Blueprint Medicines at H.C. Wainwright Conference: Strategic Pipeline Expansion

On Thursday, 27 March 2025, Blueprint Medicines (NASDAQ: BPMC) presented at the H.C. Wainwright 3rd Annual Autoimmune & Inflammatory Disease Virtual Conference. The company outlined its growth strategy, highlighted its robust pipeline, and shared its financial outlook. While focusing on international expansion and innovative therapies, Blueprint Medicines also addressed challenges in market competition.

Key Takeaways

• Blueprint Medicines projects revenue between $680 million and $710 million this year, with AvaKIT expected to reach $2 billion by 2030.
• The company is expanding its international presence, with significant contributions from the US and Germany.
• Blueprint’s pipeline includes promising therapies like elanestinib and BLU-808, targeting various allergic and inflammatory diseases.
• AvaKIT’s market strategy focuses on increasing both prescriber breadth and depth, particularly among specialists.
• The company maintains a strong financial position, emphasizing disciplined capital allocation.

Financial Results

• Revenue for the previous year was $479 million, with a guidance of $680 million to $710 million for the current year.
• AvaKIT is forecasted to achieve $2 billion in revenue by 2030, contributing to an overall peak opportunity of $4 billion across the franchise.
• International business accounted for 10-15% of top-line revenue in 2024, highlighting the importance of global expansion.

Operational Updates

AvaKIT Commercial Strategy

• Blueprint Medicines is focusing on expanding its prescriber base and deepening utilization among existing prescribers.
• Efforts are underway to engage hematology, allergy, and dermatology specialists.
• International expansion includes approvals and reimbursement in the US and Germany, with additional EU markets expected soon.

Elanestinib Development

• The HARVER study has been initiated, targeting disease modification and improved bone health.
• Elanestinib aims to shift the treatment focus from symptom management to disease modification.

BLU-808 Development

• Preclinical data has been promising, with several proof-of-concept studies planned in conditions like urticaria and asthma.
• The therapy’s pharmacokinetic profile is optimized for once-daily oral dosing, with confirmed potency and tolerability.

Future Outlook

AvaKIT Market Penetration

• Blueprint Medicines aims to sustain AvaKIT’s leadership by promoting its efficacy and safety over symptom-directed therapies.

Elanestinib Market Strategy

• The therapy is positioned as an option for new patients and those switching from AvaKIT, with a focus on expanding the patient base.

BLU-808 Potential

• The company plans to explore the therapy’s profile in large patient populations, with the potential for multiple treatment approaches.

Q&A Highlights

• Blueprint Medicines continues to see growth in diagnosed patients and international expansion as key revenue drivers.
• Elanestinib is expected to impact the long-term disease course, particularly in bone health for ISM patients.
• BLU-808 is being developed with a focus on chronic urticaria and other allergic conditions, with promising preclinical data.

For a detailed overview, please refer to the full transcript.

Full transcript - H.C. Wainwright 3rd Annual Autoimmune & Inflammatory Disease Virtual Conference:

Unidentified speaker: one as we head deeper into 2025.

Christina, Blueprint Medicines: Great. Thank you. So thank thank you, Andres, and thank you to HC Wainwright for for having us. Blueprint Medicines is a growing independent biotech company with a leadership position in mast cell biology, that we are leveraging first to drive, a really compelling commercial opportunity in a disease called systemic mastocytosis, through the launch of AvaKIT, which is our our flagship medicine that was approved for, indolent systemic mastocytosis about, eighteen months ago. We are, in the middle of a compelling launch where we’ve been driving significant revenue growth.

We achieved $479,000,000 in revenue last year, and have guided to 680 to $710,000,000 in revenue this year, against what we see as a opportunity that will get Avakit to $2,000,000,000 in revenue by 02/1930, and an overall peak opportunity across our franchise of $4,000,000,000 So we think, the first value driver is really around our launch in in systemic mastocytosis and, continued commercial execution with Avakits. We have a next generation, product for systemic mastocytosis, elanestinib, which we are, also bringing forward and have just initiated, registration enabling study called HARVER. And we’re bringing that forward to really expand and extend our franchise into the next decade and beyond. And so we’ll continue to deliver innovation in SM. And then beyond that, we’ve leveraged our capability in mass cell biology and the experience that we’ve built over over several years, to develop BLU eight zero eight, which is an oral wild type kit inhibitor, which has the potential to address a really broad range of, highly prevalent diseases that are driven by mast cells.

So BLU eight zero eight, we just reported healthy volunteer data at the beginning of this year, where, you know, the profile really started to demonstrate, our hypothesis, which was that we could develop a highly tunable, potent, selective oral wild type inhibitor, where we could really tune dosing to be able to, address symptoms and also manage, on tar on target side effects. So we are looking forward to continuing to bring BLU eight zero eight forward. We have several proof of concept studies planned this year, to better understand the profile of of the therapy as well as the role that mast cells play in disease biology and several important allergic and inflammatory conditions. And then, of course, beyond BLU eight zero eight, we have a robust pipeline. I’m joined by Percy Carter, who’s our CSO.

We have a long track record of delivering scientific innovation through our discovery engine. And so, you know, continue to invest in in that innovation and look forward to bringing, you know, other potential therapies for beyond 08/2008. The last step point I would make is is that our successful launch of Avakit along with a really disciplined approach to capital allocation have put us in a very, very strong financial position where our operating cash burn has been, coming down substantially. And we’re really in a position where we control our own destiny in terms of, being self sustaining and able to advance our priorities forward based on on the strong financial profile that we have.

Unidentified speaker: Great. Thank you for that, great overview. So starting with Avakit, maybe one question on The US market growth strategy. You know, with prescriber base, in breadth increasing, you know, is the next phase of growth focused on expanding to new prescribers or deepening of utilization? How should investors be thinking about that opportunity there?

Christina, Blueprint Medicines: Yes. I think there’s substantial opportunity on both fronts. So what we have seen through through the launch of AvaKit is very steady growth in first breadth of prescribers. So new prescribers coming on board, treating their first ISM patient, getting experience, with the profile of Avakit. And this is on the backdrop of a rapidly expanding patient base and market opportunity where we’ve seen the number of diagnosed patients continue to grow at double digit rates.

So the market is growing as we launch the drug. Prescribers are having more and more patients coming into their into their offices. So growing breadth is something that we think is going to continue to be important, in the near term. We’ve seen growth, both in hematology as well as importantly in allergy. And our initial strategy was very much focused on those two specialties, which tend to play an important role in managing indolent systemic mastocytosis.

What we have seen in a in a very, I would say, consistent way is that with breath then comes depth. So depth that follows breath. Once somebody has their first prescription, they tend to find more patients and prescribe more deeply within their practice. And there’s really two factors that drive that. One is increasing comfort with the profile, increasing comfort managing ISM, which leads to a widening lens on, you know, who an appropriate advocate patient is.

And so we see prescribers looking within their existing pool of patients and going deeper in terms of who they who they, initiate therapy with. We also see that, again, because more of these patients are being diagnosed and patients are finding their way to experienced providers that, you know, patient pools are broadening within within, health care providers who have experience treating ISM. And so I would expect both breadth and depth to continue to grow. One important strategy for us as we go forward is, you know, in addition to continuing to drive that that growth in breadth and depth within allergy, particularly, as well as, hematology, we are increasingly seeing other specialists, who who see these patients and manage them expressing an interest in in prescribing. And so dermatology is an example that, you know, we’ve used recently.

We’ve engaged with that specialty much, more significantly over the past, year. We’re expanding our field footprint this year to enable us to go even deeper there. And they, you know, they tend to play a role diagnosing these patients and have, expressed interest in managing. So we think that’s a really, compelling opportunity for us to continue to drive growth over the long term.

Unidentified speaker: Great. Very helpful. And then regarding the experience you have garnered in The United States with the successful launch in both of these indications, how should investors be thinking about ex US growth and how can you leverage what you’ve learned here in The United States to really expand that global footprint in the years to come?

Christina, Blueprint Medicines: Yeah. We’re, continuing to to grow, the footprint of Avakit and ISM globally. I do think our international expansion is an important driver of top line revenue growth as we as we continue to go forward. Right now, Avakit is approved and reimbursed, obviously, in United States and then Germany, where we have reimbursement, in ISM. We have reimbursement in advance SM and a number of other markets that are are still, executing, you know, launches outside The US, in advance SM.

But ISM, we expect to have additional markets coming online this year, mostly in Western Europe, some of the other major EU markets as we work through pricing and reimbursement, negotiations. The market dynamics obviously vary from market to market, but there are some things that are pretty consistent. The epidemiology looks similar as we think about Europe versus The United States. So, patient opportunity as you normalize for the population is, you know, looks similar and is significant outside The US. Treatment approaches, are more similar than different.

So there’s a lot of, a lot of our learning in The United States that we can leverage as we as we launch outside The US. Our international business in 2024 contributed, you know, roughly was sort of in that 10 to 15% of top line revenue depending on the quarter. And as we think about this year, I would expect it to continue to sort of be in that range. So we’re growing revenue globally. We’re growing it in The US.

And then outside The US, you know, we expect to see additional growth as well.

Unidentified speaker: Very helpful. And then, you know, one question on competitive positioning in the future differentiation, you know, as new therapies, you know, enter the SM market, could you highlight for investors, you know, how AvaKitt’s, you know, how are you planning on sustaining AvaKitt’s, you know, leadership position in this in this market, and, you know, what are the key drivers for maintaining that long term growth?

Christina, Blueprint Medicines: Yes. We are, you know, at the very beginning stages of penetrating the ISM opportunity. You know, we have still a minority of patients being treated. And as I mentioned, the market itself is growing underneath the launch, which, you know, is is in some ways what you would hope and expect to see with a rare rare disease disease like this where the advent of effective therapy catalyzes diagnosis and treatment. So our focus and objective this year, and I suspect that will be the case, for, you know, several years to come, is to continue to catalyze treatment with disease modifying therapy.

The biggest, you know, competition to AvaKit is symptom directed therapy only. And and that is really the discussion that we’re having is is the benefits of treating with a disease modifying therapy. You know, Avacad has a profile that sets an incredibly high bar. You know, we had very compelling efficacy in Pioneer. We’ve seen that efficacy sustained as we’ve, you know, followed patients out now over the long term.

We just presented data, recently at Quad AI with a median of three years of follow-up with some patients significantly longer than that, where we’re seeing sustained efficacy. And what’s really, really important in this market is safety, and being able to see very consistent safety tolerability, no adverse events coming up, for the first time that are different. The profile has very much held. And that’s been as we’ve just talked to, you know, particularly community allergists who are seeing the majority of these patients, that is such an important driver. And so, you know, we think we’re really well well positioned there.

When patients start on Avakit, we’re seeing very positive trends in terms of staying on therapy and also patients are very, very compliant. And so, you know, again, I think, the bar we’re setting is high, and I think it’s gonna take the differentiated strategy that we’re bringing forward with alanestinib, frankly, to to challenge, Avakit’s, you know, sort of position on the market.

Unidentified speaker: Absolutely. And that’s a perfect segue to my next question, wanted to highlight to investors. It’s not, surprise, surprise, it’s not all about Avakit. Mhmm. The company has a robust pipeline.

So moving on to maybe elanestinib, which is the next generation KIT inhibitor. So it would be great to set a baseline about some of the critical differences between elanestinib and AvaKIT for investors in terms of efficacy and safety profiles that we should expect, and maybe even, commentary on patient selection, how certain populations might be more amenable here.

Christina, Blueprint Medicines: Great, happy to happy to start. Percy may wanna chime in on this one as well. You know, from a chemical matter perspective, elanestinib is also a very highly potent selective, KIT D sixteen b inhibitor. So we’re starting with raw material that I think gives us a lot of optionality to be able to, you know, develop this in the right way to to impact the disease. As we have developed Avakit, learned a lot about ISM, we really challenged ourselves to think about how to develop l anosinib in a way that is differentiated.

Because as I said before, the bar that Avakit’s setting from a product profile perspective is is a very high bar. And so, you know, we need to think about innovation in terms of how to impact the disease. We know a lot more about ISM now than we did when we first started pioneer. And so the way that we’re thinking about, development for elanestinib is to really shift the narrative from, addressing symptoms, which is incredibly important. And, of course, these patients need need a therapy that can effectively address symptoms, but also modify the longer term course of the disease.

And so what we what we know is that, you know, ISM can have, really significant impact on patient morbidity, in some cases, mortality over time. And so some of the, you know, endpoints that we’re exploring with Harbor are really, providing a, you know, solid base of evidence around the ability to impact that over the long term. So bone health is one aspect of this disease that has become, really, really important for providers and patients. You know, forty to sixty percent of ISM patients, if not more, may have osteoporosis, osteopenia. These are often young patients.

You hear stories of patients presenting with fractures, where maybe the disease hasn’t even been identified, and that can be the presenting symptom. And so bone health in some ways is a marker for the the impact that ISM can have, you know, really on patients’ long term health and wellness. And so, you know, looking at the ability to impact that, we think, is gonna be important. We have some data suggesting even Avakit, does impact, bone. We we presented that at at Quad AI, but we’ll be looking at this in a much more rigorous way with elanistinib again to really speak to that disease modification profile.

We’re looking at other endpoints around allergic reactions, etcetera. So it’s really about, a differentiated clinical profile that we will bring forward versus, you know, redoing the pioneer study and, you know, kind of coming out with a dataset that we ultimately don’t think is gonna be enough to to really differentiate over the long term.

Unidentified speaker: Great. So maybe digging into a little bit of more of the nuance in El Anestin, could you maybe from a mechanistic potential for improving bone turnover and anaphylaxis reduction and compared to Avakit?

Christina, Blueprint Medicines: Yeah, that may be a good one for, Percy, you want to speak to?

Percy Carter, CSO, Blueprint Medicines: Sure. Yes. So the what I would highlight with the molecule is the excellent potency, very high selectivity and excellent distribution properties, which we think give it the sort of fundamental characteristics you know, in the patient context to cover the target safely, and to allow us to see the effects that, we believe will be driven by inhibiting this target, right, as as Christy said at QuadAI, you know, we’ve with Avapritinib, we’ve been able to see sort of initial views, for example, in these new endpoints that we’re going to be studying now more rigorously in this trial. So we’re basically building on that foundational strength we have with Avapritinib with a molecule that we think has the characteristics we need to demonstrate that in

Unidentified speaker: humans. Very helpful. And then one hypothetical for investors who are really projecting out, the potential of elanestinib. You know, if elanestinib does secure a broader label, do you expect it to primarily capture new patients or will it new patients or will it drive switching from Avocat? Can you highlight some of those dynamics that you’re thinking about in this very early stage program?

Christina, Blueprint Medicines: Of course, and and, you know, as as you would expect, you know, as we get closer to actually having data available from harbor, you know, an approval launch, I’m sure we will be talking about all of these dynamics a lot a lot more detail. But at a high level, you know, our strategy allows us to have two options on the market for some period of time. We know that there’s going to be, a large base of patients on Avakit who are doing really well, right, which is great. And elanestinib, I think, could be another option certainly for new patients starting therapy. Over time, you know, you you may see migration of, some of the business to Avakit, really, even just based on new patients coming in.

Again, we’ll be growing the market, over this period of time. We’ll still be you know, we talk about $2,000,000,000 for Avakit in 2030. You know, that, by my back of the envelope math, is maybe 6,000 patients on therapy in The US. And so if you think about, you know, twenty five thousand patients diagnosed today plus, we’re talking about a minority of patients being treated with Avakit even at that level. So I think it will be continuing to grow the market, having new patients come on, and it’ll be another option, obviously, for patients who are on therapy as well.

But we will have, I think, a good amount of time where you’d have both options on the market together.

Unidentified speaker: Very well, very helpful, thank you for that clarity there. So moving on to BLU808, BLUPRINCE’s wild type KIT inhibitor for allergic and inflammatory diseases. Now it would be great to just get an overview of some of the insights you’ve generated from the phase one study that are shaping the dose selection and proof of concept of trials, and just an overall, overview of eight zero eight before we get into more of the nuances.

Christina, Blueprint Medicines: Sure, so I’ll let Percy, speak to this. I mean, a very high level, what I’ll just say is that, you know, as we set out to execute that phase one study, we obviously had sort of a, almost a set of attributes we were hoping to see from the molecule. And in many ways, that dataset exceeded, what we were what we were hoping to see. And and that, you know, initial phase one study, obviously, we need to continue to move it forward and and look at, further proof of concept studies in patients. But do you wanna talk more about the program?

Percy Carter, CSO, Blueprint Medicines: No. That’s great. We can just start out right there actually with Christy’s comments. So when we bring forward a program into the clinic, we have a target product profile. So what we want the compound to do to sort of achieve a certain set of results clinically.

And as Christy just said, I think BLU-eight zero eight really the actual data sort of met all those criteria we had set out. So what were we looking for specifically? So I would say I would call attention specifically to three things. So the first was, of course, the fundamental pharmacokinetic profile. That was a crucial attribute in our optimization scheme, because as you know, in the context of wild type kit, given that there will be target mediated impacts, we wanted to have as close as flat a pharmacokinetic profile as possible with once daily oral dosing.

And the advantage that gives us is, control over dose, dose regimen, as we move forward into these clinical POCs that just gives us a ton of flexibility in terms of how we conduct those trials and what doses we assess. And that’s exactly the PK profile that we showed at JP Morgan and then subsequently. So the second thing, of course, we were looking to see is to confirm our estimates of the potency of the molecule right on the target and again our potency of the molecule right on the target. And again, our pharmacodynamic readouts showed that unambiguously in a fashion that was well aligned with exposure. And so confirming what we had seen in vivo preclinical studies and in vitro preclinical studies, that profile was borne out in human volunteers.

And then the third aspect, of course, in a fundamental underpinning of any Phase one study is to ensure that the tolerability of the molecule is where we expect it to be. And again, this is something we spend a lot of time doing. I apologize for being late, but in fact, the conversation we’re just having is a very different program, but on this crucial topic. And, we were delighted to see that the tolerability profile of eight zero eight, was what we hoped for, in volunteers, giving us the confidence to advance to patient studies for a proof of concept.

Unidentified speaker: Very helpful and a perfect lead to my next question. So, you know, my next question concerns with, you know, competitive positioning in the allergy market and with the caveat of, you know, all these studies are early stage and cross trial comparisons, you know, you need to be very cautious. Can you help us understand, you know, how BLU eight zero eight differentiates from other biologics and small molecules in the allergy and inflammation space to help us maybe sort of navigate some of those pitfalls of cross trial compare comparisons at such an early stage?

Christina, Blueprint Medicines: Sure. Maybe maybe I’ll start generally and, you know, we can definitely go into more more specifics here. I think, you know, if you think about Blue eighty eight, it really is, in my mind, an example of when people say pipeline in a product or pipeline in a pill. This is what what we’re what we’re dealing with, we think, here, where you’ve got a mechanism that could really apply quite broadly across a number of allergic and inflammatory diseases. We are learning more about the role of kit, you know, as really the master control switch of the mast cell and sort of what what, you know, mast cell the role of mast cells in the biology of of a number of these diseases.

And, you know, part of our strategy with our proof of concept studies is to both learn more about the profile of beta weight, but also to learn more about the biology of the disease, particularly indications where, we are still learning. Right? So I think about chronic urticaria is one where there’s a lot of data already to suggest that, you know, KIT plays clearly, you know, important role. Mast cells are obviously an important driver of of disease there. We have data from antibodies, targeting targeting KIT that are are further ahead.

I think when we’re comparing an oral agent versus maybe some of the biologics, you know, there’s obvious benefits to, first of all, having, you know, an oral therapy for for some of these indications where patients are taking therapy chronically.

Percy Carter, CSO, Blueprint Medicines: Mhmm.

Christina, Blueprint Medicines: But also importantly, I think the ability to tune is different. With an oral, Percy can can talk more about this. But with biologic, you’re much more sort of in an on or off type of situation where, you know, we think with with an oral small molecule, we can tune the profile. If you expand the lens beyond, you know, kit and mass cells, obviously, there’s other approaches targeting a number of these diseases. You know, I think, we’ll see as the data plays out exactly how profiles differ, but, you know, what you often see in large immunology markets is that, you know, some patients may respond differently to different mechanisms of action.

We’re dealing with very large patient populations. You know, certainly multiple orders of magnitude, what we’re talking about with ISM. And so, you know, the commercial opportunity here is is pretty compelling, and I think there’s going to be room for for multiple approaches, particularly if you have a a highly effective oral therapy that does have that balance of benefit risk. I don’t know if you want to talk more about the sort of kit space specifically and

Unidentified speaker: Please, please, no. If you’d like to add anything, that’d be great.

Percy Carter, CSO, Blueprint Medicines: I think Christy, covered it very well. I mean, I think in in brief, the only thing I’ll add is just if we come back to a biologic versus a small molecule, we have the ability to explore three different sort of fundamental hypotheses about the relationship between exposure and benefit to patient. And for us, we can do that with a dose that allows us to cover the pharmacological activity of kit, but not to the extent that would lead to the the the death of mast cells through apoptosis. We could alternatively go to a level of exposure, that the biologics have achieved and in fact induce apoptosis of mast cells. So we can do either of those.

So we can explore a unique hypothesis, or we could do sort of an oral biologic type approach if you wanted. And then the third thing we can do is to take a page out of, sort of a more nuanced view here, and you can imagine a sort of an approach where we first initially explore a high dose or a sort of a saturating dose with a molecule and then reduce that dose to spare the patient the target mediated effects while benefiting from the induction of the mast cell killing. So, we can explore two unique hypotheses in addition to the sort of oral biologic profile.

Unidentified speaker: Great, very helpful. And in the absence of time, my last question, beyond chronic urticaria, which is ready in the cross years for 08/2008, you know, how should we be thinking about additional indications, which offer kind of the best opportunity for demonstrating, you know, the breadth and the possibility of wild type kit inhibition moving forward?

Christina, Blueprint Medicines: Yeah. So we, we have four proof of concept studies, that we’ve initially planned, right, in the short term. So, we talked about urticaria. We’re looking at, allergic rhinitis and allergic conjunctivitis. Conjunctivitis.

We’re looking at asthma. And then, MCAS, which, you know, is a really interesting opportunity that I think blueprint is, uniquely positioned to to pursue and has benefits across our portfolio, both, our, you know, SM portfolio as well as potentially a new opportunity for eight zero eight. There are a number of additional indications beyond that where we think mass cells can play an important role. And I’ll give a little plug if anyone hasn’t, gone back. We did a few seminars, last year, where we explored some of the biology and potential indication opportunities, and those are available on our website if anybody’s interested.

We are planning another, you know, in the first half of this year. And And so, you know, what we hope to do with the proof of concept data, as I said, is to get an initial read both on, you know, the profile, as well as understanding biology and some of these indications. And then, you know, we’ll have more to say in terms of how do we think about strategically bringing 08/2008 forward. You know, what indications do we prioritize? What do we do in parallel versus what do we do in sequence?

There’s obviously a lot of opportunity here for us to to think about exploring.

Unidentified speaker: Great. No. This is very helpful. Thank you very much. That’s all the time we have today.

Obviously we could continue talking for another couple of hours here, but I really appreciate the insight, that the team has given us. I’d really like to thank Percy, Christina, and again Cassie for joining us today. Really looking forward to a great year for you guys and looking forward to future updates.

Christina, Blueprint Medicines: Thank you so much. Thanks for having us. Thank you.

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