviction in the market opportunity that's really, triggering us, I think, to invest in the field in expanding the field force, which enables us to increase reach and frequency across the allergists and hemop, where we've we've targeted primarily to date, as well as expand into the the derms and GIs. I would say there's incredible headroom across all of these specialties to continue the growth.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

Most of the prescribing to date has, of course, been in allergists and hemox. 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 for germs and GIs, we are just getting started. And, you know, we we know that the they are both treating an already diagnosed number of of SM patients, but there are also patients coming in who can 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
Kate Haviland
President & CEO at Blueprint Medicines

Maybe one thing I'll just add is that, you know, as we think about, you know, 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 the treatment. And so we all use these kind of reg you know, measures of TSS score, and we try to classify patients as moderate, mild, severe, all these things. That 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 wanna 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.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

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 continue to expand in allergy and moving into GI and derms who we know will have, will be very, positively received that that clinical long term profile.

Operator

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

Analyst

Hey, good morning. It's Alex on for 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
Kate Haviland
President & CEO at Blueprint Medicines

So you wanna talk about the size increase? Yeah. So this is an incremental field force expansion that will enable us to both increase our our reach and frequency on the current prescriber base as well as to expand to these other other 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. You know, I think the key the key piece sort of 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 Thigh with CVS. Your line is open.

David Dai
David Dai
Director at UBS Group

Great. Thanks for taking my questions. I just wanted to drill down on the AYVAKIT prescriber base. So after you mentioned that, know, as you're extending to the dermatology and GI prescribers, how should we think about the compliance rate of these patients given that these patients are could be them have milder symptoms?

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

Yeah, David. That was a little bit to what I was mentioning is that it's the idea of kind of milder symptoms is not really the pull through here. Right? In in a clinical care setting, it's patients who are just not do not have enough control over whatever their symptoms may be to be able to do the things that they wanna do in life. And so, really, in the commercial context, that is the is the context.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

It's around is a patient well controlled or not. And so, you know, the the rubric of patients being kind of more severe versus less severe across specialties is really not is really not relevant for the commercial setting like it is for a clinical like a clinical development setting.

Philina Lee
Philina Lee
Chief Commercial Officer at Blueprint Medicines

So The only thing I would add to that is that, you know, our commercial compliance across the board has been exceptionally high, which I think just speaks to the the profile of advocate. Once the patient, you know, 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
Judah Frommer
Executive Director - Senior Equity Research Analyst at Morgan Stanley

Hi, thanks for taking the question. Just a follow-up on the urticaria indication for eight zero eight. I'd be curious how you're viewing unmet need in Sindu and CSU, whether the Dupixent approval and CSU affects that and whether risk benefit profile could be viewed differently by derms versus allergists? Thanks.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Hi.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Yeah. So just first of all, I think that in most of these 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
Managing Director at Stephens Inc

Hi, good morning. Thank you for taking my questions and congrats again on the sales results for AYVAKIT. My question is, in regards on the progress with elanestinib, BLU-eight zero eight and post nominate, first approach integrated 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 AVIC get to be reinvested into the pipeline?

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

This Mike, do wanna take that? Yeah. Sorry. Go ahead.

Becker Hewes
Becker Hewes
Chief Medical Officer at Blueprint Medicines

Yeah. Yeah.

Mike Landsittel
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

This is Mike.

Mike Landsittel
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

Yeah. So, I mean, as we've we've talked previously, like, capital allocation and prioritization has been one of our key priorities really over the last couple years to make sure that we are investing in what we see are the greatest opportunities to drive, you know, top line growth, both now with AYVAKIT and in the future with pipeline. And so we've been really disciplined on, you know, both being able to make sure that we're investing appropriately in AYVAKIT, but also targeting within our pipeline where we think we we have, you know, the greatest potential to drive growth. So it's not so much like we're we're expecting to see, you know, modest increases, as I mentioned, in both s g and a spend and r and d spend because we'll be investing in both areas to drive to drive again both near term and long term growth. And I think, you know, specifically, we don't typically break down, like, within the pipeline where that program spend is going.

Mike Landsittel
Mike Landsittel
Chief Financial Officer at Blueprint Medicines

But, clearly, elenestinib and eight zero eight are gonna be the top priorities, in the near term to drive that.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

And one thing I'll just add is that I think we we have a really good track record here at Blueprint at as I don't in terms of our business development strategy. And, you know, 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, you know, prioritized drivers of growth. And if there is if there are programs that, you know, either, we we believe we would benefit from a partnership in terms of execution and or maybe not, you know, or not our our highest strategic priority. We have a track record of having out licensed those and, you know, for instance, just recently, we put a program in the hands of IDRX and, you know, we've just received $80,000,000, at beginning of this year due to that transaction. And that was a program that we chose not to move forward ourselves, but certainly need you know, was beneficial to move forward in someone else's hands.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

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 question we have time for today. Kate Havanan, I will turn the call back over to you.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

So thank you, everybody. We are 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.

Kate Haviland
Kate Haviland
President & CEO at Blueprint Medicines

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.

Executives
    • Jenna Cohen
      Jenna Cohen
      Vice President of Investor Relations and Global Business Communications
    • Kate Haviland
      Kate Haviland
      President & CEO
    • Philina Lee
      Philina Lee
      Chief Commercial Officer
    • Becker Hewes
      Becker Hewes
      Chief Medical Officer
    • Mike Landsittel
      Mike Landsittel
      Chief Financial Officer
    • Christina Rossi
      Christina Rossi
      Chief Operating Officer
Analysts
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Key Takeaways

  • Blueprint reported Q1 AYVAKIT revenues of $149.4 million, up 61% year-over-year, and raised its 2025 AYVAKIT guidance to $700–720 million based on strong patient demand and a favorable free-versus-paid goods mix.
  • The company advanced two mast cell–targeted programs: elenestadem entered the pivotal HARBOR study exploring disease modification and organ healing in systemic mastocytosis, and BLU-808 initiated proof-of-concept trials in allergic rhinoconjunctivitis and chronic urticaria with early data expected this year.
  • PIONEER long-term data showed AYVAKIT’s safety profile remained stable over three years with low discontinuation (<3%) and durable efficacy at the 25 mg dose, supporting high patient compliance and satisfaction.
  • Blueprint is expanding its commercial footprint beyond hematology/oncology by adding field teams focused on allergists, dermatologists, and gastroenterologists, while targeted direct-to-consumer and peer-to-peer campaigns are increasing patient activation and new starts.
  • The company ended the quarter with a $900 million cash balance, expects modest increases in R&D and SG&A to support prioritized programs, and anticipates significant year-over-year declines in operating cash burn.
AI Generated. May Contain Errors.
Earnings Conference Call
Blueprint Medicines Q1 2025
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