Q4 2024 Blueprint Medicines Corp Earnings Call
Good morning, My name is not yet and I'll be your conference operator today.
Speaker Change: This time I would like to welcome everyone to the blueprint medicines full queue in FY 'twenty 'twenty four earnings release and conference call.
All lines have been placed on mute to prevent any background noise.
Speaker Change: After the Speakers' remarks, there'll be a question and answer session.
Speaker Change: I would like to ask a question. During this time simply press star followed by the number one on your telephone keypad.
Speaker Change: If you would like to withdraw your question. Please press star followed by Chase.
Speaker Change: Please pan limit yourself to one question.
Kathy: Thank you Kathy.
Speaker Change: So if you say to you may begin your conference.
Speaker Change: Thank you Nadia good morning, everyone and welcome to blueprint medicines fourth quarter, 'twenty 'twenty, four financial and operating results conference call.
Speaker Change: This morning, we issued a press release, which outlines the topics we plan to discuss today.
Speaker Change: You can access the press release as well as the slides, we will be reviewing today by going to the investors section of our website at www Dot blueprint medicines dotcom.
Speaker Change: Joining me today are Kate Haviland, Chief Executive Officer, Lina, Li Chief Commercial Officer Vega.
Speaker Change: Becker Hughes, Chief Medical Officer, and Mike Lance Lauck, Chief Financial Officer, who I know Mooney, President research and development and Christy Rossi Chief operating officer are also on the line and available for 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.
Speaker Change: And blueprint disclaims any obligation to update such statements with that I'll hand, the call over to Keith.
Thank you Kathy and good morning, everyone.
Keith: Absolutely print medicines, we are driving growth and innovation with operational excellence.
Keith: Our aspiration is to fundamentally shift the way allergic and inflammatory diseases are treated by targeting the myself.
Keith: This begins with advocate a breakthrough medicine for patients with systemic mastocytosis.
Keith: And 'twenty 'twenty, four we delivered $479 million in product revenue.
Keith: Representing impressive annual growth of 135%.
Keith: If it gets deep and sustained clinical impact in very strong safety profile have transformed the treatment paradigm and I guess I'm.
Keith: Today, we are pleased to announce our revenue guidance for 'twenty 'twenty five.
Keith: We expect advocate to achieve $680 million to $710 million in revenue this year.
Keith: Placing us firmly on the path to realize $2 billion in revenue by 2030.
Keith: With a 45% growth rate at the midpoint of our expected 2025 revenue continues to position advocate among the most successful rare disease launches to date.
And yes, I'm marker market is bigger than we thought and is growing faster than we expected as we talked about at the JP Morgan Conference in January.
Keith: We now project our blueprints are some franchise will reach a peak value of $4 billion with advocate other anchor.
Keith: We've got a lot of questions on what makes us confident to increase the peak S N franchise opportunity to $4 billion at this point.
Keith: Our confidence is based on a few key factors.
Keith: First only a fraction of currently diagnosed I send patients today are being treated with advocate.
Keith: We estimate that we have penetrated less than 10% of the existing pool of diagnosed S N patients, giving us tremendous headroom for growth.
Keith: Second.
Keith: This pool of diagnosed patients is growing.
Keith: And it is growing faster than we expected.
Keith: We have seen consistent double digit growth over the last five years and as of today, we can see more of that 25000 diagnosed patients in U S claims data.
Keith: Our team has done a tremendous job increasing awareness in the clinical suspicion about that among providers as well as establishing the commercial testing infrastructure needed to enable this growing number of providers, where catalyzed to pursue an S. M diagnosis.
Keith: And importantly, we expect this growth and diagnose I send patients to continue for years to come.
Keith: Based on our view that the true prevalence about them is much larger than we originally thought.
Keith: This larger estimated prevalence is informed by both new epidemiology data published in 'twenty, 'twenty, four and our own real world experience.
Keith: The new epidemiology data suggest a twofold increase in the number of asthma patients relative to prior estimates, bringing the number of potential SM patients in the U S up from approximately 30000 patients to 60000 patients.
Keith: In our real World experience, we have seen as in patients being diagnosed from a broader range of providers than we expected at this point in the launch.
Keith: Most notably from medical Dermatologists and Gastroenterologists.
Keith: As both med derm and GI is start to actively look for higher some patients they are finding them in their practice.
Keith: Many of these health care providers are motivated to manage these SM patients directly and build their expertise in at that.
Keith: And this leads me to the last factor.
Keith: The critical mass of experienced hematology oncology and allergy immunology providers that we have established.
Keith: This foundation of Ava can experience providers will be an engine for growth this year.
Keith: And we have a significant opportunity to continue to increase the depth of prescribing amongst this group, while we also grow the breadth of new prescribers.
Keith: In 2025, we are increasing our investments in education, and marketing building initiatives as well as in the expansion of our commercial and medical field infrastructure.
Keith: In order to drive multi dimensional growth as we make progress towards our goal of $2 billion in advocate revenue by 2030.
Keith: Helena, it's going to go and more is going to spend more time and go into more depth on our planned investments.
Keith: And the factors that inform our 2025 guidance later on the call.
Keith: Yeah.
Keith: Beyond driving revenue growth, we have clear areas of focus to create value this year.
Keith: This includes building a durable awesome franchise with Ela messing up that will enable us to drive growth throughout the next decade.
Keith: <unk> is our next generation kit D 16, B inhibitor.
Keith: With our phase III trial of elements now underway, we are driving innovation in the treatment of S. M by moving beyond symptom control to disease modification.
Keith: We are prospectively measuring the impact of bottleneck on significant I S. M disease Morbidities in the Harbor registration study.
Keith: And we are confident we will be able to clinically differentiate enough nib and deliver improved outcomes fryers and patients.
Putting blueprint in the driver's seat of innovation and that's M for years to come.
This year, we are also initiating a range of proof of concept studies to further define the broad potential of blue eight O H, our next blockbuster opportunity.
Keith: The healthy volunteer data, we presented last month showed that blew it always has a wide therapeutic index and impressive pharmacodynamic impact.
Keith: Lew, Italy, its clinical profile positions it as the best in class and likely the first in class oral kit inhibitor across a range of mast cell mediated allergic inflammatory diseases that impact many thousands of patients.
Keith: Later on the call Becker will talk more about our next steps as we stand up our proof of concept studies, which we expect to begin generating data later this year.
Keith: And last but not least we are advancing our portfolio of earlier stage programs that will be the core value drivers of tomorrow for blueprint.
Keith: Our 2025 capital allocation strategy is aligned with our plan for value creation.
Keith: We are prioritizing investments that will enable us to drive significant near term growth, while solidifying that says leaders and developing novel mast cell therapies.
Keith: Importantly, we are leveraging our clinical development and commercial infrastructure as well as our deep scientific expertise to drive growth and innovation, while at the same time, maintaining our financial discipline. The only allows our best programs to advance.
Keith: You can see the impact of our approach as evidenced by the significant reduction in cash burn we achieved in 2024.
Keith: Mike will talk more about our investment plans and the strength of our financial profile at the end of the call.
Keith: Altogether, we have an industry, leading expertise and targeting muscle biology.
Keith: We have a unique portfolio of novel mast cell targeting commercial and investigational programs.
Keith: And we have a track record of operational excellence.
Keith: Which placed us in the strongest position we've ever been in as a company as we've set up 2025 to be another great year.
Speaker Change: With that I'll turn it over to <unk> to dive deeper into EBITDA performance.
Speaker Change: Thanks Kate.
Speaker Change: Last year I highlighted the importance of the first quarters of launch in setting the growth trajectory for a new product.
Speaker Change: Reflecting on 2024, I'm proud to say that our team has truly hit it out of the park, establishing a rock solid foundation for Ava kit and blueprint.
Speaker Change: Let's look at our results.
Speaker Change: In 'twenty 'twenty, four we achieved $479 million in Ava kit net product revenues.
Speaker Change: Fourth quarter revenue was $144 million with 124 million in the U S and $20 million ex U S.
Speaker Change: Growth in the fourth quarter was driven by continued strength in the business fundamentals, we've been reviewing throughout the year across our key stakeholders patients payors and providers.
Speaker Change: Starting with patients.
Speaker Change: We continued to see strong and steady new patient starts and low discontinuation rates compliance.
Speaker Change: Compliance remains high and we saw a trend towards multi year durations of therapy, underscoring Ava kids sustained benefit and Tolerability, which many patients have described as life changing.
Speaker Change: In recent market research, 95% of patients strongly agreed they're satisfied with either kit and more than 80% said they would recommend it to another patient.
Speaker Change: Now turning to payers and access.
Speaker Change: Access remains strong in Q4 with robust payer coverage and fast times to fill.
Speaker Change: Our mix of free and commercial goods remained stable and similar to what we saw in Q3 as we expected.
Speaker Change: In Europe, our international team recently obtained ISO pricing for Ava kit in Germany.
Speaker Change: The price reflects Ava kids proven benefit risk profile and sets a benchmark as we move through additional price negotiations and lunch I S. M in more countries. This year.
Speaker Change: Left let's look at health care providers.
Speaker Change: In Q4, new and repeat advocate prescribers grew significantly in both academic and community sites of care let's.
Speaker Change: Let's take a closer look at how we're growing breadth and depth and this next slide.
Speaker Change: In prior quarters, we've talked a lot about breadth and depth of Eva could prescribing in the top 400 providers by patient volume to show how first positive experiences with the Kid lead to increased use overtime.
Speaker Change: Over the course of this launch our field teams have been engaging several thousand providers, establishing a large and growing base of prescribers, who have experience and are motivated to diagnose and care for additional S. M patients in their practice.
Speaker Change: These new charts now capture the school experience from all the providers, who have prescribed advocate for us and since I am approval.
Speaker Change: There are a couple of important points to highlight here.
Speaker Change: First you can see in the chart on the left that the total number of Eva kit prescribers has grown significantly.
Speaker Change: Well, we've seen growth in prescribing across the thousands of Haemonchus allergy specialists, we've been targeting the number of allergists with Eva could experience has grown tenfold since ice M. Approval. This is crucial because many of the providers with the highest volume of ISR patients in their care our allergist both in.
Speaker Change: <unk> and community sites of care.
Speaker Change: Second.
Speaker Change: Positive first experiences with Eva kit are driving repeat prescribing and broadening the lens on who was an Eva kept patients.
Speaker Change: In the right hand chart, you can see we're growing the number of prescribers with multiple patients on therapy.
This is predominantly by allergists, who are moving quickly to treat more of their patients.
Speaker Change: They're finding Ava kids profile easy to manage with nothing beyond their typical check ins two or three times a year.
Speaker Change: The large and growing base of advocate prescribers across all specialties and settings is an important lead indicator.
Speaker Change: Hmm.
Speaker Change: When we activate a new prescriber, we're planting a seed for future growth as we know that prescribers deepen use of Eva kit overtime.
Speaker Change: Taken together these results exemplify how we built the engine for our continued growth.
Speaker Change: As we enter the next phase of launch we are making strategic high return investments to drive growth over the course of this year and beyond.
Keith: As Keith said, we're scaling our infrastructure and expanding capabilities to reach more providers and patients in line with the larger S. M opportunity, we see in front of us.
Keith: These investments are focused on four key priorities.
Keith: First driving urgency to treat with patients and providers through tailored peer to peer offerings and direct to patient initiatives.
Keith: Second generating and disseminating data on real world, Eva could experience and I S M disease burden.
Keith: Third, enabling ISR launches in additional geographies and finally.
Keith: Expanding our commercial and medical field teams in the U S to broaden our reach to additional providers and specialties.
Keith: The field team expansion is a key investments that will enable us to increase our interactions with <unk> and allergists and also broaden our reach to medical dermatologists and Gastroenterologists, who are managing S. M.
Keith: This will enable us to engage several thousand more providers, increasing the pool of diagnosed patients we can directly impact by 40%.
Keith: We plan to have our expanded team hired them in the field by the second half of 2025.
Keith: We are confident these investments will continue to build this market drive growth and sustained Ava kit as the durable market leader across the spectrum of S. M.
Keith: Turning now to our guidance.
Keith: The strong foundation and trajectory of growth we established in 2024 informs our revenue expectations for this year.
Keith: As Keith mentioned, we expect to achieve $680 million to $710 million in Ava kit revenue in 2025, representing a 45% increase at the midpoint and robust year over year growth.
Keith: At this point in the launch we have good insight into the factors that drive our business overall and impact quarterly revenue.
Keith: This includes more fundamental drivers of growth over the long term such as new patient starts and discontinuation rates.
Keith: And the growth of our international business, as we achieved pricing and reimbursement outcomes in additional markets through the year.
Keith: It also incorporates factors, which can impact shorter term revenue performance, such as compliance and free goods right.
Keith: Our guidance range is narrow and accounts for the variability we've come to anticipate across these key factors.
Keith: With this guidance range, we expect to see strong and steady growth in patients on therapy through the year as more patients are prescribed Ava kit and stay on therapy, putting us further down the path towards realizing $2 billion in Ava kit revenue by 2030.
Keith: Overall, we couldn't be happier with where we sit today with another year of tremendous achievement under our belt and a proven track record of commercial and medical execution.
Keith: With the vast majority of S. M patients still untreated today, we have important work to do and incredible growth opportunities ahead.
Becker: With that I'll turn it over to Becker.
Becker: Believe me.
Becker: And I assume we know that providers and patients find long term safety and efficacy data meaningful and motivating and aesthetic setting of a chronic lifelong disease.
Becker: Later this month, we will share 14 presentations at the American Academy of allergy asthma and immunology annual meeting.
Becker: A key update will be three year follow up data from the pioneer study of Eva kicked in ISS.
Becker: We now have a very large body of either get data, including pioneer results beyond three years that we can now combine with real world evidence beyond 18 months.
Becker: Other these data confirm that 25 milligrams strikes the right balance of benefit and Tolerability for the vast majority of <unk> patients, enabling patients to stay on therapy chronically and continue to benefit over time.
Becker: Quite a while we will also present new data for the minority of patients with high disease burden, who escalate to 50 milligrams in the pioneer study with a median follow up of 10 months post escalation.
Becker: These patients 50 milligram had a similar safety profile as 25 milligrams bid in the six month Registrational portion of the pioneer trial.
Becker: No patients discontinued treatment due to side effects.
Becker: In addition, we will share data on the positive impact of advocate on bone health from a subset of patients in pioneer who had bone scan data available.
Becker: And Selena discuss these types of data highlights the importance of early treatment to prevent significant morbidities driven by the chronic effects of the disease and provide additional reasons for both providers and patients to start treatment with advocate.
Becker: These data also underscore our decision to prospectively include rigorous assessments of bone density and the harbor study of all enough to know which redesigned in collaboration with the FDA.
Becker: Now moving to Blue aliens.
Becker: At the Jpmorgan Conference last month, we were incredibly pleased to share positive healthy volunteer data showing blew right away was well tolerated with dose dependent pharmacokinetics and rapid robust trip days' response.
Becker: These data were exactly what we were hoping to see from blue anyway, and the crop in the clinic and positively differentiate it from other programs targeting wild type kit.
Becker: Laredo has unique clinical profile and wide therapeutic index support our differentiated approach to development.
Becker: Flooring innovative dosing regimens to achieve the right balance of efficacy and Tolerability.
Becker: Our goal is to provide meaningful clinical benefit to patients across multiple allergic and inflammatory diseases with an oral medication that is well tolerated and can be taken chronically.
Becker: Today, we're working to initiate proof of concept studies in chronic urticaria and allergic rhinitis conjunctivitis and the first half of 2025 with additional studies in allergic asthma and mast cell activation syndrome or impact in the second half.
Becker: Proof of concept studies are designed to inform the most efficient next steps in developing and development by exploring various doses and dosing regiments.
Becker: In chronic urticaria, we're wildly targeting has been derisked by others. Our studies will include several arm to measure the impact of various dosing approaches.
Becker: An allergic rhinitis and conjunctivitis will also be looking to derisk muscle biology in respiratory disease to inform additional development.
Becker: Importantly across all of our proof of concept studies there are opportunities to rapidly generate an informative data and we expect to report initial data later this year.
Becker: Mmhmm cast, we see an exciting opportunity to tie together much of what we know about mass cells across our portfolio here were working to utilize advance diagnostic testing to help us better characterize patients with symptoms of mast cell activation, who may benefit from our efforts.
Becker: In both <unk> and blew it away programs.
Becker: And the next installment of our scientific seminar series planned for the first half of this year, we will dive deeper into the biology of a M castle and the potential impact to the large number of patients suffering from an underserved disease.
Becker: We believe bleed blue radar its unique profile and our differentiated approach can unlock true pipeline and a product potential.
Becker: With the benefit of strong early data, our investigators and all of US at blueprint are really excited to embark on this next phase of the development as we pursue new treatment approach that could have tremendous patient impact.
Mike: Now over to Mike for more than a financial thing.
Mike: Thanks Becker earlier. This morning, we reported detailed financial results in our press release.
Mike: For today's call I'll touch on a few highlights.
Mike: 2024 was a remarkable year of progress with significant revenue growth and continued opex discipline, leading to further strengthening of our financial profile.
Mike: As previously mentioned, we achieved total revenues of $479 million from net product sales of advocate representing robust year over year growth.
Mike: In addition, we reduced our operating expenses in 2024 compared to 2023, as we focused investment on our mass cell franchise programs and efficiently operated the business.
Mike: As Polina noted, our 2025 revenue guidance and the relative narrowness of our guidance range reflects our confidence in our understanding of the fundamental drivers of our business.
Mike: We've also incorporated our view of the quarterly revenue dynamics inherent in our business into this range.
Mike: For example, we expect Q1 to be more challenging due to a number of financial factors, including the typical insurance Rivera frication process challenges common across our industry that are now impacting a much larger base of patients on <unk>.
Mike: As well as potential FX headwinds given the larger contribution from our international business.
Mike: Looking at operating expenses, we anticipate a modest increase in both R&D and SG&A expenses in 2025, as we increased investment in commercial infrastructure and progressed development of Ellen estimate and blew it away.
Mike: Importantly, the inflection in advocate revenue combined with a focused disciplined approach to investment led us to further reduce our operating cash burn by more than half last year and in 2025, we expect our operating cash burn to continue to decline significantly.
Mike: We entered 2025 with a cash position of $864 million and we expect our cash balance to be further strengthened by the approximately $80 million payable to us upon the closing of Gsk's acquisition of <unk> as a result of our equity stake.
Mike: Together this puts us in a position to be able to sustainably invest in compelling growth opportunities and drive multiple value inflect in catalysts across our portfolio.
Mike: With that I'll now turn the call back over to the operator for questions operator.
Mike: 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 your telephone keypad.
Mike: Please limit yourself to one question.
Mike: So just a moment to compile the Q&A roster.
Speaker Change: The first question guys. She brought Kinney know of Stifel. Please go ahead.
Speaker Change: Hi, good morning, and thank you.
Matt on the guidance calls for adding $215 million in <unk> sales. This year, but then to straight line from the end of this year to the $2 billion number in 2030, youll need to step that up to around $260 million and absolute sales addition, each year from 26% to 30.
Speaker Change: One am I thinking about that right to what is the most important lever you have to pull to increase the absolute dollar edition and year for the ISR launch and beyond thank you.
Brad: Thanks, Brad for your question Yeah, we are incredibly pleased to be able to project. The midpoint of our R. 22 me by range of 45% growth rate, which as you know really puts us again in and pretty rare rarefied air them relative to other rare disease launches and and to see this type of growth is really incredibly.
Brad: Lighting, Chris do you want to talk little bit more about brad's questions relative to ramp yeah. Sure. So you know as Kate said, we are really pleased with the growth that we see coming for the kit in 2025 mm. That's growth rate you know puts us very much in line and actually at the upper end in terms of analog rare disease launches that we see.
Brad: I'll also note that you know if you just look at external views on you know consensus now and in 2030 I think this is very much in line with that wrap so a very reasonable ramp them. You know what we are seeing in terms of steady growth in patients on therapy is really being driven by you know the growing base of experience that we have with <unk>.
Brad: We've spent the last 18 months I'm building, a really strong commercial foundation and really an engine that will drive the continued growth. We now have more prescribers that our experience. We know that once the prescriber has that positive first experience. They go deeper and so we've planted a lot of the themes that are going to drive that growth over the next.
Brad: Several years Am I also think some of the investments that flea and I mentioned in terms of expanding our commercial field and infrastructure are really putting that as foundation in place to drive growth into 'twenty six 'twenty seven beyond right. So it's it's really about building that foundation that will enable us to reach more of the patient.
Brad: Who are diagnosed with I S M them and get them on therapy. So you know we're very much at the early stages of that there's a ton of headroom for growth and you know, we really see a strong steady growth to get to you know 2030, and the $2 billion I'm Guardedly Sac.
Speaker Change: Thank you. The next question gets his salary and a rich guy off Goldman Sachs selling. Please go ahead.
Speaker Change: Good morning, Thanks for taking my question post reporting 808.
Speaker Change: Data in healthy volunteers could you just speak to us about how Derisked do you believe the safety profile is and how you're thinking about potential dosing strategies and the different indications and as we look at these indications of <unk> asthma and others, you'll read through as well as when we might see these datasets. Thank you.
Speaker Change: Yeah. Thank you. So thanks for that question and you know as we as we said at Jpmorgan and in fact, we just mentioned weird, which is incredibly pleased with the profile of 808 is exactly what we were hoping to see from a healthy volunteer study and now we're embarking on on you know moving into Ah patients, but could you talk a little bit more about how you want to think about the derisking of the healthy volunteer data and then also.
Speaker Change: What we hope to see this year, yeah sure with all being the the healthy volunteer data gave us a very wide therapeutic index. We have multiple doses that we can work with and we have multiple different strategies that we've talked a little bit about before to the molecule and this includes a single dose dosed chronically.
Speaker Change: It includes dosing strategies, where we start with a brief high dose and then go to alert us to maintain a response or one will repatriate to effect and in fact, you're doing a patient or a patient population.
Speaker Change: What we're doing in the first half of the year, particularly in the chronic or to carry a space in the inducible urticaria spaces, we're using a.
Speaker Change: Purely kit driven disease to better understand some of these dosing strategies that we can then apply differently to different disease indications and so in the second half of the year, we will get into asthma and into impasse and better understand both in and those diseases how to use them.
Speaker Change: <unk> dosing strategy has been very elucidating and the first half of the year and as I mentioned in the prepared remarks, our expectation is to be able to share. Some of the early data from the first to file at least one of the first two programs.
Speaker Change: In the latter half of this year.
Speaker Change: Thank you. The next question goes to Michael Schmidt of Guggenheim Michael. Please go ahead.
Michael Schmidt: Hey, guys. Thanks for taking my question I just had a.
Speaker Change: A follow up regarding 2025.
Speaker Change: How much of the expected growth in 2025 is driven by your expectation.
Speaker Change: Okay didn't existing patients versus adding new patients.
Speaker Change: Look at the chart of slide seven it looks like a lot of the prescribers have only one patient on therapy.
Speaker Change: How much opportunity is there.
Prescribed the drug.
Speaker Change: Additional patients.
Prescribers.
Speaker Change: Sure.
Speaker Change: So.
Speaker Change: Thank you.
Speaker Change: Yeah. Thank you Michael you know I think I think what we're really pleased about is that we're off to an incredibly strong start and as we said you know, we we believe that less than 10% of the currently diagnosed that same population is currently on an EBA cat and to your point, we have a tremendous number of prescribers.
Speaker Change: They have an opportunity to both deepen their experience as well as adding new prescribers play did you want to talk a little bit more about that yeah.
Speaker Change: As Kate mentioned, we see tremendous potential to continue to drive growth both from breadth and depth and importantly, when we look at that chart of providers am I you know I think the most exciting piece that jumps out to me is how it shows that.
The kids experience appeal to a broad base of customers across all specialties and setting some we see adoption across both he he marks.
Speaker Change: As well as growth in the allergy immunology setting.
Speaker Change: And as we expand our field force will also be broadening the range of providers that will be able to engage so we would expect to see breadth to continue to grow and we also expect to see deepening because with every provider who starts a patient on eve of kit.
Speaker Change: We know that that first positive experience will lead them to identify additional patients over time, it's really like planting a seed for further growth and so you know.
Speaker Change: In short I would say, we would expect to see growth across all of these are all all of these dimensions and one thing just to note for you guys. I mean, I know we have shown this type of visual representation of the breadth and depth. Previously this is a little bit of a different view and fleet I mentioned this in her prepared prepared remarks, we had typically looked at the top 400 providers by.
Speaker Change: What we look at it as EM volume were now showing you everyone. Since I S. M approval, who has been prescribing prescribing Ava cat and I think what you see here and what we've always said is we're calling on you know multiple thousands of these providers and we're seeing uptake not only in those highest concentration providers, but also throughout the middle and tail of of the chips Linda's point, so if it hits.
Speaker Change: <unk> is very conducive to a range of specialties in community settings, as well as academic centers and.
Speaker Change: And we are excited to see that continued growth on both of those parameters are this year.
Speaker Change: Thank you. The next question because you're buying at Chang of J P. Morgan Brian. Please go ahead.
Brian: Thanks for taking my question this morning.
Brian: As you think through your first set of POC indications.
Brian: What's your latest thoughts on that and also duration of Tryptase reduction that will ultimately deliver a response and also have the minimal safety package. Thanks.
Becker: Yeah, Yeah. Thanks, Brian Yeah. We've we have blueprint have had been good students of trip case over the last decade and have probably gone deeper than most on that so I'm going to hand that over to Becker now based on that.
Becker: So it's important to remember that Tryptase is not a mediator.
Becker: It is an indication of target engagement, but as we've seen in systemic mastocytosis and expect to see throughout multiple diseases, particularly the more complex diseases like asthma that there will probably not be a one to one correlation between symptom reduction or disease reduction and trip days so well.
Becker: We've used the trip phase four is to define a therapeutic range and we also know from I assume that once you get a massage under control you can maintain that control oftentimes without killing it. So I think the what I would say to that is stay tuned for our results for the first few proof of concept studies and we'll be able to explain more.
Becker: With respect to the level of drug needed over time, but I don't think it's going to be a trip they story throughout the program.
Becker: Yeah.
Speaker Change: Thank you. The next question goes to Amy <unk> of Needham and company Amy. Please go ahead.
Speaker Change: Hi, This is Dylan on for Amit.
Speaker Change: What assumption had been building one 2 billion dollar guidance examples like the sequential patient growth market expansion, except for U S and in other indications other than I assume.
Speaker Change: Well so in your 2 billion guidance have you made any assumption on your competitor and our competitor in Chi.
Speaker Change: And lastly, do you expect to expand your current commercial something to achieve a stupid and killed.
Speaker Change: Thank you for that question could you maybe if you'll talk about how we're thinking about the ramp to the $2 billion in the competitive dynamic and then flint or do you want to take the field expansion and what you're hoping to see that.
Speaker Change: So you know our our view is that are we have a lot of headroom to continue to grow Eva cat penetration and if we think about you know with $2 billion opportunity in 2030 and in many ways I think that's a conservative view of what can be achieved right. We see more than 25000 diagnosed patients the United States as of today.
Speaker Change: We know that that diagnosed patient population has been growing at double digit rates and we believe it's going to continue to grow robustly. Given you know an updated view of what we think that youre prevalence of this disease as them with more than 60000 patients in the United States and so if you just make some kind of reasonable assumptions about you know patients.
Speaker Change: On therapy added $2 billion run rate.
Speaker Change: You're talking about a minority of patients still even in 2030 being treated with either Canada, United States and.
Speaker Change: Our view is that you know for the foreseeable future. The most significant competition for advocate is Simpson directed therapy and you know we are really catalyzing, a new market, where prescribers and patients are understanding the benefits of treating with a disease with a therapy that effects the underlying driver of the disease.
Speaker Change: So you know, we're very confident in our ability to continue to get to that I get to that number with very much strong steady growth and and patients being treated and you know given the the dynamics with the Eva kept why we see that one patient start on therapy. They are staying on for a long duration of treatment.
Speaker Change: You know clearly you want talk a little bit about what you're hoping to achieve with the field extension tabs.
Speaker Change: Lately and so the things that we're focused on in the near term early two folds. One is increasing the urgency to treat and also broadening our ability to both increase the number of interactions with our core hematologists and and often allergists as well as to broaden the breadth of the providers that we're engaging with.
Speaker Change: So there's both there's investments I'd put them sort of in two buckets, where we would expect both nearer term as well as longer term impacts.
Speaker Change: The field force expansion you know, we really don't expect to see impact in 2025, that's not baked into our current guidance, we expect that to drive growth really in 2026 and beyond.
Speaker Change: In the meantime, we're also increasing our investments in really driving that urgency to treat by engaging with providers tailoring our peer to peer engagement.
Speaker Change: As well as the increasing our direct to patient programming and we expect those investments to continue to drive growth in 2025 and beyond.
Speaker Change: Altogether with these investments we feel confident in our ability to drive that patient ramp that Christy spoke to them both in.
Speaker Change: In 2025 and through to 2030.
Speaker Change: Yeah. So I think what you've heard is as you know we're investing in more surround sound building on our critical mass.
Speaker Change: And then you know as we increase the number of of our field team members and we can reach more prescribers, we expect we will increase treatment rates.
Speaker Change: And we will see that you know really take effect predominantly next year.
Speaker Change: As that team gets into place and really starts to get embedded in there in their territories.
Speaker Change: As we said this is that it's because you just mentioned it is a growing compelling large rare disease market and this is our plan to invest in capturing Umm al and be able to leverage all of those dynamics.
Gus: Thank you. The next question Gus you Derrick Chiller of wireless fall guys. Derek. Please go ahead.
Speaker Change: Yes.
Derek Chiller: Hey, good morning, and thanks for taking the questions maybe just some follow ups on the field team expansion I guess, how big is this expansion going to be I guess in terms of a percent or absolute basis, and then I guess what percent of the patients are actually being seen by the Gi and the medical derm. So that you can also target with the expanded sales force. Thanks.
Speaker Change: Yeah. So you know you want to talk about just the relative magnitude of expansion here and then also you know what what that benefit's going again, it's we're actually gonna be able to get you are a significant number of more patients directly with the expansion. Yeah. So derik. This is really an incremental field force expansion that will enable us to not only have more frequent engagements on our.
Speaker Change: Our existing provider base, which we know is important to remain filling in for S. M.
But really also expands our ability to engage with med derm N G eyes, which together we can see a couple of thousand bedrooms and G is who are managing two or more S and patience today.
Speaker Change: That piece is really important because he marks and allergists, while they manage them.
Speaker Change: They're the two specialties that manage sort of the.
Speaker Change: The larger tranche of S M patients like by by expanding the field team, we will actually be able to address providers, who are treating the majority of diagnosed S. M. Patients today, we expect that to not only increase penetration into the existing diagnosed patients, but also continue to grow the market as they are motivated to diagnose additional patients.
Speaker Change: Yes.
Speaker Change: Okay.
Speaker Change: Thank you. The next question goes to Marc from of TD, Kevin Mark. Please go ahead.
Speaker Change: Hi, Thanks for taking my questions.
Speaker Change: One just kind of following up again on this midterms and GI Docs do you expect over time that that population starts becoming either a major or the major driver of growth or is this.
Speaker Change: Overtime, just incremental opportunities relative to kind of continue to drive growth within.
Speaker Change: The original kind of target markets and then also just on the 2025 guidance just can you walk through your assumptions around ex U S.
Additional ex U S reimbursement agreements.
Speaker Change: When we might expect us.
Speaker Change: Yeah. So Glenn do you want to take marks the first part of my question just around the specialties I mean, I know we continue to think allergy immunology is going to be the most predominant specialty and managing them.
Speaker Change: Large number of patients here, but how you think about that Jameson Jasmine Christy we could talk a little bit more about international yeah.
Speaker Change: Yeah. Thanks for the question Mark I would say the growth is really multi dimensional across a lot of friends. So we have tremendous headroom to grow across both existing as well as the new specialties, we would expect to see.
Speaker Change: That breadth breadth and depth chart as those grow we would expect to see more new haemonchus and allergists come on as well as deepen we have at this point I think it's a relatively small number of of med term med germs N. G is but I wouldn't be surprised to see if we start to see a few more of those.
Speaker Change: And in addition, you know what's really important is these specialties are motivated.
Speaker Change: Motivated and they're managing and and recognizing and diagnosing Morris M patients and we expect them to be taking on a greater role in the future.
Speaker Change: And I cannot comment a little bit on on the international piece first.
Speaker Change: First of all if we if we look at 2024 I'm really pleased with the performance of our international team at which drove you know really nice strong revenue growth as we know Germany is is the only market right now outside the U S, where we have I S. M reimbursement, we have we can say at JP Morgan.
Speaker Change: We expect to add another five markets to come on line as we go through the year are you know those are many of the usual suspects rates that we're following kind of a similar cadence with I S. M. As we did with advanced a sense of some of the other major European markets and we expect to come online through the year and that sort of a steady steady pace and that's one of the.
Speaker Change: One of the variables honestly as we think about the guide that we've kind of incorporated them you know the timing around that as you know not something you want to put a firm stake in the ground on right now given you know, we certainly don't fully control those negotiations with some of our European payers and that you know certainly think we're in a good place that the.
Speaker Change: Price be green in Germany, actually I think is a really nice recognition of the value of a vacate in I S. M and I think puts us in a nice spot as we as they purchase negotiations. So I'd expect you know additional markets coming online overall from a revenue perspective, you know last year, we said that international we expect it to be sort of in that 10% to 15%.
Speaker Change: Range, that's where we work through the year, obviously with quarterly ups and downs as you would expect that I think this year, that's probably the right range as well. So we will see you know a nice growth both in the U S as well as outside the U S.
Speaker Change: And I want to actually just this is fully in again I Wanna add one additionally be color comment to your question around the potential to grow the market by engaging these broader specialties and you know as we look at medical dermatologists.
Speaker Change: A lot of SM patients the majority in fact present with skin involvement and so cutaneous mastocytosis in adults. We know is actually a reservoir of undiagnosed I S. M patients, which is immediately actionable.
Speaker Change: The med germs, we also have a number of patients who are showing up with with Gi symptoms sort of ibs like symptoms and so a lot of those educational efforts.
Speaker Change: On both a G I as well as a more systemic symptoms as a flag can continue to drive and grow this market over time.
Speaker Change: Okay.
Gustavo: Thank you. The next question Gustavo <unk> of Raymond James. Please go ahead.
Speaker Change: Hey, guys I was curious if you think it's possible to maybe have some blue 808 data you know, Pennsylvania to carry out by year end 'twenty five and then the M class angle here. It seems you know super interesting, particularly for you guys.
Speaker Change: Do you see you know a potential total symptom score like your proprietary methodologies needing to be used for blue Blue studies in <unk> and.
Speaker Change: And how are you ensuring that you know potential impacts patients with a D. 816 week mutation are being identified and you know potentially put on it because it was all.
Speaker Change: Sensitive testing involved here any color there would be helpful.
Speaker Change: Yeah. Thanks for the questions I'll take the easy one and then give back or the hard stuff in terms of data yeah. We do expect data by the end of the year you know coming out of these studies, what we're gonna give you guys more on that both the design and end and when it's more specifics around what you can expect there as we get these trials off the ground, we start rolling patients, but yes, we do expect data.
Becker: And then Becker do you want to talk about MKS is just how we're thinking about it yeah. So MKS is an amazing opportunity to really address a patient population with very little right now in terms of the understanding of their disease or therapeutic options and with respect to the symptom met the symptom measurement.
Becker: We think that there will be a need to refine the way that we look at something but these are activating ourselves very similar to what we've seen and are what drive systemic mastocytosis symptoms and so we think we're already well on our way to being able to measure the symptoms in these patients and as we select patients for our studies that will be part of that focus.
Becker: With respect to elucidating the extent to which the mutation is driving the disease versus patients who don't have a mutation and have more classic in cash.
Becker: Already with the <unk>.
Becker: Digital droplet PCR you can identify the patients that have the mutation that are being treated as ymcas patients and so really increasing the awareness of this and using not testing as the beginning we have talked about the rolling circle amplification test and is more sensitive for kit mutations and we are working with vendors to bring back.
Becker: Board and we'll say more about how we will incorporate that into some of the studies and then how it it will be available.
Becker: <unk> in the future for broader testing.
MKS Christie: That storage about really say about MKS Christie do you want to add a quick point and we're obviously very excited about <unk>. We think that that says you know a really interesting factor for growth. Both for army you paid it kept portfolio as well, especially with 808 am I think its important to remind everyone that when we talk about the U S. M opportunity the number of diagnosed patients they see the true prevalence of this.
MKS Christie: Additionally, sort of reservoir potentially of patients that may be sitting in <unk> that is not yet detected that as sort of a bother biatch right. So I think it's just important to remember that you know the headroom that we see for growth is in the current our current view of the I S. M patient population and M cast as really an opportunity I think for significant upside.
MKS Christie: It's crazy.
Speaker Change: Thank you. The next question does she Mike <unk> of Morgan Stanley Mike. Please go ahead.
Mike: Good morning, Thanks for taking the question, maybe just one on blue eight await a it looks like you shared fairly comprehensive topline data from the phase one study already so just curious any additional data points for quality, either we should be focused on thanks.
Mike: Yeah. Thank you for that question, Mike I mean, where we're kind of putting together some of the data quantity I know its a poster it's got some limited real estate. So we're gonna we're figuring out from just the higher D perspective, well, we wanted to put in there to make sure that that you know a health care provider kind of customers at that meeting and get a really good sense of the profile of our blue 808, but were.
Mike: Looking forward to presenting that data at Quad AI and importantly, we're going to see the long term safety data from an efficacy and safety data and specifically for Ava Cat 25 milligrams as well as now 10 months of experience, where you know a subset of patients, but a meaningful number of patients with advocate at 50 milligrams and and.
Again that safety data.
Mike: Continues to look really strong and very kind of consistent with what we saw very early on in the program. So that's what you'll see is the only gets worse over time. So that's a really amazing kind of an exciting presentation for us at Kodak.
Speaker Change: Thank you. The next question because she David day of UBS, David. Please go ahead.
David: Great. Thanks for taking my questions and I just had a question.
Speaker Change: A list of nib to surround view some key differentiation.
Speaker Change: Just to the computer to advocate, maybe you can share some of the key efficacy and safety benefits over it again.
Speaker Change: Yeah, and again, that's where we're really thrilled with all net net I mean, I think one of the things for us as a company is to have the assets and strategy in place to achieve our goal of continually improving outcomes for SM patients. While also maximizing the longer term potential of our overall franchise on the back of the opportunity that we've talked a lot about.
Speaker Change: It's really compelling and growing it's just you know an exceptional place for us to be back or you want to talk a little bit about what your what you guys are driving in the inner Harbor study to differentiate on that.
Speaker Change: I would first say that Eva leave little room for improvement and so the distinction here is not about a new compound that has an advantage over any of a cat, it's really a distinction in the development.
Speaker Change: And we're developing LMS to move to have a different profile than Eva kicked us in the future and as I mentioned before the two main areas that we're incorporating into harbor are a really rigorous look at both bone health and then the episodes of anaphylaxis could occur in patients with indolent pest.
Speaker Change: So our plan is to have a distinct labeled for LMS to nib and to really drive. This notion that you need to treat patients early you don't want to wait until the symptoms are unbearable and then come after a lot of the damage is already done, but you want to treat patients in a preventative manner with an LMS.
Speaker Change: Both on the development program and then the label will drive those points top quintile.
Speaker Change: Thank you. The next question because she ran at Benjamin of citizens J M. P.
Ben: Ben Please go ahead.
Speaker Change: Right.
Speaker Change: Hey, good morning, guys, congratulations on the quarter and of course those are.
Speaker Change: That's on the on the guidance.
Speaker Change: As it becomes available.
Speaker Change: Ex U S opportunity can you talk a little bit about what percentage of the 2030 revenues that you've highlighted and peak revenues comes from the X U S.
Speaker Change: Opportunity and then just as a follow up to a prior question do we expect any interim data from part one of Oak Harbor and when do we think the.
Mike: 33 portion you know, Mike ultimately read out.
Speaker Change: Yeah.
Speaker Change: As all of US Chris do you want to talk little bit about international and then Becker I'll have him talk a little bit more about the Harper study.
Speaker Change: Yeah.
So you know I think that international has become an important part of our our topline revenue is certainly an important part of our revenue growth. We expect it will slowly pick up over time in terms of contribution but even in 2038 my expectation is that the U S is driving the majority of our revenue opportunity and obviously, we won't get out.
Speaker Change: Now into the next decade, you know, we'll see how things evolve, but you know with these types of markets.
Speaker Change: The U S tends to be you know driving the most significant topline revenue.
Speaker Change: Whether that ends up being 70, 30, 70, 525, well see I think theres a number of different scenarios that get you there and obviously, how how some of these pricing and reimbursement card slip a little off the drive that up but the bottom line is under any circumstance I'm. You know we will still have a lot of headroom to penetrate both in the U S as well.
Speaker Change: Outside the U S to make these numbers you know and I view very very achievable.
Speaker Change: Do you want talk a little bit more about how the harbor studying expectations on timing there yeah. So as Keith mentioned earlier, the harbor studies underway I'm, bringing the sites up with the new design is the phase that we're in right now and then with respect to timelines and Readouts will will update you later as we get a better sense of how things are going out there.
Speaker Change: Early in the in the in the process.
Speaker Change: Thank you. The next question guys you clean it Casey of that claim. Please go ahead.
Speaker Change: Hi, good morning, and thanks for taking our question I know you made some comments on seasonality expectations for 125, but was there any seasonality you saw in <unk> and then with the derm and Gi is you'll be targeting or they typically more rapid of doctors are more conservative than their haemonchus allergist colleague and any notable differences in the type of patients that these docs are saying versus the.
Speaker Change: Hemo chronologist that might drive differences in prescription trends.
Speaker Change: Yeah. Thanks, Colleen, maybe maybe Mike you could talk a little bit about the kind of quarterly dynamics, particularly on Q1, and if we need to go to well how do we think about different providers. I mean, one thing I'll say, calling is that we now have a very good sense of these quarterly dynamics since we have a whole year of Biosimilar about from 2024 and this is a chronic disease.
Speaker Change: We see you know we do see at times around vacation points that patients do delay starting therapy for for a couple of times until they come back from vacation, we've talked about that in the summer we talked about that in Q4, we're baking that in now so where where that part of our expectation and it's incorporated into integrated generally over the course of the year, Mike I don't know you want to hit a little bit more on just the Q1.
Speaker Change: And that makes up that you know, we all experience and thanks.
Speaker Change: Thanks, Dave I think you touched on a little bit of the seasonality piece I think Q1, I think as everyone knows it's kind of unique in the industry, we often see financial headwinds and I noted that in our remarks in the script.
Speaker Change: My formal remarks, but essentially you have all the re verifications that take place for insurance purposes, and that's like we're confident in that but it is a process you have to go through and when we have a larger base of patients that's increased effort, but that's a good good problem for us to have Additionally, you know a couple of other things that we're facing we're seeing I think everyone knows it's just rip the movement in the dollar we're seeing some FX.
Speaker Change: Headwinds and I think one other one to call out it's just like quarterly ordering dynamics that we have a much better handle on now as we've learned more about the business and in Q1, a couple of our large customers actually will have like one less ordering day that we than we typically see in a normal quarter. So I think all of that kind of leads into some you know potential.
Speaker Change: Potential headwinds, but that doesn't change our outlook on the year that is absolutely baked into our annual guidance and it's just really our understanding of the seasonal and quarterly dynamics that we see.
Speaker Change: And to your question about the the broader specialties terms in G. I. So I think we would expect to see to growth impact of this one is both growing the size of the market by capitalizing even more diagnosis and the second is by growing the treatment of these patients and get sort of rec more more a recognition of the bird.
Speaker Change: And and and the urgency to treat with Eva cat.
Speaker Change: As we look at these specialties are as I as I talked about derma are gonna be seeing patients who are coming in with skin involvement as sort of part of their symptom presentation of I S. M. Cutaneous mastocytosis is an actionable steps to follow up and do the differential.
Speaker Change: I noticed this and find the S M patients within that are there.
Speaker Change: You May also play an important role in co managing these patients and so really the more arms, we can get around this to see the burden and the impact that the ISR is having on patients' lives. All of these are important weights on the lever to move more patients towards treatment.
Speaker Change: Okay.
David Lebowitz: Thank you. The next question gets you David Lebowitz of City David. Please go ahead.
Speaker Change: I guess John on for David Thanks for taking our question.
Speaker Change: From a high level can you help contextualize, how you expect your $2 billion 2038 could estimate translates to the $4 billion franchise speak.
Speaker Change: Specifically what are the key drivers for that incremental growth how should we be thinking about general timelines and how you expect to indicate no in estimated share evolves during that time.
Speaker Change: Well. Thanks, Thanks for the question I'll hit it to Christie Love that you guys are modeling out until 2040 is really excited about the beyond 2035 was just call. It that's awesome Christie do you want to talk more about that alright. So you know I think maybe just at a high level are you know the the diet around 20 are things sort of the view of the barbell you Ann.
Speaker Change: I think really just reinforces our view that in 2030, if we're you know the $2 billion opportunity for either Cat. We don't think we're at peak and we don't think we're close to peak, we think that you know that's a market they'll have a lot of room.
Speaker Change: We need to be penetrated as I said earlier, you know you're just kind of make some high level assumptions about how many patients may be on therapy in the U S and 2030. It is still a significant minority and we expect that you know diagnosis is going to continue to grow. So you know the $4 billion of you just really reflects the fact that we think we will continue to penetrate into I S. M.
Speaker Change: In the next decade, what exactly that looks like between advocate and Alan That's Nab. Obviously, you know will depend upon the strategic choices that that will make and I'm sure. We'll have more of a view on that when we have the harbor data in hand, and get a little closer I do think it's worth noting that you know, we don't really see a lot of credit for Elena.
Speaker Change: In anyone's models at this point, though we do think you know this is a really significant opportunity that will extend a multibillion dollar franchise in the next decade.
Speaker Change: So there we're getting a growing piece of a growing pie and we have all the assets and strategy in place them within the same portfolio to enable us to kind of drive this you.
Speaker Change: Two to where we need to get to them beyond so.
Speaker Change: Thank you. The next question guys. She might speak lot of Oppenheimer. Please go ahead.
Speaker Change: Hey, guys. Thanks for the question maybe another one for Mike on the financials, you're guiding to $2 billion in sales by 2030, our arena, where we can start to triangulate to cash flow positivity.
Speaker Change: Yes.
Speaker Change: Matt Great question I think you were we Havent put a firm stake in the ground into guidance as to when we're going to achieve profitability, but I think when you see our 10-K later today like we significantly reduced our operating cash burn in 2024 from 2023, I think 2023 we're well over $400 million 2024 was under 200.
Speaker Change: We expect to see another significant reduction in cash burn in 2025, and as you grow revenues like our our goal is going to be to operate this business with financial discipline and discipline and we firmly believe we're going to be able to grow revenues faster than operating expenses, while still being able to invest in the exciting growth opportunities out of this and at some point those curves crossed will be cash.
Speaker Change: For a positive, but we're not going to put a specific data on that right now.
Speaker Change: Yeah.
Speaker Change: Thank you we have time for one more question. The final question guys, Hey, David never got 10 of Wedbush Securities. David. Please go ahead.
Speaker Change: Sure.
Speaker Change: Oh, Thanks for squeezing me in I just had a.
Speaker Change: So even though.
Speaker Change: Science, and chemistry behind or either way.
Speaker Change: Was told if you could help us out how are you avoiding some of the neutral opinion, another sort of Brexit folks have seen or if it's.
Speaker Change:
Speaker Change: It's simply a.
Speaker Change: Kinetic pharmacodynamic property of your molecule.
Speaker Change: Okay.
Speaker Change: Yeah, I think David we're glad to get you in under the wire here I don't Becker, we only have like 30 seconds, sorry, we can always follow up too but.
Speaker Change: Yeah, David So as we said earlier there is a extremely wide therapeutic index.
Speaker Change: I think we all know that if you again and kit inhibitor and push it to very high doses Youll eventually get on target inhibition, but but what we saw was a really robust reduction in trip days in AR in a range, where we did not see side effects that would limit limit dosing.
Speaker Change: Compounds with different antibodies are different volume of distribution matters, and we don't always understand why we see a better profile for one from one to the other but what we do know is the OE has an optimized profile honestly, even better than expected. So we're really pleased to go forward with it.
Speaker Change: Okay.
Speaker Change: Thank you that's all the questions that we have time for today, Kate Haviland Aton Nicobar type of T. H.
Speaker Change: Thank you and and we have a really exciting year in front of us and we are more confident than ever.
Speaker Change: And our future potential so proud to be on this journey with the best team in the industry here and we appreciate your continued support so thank you all.
Speaker Change: Thank you. This concludes today's conference call you may now disconnect.
Speaker Change: Yeah.
Speaker Change: [noise].