Q3 2024 Blueprint Medicines Corp Earnings Call
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Speaker Change: And then at Cowen you May begin your conference.
Speaker Change: you may begin your conference.
Speaker Change: Well I Dunno Mooney President research and development is also on the line and available during Q&A.
Speaker Change: Before we begin I'd like to remind you that some of the statements made during the call today are forward looking statements as outlined on slide three and are subject to a number of risks and uncertainties. These may cause our actual results to differ materially including those described in our reports filed with the SEC you are cautioned not to place any undue reliance on these forward looking statements and blueprint.
Speaker Change: Claims any obligation to update such statements I'll now hand, the call over to Kate.
Kate: Thank you Jennifer and good morning, everyone.
Kate: This morning, we reported another strong quarter of advocate revenue growth.
Kate: Adding additional momentum to our impressive year to date results and providing a strong foundation for us to drive long term shareholder value creation in 2025 and beyond.
Kate: Eva kit sales trajectory has given us the confidence to raise our revenue expectations significantly over the course of this year.
And we now estimate that we will end the year between 475 and $480 million in product revenue.
Kate: We are on a half billion dollar run rate in our first full year of I S. M launch.
Kate: Leasing advocate firmly on the path to realizing its more than 2 billion dollar peak revenue opportunity.
Kate: And positioning advocates launch an ASM to be among the most successful rare disease launches to date.
Kate: To build a blockbuster medicine, we must demonstrate a significantly meaningful and differentiated clinical impact that transforms treatment paradigms in diseases with high medical need.
Kate: This is so simple to say and.
Kate: And not easy to do.
Kate: At blueprint, we have demonstrated that Eva kit drives clinically meaningful deep and sustained symptom impact for patients with I S. M.
Kate: Now over several years in the pioneer study with a consistent and well tolerated safety profile.
Kate: Treatment with Eva kit is allowing I S N patients to reclaim controls of the control of their lives and is transforming the treatment paradigm when a N I S M.
Kate: With continued long term data generation and strong commercial adoption. We believe advocate is setting up to be the durable market leader across the spectrum of both advanced and indolent SM for years to come.
Kate: Building, a blockbuster brand has never been easy.
Kate: And it has only become harder as therapeutic areas have become saturated.
As legislative regulatory and market access headwinds have strengthened.
Kate: All on the backdrop of what has been a challenging macro environment in recent years.
Kate: At Blueprint, we have successfully managed through all of these challenges.
Kate: Our commercial and medical teams have done a tremendous job navigating the range of short term quarterly dynamics, we face.
Kate: Including the industry wide dynamics, all medicine space.
Kate: The dynamics that are intrinsic to rare disease markets and launches.
Kate: As well as the dynamics are unique and specific to the us market.
Kate: While managing all these short term dynamics, we have always maintained conviction and advocates more than $2 billion peak revenue opportunity.
Kate: And we continue to be focused on building a foundation for long term growth and delivering compelling results year over year.
Kate: Molina will talk in more detail about our commercial results shortly.
Kate: Leveraging our leadership in S M and our deep expertise expertise in muscle biology, we are working to broaden our impact by addressing the significant medical needs of thousands of patients with Blue 808 are wild type kit inhibitor.
Kate: We designed Blu 808 to raise the bar on what a treatment for chronic or to Korea, and other mast cell mediated inflammatory diseases can offer.
Kate: By taking into account the full patient experience.
Kate: Efficacy tolerability and the burden associated with administration.
Kate: We see the opportunity for Blue AOA is twofold.
Kate: To command, a large share of significant established markets and to drive further growth by expanding the treated populations in those markets.
Kate: Kristina will provide an update on the progress we have made across our portfolio later on the call.
Kate: As we report our Q3 results today Ava kept strong revenue ramp coupled with our disciplined investment in our most compelling product opportunities places us in a position to realize a significant decline in cash burn this year.
Kate: While maintaining our focus on long term growth and value creation.
Kate: We are building a solid financial foundation for blueprints future.
Kate: Mike will talk more about our financial results later on the call and we look forward to reviewing our portfolio priorities in early 2025.
Speaker Change: I will now turn it over to Paulina to discuss this quarter's commercial performance in more detail.
Paulina: Kate in the third quarter, a vacate achieved $128 $2 million in net product revenue with $113 $1 million in the U S and $15 $1 million ex U S.
Paulina: This represents a 137% increase year over year and our conviction in this blockbuster brand has never been stronger.
Our team's efforts are coming to fruition as we build this market and advocates strong launch trajectory proves our success.
Paulina: Growth this quarter was driven by strength in our key business fundamentals.
Paulina: First we continue to see strong and steady growth in patients on either kit driven by new patient starts and low discontinuation.
Paulina: Growth in patients on therapy is driving Eva kids successful launch.
Paulina: At our last call, we anticipated seasonal dynamics around summer holidays might impact patient starts and compliance, but this turned out to be less of an issue than expected.
Paulina: Our team did a great job managing through these dynamics, resulting in strong demand towards the end of Q3.
We have a large base of patients on therapy, and we expect this to continue growing.
Paulina: Compliance remained high consistent with what we've seen and trends and durations of therapy remains strong demonstrating the real world benefit patients see on a ticket.
Paulina: Second our mix of free and commercial goods remained stable our average free goods rates since I S. M approval is in the mid teens and has stabilized over the past two quarters. We expect this to remain consistent for the remainder of the year.
Paulina: Finally, our international team delivered strong results this quarter driving growth in advanced of them across several geographies and an eye of them with the launch underway in Germany.
Paulina: Germany comprises the majority of international sales and the ISR launch dynamics, we're seeing their mirror, what we've seen in the U S.
Paulina: In the third quarter strong growth in volume offset a lower price accrual from a planned German price reassessment process, which will be complete by early next year.
Paulina: We anticipate geographic expansion across both advanced SM and I asked them to continue to drive growth in 2025 and beyond as we launch them in additional markets.
Paulina: Okay.
Paulina: Breadth and depth of prescribing our strong lead indicators of continued growth. This chart shows how first positive experience with advocate leads to repeat prescribing among the top 400 providers by S M patient volume.
Paulina: This is just part of the story, we're engaging with several thousand providers beyond the top 400 and adoption is growing across this broader range.
Paulina: As they have a kid experience grows we see new haemonchus, an allergist prescribing with adoption evenly split across academic and community settings.
Paulina: These dynamics are exactly what you'd want to see in a strong launch and they plant the seeds for continued growth.
Paulina: We've also seen the first examples of prescribing by additional specialties such as dermatologists.
Paulina: For example, we're seeing cases, where dermatologists can become the local S. M champion motivated to diagnose and move more patients to treatment.
Paulina: This dynamic bodes well proven yet another opportunity to further grow the market and our ability to capture it with advocate.
Paulina: Awareness of S. M continues to grow across a broadening spectrum of specialists, who touch the constellation of disease symptoms.
Paulina: We understand the S M market better than anyone else, what activates patients to start therapy that targets the root cause of ice and what drives urgency to treat among providers and how to streamline access to therapy.
Paulina: Now, let's dive into some of our latest provider and patient initiatives.
Paulina: Our team recently launched a new branded campaign for providers designed to challenge the notion of well controlled along with resources that highlight the two year safety and efficacy data we presented at the European Academy of allergy and clinical Immunology conference.
Paulina: These data are valuable to providers, considering Ava kit and Christie will share more on this.
Paulina: Having made progress educating the provider base and growing Eva could experience among haemonchus and allergists. We can now further expand on our direct to patient initiatives.
Paulina: We recently launched a patient campaign to raise awareness and spotlight, how Eva targets the source of the disease.
We also launched a new patient mentor program increased support for face to face patient events and expanded our patient to master programs.
Paulina: We know that patients find it helpful to hear from other patients as they consider starting either kit and an educated patient is a catalyst for treatment.
Paulina: Our efforts with the S. M community are working.
<unk> is helping more patients every quarter and we are continually enhancing and expanding our initiatives as we drive this launch towards its peak potential.
Speaker Change: I'll now turn it over to Christy to share more about our data generation efforts and near term catalysts as we head into 2025.
Speaker Change: Helena.
Helena: We have been treating S M patients with either cat and clinical trials since 2016.
Helena: And I've spent the last eight plus years amassing a significant body of evidence on its long term impact across the spectrum of the disease.
Helena: Advanced at them, we've been able to demonstrate significantly improved overall survival versus prior standard of care therapy.
Helena: And then I asked them, we know that Hcp's and patience science longer term data on the safety and clinical impacted eight of cat meaningful and motivating and the setting of a chronic lifelong disease.
Helena: We have continued to publish long term follow up data from the open label extension of the pioneer study.
Helena: Most recently, we presented updated data demonstrating that with median follow up of more than two years and with some patients treated as long as four years.
Helena: They kept showed durable efficacy and a favorable safety profile.
Helena: Safety data were consistent for a subset of patients who dose escalate to 50 milligrams once daily reinforcing the flexibility that hcp's have to customize treatment based on the individual needs of their patients.
Helena: What's that's blueprint apart. However is not just a mountain of evidence we continue to amass with Eva cat.
Helena: Our team has developed a tremendous command of the S M market by being at the table with a broad group of health care providers, diagnosing and treating patients every day.
Helena: We have an unparalleled depth of knowledge and expertise across the continuum from research through development to commercialization.
Helena: Which affords us an evolved understanding of patient and provider needs over the long term.
Helena: This creates a virtuous cycle, where insight fuels innovation, enabling us to drive progress with any of the cat and guiding us on how to deliver and involves value proposition with Elena to Nab, Our next generation <unk> inhibitor.
Helena: And we're establishing the same virtuous cycle and mast cell disorders more broadly.
It was insight and feedback from allergists that inspired us to pursue a potent selective and tunable oral wild type kit inhibitor, resulting in blue 808.
Helena: And just last weekend at the American College of allergy asthma, and immunology meeting in Boston I saw firsthand evidence of scientific exchange, sparking new ideas and driving strategy and impact across our myself portfolio.
Helena: As we approach the end of what has truly been an exceptional year and look ahead to 2025 I'd like to touch on some of the near term milestones and catalysts. Our team is focused on starting with our primary strategic priority advancing that's my cell portfolio.
Helena: This includes the successful global commercial launch of Eva Cat, which Helena discussed today.
Helena: It also includes the advancement of Alan Messing up where are we remain on track to initiate the registration enabling part two of the harbor study by year end.
Helena: And Blue 808, which has been living through a phase one study in healthy volunteers.
Helena: We anticipate sharing this data early next year, including initial data on Blue, Italy safety profile drug like properties and early biomarker responses that will help to inform how we think about the potential for broad disease impact.
I hope you have a chance to dial in for the second and our series a science focused seminars on November 14th where we plan to talk more about the strategic development plans for our mass cell therapy franchise.
Helena: Regarding our cell cycle portfolio, we have been closely tracking the emerging dataset, including those shared at ESMO. They continue to validate the promise of targeting CDK too.
Helena: We are nearing the end of the combination dose escalation portion of the phase one valor study and in parallel we have also advanced our next generation programs, particularly our CDK to do greater more quickly than we originally expected.
Helena: The significant investment and capabilities required to move into later stage breast cancer trials, we've been clear that we will not move Blu 222 forward beyond this phase of development on our own and have been engaging in strategic partnership discussions.
Helena: Through these conversations we are evaluating the emerging data from CDK two inhibitors as well as the profiles of our next generation assets to determine what may be the best in class approach and therefore, what the optimal structure and timing of a potential partnership would look like.
Helena: We anticipate sharing more about our plans and priorities early next year.
Helena: In 2024, our team has executed to both drive exceptional topline revenue growth.
Helena: And prioritize our investments in our highest value programs.
Helena: Our strong financial foundation for sustainable corporate growth.
Helena: I will now turn it over to Mike to discuss our financial results.
Thanks Christie earlier. This morning, we reported detailed financial results in our press release for today's call I'll touch on a few highlights from the quarter.
Mike: In the third quarter total revenues were $128 $2 million from net product sales of advocate.
Mike: And as mentioned earlier, we are raising our aggregate product revenue guidance and now expect to achieve $475 million to $480 million and net product revenue in 2024.
Mike: This updated guidance is based on continued growth in total patients on therapy.
Mike: Continued favorability in compliance and other factors.
Mike: And stronger than expected performance outside of the U S.
Mike: Our gross to net margin remained stable in the mid eighties and our international business is on track to breakeven by the end of this year.
Mike: Our total costs and operating expenses remained relatively flat at $177 $2 million for the third quarter.
Mike: We anticipate that both our R&D and SG&A expenses will remain relatively consistent as we closed out the remainder of this year.
Mike: As we look ahead to 2025, our capital allocation priorities remain squarely focused on investment in our basketball portfolio.
Mike: This is to ensure that we're capturing the clear opportunity that the SM market represents with the commercialization of advocate.
And advancing our pipeline of other mass cell therapies to drive long term growth in areas, where our conviction around disease biology is clear.
Mike: We plan to share more perspective on our 2025 capital allocation strategy early next year.
Mike: We continue to strengthen our financial position with $882 $4 million in cash on hand at the end of the quarter.
And importantly, we've seen our cash burn dropped significantly in 2024.
Mike: And we expect this trend to continue in 2025, reinforcing our clear path to financial sustainability.
Mike: With the ongoing success of the advocate watch and our commitment to disciplined investment to drive growth. We are in a great position to continue to create long term shareholder value.
Mike: With that I'll now turn the call back over to the operator for questions.
Mike: Operator.
Mike: Yeah.
Speaker Change: Thank you at this time I would like to remind everyone in order to ask a question. Please press Star then the number one on the telephone keypad, we'll pause for just a moment to compile the Q&A roster.
Speaker Change: Our first question is to you Mark from of TD, Karen Mok. Please go ahead.
Karen Mok: Hi, Thanks for taking my questions and congrats on another strong quarter.
Karen Mok: Maybe just on the.
Karen Mok: I assume side to start just youre getting a meaningful number of patients out at 612 months now any type of reauthorization criteria youre seeing or any type of.
Karen Mok: Maybe what we should expect in terms of discontinuation starting to factor into the launch trajectory and then as we get to that Walter kit inhibition data next year.
Karen Mok: Curious kind of where you think the thresholds are of target engagement or to induce a response and do you really need to hit.
Karen Mok: To hit those levels clinically or do you think more of a pulsatile or induction maintenance might be the best approach.
Speaker Change: Hey, Thanks, Mark So maybe you can talk about what we're seeing in terms of an authorization and for patients and in Florida.
Speaker Change: For more than one blew it away and what we're expecting there.
Speaker Change: Yeah, Hi, Mark and as you mentioned, we are seeing a significant and growing patient base of patients on therapy and we expect this to can you to continue to grow them in terms of re authorizations you know I think overall, we're incredibly pleased with the strength of access that continues.
Speaker Change: We have secured and maintained strong payer coverage, we have not seen problems with the reauthorization.
Speaker Change: In terms of the discontinuation rates that remains very low and you know as you know what I think the two things that continue to drive. This launch is growing that strong patient base as well as our ability to retain patients on therapy. So we're really pleased to see the trends in duration of therapy, which really connect to the potential for <unk>.
Speaker Change: Long term chronic treatment.
Speaker Change: And Mark. Thank you for the question on Wild type kit as we all know now what type it is a very well validated targets and perhaps even a lot of work from biologics there I think developing small molecule like eight O. It would give us the flexibility to really tune and titrate the developments in the schedule of this morning kind of a variety of disease.
Speaker Change: This allowed us to navigate between the two.
Speaker Change: Inhibition of the activity ended the granulation Massa all the way to the Divisional Command Center and I think having such a molecule like <unk> gives us the flexibility and we are happy to.
Speaker Change: To work with this type of small molecules.
Speaker Change: Okay. Thank you.
Thank you. The next question because she Brad Canino of Stifel. Please go ahead.
Good morning, Great quarter, I'm actually consider this to be the most aggressive guide you've provided in terms of implied growth in forward quarters, I think feedback I've gotten from investors and I imagine you've heard as well is that some of the previous guidance appeared a bit too conservative.
Speaker Change: Can you help us understand how you're forecasting views and abilities have changed particularly.
Speaker Change: Do you think about how to incorporate variables for the 2025 guidance coming up next year. Thank you.
Speaker Change: Yes, Brian. Thank you I mean, we're incredibly pleased to be at a plane at this point in the year, where we can now say that where we're gonna be.
Speaker Change: Nearly $500 million in revenue, which is really a tremendous place to be and as you said we've raised the guide. We've also tightened the range to $475 million to $70 million would cause you to want to talk more about philosophy on guidance, what I know.
Speaker Change: We've talked about before that and how we think about that as far as this year is all of that yeah. Yeah. Thanks for the question Brad. So you know our philosophy throughout the year has been to try and look at the range of variables that influence revenue <unk>.
Speaker Change: Providing our best estimates on how we think you know what the range of outcomes on those barrels variables may look like and then you know what do we think that could imply for revenue.
Speaker Change: You know in the context of a launch into a category that did not exist before a market that we are building for the first time and you know the first disease modifying therapy in that market, which is an incredible privilege and such an opportunity right. So many markets you know you're fighting for market share and trying to have patients switch off.
Speaker Change: Therapies, and we are really building a you know an entirely new market opportunity, which is which is compelling but has it has its challenges and certainly I think forecasting the first year of sales in that context can be a little bit difficult.
Speaker Change: We've definitely done our best and we are thrilled that we have exceeded our own view of frankly of of how these variables my might fall through the year. We're now in a place where you know we're 10 months into the year and so we are providing a guide essentially on Q4.
Speaker Change: Our ability to forecast, how we will land one quarter is obviously much tighter than when we're looking at a whole year and so you know this guidance really represents our best thinking on how we will end the year and it's really as simple as that.
Speaker Change: As we go into guidance for next year, you know clearly we will now be more than a year well more than a year into the launch. So we you know we know this market quite well we're still of course forecasting you know new therapy in a new market, but I think have a good sense of some of the key variables that will impact.
Speaker Change: We typically if that guidance as you know on our Q4 call I'm, having some early experience next year with aspects like for example, free drug in the context of the eye or any changes will be very informative and helpful to us as we think about setting that guidance.
Speaker Change: The philosophy will stay the same.
Speaker Change: Great. Thank you.
Speaker Change: Thank you. The next question go to Ryan Benjamin of Fish citizen J P. M. <unk>. Please go ahead.
Thanks, very much guys for taking the questions and congratulations on a great quarter can you talk a little bit about the direct to patient AD campaign, and the new patient mentor program how does it.
Speaker Change: Actually impact SG&A and how do you evaluate whether this is working or not and if it isn't working what are the other options that you have at your fingertips to grow market share.
Speaker Change: Thanks, Thanks, Brent for the question I think first and foremost I think we're at such a wonderful place in this launch where we have critical mass where it now makes a lot of sense for us to be kind of doubling down in pushing forward on some of these direct to two patient campaigns and initiatives with when you had talked about before did you want to talk a little bit about how you think about.
Speaker Change: That relative to SG&A spend and the impact of these types of efforts as we look to kind of activate educate patients.
Speaker Change: Yeah on the Iran. I think maybe just first off starting at the enterprise level, we've talked about how are our top priorities for for investment and blueprints growth or really across our mass cell portfolio, including the Ava kit launch <unk> and Ada wait and we will certainly look to continue to find opportunities to invest where we.
Speaker Change: See the the most impactful benefit in terms of return on investment.
Speaker Change: So in terms of how that translates into the Ava kit launch you know you've just seen with our updated guidance that we are well on course to deliver nearly half a billion in revenue firmly on the path to achieve that peak conviction has never been this strong now is the time for us to be leaning in further into these direct to patient initiatives now that we have secured and activated and.
Speaker Change: Growing prescriber base that continues to grow we have the opportunity to lean in and further engage and activate patients. Why that's important is you know we we continue to see that challenging that notion of well control is critically important to open Ah patients aperture to what may be possible in.
Speaker Change: In terms of starting on a disease modifying therapy, while patients may have become acclimated to the sense of a new normal or sort of coping with the disease and like restricting their behaviors and their activities.
Speaker Change: Theres nothing more compelling than just hearing from another patient their experience on Ava kit and what what it can feel like how transformative that can be to reclaim what that new normal is like and so going into those direct to patient initiatives.
Both in terms of raising awareness talking about the importance of targeting the source of the disease and creating more of these opportunities for patients to hear from other patients will be a critically important component of continuing to drive in this launch.
Speaker Change: Thanks, guys and congrats.
Thank you. The next question goes to Michael Schmidt of Guggenheim. Please go ahead.
Speaker Change: Hi, This is Paul on for Michael Thanks for taking my question and congrats on the quarter just a quick one on <unk>.
Speaker Change: And third quarter with the sort of thing.
Speaker Change: It is summer seasonality actively counterbalanced by efforts on your end or does it simply play out less than expected.
Speaker Change: And in general over the balance of a full 12 months.
Speaker Change: And have on the market and then my second one is just on Hulu.
Speaker Change: Early signals of activity that we might be.
Speaker Change: Do you think about some of the classic markers like muscle appreciate that introduction for the oral agents compared to what's been shown by the antibody and what's the right way to think about correlation between those biomarkers and clinically relevant endpoints like the <unk> activity score.
Speaker Change: Yeah. Thanks, Thanks, a lot there's a lot of that so maybe if you could take the first piece and then I'll hand it to.
Speaker Change: To have Koolade, one thing I'd, just say is that you know the commentary that we have been making around as we think about new patient starts since before we even launched as this is a rare disease and we expect there to be variability or lumpiness on a week by week basis, but over the long you no longer perspective, we can we continue to believe there will be strong and steady growth in patients on therapy.
Speaker Change: And you know the commentary made on the last quarter was that because it's not about if patients start it's about one and I think what has been really clear as both of those things have played out exactly as we have described yourself. We noted would you like to talk with them.
Speaker Change: Yeah, So I think I didn't hate teed, it up really well we've alluded to some of the short term quarterly dynamics that that can impact a particular quarter. We know that holidays have the potential to impact the timing of patient starts to go to the next visit what we saw in the third quarter is that our team has done an incredible job managing.
Speaker Change: Through these dynamics with a strong increase of demand through the back half of the year, but I think most importantly sort of laser ring out from quarter to quarter is really that year over year trajectory and we can see from the updated guidance. We are on a path to deliver over 100% growth year over year, and again I think that places us firmly.
Speaker Change: <unk> on the path of marching towards that greater than $2 billion peak.
Speaker Change: And that's what you want to talk a little bit about wild type kit and what we're thinking about from a biomarker in correlation prescriptive. Thank you. Thanks. Thank you for the question.
Speaker Change: We.
Speaker Change: All developing because normally you could as I said earlier I was sort of marks question.
Speaker Change: The idea that with small molecules you can read.
Speaker Change: Two the build it on the schedule to really navigate the wide range of the activity of the mast cell and target a number of type two inflammatory diseases.
Speaker Change: As Christie mentioned, we anticipate to report the data from the Sad and Mad study early in 2025, and you look at overall subject safety.
Speaker Change: We'll look at all of them from a cold here.
Speaker Change: So we wouldn't expect it from according to a very good one and really allowing us to do the tune of beauty and the penetration that I talked about earlier and we will report a number of biomarker, including the level of therapies.
More specifically to your question Tryptase is a supportive pharmacodynamic marker for the activity of the Asians.
Speaker Change: And varies from Dcs to our mother so wood.
Speaker Change: We would like to see Tryptase decrease.
Speaker Change: These.
Speaker Change: It wasn't years, we also know that in some more complex diseases, where there is already a POC you like chronic urticaria tryptase is a much a part of the story, but theyre much more and much more complex environment. As an example, when you when we go to type two asthma diseases are more complex than just looking at the trip data that you can look at all of our mortgage so we have the opportunity.
Speaker Change: Sure.
Speaker Change: Data tryptase and beyond their base.
Speaker Change: And this is where I'll, maybe I'll just put a plug in again for our seminar coming up in November.
Speaker Change: November 14th where we'll talk more about how we're thinking about eight O aid them in our overall kind of development strategy and myself. So please stay tuned and if you can.
Speaker Change: Thank you. The next question guys. She Sal <unk> of Goldman Sachs. Dowling. Please go ahead.
Speaker Change: Thanks for taking our questions is Tommy on for something and then congrats on the quarter then a follow up from some of the previous questions on Italy overall, how derisking do you think that the healthy volunteer data will be with regard to navigating the therapeutic window and what's your view on the differentiation here.
Speaker Change: From a strategy such as at March <unk> P. R X too and then just one on I assume as it relates to 2025 mm maybe lay out some of the moving parts that we should be aware of here.
Speaker Change: Especially as the X U S starts to play a larger role. Thank you.
Speaker Change: Yes. Thank you very much for the question and <unk> will talk about 808 and of course, we'll be talking more about point, whereby you know on the Q4 call, but maybe you can just give a high level of that but yes.
Speaker Change: So from my perspective.
Speaker Change: Our development strategy is really to target wildfire did to fine tune the mast cell and go to a variety of diseases.
Speaker Change: In terms of Derisking to your question on <unk>.
Speaker Change: If we look today at one type of kit.
Speaker Change: See I think biologics did a very good job showing strong proof of concept in chronic educators continues I'm also called in use. It. Therefore, the data that we anticipate to share early in 2025 would be a major inflection points, where for OSM for patients could really benefit from.
Speaker Change: And what we hope and expect to be a good.
Speaker Change: Drug profile for eight O is now there is a lot of room to improve from the.
Speaker Change: Overall therapeutic index for <unk>.
Speaker Change: For these therapies and we believe having a small molecule that we can fine tune with completely different type of pharmacology from biologics will help us achieve such a goal not only in the chronic or acute care, but in a variety of diseases and as Keith mentioned earlier, we will talk about these diseases of our seminar in a few weeks.
Speaker Change: Regarding your question on different mast cell targets in numerous specifically amongst your parents to them.
Speaker Change: There are a number of targets on the mat. So we believe in the scientific community does too that such a target is probably Oregon restricted bolt.
Speaker Change: <unk> constantly to the skin.
Speaker Change: And when we need to see more clinical data because I mean, it's it's it's some it's a really data.
Speaker Change: It's a bit of free zone, if I can use this analogy.
On the our strategy by go into Wild type kit, we know that we would target the message. We're river. It is either in the lungs or in the gut or in the skin and so we believe going after the inhibition of wild type kit is a much broader strategy around how the products.
Speaker Change: Yeah, and then Christian you want to talk a little bit about just at a high level, what do we think about it like way Bob Obviously, we'll spend a lot more time with you all on this one as mentioned yeah. So we'll provide guidance again on the Q4 call. I think you know the variables that we'll be thinking about are going to be you know in broadly very similar to sort of what we've been what we've been talking about this year.
Speaker Change: You know at a macro level, what's driving this launches new patients starting on therapy, and you know keeping those patients on therapy over time, and you know what Oh of course, it's becoming more and more important as we progress through the launch as you know we now have a very substantial number of patients being treated with Eva cat that we won't be carrying into next year and then of course growing.
Speaker Change: From from that base you know what.
Speaker Change: New markets coming on internationally as you mentioned as we negotiate pricing and reimbursement and move through a national launch as you know every year has its own factors I'm sure, we'll talk a bit about free drug I R. A M passed et cetera, as we get into next year and sort of see how the first couple of months of the year play out, but as you know the big picture again is that we are in the early innings of a launch.
Speaker Change: With a substantial amount of room for continued growth and that you know look forward to providing more on that and you know as we go into next year.
Speaker Change: Thank you. The next question guys cheek, Eric Coachella or flowers fall Guy Derek. Please go ahead.
Speaker Change: Hey, good morning, and congrats on the updates here and on the quarter just two one clarifying and then just one question. So just on <unk> relative to the second quarter. Just in terms of the net patient adds I guess, what does that look like Q over Q and then I guess in terms of the seasonal factor here.
Speaker Change: Can you kind of just kind of give us some qualitative information on the magnitude and how might be back to work you.
Speaker Change: And then just on <unk>.
Speaker Change: You guys have talked a lot about on the call very exciting I guess is your expectation tryptase reductions in line with antibodies and safety is better than the antibody or how do you think the overall profile will look.
Speaker Change: Thanks.
Speaker Change: Yeah. Thanks, Doug for the question so far that we as we moved away from talking about kind of that projected estimated number on therapy at end of the quarter last year, Darren but we can certainly provide kind of qualitative commentary around how we're thinking about seasonality and what we've seen there and I know we've touched on that before I do you want to kind of reiterate there how youre thinking about seasonality yeah.
Speaker Change: Think I would just reiterate by starting with the expectation that we continue to expect strong and steady growth in the number of patients on therapy. This is what we have always said there might be variations month to month based on factors like holidays. We know that in Q4. For example, there are few fewer business days.
But I think maybe going back to what Christy just articulated them you know the most the most important piece. That's driving this launch is that continued growth in in patients on therapy strong and steady.
Speaker Change: Quarters are going to be more studies in months in a rare disease launch such as this one.
Speaker Change: And most importantly, the arc of that overall trajectory is that we are showing significant growth on a year to year basis significant headroom to continue to grow and we're really focused on marching towards that peak potential.
Speaker Change: And I think you know that now that we're kind of coming into our like the end of our first full year of launch we have a lot of confidence around the dynamics that we see in any given quarter and those are as I said in my prepared remarks like dynamics that everyone faces dynamics that are specific to rare diseases I know that our U S. M and I think we now have shown and demonstrated that our team is able to understand that.
Speaker Change: And work through those in a very constructive way you know.
The goal here is to move towards the $2 billion plus product and we're going to do that year by year right. So that is our focus and will continue to be our focus.
Speaker Change: What do you want to talk a little bit about blue 808, and and how you're thinking about that relative to what we've seen from some of the data that's come out from the how about yours.
Speaker Change: I think the.
Speaker Change: Clearly the data from the antibody approaches validates the target and specifically in two subsets of or to carry out which is chronic spontaneous urticaria uncalled and use those who carry which is really a good thing and I mentioned this makes our sad and Mad data report and anticipate early next year extremely important and a meter.
Speaker Change: A major inflection point for patients and for for Blueprint medicines.
Speaker Change: Developing small molecule in this area. It gives us the opportunity to really navigate the targeting of mass. So all the way from depleting the method when we need to do that to inhibiting the activation ended the granulation without with getting full depletion.
Speaker Change: Allow us also to have a very good therapeutic index by on one hand, having the activity on the disease.
Speaker Change: Not reaching that.
Speaker Change: And that leads to my little myeloid progenitor to generate adverse events or toxicity that we don't need to it is very difficult to do with biologics. We believe our strategy with a small molecule is very differentiated and will allow us to fine tune and titrate and achieve these goals.
Speaker Change: Thank you. The next question goes to Mike <unk> of Morgan Stanley. Please go ahead.
Mike: Hi, good morning, Thanks for taking the question and congrats on the quarter as well.
Speaker Change: Maybe just on <unk>.
Speaker Change: You highlighted sort of strengths.
Mike: S and being better than expected, maybe you could provide some more color there and how you expect those trends to sort of evolve as we get into next year. Thanks.
Yeah, I think we're incredibly pleased with how the international launch has been going and the team has done just a tremendous job in terms of kind of dealer and delivering well beyond expectations and because there are no. If you want to talk a little bit more about that international launch. Yes. We were really really pleased with the strength of the quarter of course, we talk a lot about Germany, which.
Mike: As you know the first out of the gate on an I S M and where we've seen absolutely no sad dynamics that look very similar to the U S. In terms of uptake, which has been really gratifying to see including prescribing coming from both academic centers and in the community. So very very similar dynamic.
Mike: And in the quarter, what we saw was the strength of that I pay them really overcoming a you know.
Mike: Some of the price headwinds that we had referred to them of course, we're still working through the process with negotiations in Germany, and we'll have a sense of where that lands early next year, but I'm you know the demand growth is really more than offsetting that which has been great to see and then of course, we continue to see growth in other markets, which are still watching and events with them and as we get into next year.
Mike: We expect to see more of the major markets in Europe start to come online as we worked through I S. M. A pricing negotiation. So you know we've talked about international being an important driver of topline revenue growth I expect that to continue and of course the U S will represent the lion's share of the opportunity in the short term, but yeah, we would expect.
Mike: <unk> continued to become more.
Mike: More and more important part of the opportunity and certainly when we think about the peak I'm. You know we're we're excited about the potential we see both in the U S is all about the outside the U S.
Speaker Change: And then one thing to add to that and in Mike's commentary. He mentioned that actually at the end of this year. Our international organization is going to break even and so this team is really driving kind of tremendous tremendous top line growth in a very financially efficient and disciplined way.
Speaker Change: So that is a I think that's a great place for us to be.
Speaker Change: Great. Thank you.
Speaker Change: Yeah.
Speaker Change: Thank you. The next question, Yes, you Amit <unk> of Needham Ami. Please go ahead.
Hi, good morning, Congrats on the nice quarter. Two quick questions is supposed to be on and inactivate and an estimate would be a path to Oklahoma study initiating by the end of the year can you talk about.
Speaker Change: How you are developing strategy differs from that of Eva kit and how do you see it.
Speaker Change: Being differentiated from either kit and with regards to Ito eight.
Speaker Change: The ability of a small molecule strategy.
Speaker Change: <unk> talked about quite a bit in the Q&A.
Speaker Change: As we see the data from the handling about one per study a dose escalation study.
Speaker Change:
Speaker Change: How would you take that information and then think about.
Speaker Change: Different diseases, where you could apply it and maybe sort of what are some of the indications that you're thinking about taking it into.
Speaker Change: Some initial thoughts that would be helpful. Thank you.
Speaker Change: Yeah. Thank you. Thank you I mean, I think I'm, maybe we'll start I'll hand, because he can talk a little bit more oh, that's it and flood will take the.
Speaker Change: So it's a good question about Blue anyway, I think just stepping back for one moment as that.
Speaker Change: The position we're in at Blueprint medicines with Ava can be.
Speaker Change: Becoming the standard of care in a durable leader across advance S. M. S. M. And then the opportunity we now have to bring Alan afternoon for it to really maximize our longer term performance and innovation across the S. N franchise is really a unique and an incredible position.
Speaker Change: Talk a little bit crazy about how we're thinking about bringing forward to do just that yeah.
Speaker Change: So you know as Kate is that our strategy with <unk> is really to extend our you know our franchise that we think is incredibly important both for patients and for blueprint as we get into you know really the next decade and beyond them. This is an opportunity that we are growing into and we think we have a cigna.
Speaker Change: Amount of headroom and ask them to continue to grow our expectation is that ever kept will be you know the durable market leader for a quite some time and we'll be growing our you know for years to come and the value of all or nothing that is really to be able to bring a therapy that delivers a additional differentiated clinical.
Speaker Change: Impact over the long term so that we can continue to drive growth in the franchise and say you know the latter part of the next decade and so.
Speaker Change: So you know we will be initiating part two of harbor, we'll obviously share more about what the design of that study looks like but we know that the only way that you get to extend the value of a franchise like this is by delivering clinical differentiation.
Speaker Change: We understand more about you know the full manifestations of the disease now.
Speaker Change: And much more about that than we did you know five or 10 years ago, and so I think our ability to measure clinical impact and really demonstrates what a targeted therapy can do in the space is is different now than it was when we started the pioneer studies out you know in terms of what the actual design of Harvard will look like well.
Speaker Change: They have more to say about that as we open the study and again I would point everybody to our Nassau webinar that we have scheduled on November 14th where we're planning on sharing more about our development strategy across our MSR portfolio.
Speaker Change: Thank you Christy if they couldn't thank you Andrew for the question. So your first question is how.
Speaker Change: With the data from the Sad and Mad study help us understand the tune ability profile.
Speaker Change: Ada with I would say as we all know sediments steady we are exploring a number of those as a variety of cohorts of patients over time and we are also looking at Pharmacodynamic marker. So when we look at the totality of the pharmacology and the data in healthy volunteers of the variety of those has a different level of inhibition and their impact on the pharmacodynamic marker.
Speaker Change: Can you give us really a good understanding of how can we can develop tunable and digestible.
Speaker Change: Schedules for a variety of diseases for a second part of your question on what is the breadth in terms of number of disease or other diseases. We will go to I think we're targeting types of inflammation.
Speaker Change: And in a broad way, we will have the opportunity in a few weeks from now at our similar to talk with you about these diseases and how we think about them, but you can think about a really a targeting a broad variety of type two inflammation, how we will do it.
Speaker Change: As we mentioned in the past is through some or a number of you will see them.
Speaker Change: Cohorts or studies to really early on CD activity of course.
Speaker Change: <unk> of diseases and start step by step Derisking ate away toward the selection of the major indication for Registrational development.
Speaker Change: Thank you. The next question guys Schumacher Streicher of Oppenheimer. Please.
Speaker Change: Please go ahead.
Speaker Change: Hi, This is mark something from US just getting your questions.
Speaker Change:
Speaker Change: You saw already some preclinical data on <unk> is it fair to say that this is.
Speaker Change: This will be one of your focus there.
Speaker Change: Great question, obviously asthma type two asthma more precisely is one of the key tied to employment uses will be within a range of diseases. Obviously I think in a matter of a few weeks, we'll be able really to get in in a little bit more specific about the diseases, but I mean.
Speaker Change: It is safe to say that many diseases in the type of inflammation range or or area will be a part of our thinking.
Speaker Change: Thank you. The next question goes to Pizza Hut Olson of Barclays. Peter. Please go ahead.
Speaker Change: Great. Thank you thanks for the update really appreciate it.
Speaker Change: On slide six for Ya data rooms, breadth and depth.
Speaker Change: So it was an interesting dynamic.
Speaker Change: The number of physicians with greater than 10 patients just wondering if you could kind of talk through that if there is.
Speaker Change: Drew there or if this is.
Speaker Change: Alright.
Speaker Change: <unk>.
<unk> greater than 10 patients.
Speaker Change: If these are centers of excellence.
Speaker Change: Thank you.
Speaker Change: Yes. Thank you for the question Peter and I'll explain that has as we've been saying is kind of across the launch of the breadth and depth dynamics that we're seeing here has just been a tremendous strength and in the launch and and that that graph is a snapshot of some of the some of the providers, but did you just want to talk about our opportunity and I know that.
Speaker Change: We continue to believe we will drive quite a bit of depth as well. So yeah. Peter to your question I think the bottom line is significant headroom to grow across all metrics and measures that we see across this market in both providers and patients.
Speaker Change: So if we look at our provider base and so we're really excited to see the uptick in the number of providers, who are treating 10 or more patients again that that even within those top 400 providers substantial headroom to continue to grow and treat more patients on therapy within those top 400.
Speaker Change: As I said on the call that that's just a tiny part of the picture. There. There are additional many additional providers across that top 400, as well, where we continue to see growing breadth as well as depth and we haven't even really yet.
Speaker Change: The potential of additional specialties, such as dermatology and others, who are treating the broader constellation of Ah Ah Ah patients based on their on their symptomology. So bottom line significant headroom to grow and we're confident that places us on a path to achieve really more than 2 billion dollar peak.
Speaker Change: Thank you. The next question goes to your Prendergast of Raymond James.
Speaker Change: Go ahead.
Speaker Change: Hey, guys congrats on a great quarter.
Speaker Change: But do you expect free drug dynamic that generally remained steady below 20 odd 20 per ton for the team as he said on average but are you seeing or do you expect any in month to month or quarter to quarter variability in these dynamics.
Speaker Change: And then just a quick second question for me any guidance on how to think about gross margin moving forward. Thanks.
Speaker Change: Yeah. So so thank you Laura for those questions. When did you want to talk about how we think about free drug for as we close out this year like of course. This is all resets in 'twenty 'twenty five and as Kristie mentioned, we're gonna be painted to close attention to that and in January as we look at the impact for the rest of the Iraq, but how do you want to think about it for the rest of this year yeah.
Speaker Change: So we've talked about how launched today the proportion of patients on free drug has reached the mid teens and that has been relatively consistent over the past couple of quarters, and we expect that to remain consistent through the end of this year.
Speaker Change: As Keith alluded to I think one thing we would want to keep our eye on is this resets next year due to the changes in sort of part D from the IR E and really at this point, we have such a substantial a patient based on therapy that small small changes in these factors like like free drug can can have impacts on revenue. So another piece to look at along with all the other variables that could.
Speaker Change: You alluded to that will influence how we think about 2025.
Speaker Change: And Mike you want to talk a little bit about the gross margin question that Laura yeah. Thanks, Laura just with respect to gross margin like first of all like we think we are in a solid position with relatively low cost of goods sold and so we would expect our gross margins to be high and remain relatively stable at this point in the launch that we have enough volume coming through.
Speaker Change: We do see some variation due to sales of product to partners like system, So a month or quarter to quarter, sorry, you might see some variability, but fundamentally we expect margins to be stable and strong.
Speaker Change: Great. Thank you very much.
Speaker Change: Yeah.
Speaker Change: Okay.
Speaker Change: Thank you. The next question Gucci, David Lebowitz of Sippy, David. Please go ahead.
David Lebowitz: Thank you very much for taking my questions I have two for you first on positioning mix in the past few broken down the academic versus community doctors.
David Lebowitz: If you could just update us on that.
David Lebowitz: I'm sure if I missed it and then number two I was just wanted to ask about an estimate in the harbor to trial. It is being studied in ISR my nose getting underway soon.
David Lebowitz: Competitive therapy out there and studying it but also.
David Lebowitz: And smoothly.
David Lebowitz: Mastocytosis and I'm just curious as to.
David Lebowitz: Why it.
David Lebowitz: It would be higher for a moment as opposed to include eds in patients.
Speaker Change: Thanks for taking my question.
Speaker Change: Yeah, Yeah, Thanks, David and maybe maybe if you could just talk about the mix of physicians and then I'll hand, it to Bob to talk a little bit more about on S. Nib.
Speaker Change: And and how we're thinking about the kind of inclusion criteria.
Speaker Change: Yes.
Speaker Change: One of the most important leading indicators is is the sort of prescriber base, which continues to grow for Ava can we see that across all specialties and settings.
Speaker Change: So in terms of the mix continues to be pretty evenly split between the academic and community setting we.
Speaker Change: We continue to see additional he he marks as well as allergist immunologist I'm trying to get for the first time and as they have that first positive experience going onto repeat prescribe and really broadening the lens on the patients that they see as addressable by us for treatment.
Speaker Change: And importantly, we're really encouraged to see the growing interest and receptivity from a broader range of specialties as well we alluded to an example in in dermatology and we see potential there for <unk>.
Speaker Change: Continued growth and in interactions to really grow this market and move more patients towards treatment.
Speaker Change: And David to your question.
Speaker Change: The design of Hardboard brought to our People's hold Registrational study you didn't get it.
Speaker Change: Kristi mentioned earlier over the last many years, we have been really working with the U S.
Speaker Change: A world community on this disease, all the way from indolent to.
Speaker Change: And C and exploring all the spectrum of.
Speaker Change: Of this the subtypes of the disease and we have a good understanding of it.
Speaker Change: S M as a disease not only from subtypes of patients, but what's really patients and physicians will be looking for in the next many years.
Speaker Change: I think all of these data and this knowledge.
Speaker Change: It is factored in our developments were able be able to share more in the next weeks and months.
Speaker Change: These data small during to your specific question as you. All know is a very very small group of patients.
Speaker Change: About 2% or two 5% of the of the same and.
Speaker Change: Clearly dedicated studies for these.
Speaker Change: Those are very difficult to execute but it is a group of patients that are very interesting that we know very well, including <unk>.
Speaker Change: The way, we work with and all of our.
Speaker Change: Our data are influencing the churn and the changes to the definitions of these disease with the expert community.
Speaker Change: Thank you we have time for one more question before the top of the hour. The next question guys to Sudan like Nathan of Stephens Sudan. Please go ahead.
Speaker Change: Hi, Thank you for squeezing me in and congrats on a great quarter.
Speaker Change: Taking my question.
Speaker Change: Clearly driving that operating leverage both based on and financial efficiency to continue to bring innovation forward for patients who have significant medical needs.
Speaker Change: Thank you that concludes today's Q&A with Kate Haviland icon nickel that type of tea.
Speaker Change: Thank you everyone for joining US today, you know as we as we come into closing outweighed ramp or really advocate strong revenue ramp coupled with our disciplined investment our most compelling product opportunities as we just talked about with the last question I have put us on a path to drive sustainable growth, while we enhance long term shareholder value.
Speaker Change: And we're setting up for a very very exciting 2025 at blueprint medicines and we look forward to in a few short weeks, you're talking more about that when we kick off the year. So thank you all and thank you for your continued support of blueprint.
Speaker Change: Thank you. This concludes today's conference call you may now disconnect.
Speaker Change: [music].