Q1 2024 Blueprint Medicines Corp Earnings Call

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Faith: Good morning. My name is Faith, and I will be your conference operator today. At this time, I would like to welcome everyone to the Blueprint Medicines First Quarter 2024 Financial Results Conference Call. All lines have been placed on mute to prevent any background noise.

Faith: Good morning, My name is faith and that will be your conference operator today at this time I would like to welcome everyone to the blueprint medicines first quarter 2024 financial results call.

Faith: Call all lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question and answer session. If you would like to ask a question. During this time simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question press. The star followed by the number twos. Please plan to limit yourself to one question.

Faith: After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press the star followed by the number one on your telephone keypad. If you would like to withdraw a question, press star followed by the number two. Please plan to limit yourself to one question. Thank you. Jenna Cohen, you may begin your conference.

Faith: Thank you Jenna Cohen you may begin your conference.

Jenna Cohen: Thanks, Faith. And good morning, everyone. Welcome to Blueprint Medicine's first quarter 2024 financial and operating results conference call. This morning, we issued a press release that outlines the topics we plan to discuss today. You can access the press release as well as the slides that we'll be reviewing today by going to the investors section of our website at www.blueprintmedicines.com. Also joining me today are Kate Haviland, Chief Executive Officer, Philina Lee, Chief Commercial Officer, Fouad Namouni, President, Research and Development, and Mike Landsittel, Chief Financial Officer.

Thanks, Dave and good morning, everyone welcome to blueprint medicines first quarter 2024 financial and operating results Conference call. This morning, we issued a press release, which outlines the topics. We plan to discuss today you can access the press release as well as the slides that we'll be reviewing today by going to the investors section of our web.

Faith: Site at Www Dot blueprint medicines Dot com.

Faith: Joining me today are Kate Haviland, Chief Executive Officer, Galena, Lee Chief Commercial officer, So Adam.

Faith: Adam Rooney, President research and development, and Mike <unk>, Chief Financial Officer, Chris.

Faith: Christy Rossi, Chief operating Officer, and Becker Hughes Chief Medical Officer are also on the line and available during Q&A.

Jenna Cohen: Christy Rossi, Chief Operating Officer, and Becker Hughes, Chief Medical Officer, are also on the line and available during Q&A. Before we begin, I'd like to remind you that some of the statements made during the call today are forward-looking statements, as outlined on slide 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 disclaims any obligation to update such statements. I'll now hand the call over to Kate.

Faith: Before we begin I would like to remind you that some of the statements made during the call. Today are forward looking statements is outlined on slide three and are subject to a number of risks and uncertainties.

Faith: 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 disclaims any obligation to update such statements I will now hand, the call over to Kate.

Kathryn Haviland: Thank you, Jenna, and good morning, everyone. We delivered another very strong quarter in our launch of AvaKid and endolymph systemic mesocytosis, and we are entering 2024 in a position of strength. We have great momentum across all aspects of our business as we execute on our three priorities that we laid out in January. Our first priority is the Longevity Kit and indolence systemic meso

Kathryn Haviland: Thank you Jennifer and good morning, everyone.

Kathryn Haviland: We delivered another very strong quarter, and our launch of Eva kit and <unk> system systemic mastocytosis and we are entering 2024 and a position of strength.

Kathryn Haviland: We have great momentum across all aspects of our business as we execute on our three priorities that we laid out in January.

Kathryn Haviland: Our first priority is the launch of <unk> in indolent systemic mastocytosis.

Kathryn Haviland: We have driven impressive revenue growth, and importantly, we are building the foundation for top-line revenue growth well into the next decade. We are also making significant progress in advancing our pipeline of innovative programs that are focused on our most compelling opportunities, where we believe we have the greatest prospect of improving patient outcomes. And third, we are maintaining a strong and durable financial position with a self-sustainable financial profile. I'll briefly touch on each of these.

Kathryn Haviland: We have driven impressive revenue growth and importantly, we are building the foundation for top line revenue growth well into the next decade.

Kathryn Haviland: We are also making significant progress in advancing our pipeline of innovative programs that are focused in the motor and our most compelling opportunities where we believe we have the greatest prospect of improving patient outcomes.

Kathryn Haviland: And third we are maintaining a strong and durable financial position and a self sustainable financial profile.

Kathryn Haviland: I'll briefly touch on each of these.

Kathryn Haviland: Starting with the exceptionally strong commercial performance we delivered in Q1. We achieved $92.5 million in AvaKit Net Product Revenue for the quarter. This result surpassed external consensus as well as our own internal expectations. Philina will discuss in more detail the components of our continued commercial success.

Kathryn Haviland: Starting with the exceptional.

Kathryn Haviland: Definitely strong commercial performance, we delivered in Q1, we.

Kathryn Haviland: We achieved achieved $92 $5 million in Ava kit net product revenue for the quarter.

Kathryn Haviland: This result surpassed external consensus as well as our own internal expectations.

Kathryn Haviland: <unk> will discuss in more detail the components of our continued commercial success, but I want to take a moment to say how incredibly proud I am of our hardworking and dedicated commercial and medical team members.

Kathryn Haviland: But I want to take a moment to say how incredibly proud I am of our hardworking and dedicated commercial and medical team members. Their commitment to delivering for patients with ASIM globally is resulting in this type of performance. These results also reflect the profound impact that AvaKit is having on patients, as well as our strong and growing prescriber base. Today, we are also increasing our advocate guidance for the year from $390 million to $410 million.

Kathryn Haviland: Their commitment to delivering for patients with SM globally is resulting in this type of performance.

Kathryn Haviland: These results also reflect the profound impact the advocate is having on patients as well as our strong and growing prescriber base.

Kathryn Haviland: Today, we are also increasing our <unk> guidance for the year to 390 million to $410 million.

Kathryn Haviland: Later in the call, Mike will share more of our thinking around today's Advocate Guidance Increase, which is setting us squarely on a revenue trajectory to peak sales of greater than $2 billion globally. This revenue trajectory makes AvaKit one of the most exciting rare disease launches happening right now and puts us on a similar path to other notable rare disease product launches of first-in-class medicine that built new multi-billion dollar markets. We are also strengthening our presence in allergy and inflammation with Blue 808, our wild-type kit inhibitor.

Later on the call Mike will share more of our thinking around todays advocate guidance increase which is setting us squarely on a revenue trajectory to peak sales of greater than 2 billion globally.

Kathryn Haviland: This revenue trajectory it makes <unk> one of the most exciting rare disease launches happening right now and puts us on a similar path to other notable rare disease product launches of first in class medicines that built new multibillion dollar markets.

Kathryn Haviland: We are also strengthening our presence in allergy and inflammation with Blue 808 are wild type kit inhibitor.

Kathryn Haviland: The Blue 808 program builds on the significant insights we have gained in mast cell biology and in targeting KIT, the master control switch on mast cells, which we believe has the greatest prospect of improving outcomes for a large number of patients across allergic and inflammatory diseases. Our recent webinar, titled The Powerful Mat Cell, highlighted the scientific rationale for our aspiration to fundamentally shift the way many allergic inflammatory diseases are treated by targeting this primary effector cell, the mat cell, with both mono and combination therapeutic approaches.

Kathryn Haviland: The Blue 808 program builds on the significant insights we have gained in muscle biology, and targeting kit the master control switch under ourselves, which we believe has the greatest prospect of improving outcomes for a large number of patients across allergic inflammatory diseases.

Kathryn Haviland: Our recent webinar titled the powerful Marcel highlighted the scientific rationale for our aspiration to fundamentally shift the way many allergic inflammatory diseases are treated by targeting this primary affect yourself the mast cell.

Kathryn Haviland: Both mono and combination therapeutic approaches.

Kathryn Haviland: We are on track to file the IND for Blue 808 this quarter to enable the initiation of a Phase 1 study in Healthy Volunteers. We've established a successful track record with AvaKit, and you can continue to expect Blueprint to discover and develop potent and highly selective molecules as we expand our mast cell franchise beyond systemic mastocytosis. Turning now to the oncology side of our portfolio, next month at ASCO, we will be presenting data demonstrating that Blu222 is the first CDK2 inhibitor to be well tolerated in combination with an approved CDK4-6 inhibitor.

Kathryn Haviland: We are on track to file the IND for Blue Iterate this quarter to enable initiation of a phase one study in healthy volunteers.

Kathryn Haviland: We've established a successful track record with <unk> and you can continue to expect blueprint to discover and develop potent and highly selective molecules as we expand our mask franchise beyond systemic mastocytosis.

Kathryn Haviland: Turning now to the oncology side of our portfolio.

Kathryn Haviland: Next month at <unk>, we will be presenting data demonstrating the Bluetooth you too is the first CDK <unk> inhibitor to be well tolerated in combination with an approved CDK <unk> inhibitor.

Kathryn Haviland: With this data, we now have the clinical evidence that BLU222 is a differentiated, best-in-class CDK2 inhibitor. Blu222's ability to combine with approved CDK4-6 inhibitors also positions it to move quickly with the potential to also become the first-in-class CDK2 inhibitor approved for patients with hormone receptor-positive HER2-negative breast cancer.

Kathryn Haviland: With this data we now have the clinical evidence that blue to do too is a differentiated best in class CDK <unk> inhibitor.

Kathryn Haviland: Bluetooth to the ability to combine with approved CDK four six inhibitors also position it to move quickly with the potential to also become the first in class CDK <unk> inhibitor approved for patients with hormone receptor positive her two negative breast cancer.

Kathryn Haviland: I remain confident that we will execute a strategic partnership in the second half of this year to rapidly move Blue 222 forward into registration-directed trials. Importantly, we are executing across the business while maintaining financial discipline. Our total costs and operating expenses continue to decline, and with $735.6 million in cash on our balance sheet, we are in a strong and durable financial position. I am proud of the tremendous progress Blueprint has made in the first quarter of this year and continue to be impressed by the heads-down executional focus I see across all of our teams to make sure we're achieving our goals quarter after quarter. Now, let me turn it over to Philina to discuss this quarter's commercial performance. Okay, Philina? Thanks, Kate.

Kathryn Haviland: I remain confident that we will execute a strategic partnership in the second half of this year to rapidly move Bluetooth to forward into registration directed trials.

Kathryn Haviland: Importantly, we are executing across the business, while maintaining financial discipline.

Kathryn Haviland: Our total cost and operating expense continued to decline.

$735 $6 million in cash on our balance sheet, we are in a strong and durable financial position.

Kathryn Haviland: I am proud of the tremendous progress blueprint has made in the first quarter of this year and continue to be impressed by the heads down execution focus I see across all of our teams to make sure. We're achieving are cool goals quarter after quarter.

Kathryn Haviland: Now, let me turn it over to Colleen to discuss this quarter's commercial performance later.

Philina Lee: Thanks, Kate. And good morning, everyone. We had an exceptional first quarter, generating AvaKit net product revenue of $92.5 million, including $83.1 million in the U.S. and $9.4 million outside the U.S. Avocat revenue has grown more than 135% year over year. We saw positive trends across all key business fundamentals, continued strong and steady growth in patients on AvaKit, reflecting a strong pace of new patient starts and low discontinuation rates. Our mix of commercial versus free goods also skewed more favorable than we anticipated. However, compliance remains high.

Kate and good morning, everyone. We had an exceptional first quarter generating aggregate net product revenue of $92 $5 million, including $83 $1 million in the U S and $9 $4 million ex U S.

Speaker Change: <unk> revenue has grown more than 135% year over year.

Colleen: We saw positive trends across all key business fundamentals continued strong and steady growth in patients on either kit, reflecting our strong pace of new patient starts and low discontinuation rates.

Colleen: Our mix of commercial versus free goods also skewed more favorable than we anticipated compliance remains high our international ISR launch is off to a robust start with Germany off to the races.

Philina Lee: Our international ISM launch is off to a robust start with Germany off to the race. Let's look at our key fundamentals in more detail, starting with growth in patients. We drove a really strong pace of new patient starts coming out of the holidays throughout the first quarter of the year. We continue to see low discontinuation rates, driving positive trends in duration of therapy. For advanced SM patients, duration of therapy is trending even longer than our last update, now at an average of 25 months.

Colleen: Let's look at our key fundamentals in more detail.

Colleen: Starting with growth in patients on either kit we.

Colleen: We drove a really strong pace of new patient starts coming out of the holidays throughout the first quarter of the year, we continue to see low discontinuation rates driving positive trends in duration of therapy.

Colleen: For advanced SM patients duration of therapy is trending even longer than our last update now at an average of 25 months.

Philina Lee: And while it's still early days, duration of therapy in ISM patients is trending significantly longer. This is exactly what we expected based on AvaKit's benefit-risk profile. Q1 tends to be a tough quarter in our industry, with patients' benefits re-verifications, lower growth to net, and impacts on compliance.

Colleen: And while it's still early days duration of therapy in ISR patients is trending significantly longer.

Colleen: This is exactly what we expected based on Eva kits benefit risk profile.

Colleen: Q1 tends to be a tough quarter in our industry with patients benefits re verifications lower gross to net and impact on compliance.

Philina Lee: Our market access team did a great job managing through all of this. Our free goods share, once again, has been a surprise to the upside and is now averaging about 20% since ISM launch. The favorability of commercial versus free goods is really driven by two things. First,

Colleen: Our market access team did a great job managing through all of this.

Colleen: Our free goods share once again has been a surprise to the upside and is now averaging about 20% since I S. M launch.

Colleen: The favorability in commercial versus three goods is really driven by two things first the payer mix of ISR patients skews more commercial and we estimate that ISR patients now comprise the majority of patients on therapy.

Philina Lee: The payer mix of ISM patients skews more commercial, and we estimate that ISM patients now comprise the majority of patients on therapy. Second, Medicare patients were able to access paid therapy, as we saw last year. Importantly, unlike last year, we expect these patients will be able to stay on paid therapy for the rest of the year due to changes to the out-of-pocket cap as part of Medicare Part D benefits redesign to address inflation.

Colleen: Second medic.

Colleen: Medicare patients, we're able to access paid therapy as we saw last year importantly, unlike last year. We expect these patients will be able to stay on paid therapy for the rest of the year due to changes to the out of pocket cap as part of Medicare part D benefits redesign in the inflation reduction Act.

Philina Lee: In this third full quarter of launch, we are seeing strong and steady performance against our key revenue drivers, just as we expected. The favorability and commercial mix exceeded our expectations and provides a tailwind for the remainder of the year.

Colleen: In the third full quarter of launch we are seeing strong and steady performance against our key revenue drivers just as we expected.

Colleen: The favorability in commercial mix exceeded our expectations and provides a tailwind for the remainder of the year.

Philina Lee: This, along with confidence in our strong continued execution, is a key driver for our guidance update today. We will continue to watch the fundamentals that drive revenue over the course of the year, such as the ongoing pace of new patient starts, duration of therapy, compliance, and free goods, as well as our ongoing European launch performance. Taken together, our ISM first three launch quarters are showing a clear inflection point for AvaKit revenue growth. Now, let's talk about why we expect to sustain this growth for years to come.

Colleen: This along with confidence in our strong continued execution is a key driver for our guidance update today.

Colleen: We'll continue to watch the fundamentals that drive revenue over the course of the year such as the ongoing pace of new patient starts duration of therapy compliance and free goods as well as our ongoing European launch performance.

Colleen: Taken together, our first our ISN first three launch quarters are showing a clear inflection point for Ava kit revenue growth.

Colleen: Now, let's talk about why we expect to sustain this growth for years to come.

Philina Lee: Our team is executing well against our commercial strategy, and we continue to make headway across multiple paths to drive sustained growth. We continue to grow breadth and depth in the AvaKit prescriber base across all specialties and settings. Prescriber breadth is one of the most important lead indicators for revenue growth. The number of new AvaKit prescribers continued to grow this quarter, including a growing number of allergists who have been activated to treat SM patients. Overall prescribing is still split evenly across academic and community settings.

Colleen: Our team is executing well against our commercial strategy and we continue to make headway across multiple paths to drive sustained growth.

Colleen: We continue to grow breadth and depth in the Ava kit prescriber base across all specialties and settings Chris.

Colleen: Prescriber breadth is one of the most important lead indicators for revenue growth.

Colleen: The number of new advocate prescribers continue to grow this quarter, including a growing number of allergists, who have been activated to treat S. M patients.

Colleen: Overall prescribing is still split evenly across the academic and community setting.

Philina Lee: The chart on the left shows AvaKit adoption by the top 400 providers by SM patient volume. And as you can see, we're starting to see even more depth from repeat prescribing as positive first experiences lead providers to start their second, third, or more patients on AvaKit. Because the chronic burden of ISM is often underappreciated, a key part of our ongoing execution is redefining what disease control means for providers and patients, and we continue to expand our direct-to-patient and provider educational initiatives to foster greater awareness of the burden of disease with the goal of activating patients to ask about vocation. Living with ISM can be very isolating.

Colleen: Okay.

Colleen: The chart on the left shows Ava kit adoption into the top 400 providers by S. M patient volume and as you can see we're starting to see even more in depth from repeat prescribing as positive first experiences lead providers to start their second third or more patients an advocate.

Colleen: Because the chronic burden of ice M is often underappreciated a key part of our ongoing execution is redefining what disease control means for providers and patients and we continue to expand our direct to patient and provider educational initiatives to foster greater awareness of the burden of <unk>.

Colleen: <unk> with the goal of activating patients to ask about advocate.

Colleen: Living without them can be very isolating.

Philina Lee: Establishing patient-to-patient connections is a critical part of the journey to treatment. In Q1, we launched a monthly educational series where patients can learn from the experiences of other patients who are taking AvaCare. As part of our ongoing community-building efforts, we held a first-of-its-kind summit, bringing together patient advocates, patients, and multidisciplinary thought leaders. And just this month, we launched an unbranded direct-to-patient campaign to drive further awareness of the toll of living with ISM and the availability of a new treatment option.

Colleen: Establishing patient to patient connections is a critical part of the journey to treatment in.

Colleen: In Q1, we launched our monthly educational series, where patients can learn from the experiences of other patients who are taking Ava kit.

Colleen: As part of our ongoing community building efforts, we held a first of its kind summit, bringing together patient advocates patients and multi disciplinary thought leaders.

Colleen: Just this month, we launched an unbranded direct to patient campaign to drive further awareness of the toll of living with is and the availability of a new treatment option.

Philina Lee: All of these efforts are yielding impressive results. Unaided awareness among patients has grown nearly eight-fold since approval, and we expect our expanding peer-to-peer and patient initiatives will continue to activate more patients and providers to try AvaKit. In closing, the momentum we've shown in our first three quarters of launch makes us incredibly confident about the path we are on to achieve a more than $2 billion dollar peak opportunity with AvaKit. We are building and shaping this market.

Colleen: All of these efforts are yielding impressive results.

Colleen: Unaided awareness among patients has grown nearly eightfold since approval and we expect our expanding peer to peer and patient initiatives will continue to activate more patients and providers to try Ava kit.

Colleen: In closing the momentum we've shown in our first three quarters of launch make us incredibly confident about the path we're on to achieve a more than $2 billion peak opportunity with Eva kit.

Colleen: We are building and shaping this market we are growing the prescriber base, we are activating more patients to seek treatment. We're growing the number of diagnosed patients and there is plenty of headroom to continue to grow we knocked it out of the park this quarter.

Philina Lee: We are growing the prescriber base. We are activating more patients to seek treatment. We're growing the number of diagnosed patients, and there is plenty of headroom to continue to grow. We knocked it out of the park this quarter, and we remain laser focused on our mission to help more patients in need. With that, I'll hand it to Fouad, who will share how we're expanding our efforts in mast cell disorders to help more patients with allergic and inflammatory conditions beyond SM.

Colleen: And we remain laser focused on our mission to help more patients in need.

Speaker Change: With that.

Speaker Change: I'll hand, it to <unk>, who will share how we're expanding our efforts in mast cell disorders to help more patients without with allergic and inflammatory conditions beyond S. M.

Fouad Namouni: Last week, Dr. Mariana Castells, a renowned researcher and clinical expert in mast cell diseases, joined us for a webinar titled The Powerful Mast Cell, a promising and yet underappreciated target for treating allergic and inflammatory diseases. The webinar was the first in a new series we have planned to keep you updated on how we are thinking about the evolving science behind our portfolio and R&D strategies. If you haven't yet had a chance to listen to the discussion, I encourage you to check out the replay, which is live on our website.

Speaker Change: Thankfully now lastly.

Speaker Change: Last week, Dr. Mariana Garsten's renowned researcher in clinical expert in Mexico diseases joined US four webinar titled the powerful myself.

Speaker Change: A promising and yet underappreciated target for treating allergic inflammatory diseases.

Speaker Change: It wasn't or was the first in a new series, we have plan to keep you updated on how we are thinking about the evolving science behind our portfolio and R&D strategy.

Speaker Change: If you haven't yet had a chance to listen to the discussion I encourage you to check out the replay which is live on our website.

Fouad Namouni: The mast cell is a key driver cell responsible for the pathogenesis of a wide range of allergic and inflammatory conditions. Research into mast cell biology and its involvement in inflammatory diseases has recently increased with the goal of identifying novel therapeutic targets in allergy and inflammation. This webcast focused on the biology of mast cells, including their essential roles as drivers of and contributors to inflammatory responses, their core involvement in biological pathways relevant to an array of allergic and inflammatory diseases, and Blueprint's approach to modulating mast cells and building a pipeline for allergic and inflammatory diseases. Blueprint has a long and proven track record of leadership in this space, as evidenced by the success of AvaKid and the ongoing development of Allenospine.

Speaker Change: The message is a key driver so irresponsible for the pathogenesis of a wide range of allergic inflammatory conditions.

Speaker Change: To Master biology, and its involvement in inflammatory diseases has recently increased with the goal to identify novel therapeutic targets in allergy and inflammation.

Speaker Change: This webcast focused on the biology open access, including their essential roles as drivers and contributors to inflammatory responses.

Speaker Change: Their core enrollment in biological pathways.

Speaker Change: Relevant to an array of allergic and inflammatory diseases.

Speaker Change: And blueprint approach to modulating massive and building a pipeline in allergic and inflammatory diseases.

Speaker Change: <unk> has a long and proven track record of leadership in this space.

Speaker Change: As David has said by the success of Eva Kid and the ongoing development of Atlanta.

Fouad Namouni: And we have built what we believe is one of the most advanced mass cell drug discovery capabilities in the industry. Blue 808 is poised to help us beyond systemic mastocytosis. Tackling the challenge of developing a potent and highly selective, tunable, oral, wild-type kit inhibitor is a logical evolution of our capabilities in this space, and the opportunity we have ahead of us is significantly larger than any we've pursued in the past

Speaker Change: And we have been what we believe is one of the most advanced with mass and drug discovery capabilities in the industry.

Speaker Change: <unk> 808 expose it to help us beyond systemic mastocytosis.

Speaker Change: Tackling the challenge of developing a potent and highly selective tunable or wild type kit inhibitor is a logical evolution of our capabilities in this space.

Speaker Change: And the opportunity we have ahead of us is significantly larger than any with pursued in the past.

Fouad Namouni: Our vision for scientific leadership in mast cell diseases is built on four key pillars. First, a deep understanding of mast cell biology and how to modulate its activity. Second, select the best target modalities for monotherapy and combination strategies to achieve first and best in-class positions.

Speaker Change: Our vision for scientific leadership in Madison diseases has been on four key pillars.

Speaker Change: First love her deep understanding of the mast cell biology to moderate its activity.

Speaker Change: Second to select the best targets modalities for monotherapy and combination strategies to achieve first and best in class positions.

Fouad Namouni: Third, establish a strong preclinical and early clinical POC, or proof of concept, to de-risk development, and lastly, pursue pipeline opportunities in major mast cell-associated disorders where there is a medical need. We will continue to provide updates on our mast cell franchise throughout the course of the year, including Blue 808 IND submission on track for this quarter, which will enable us to initiate the Healthy Volunteer Study. We will also initiate part two of the Harvard trial for l-anesthenib in indolent systemic mastocytosis in the second half of this year.

Speaker Change: Third.

Speaker Change: Stablish strong preclinical and early clinical POC or proof of concept to Derisk development lastly, pursue pipeline in a pill opportunities in laser them asset associated disorders, where there is a medical need.

Speaker Change: We will continue to provide updates on our mass cell franchise throughout the course of the year, including Blue April eight IND submission.

Speaker Change: On track for this quarter, which will enable us to initiate the healthy volunteer study.

Speaker Change: We will also initiate part two of the harbor apply them for element in indolent systemic mastocytosis in the second half of this year as we expand or myself focus we continue to drive innovation in systemic mastocytosis as our understanding of the spectrum of patients and the underlying biology of the disease evolves.

Fouad Namouni: As we expand our mast cell focus, we continue to drive innovation in systemic mastocytosis as our understanding of the spectrum of patients and the underlying biology of the disease evolves, moving from allergy and inflammation to our oncology part of the portfolio. We are happy to report for the first time at the 2024 ASCO meeting the safety and signal of early clinical activity of Blue 2-2-2 in combination with ribocyclib and fulvestrant in hormone positive, HER2 negative breast cancer patients.

Speaker Change: Yeah.

Speaker Change: Moving from allergy and inflammation two hour oncology part of the portfolio.

Speaker Change: We are happy to report for the first time at the 'twenty 'twenty four Astro meeting the safety.

Speaker Change: And the signal of early clinical activity of Blu 222 in combination with rigor cyclic and full restaurant in hormone positive <unk> negative breast cancer patients. We believe these data clearly demonstrate the first and best in class potential of Blu 222 to become the combination partner of choice with CDK four six.

Fouad Namouni: We believe these data clearly demonstrate the first and best-in-class potential of Blue 2-2-2 to become the combination partner of choice with CDK4-6 inhibitors in hormone-positive, HER2-negative breast cancer. With this, I will turn it over to Mike to discuss our financial results.

Speaker Change: Inhibitor in hormone positive <unk> negative breast cancer.

Speaker Change: With this I will turn it over to Mike to discuss our financial results.

Speaker Change: Good.

Michael Landsittel: Earlier this morning, we reported detailed financial results in our press release. For today's call, I'll touch on a few highlights. In the first quarter, total revenues were $96.1 million, including $92.5 million in net product revenues from sales of AvaKit and $3.6 million in collaboration and license revenue. As Philina discussed, AvaKit revenue was driven by strong, continued strong, and steady growth in patient starts. Positive Trends Underlying Extended Duration of Therapy and Favorability in the Mix of Commercial Patients

Mike: Earlier. This morning, we reported detailed financial results in our press release for today's call I'll touch on a few highlights.

Mike: In the first quarter total revenues were $96 1 million, including $92 5 million in net product revenues from sales of <unk> and $3 $6 million in collaboration and license revenues.

Mike: That's philina discussed Ava kit revenue was driven by strong continued strong and steady growth in patient starts positive trends underlying extended duration of therapy and favorability in the mix of commercial patients.

Michael Landsittel: Given the strength in Q1, we are raising our AvaKit product revenue guidance and now expect to achieve $390 to $410 million in net product revenue in 2024. We've had a stronger than expected start to the year, and we are still learning about the seasonal trends and impacts in ISM as we make our way through the first year of launch. Our guidance philosophy is to provide our best understanding of where we may end the year, given the various puts and takes on revenue both in the U.S. and EU each quarter.

Mike: Given the strength in Q1, we are raising our Ava kit product revenue guidance and now expect to achieve $390 million to $410 million and net product revenue in 2024.

Mike: We've had a stronger than expected start to the year and we are still learning about the seasonal trends that impact in <unk> as we make our way through the first year of launch.

Mike: Our guidance philosophy is to provide our best understanding of where we may end the year given the various puts and takes on revenue both in the U S and EU each quarter.

Michael Landsittel: This update relatively early in the year reflects that commitment. The increase in today's guidance reinforces that we are on our path to capturing AvaKit's peak opportunity of greater than $2 billion. This revenue growth, coupled with expense discipline and focused investment, is what continues to give us confidence in achieving a financially self-sustaining profile. Our total costs and operating expenses continue to decline, and we're $174.9 million for the first quarter. We anticipate that our research and development expenses will remain relatively flat for the remainder of the year with some quarter-to-quarter variability.

Mike: This update relatively early in the year reflects that commitment.

Mike: The increase in today's guidance reinforces that we are on our path to capturing Ava kits peak opportunity of greater than $2 billion.

Mike: This revenue growth coupled with expense discipline and focused investment is what continues to give us confidence in achieving a financially self sustaining profile.

Mike: Our total costs and operating expenses continued to decline and were $174 9 million for the first quarter.

Mike: We anticipate that our research and development expenses will remain relatively flat for the remainder of the year with some quarter to quarter variability.

Michael Landsittel: We also expect our SG&A expenses to remain relatively stable as we continue to gain operating leverage from our commercial infrastructure. We believe that the current full-year sell-side consensus for total costs and expenses of approximately $715 million, which includes non-cash stock-based compensation expense, reflects an appropriate estimate for 2024. We remain in an exceptionally strong and durable financial position with $735.6 million in cash on hand. Advocates Revenue Performance and Today's Guidance Increase Our continued focus on managing operating expenses will result in our goal of further reducing our cash burn in 2024. Our solid financial profile drives our ability to generate long-term value as we invest in the commercial success of AvaKit and advance an innovative portfolio of medicines.

Mike: We also expect our SG&A expenses to remain relatively stable as we continue to gain operating leverage from our commercial infrastructure.

Mike: We believe that the current full year sell side consensus for total costs and expenses of approximately $715 million, which includes noncash stock based compensation expense.

Mike: Reflects an appropriate estimate for 2024.

Mike: We remain in an exceptionally strong and durable financial position with $735 $6 million in cash on hand.

Mike: <unk> revenue performance in today's guidance increase couple.

Mike: Coupled with our continued focus on managing operating expenses will result in our goal of further reducing our cash burn in 2024.

Mike: Our solid financial profile drives our ability to generate long term value as we invest in the commercial success of <unk> and advanced and innovative portfolio of medicines.

Operator: With that, I'll now turn the call back over to the operator for questions. Operator. Thank you.

Speaker Change: With that I'll now turn the call back over to the operator for questions.

Speaker Change: Later.

Speaker Change: Okay.

Operator: Thank you. At this time, I would like to remind everyone, in order to ask a question, press star, then the number one on the telephone keypad. We'll pause for just a moment to compile the Q&A. Ross. Your first question comes from the line of... Brad Canino from Stifel. Brad, the line is open. Please ask your question.

Speaker Change: Thank you at this time I would like to remind everyone in order to ask a question Press Star then the number one on your telephone keypad well pause for just a moment to compile the Q&A roster.

Speaker Change: Your first question comes from the line of.

Bradley Patrick Canino: Brad Canino from Stifel.

Unknown Executive: Thanks for that question, Brad. And to your point, I think one of the things that were very notable about Quad AI this year was just in comparison to the year before, where we were kind of introducing Blueprint for the first time and showing the AvaKit pivotal data for the first time. And then here we were kind of sitting with a large group of now people who had clinical experience with a drug, and we're treating patients, which was such a kind of notable step change to your point.

Bradley Patrick Canino: Brad Your line is open please ask your question.

Bradley Patrick Canino: Thank you great quarter.

Bradley Patrick Canino: I remember back at the quarter meeting in February you referenced a strong sense of iOS them awareness and the desire to treat patients and the company also had a strong presence at the meeting so if in your view how much of that awareness momentum is baked into the trajectory exiting the quarter and what is the team still see us headroom. Thank you.

Speaker Change: Yeah. Thanks for that question, Brad and and to your point I think one of the things that was very notable about quality I did here was just in comparison to the year previous where we are and are introducing blueprint for the first time in showing the advocate pivotal data for the first time and then here we were kind of sitting with a large group of now people, who had clinical experience with the drug.

Speaker Change: And we're treating patients which was such a notable step change to your point so.

Speaker Change: So basically and we don't talk a little bit about how our awareness.

Speaker Change: The impact of awareness and health care providers, a growing prescriber base and how that's really fueling our future growth this year and beyond.

Unknown Executive: Yeah, thanks for the question, Brad. And, you know, to your point, quite AI, certainly we could; the excitement was palpable from treaters across the academic and community setting. That said, I would still say we're still just getting started with significant headroom to grow. You know, the team has the ongoing pace of interactions of regional meetings, and the cadence of Congresses throughout the year. And I think as we really look at our lead indicators on the provider side, you know, with our breadth and depth of prescribing, this is a really strong foundation for continued growth and certainly leaves significant headroom for continued penetration, not just into the top 400 but well beyond that into the broader treating community.

Unknown Executive: So, maybe we want to talk a little bit about how our awareness, you know, the impact of awareness and healthcare providers, the growing prescription cyber base, and how that's really fueling our future growth this year and beyond. Yeah, thanks for the question, Brad, and

Speaker Change: Thanks for the question Brad and.

Speaker Change: To your point I'm quite a I certainly we could week the excitement was palpable from treaters across the academic and community setting you know that said I would still say, we're where we're still just getting started with significant headroom to grow.

Speaker Change: The team has the ongoing pace of interactions of regional meetings, the cadence of Congress. This throughout the year.

Speaker Change: And I think as we really look at our lead indicators on the provider side.

Speaker Change: With our breadth and depth of prescribing. This is a really strong foundation for continued growth and certainly leave significant headroom for continued penetration not just into the top 400, but well beyond that into the broader treating community.

Unknown Executive: In addition, touching on some of the direct-to-consumer initiatives, we're really excited to be rolling out the next wave of engagement for patient activation, and that also leaves significant headroom to grow in sort of patients' awareness and likelihood to ask their providers about AvaKit. All of this, we think, bodes exceptionally well that we are well on that path toward that over $2 billion peak opportunity for AvaKit.

Speaker Change: In addition, touching on some of the direct to consumer initiatives, we're really excited to to really be rolling out. The next wave of of engagement for our patient activation.

Speaker Change: And that that also leaves significant headroom to grow in sort of patients' awareness and likelihood to ask there are providers of Aveva kit.

Speaker Change: All of this we think bodes exceptionally well on that we are well on that path towards that over $2 billion peak opportunity for advocate.

Speaker Change: Okay.

Operator: Thank you. Your next question comes from the line of Marc Frahm from TD Calvin. Marc, your line is open.

Speaker Change: Thank you. Your next question comes from the line of Marc Frahm from TD Cowen Martin Your line is open.

Unknown Executive: Hi, thanks for taking my questions and congrats on the quarter as well. Maybe following up on Brad's question, just how are you seeing kind of maybe trends leading indicators to get into that bigger population that, you know, hasn't been well diagnosed historically, things like testing rates and stuff like that, to really access maybe, you know, the next kind of 10,000 patients that you've kind of talked about being out there that haven't been cared for historically?

Marc Alan Frahm: Hi, Thanks for taking my questions and congrats on the quarter as well.

Marc Alan Frahm: Maybe following up on Brad's question.

Are you seeing maybe trends.

Marc Alan Frahm: Leading indicators to get into that bigger population that hasnt been well diagnose historically things like testing rates and stuff like that to really access maybe the next group of 10000 patients that you've kind of talked about being out there.

Marc Alan Frahm: Haven't been cared for historically and then.

Unknown Executive: And then related to some of that, just with the top line really taking off here, could maybe Kate and Mike talk about kind of the goal of getting profitability, how important is that for the company, particularly as you start thinking about things like 808 coming in that, you know, could have a very broad development program ultimately?

Marc Alan Frahm: Related to some of that.

Marc Alan Frahm: With the top line really taking off your <unk>.

Marc Alan Frahm: Maybe Keith and Mike talk about because the goal of getting to profitability. How important is that for the company, particularly as you start thinking about things like <unk> coming in that could have a very broad development program up there ultimately.

Unknown Executive: Yeah, thanks for the questions, Marc. Maybe we'll start by talking about how we see, I mean, we're building a brand new market here with AvaKit and SM, and how we think about the different avenues of growth of building that market, some of the things you mentioned already, which is the increasing rate of diagnosis, which is a critical part, but also the fact that we know that there are a number of patients who are diagnosed, and you know, that we are just So, Philina, how do you think about both penetrating the currently diagnosed group versus the opportunities to continue to grow the overall size of the market?

Keith: Yes, thanks for the questions Marc maybe I'll start with talking about how we see I mean, where we are building a brand new market here with Eva CAD and <unk> and how we think about the different avenues of growth in building that market that some of the things you mentioned already which is the increasing rate of diagnosis, which is a critical part but also the fact that we know that there is a number of patients who are diagnosed.

Keith: And you know that.

Speaker Change: We are just kind of starting to penetrate and we have a lot of room, there as well. So how do you think about both penetrating the currently diagnosed group versus the opportunities to continue to grow the overall size of the market Yeah, I would say and thanks for the question Mark you know first and foremost if we just look at the patients who are already diagnosed which continues to steadily increase.

Philina Lee: Yeah, I would say thanks for the question, Marc. You know, first and foremost, if we just look at the patients who are already diagnosed, which continues to steadily increase, penetration into that patient population already represents an over $1 million opportunity, and that's where, sort of like, the primary focus is dedicated right now. And you spoke of sort of the, you know, there are multiple prongs for continued growth, and you spoke specifically of continued growth and diagnosis.

Penetration into that patient population already represents an over $1 billion opportunity and that's that's where sort of like hearing now our primary focus is is dedicated.

Speaker Change: And you spoke to sort of the you know what there there are multiple prongs for continued growth.

Speaker Change: And you you spoke specifically to the continued growth in diagnosis.

Philina Lee: We are really focused on continuing to drive initiatives like the high sensitivity kit testing, disease awareness, and sponsored testing from Blueprint, and we're really encouraged to see significant increases in the volumes of high sensitivity kit testing, especially in the allergy segment where that's really important. And even beyond that, we know there are further levers for growth for AvaKit, and that's adoption into a broader range of patients, you know, sort of widening that line of who is seen as not well controlled, as well as expansion into further geographies. And we're seeing, as I said, really strong initial performance in our international launches. All of this bodes really well for that peak opportunity.

Speaker Change: There we are really focused on continuing to drive initiatives like the high sensitivity kit testing the disease awareness.

Speaker Change: Bonds are testing from from blueprint and where were really encouraged to see significant increases in our in the volumes of high sensitivity kit testing, especially even in the allergy segment, where that's really important.

Speaker Change: And even beyond that we know there are further levers for us for growth for Ava cat and that's adoption into a broader range of of patients.

Speaker Change: You know sort of widening that line of who is seen as not well controlled.

Speaker Change: As well as expansion into a into further geographies and were seeing as I said, you know really strong initial performance in our in our international launches all of this bodes really well towards that peak opportunity.

Kathryn Haviland: Yeah, one thing maybe to add there is that this feels very similar to other rare disease launches we've seen where, you know, the epidemiology and these disease states tend to be kind of dated, and there are very small studies that inform that. And, you know, what we're seeing is even in our sponsored testing, patients coming out of other pools of patients, be that MCATs or others, that I think have been very much underrepresented in the current epidemiology. So, you know, we'll be providing more views on that as it comes, but, you know, our view is this market has a long way to go in terms of continuing to grow the number of patients on therapy, as well as Perhaps now I can turn to your question about, you know, the importance of profitability and how we think about that. Mike, do you want to start? Yeah, I'll start.

Speaker Change: Yeah, and one thing maybe to add there as you know this feels very similar to other rare disease launches, we've seen where.

Speaker Change: The epidemiology in these disease states tend to be kind of dated and very small studies that inform that and what we're seeing is even in our sponsored testing patients coming out of other pools of patients either <unk> or others that I think I've been very much under represented and that current epidemiology, though it will be providing more views on that as it comes but R. R.

Speaker Change: As this market has a has a long way to go in terms of continuing to grow the number of patients on therapy as well as the number of patients who are diagnosed with this disease.

Speaker Change: Now turning to your question about the importance of profitability.

Speaker Change: And how we think about that by doing a start yeah I'll start I think Mark I think the key is we're trying to build a sustainable business for the long term and so.

Michael Landsittel: I think, Mark, I think the key is, you know, we're trying to build a sustainable business for the long term. And so, part of that is making sure that we stay laser focused on our top priority investment areas, such as Blue 808, which is upcoming, and that requires financial discipline and focus. But, like, long term, we're looking to find these key value drivers where we can add long-term value to the company, and that's the long-term goal.

Speaker Change: Or that is making sure that we stay laser focused on our top priority investment areas, such as blue anyway, which is upcoming and that requires financial discipline and focus but like long term. We're looking to find these key value drivers, where we can add long term value to the company and that's that's the long term goal in just adding I completely agree and I think one of the things we've been known for.

Michael Landsittel: Yeah, and just adding, I completely agree, and I think, you know, one of the things we've been known for at Blueprint Medicines is our research and development capabilities, and we have a tremendous opportunity to continue to drive innovation and make really meaningful impacts on patients. I think Blue 808 is certainly on everyone's radar, but we have, you know, some of our favorite programs we haven't had a chance to talk to anybody about yet. And so, you know, we're looking forward to talking about our continued innovation in this space.

Speaker Change: At blueprint medicines, as our research and development capabilities and and we have a tremendous opportunity to continue to drive innovation and to make really meaningful impact on patients I think blew it away. It is certainly on everyone's radar, but we have you know some of our favorite programs. We haven't had a chance to talk to anybody about yet and so we're looking forward to talking about our continued innovation in.

Speaker Change: This space.

Speaker Change: Okay.

Speaker Change: Great. Thank you.

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

Speaker Change: Thank you. Your next question comes from the line of Michael Schmidt Michael Your line is open.

Operator: Good morning, this is Paul on behalf of Michael. Thanks for taking our question and congrats on the quarter. Mine's on advocates for the ex-US opportunity.

Speaker Change: Hey, Good morning. This is Paul on for Michael Thanks for taking my question and congrats on the quarter.

Speaker Change: An advocate for the ex U S opportunity I know you mentioned, Germany sort of drive the European sales. This year do you still see a roughly 10% contribution to this year's full year sales and then how are you currently thinking about the longer term trajectory in Europe, perhaps beyond this year and then maybe just squeezing one on CDK to any updates on sort.

Speaker Change: The color or the tenor over the progress of your partnership discussions for that or these exclusively on blue to do two or perhaps more broadly for the CGM franchise. Thank you.

Unknown Executive: I know you mentioned Germany sort of driving European sales this year. Do you still see a roughly 10% contribution to this year's full year sales? And how are you currently thinking about the longer term trajectory in Europe, perhaps beyond this year? And then maybe just squeezing one on CDK2, any updates on sort of the color or the tenor of the progress of your partnership discussions? For that, and are these exclusively on blue to do two or perhaps more broadly for the CDK franchise? Thank you.

Speaker Change: Yeah, Michael Thank you for those questions and I think one thing is that the European team has done they've done a tremendous job out of the gates with I S. M approval at the end of December with Germany, really coming on strong in terms of getting patients under therapy, and so they've they've done a tremendous job there Kristine how do you think about the overall contribution for Europe in its growth trajectory and maybe also could you take that BD.

Kathryn Haviland: Yeah, Michael, thank you for those questions. I think one thing is that the European team has just done a tremendous job out of the gates with ISM approval at the end of December, with Germany really coming on strong in terms of getting patients under therapy. And so they've done a tremendous job there.

Kristine: A question as well sure. Thanks, Paul Yeah, we're really pleased with the performance of the international team as Kate said Theyre very much off to the races, and I think it's been particularly encouraging to see that some of the underlying dynamics in the German launch early on have looked very similar to the United states, including where ever seen prescribing etcetera.

Christina Rossi: Christy, how do you think about the overall contribution for Europe and its growth projector? And maybe also, could you take the BD question as well? Sure. Thanks, Paul. Yeah, we're really pleased with the performance of the international

Christina Rossi: Sure. Thanks, Paul. Yeah, we're really pleased with the performance of the international team. As Kate said, they're very much off to the races.

Christina Rossi: And I think it's been particularly encouraging to see that some of the underlying dynamics in the German launch early on have looked very similar to the United States, including where we're seeing prescribing, etc., lots of patient demand. So really encouraging to see. Of course, there are other dynamics in Europe that are different from the United States, and most notably, that would be price negotiations. So some of those cards will start to flip this year, and then into next year as well.

Kristine: Lots of that patient demand, so really encouraging to see and of course, there's other dynamics in Europe that are different from the United States and most notably that would be price negotiations. So some of those cars will start flipping this year and then into next year as well. So you know when I think about the overall opportunity in Europe. It's it's certainly significant it's going.

Christina Rossi: So, you know, when I think about the overall opportunity in Europe, it's certainly significant; it's going to be a contributor to our overall top line. But for this year, as I said, that 10 to 15 percent of revenue, as a rough estimate, is probably not a bad place to be. This, frankly, is one of the levers that we think about when we're trying to set guidance ranges, right? I mean, this is a variable that informs how we may perform over the year. But I think 10 to 15 percent is probably a good estimate.

Kristine: Be a contributor to our overall topline them, but for this year. That's that you know that 10% to 15% of revenue as a rough estimate is probably not a bad place to be.

Kristine: Frankly, as one of the levers that we think about when we're trying to set guidance ranges right. I mean this is a variable that informs how we may perform over the year, but I think 10% to 15% is probably a good a good estimate and then at peak in markets. Like this you know, we'll see we'll see how things evolve in the coming years as we continue to grow the opportunity, but I. You know you often would see the U S still representing.

Christina Rossi: And then at a peak in markets like this, you know, we'll see how things evolve in the coming years as we continue to grow the opportunity. But, you know, you often would see the U.S. still representing, certainly, the majority of the dollar value of the ESM opportunity. And as Felina said, you know, it's very easy math to get to a multibillion-dollar opportunity just in the U.S. based on what we are seeing.

Kristine: The majority of the dollar value of the S M opportunity and it's filling a sad.

Kristine: You know, it's very easy math to get to a multibillion dollar opportunity just in the U S. Based on based on what we are what we are seeing.

Christina Rossi: In terms of CDK2, you know, as we said in the prepared remarks, we are very, very excited about the data set that we're going to be sharing at ASCO. We really see this as, you know, the first data that will show that you can safely combine a CDK2 inhibitor with a 4. Our partnership discussions are progressing well. We've always been very clear that, you know, the second half of this year is when it makes sense to have a partner on board strategically based on where the program will be heading as we exit this year and into next.

Kristine: In terms of CDK till I, you know I think that in the prepared remarks very very excited about the data set that we're gonna be sharing at <unk>, we really see this as you know the first the first data that will be showing that you can safely combine a CDK <unk> inhibitor with a four six am our partnership discussions are progressing well, we've always been very clear.

Kristine: You know second half of this year is when it makes sense to have a partner on board strategically based on where the program will be heading as we exit this year and into next and certainly you know as we think about the tenor of those conversations.

Christina Rossi: And certainly, you know, as we think about the tenor of those conversations, our focus is on CDK2. That's where, you know, having Bluetooth 2.2, having a partner on board to advance the program makes the most sense. Of course, there's a lot of innovation in our pipeline that, you know, partners may be interested and excited about. We always have those conversations, and we'll continue to do that. Our next question comes from the line of

Kristine: Our focus is on CDK to that's where you know having a a bluetooth due to having a partner on board to advance. The program makes the most sense of course Theres a lot of innovation in our pipeline that you know our partners may be interested and excited by we always have those conversations and we'll continue to do that.

Speaker Change: Thank you.

Operator: Our next question comes from the line of Laura Prendergast from Raymond James. Laura, your line is open.

Speaker Change: Our next question comes from the line of Laura Prendergast from Raymond James Laura Your line is open.

Laura Anne Prendergast: Hey, guys congrats on a good trend today.

Laura Anne Prendergast: Another one on CDK to thinking about how the cost of CDK to impact your opex box throughout the year.

Laura Anne Prendergast: I'm, assuming you guys are looking to out license or not kosher and if you could just provide any any input here about how youre thinking about a.

Laura Anne Prendergast: CDK team moving forward.

Operator: Yeah, thank you, Laura. Maybe we'll quickly, we'll start with just how we're thinking about BD, and then Mike, maybe you can weigh in on just how you have, you know, any kind of impact on financial aid this year. Yeah, sure.

Speaker Change: Yeah. Thank you Lord David can we just start with just strategically how we're thinking about BD and then Mike maybe you can weigh in on just how you you know any kind of impact on like yeah. Yeah sure. So from a strategy perspective, we've always been very intentional easing business about that's you advanced programs and really complement them you know what our own capabilities and in our.

Unknown Executive: Sure. So from a strategy perspective, we've always been very intentional about using business development to advance programs and really complement what our own capabilities and our own internal priorities may look like. And certainly, at this stage, as we said, we are very focused on continuing to really develop a broad portfolio of mast cell-driven disorders, certainly executing the ongoing Entreviva kit. We have LNF SNIB and Blue 808 coming behind that.

Mike: And sort of internal priorities may look like and certainly at this stage and as we said we are very focused on continuing to really develop a broad portfolio and mast cell driven disorders, certainly executing the ongoing launch of David Cat, We have Alan that's never been blew it away coming behind that CDK too is a very very sick.

Unknown Executive: CDK2 is a very, very significant commercial opportunity and one that, because it's primarily in breast cancer, is obviously in a somewhat different space. I always think about having, you know, co-development, and co-commercialization make sense in places where you're trying to build and leverage scale. In the short term, you know, breast is not a place where we're trying to build and leverage scale. So again, we'll continue to advance those conversations as they make sense. But we have a very clear idea strategically of what we're trying to accomplish. Unknown Speaker Yeah, just with respect to the financial

Mike: Again, our commercial opportunity and one that because its primarily in breast cancer is obviously in a somewhat different space and so that's really the driver behind us having these conversations will be open as we go through you know in terms of what the exact structure will look like but I'm you know we have a partner on board because we.

Speaker Change: Thank you now we think the capabilities of the partner brands are are irrelevant to how we advance it I always think about having you know a co development co commercialization makes sense in places, where you're trying to build and leverage scale them in the short term that you know breast is not a place where we're trying to build and leverage scale. So again, we'll continue to advance those conversations.

Speaker Change: As they make sense, but we have a very clear idea strategically of what we're what we're trying to accomplish.

Michael Landsittel: I think with respect to the financials, you know, there's, there's, we obviously don't comment specifically on partnership structures or financials at this point; it's too early. But for 2024, you know, we're continuing to execute on our phase one study that's baked into all of the financial guidance that we've given. We feel very confident about that. And then longer term, you know, as Christy mentioned, strategically, it makes a lot of sense to bring a big partner on board, you know, and part of that is driven by the fact that these studies to move forward in breast cancer are going to be very expensive. And we want partners to, you know, both strategically as well as financially help support us there.

Mike: Finally, I, just I think with respect to the financials.

Mike: We obviously don't comment specifically on you know partnership structures are financials at this point, it's too early but for 2024, we're continuing to execute on our phase one study that's baked into all of the financial guidance that we've given we feel very confident about that and then longer term as kristie mentioned strategically it makes a lot of sense to bring a big partner on.

Mike: And part of that is driven by the fact that these studies to move forward in breast cancer, we're going to be very expensive and we want a partner to both strategically as well as financially help support us there.

Speaker Change: Thank you.

Mike: Jeffrey.

Operator: Thank you. Our next question comes from the line of Salveen Richter from Goldman Sachs. Salveen, your line is open.

Jeffrey: Thank you. Our next question comes from the line of solving enriched or from Goldman Sachs solving your line is open.

Operator: Thank you. Thank you. Our next

Solving: Alright, Thanks, Matt on personal view on Blue 808 could you talk about your strategy for determining which.

Solving: Zero.

Solving: And Star Wars honor for him.

Solving: Thanks, so much.

Solving: Yes.

Fouad Namouni: Fouad, do you want to start and talk about 808? Thank you. Yeah.

Speaker Change: Hello, do you want to start and talk about anyway. Thank you yes.

Fouad Namouni: Thank you. Yeah, I mean, for Blueprint to wait, and I actually would encourage you and others to really go back and listen to the live replay of our powerful mass cell from last week, where we discussed this topic in depth. The way we are thinking about it is, as we said, chronic urticaria is our leading indication. But, given the profile of the molecule, which we believe is really a very good profile, and we are filing the IND and taking it to the clinic this year, to really look at a number of other indications, because the involvement of mass cells in the biology of numerous diseases is clearly and well established now.

Speaker Change: The weight and I actually you would of course you.

Solving: You and others to really go back and listen to the live replay.

Solving: A replay of our powerful mass settled from last week, we're really treated this topic in depth. The way we are thinking about it is as we said conical actually carry is our leading indication on the other hand, given the profile of the molecule that we believe is really a very good profile and we are filing the IND and taken into the clinic.

Solving: This year is to really look at a number of other indications because the environmental mess in the biology of numerous diseases is clearly well established now we'd be doing a lot of preclinical work in other indications, but also will be doing.

Fouad Namouni: We'll be doing a lot of preclinical work in other indications, but we're also doing proof of concept clinically for a number of indications, and we see where we really think the full scope is and how to line up the additional indications beyond chronic urticaria. So we believe this molecule, if the clinical profile is consistent with the preclinical data that we are seeing, has the opportunity to really tackle a number of diseases, and therefore, we'll have to look at the POC for many of them. Thanks, Matt. I may just add that I...

Solving: Of concept pre clinically for a number of indications and we see where we really think.

Solving: The full scope is and how to align the additional indication beyond chronic quota carriers.

Solving: We believe this molecule F. The clinical profile, that's consistent with the preclinical data that we are seeing has the opportunity to really talk about a number of diseases and therefore, we'd have to look at the PUC for many of them.

Unknown Executive: Thanks. I may just add that we also believe that the profile of the compound and the fact that we have an oral therapy here are going to enable us to kind of tune the therapy to address different patient populations across all these different disease states. We think we're really well positioned in terms of the target product profile of the compound to evaluate this and bring forward what will truly be a pipeline and a pill opportunity.

Speaker Change: Thanks, Matt and maybe I'll just add that we also believe that the the profile of the compound and the fact that with an oral therapy here is going to enable us to be able to kind of tune that therapy to address different patient populations across all these different disease States. We think we're really well positioned in terms of the target product profile of the compound to evaluate this and.

Solving: Going forward, what will truly be a pipeline in a pill opportunity.

Speaker Change: Thank you.

Solving: Sure.

Operator: Thank you. Your next question comes from the line of Ren Benjamin from Citizens JMT. Your line is open.

Solving: Thank you. Your next question comes from the line of Ren Benjamin from citizens JMP. Your line is open.

Operator: Hey, good morning, guys. Thanks for taking the questions and congrats on an amazing quarter and really good guidance. The question for me is, of the top 400 or so prescribing physicians, how many are prescribing, and what's the split between allergists and hematologists? And as we think about reasons why eligible patients might not be being prescribed to advocate, are there any reasons? Is that a potential group of patients that is incorporated into that kind of headroom calculation of how the area could grow? And how do you plan on addressing that? Thank you.

Reni John Benjamin: Hey, good morning, guys. Thanks for taking the questions and congrats on an amazing quarter.

Reni John Benjamin: And a really good guidance.

Reni John Benjamin: The question for me is of the top 400 or so.

Reni John Benjamin: Physicians, how many are prescribing and what's the what.

Reni John Benjamin: Flip between Allergists, and Hematologists and as we think about reasons that eligible patients might not be being prescribed as a kid can you you know.

Ren Benjamin: Are there any reasons is that a potential group of patients that you know it was incorporated into that kind of headroom calculation.

Ren Benjamin: The area could grow and how do you plan on addressing that thank you.

Philina Lee: Yeah, thanks, Ren. I mean, Philina, maybe to address both how we see kind of demand coming from the physicians who have the most patients as well as from, you know, positions from the tail and how you see that kind of dynamic in terms of demand and penetration, as well as to talk a little bit about kind of the cadence of new patient starts, right, and how that is informing our growth into the future. Yeah, thanks for the question, Ren. I would

Speaker Change: Yeah, Thanks, Ryan maybe too to address both how you see kind of demand coming from the physicians will have the most patients as well as out of positions on the tail end and how you see that kind of dynamic in terms of demand and penetration as well as talking a little bit about kind of the cadence of new patient starts right and how that is informing our.

Ryan: Our growth into the future.

Philina Lee: Yeah, thanks for the question, Ren. I would start with, you know, the initial breadth and depth of prescribing that we've established in these first three full quarters of launch in ISM sets a really strong foundation for us, both in terms of the breadth of the penetration, you know, what you want to see in rare disease is not that it's concentrated in just a handful of centers, like what really fosters, you know, plants the seeds for sustainable growth and continued growth is that breadth of prescribing and the fact that we're seeing adoption across both hematology, oncology, as well as allergy, immunology and split across academic and community settings is extremely important, and is a great lead indicator for continued growth.

Ryan: Yeah. Thanks for the question Ren I would start with.

Ryan: The initial breadth and depth of prescribing that we've established in these first three full quarters of launch in ISR sets, a really strong foundation for us.

Ryan: Both in terms of the breadth of the penetration.

Ryan: You know what you want to see and rare disease is not that its concentrated in just a handful of centers like what really fosters plants. The seeds for sustainable growth and continued growth is that breadth of prescribing and the fact that we're seeing adoption across both hematology oncology as well as allergy immunology and split across academic and community.

Ryan: Settings is extremely important and.

Ryan: And is a great lead indicator for our continued growth when.

Ryan: When we look at the top 400, that's really something that we are laser into to illustrate what we see as more of the kinetics of prescribing it doesn't speak to the full penetration because that's really just a snapshot of some of the highest volume providers.

Philina Lee: When we look at the top 400, that's really something that we laser into to illustrate what we see is more the kinetics of prescribing; it doesn't speak to full penetration because that's really just a snapshot of the highest volume providers. But so what's really important, I think, to see from this is that first prescribing is really leading to positive experiences that lead to subsequent repeat prescribing for two, three, or even more patients.

Ryan: So what's really important I think to see from them from that is that first prescribed first prescribing is really leading to positive experiences that lead to subsequent repeat prescribing for for two three or even more patients.

Philina Lee: You know, even within that mix, we see a really great mix of allergists, immunologists, and hematologists, but you know, I think there is headroom just across, you know, all segments and deciles of providers to continue to grow there. In terms of your question around eligible patients and prescribing there, the most important thing we're focused on is identifying when a patient is not well controlled. And that is really, that's sort of like a crux across both our provider engagements as well as patient engagements.

Ryan: Even within that mix, we see a really great mix of allergist immunologist hematologists.

Ryan: But you know I think headroom just across them, all all segments and F Isles of providers to to continue to grow there.

Ryan: In terms of your question around eligible patients and prescribing. There you know the most important thing we're focused on is identifying when a patient is not well controlled and that is really that's sort of like a crux across both our provider engagements as well as patient engagement.

Philina Lee: From a patient perspective, you know, patients can also become acclimated to living with a new normal; they may be avoiding triggers. And so it's really important to make them aware of a new treatment option. And that reclaiming some of the freedoms of their prior life is actually possible. And so our direct to consumer efforts are really focused on driving that. And again, there is a lot of headroom to grow. Our next question comes from the line of Derek Archila.

Ryan: From a patient perspective patients can also become acclimated.

Ryan: Two living with a new normal they may be avoiding triggers and so it's really important to make them aware of a new treatment option and that.

Ryan: Claiming.

Ryan: Some of the the freedoms of their prior life is actually possible and so really our direct to consumer efforts are really focused on driving that and again there are a lot of headroom to grow.

Speaker Change: Thank you.

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

Speaker Change: Our next question comes from the line of Derrick Our Chiller from Wells Fargo. Your line is open.

Speaker Change: Hey, good morning, and congrats on the quarter was hoping maybe you could discuss some of the assumptions behind the updated guidance. It seems to suggest a slowdown in net patient adds relative to the prior quarter.

Speaker Change: Trying to reconcile that with the very bullish commentary in the prepared remarks.

Operator: Yeah, thanks, Derek. I think, you know, just to start there, when we think about it, Mike kind of talked about our philosophy of guidance. And, you know, we are in the early days of building a brand new market. And so, as we think about the low and high end of our updated range, it is driven by, you know, ranges around a multitude of different inputs across numerous variables. I don't know, Philina, if you want to talk a little bit about how you think about all those variables. But, you know, we certainly expect continued growth and overall opportunity. Yeah, Derek, I think the first answer to your question is no, we do not.

Speaker Change: Yes, I think Derek I think just to start there yeah. When we think about my time talking about our philosophy on guidance.

Speaker Change: And you know we are we are in the early days of building a brand new market and so as we think about the low and high end of our updated range is driven by you know ranges around a multitude of different inputs.

Speaker Change: Across numerous variables are no point, if you want to talk a little bit about what you think of all those variables, but yeah. We certainly expect continued growth in the overall opportunity yeah, Derik I think the first answer to your question is no. We do not expect a slowdown in net patient adds.

Philina Lee: Yeah, Derek, I think the first answer to your question is no, we do not expect a slowdown in net patient ads. You know, we see strong and steady growth in net patient ads, and we expect that to continue. This past quarter, we saw that really driven by a strong cadence of new patient starts and very low discontinuation rates, which really works well, I think, for chronic durations of therapy.

Speaker Change: We see strong and steady growth in the net patient adds and we expect that to continue.

Speaker Change: This past quarter, we saw that really driven by a strong cadence of new patient starts very low discontinuation rates.

Speaker Change: Which really pen 10, dwell I think for a chronic durations of therapy.

Speaker Change: And that's that's also there are a number of additional key.

Philina Lee: And, you know, that's also, there are a number of additional key revenue drivers that contribute to our guidance, in addition to that continued strong and steady growth of net patient ads that we expect. So it's things like our compliance, the proportion of free goods, which exceeded for this quarter, and is something we'll be looking to see how that evolves over the course of the year, as well as things like our ongoing European launch.

Speaker Change: New drivers that contribute to our guidance. In addition to that continued strong and steady growth.

Speaker Change: Net patient adds that we expect so it's things like our compliance.

Speaker Change: The proportion of free goods, which exceeded for this quarter and it's something we'll be looking to see how that evolves over the course of the year.

Speaker Change: As well as things like our ongoing European launch and so it's the inherent variability in these key revenue drivers moving forward that factor into our guidance range.

Speaker Change: All of that said you know if we look at sort of the midpoint of that range that represents nearly a doubling of the revenue that we achieved last year and.

Speaker Change: And we believe it firmly does put us on that trajectory towards the peak.

Speaker Change: Got it thank you.

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

Philina Lee: And so it's the inherent variability in these key revenue drivers moving forward that factor into our guidance range. All of that said, you know, if we look at sort of the midpoint of that range, that represents nearly a doubling of the revenue that we achieved last year. And we believe it firmly does put us on that trajectory towards the peak. Your next question comes from Peter Lawson, from Barclays.

Speaker Change: Your next question comes from the line of Peter Lawson.

Peter Richard Lawson: From Barclays. Your line is open.

Peter Richard Lawson: Thanks, so much thanks for taking my questions.

Peter Richard Lawson: Yeah.

Peter Richard Lawson: As we think they are at ICU patients you mentioned, there was a kind of a.

Peter Richard Lawson: Discontinuation rate.

Barclays: Anything else you can kind of comment around those patients whether it is.

Barclays: And then shifting compliance after essentially 10 minutes of use.

Peter Richard Lawson: Any sense of.

Peter Richard Lawson: Drug holidays for instance.

Peter Richard Lawson: Okay.

Operator: Yeah, thanks, Peter, for the question. I think one of the things I think the team has just done a tremendous job in Q1 is, and we know that Q1 can often be very challenging kind of across, you know, as Polina mentioned, her prepared remarks across the pharmaceutical industry and biotech. And they just did a tremendous job of executing in the re-verification process and making sure that the compliance, you know, stayed very high. But I know, Polina, since launch, have you thought about seeing anything that you would say is thematic from a patient discontinuation perspective?

Speaker Change: Yeah. Thanks, Peter for the question I think one of the things I think the team has just done a tremendous job in Q1 is and we know that Q1 can often be very challenging kind of across as Blaine mentioned in her prepared remarks across the pharmaceutical industry and biotech and they just did a tremendous job on on <unk>.

Speaker Change: Executing the re verification process and making sure that that compliance state.

Speaker Change: The state has stayed very high but I don't really know since launch have you ever thought about or seen anything that you would say that's thematic for it from a patient discontinuation perspective.

Philina Lee: Yeah, thanks, Peter. So what we've seen in terms of discontinuation rates so far has been very much in line with what we've expected when we see AvaKit's really favorable benefit-risk profile from Pioneer. So discontinuation rates have been low. And you know, the very early trends toward duration of therapy have been very, very positive. And, you know, we've even seen a lengthening on the advanced SM side.

Speaker Change: Yeah. Thanks, Peter So what we've seen in terms of discontinuation rates. So far has been very much in line with what we've expected when we see a that gets really favorable benefit risk profile from pioneer so discontinuation rates have been low.

Speaker Change: The very early trends towards duration of therapy has been very very positive and you know we've even seen a lengthening on the advanced SM side.

Philina Lee: You know, to your question around sort of compliance and holidays, like, in fact, that has also trended extremely positively in terms of patients staying on therapy. When we think about this patient population, you know, they're very sticky, right? Like, they have a regimen that they are adapting to, and they're really not taking dose holidays. And so, you know, this is a really important point, because as we progress in the launch and we increase the total number of patients on therapy, it's not just new patient starts anymore but also the cumulative effect of these patients remaining on therapy that will contribute to overall revenue growth. Yeah, I think that last point is really important in the sense that as that pool of candidates

Speaker Change: You know to your question around sort of compliance and holidays like and in fact that has also trended extremely positively in the in terms of patients staying on therapy. When when we think about this patient population.

Speaker Change: You know, they're there they're very they're very sticky right like they they have a regimen that they are adapting to and they're really not taking Jewish holidays.

Speaker Change: And so you know this is this this is a really important point because as we progress in the launch and we increased the total number of patients on therapy, it's not just new patient starts anymore, but also the accumulative effect of.

Speaker Change: Of these patients remaining on therapy that will contribute to overall revenue growth and I think that last point is really important in the sense that as as that pool of patients on therapy, you get so much larger.

Unknown Executive: And I think that last point is really important in the sense that as that pool of patients on therapy gets so much larger, point changes and compliance and or free goods make a big difference from a revenue perspective. So I think that, you know, we're certainly seeing that. Your next question comes from the line of Matt Biegler from Oppenheimer.

Speaker Change: Changes in compliance <unk> for you guys to make a big difference from a revenue perspective. So I'm. So I think that you know, we're certainly seeing that.

Speaker Change: Okay.

Operator: Your next question comes from the line of Matt Biegler from Oppenheimer. Your line is open.

Speaker Change: Your next question comes from the line of Matt Biegler from Oppenheimer. Your line is open.

Matthew Cornell Biegler: Hi, there congrats from us as well in the quarter I'm curious, if you're seeing any signs of growth in advanced of them, maybe because of the increased prescriber awareness from the England launch.

Matthew Cornell Biegler: Or I guess should we still think of advanced it's pretty flat going forward. Thanks.

Kathryn Haviland: Yeah, I'll just start quickly to say, we are so pleased to see the duration of therapy in Advanced SM being at 25 months. I mean, I think these are patients with very, very aggressive diseases, and I think that bodes well for both the Advanced SM opportunity as well as the research, the ISM opportunity. But do you want to talk about the growth and number of new patients in Advanced SM? Yeah, Matt, so I would say we do continue to see growth on the

Speaker Change: Yeah, maybe I'll just start quickly to say I mean, we are so pleased to see that duration of therapy and advance F N b.

Speaker Change: I mean at 25 months anything these are patients with very very aggressive disease, and I think that bodes well for both the advanced SM opportunity as well as the reason it would be awesome.

Speaker Change: Some opportunity, but you asked about the growth in number of new patients in France or something like that.

Philina Lee: Yeah, Matt, so I would say we do continue to see growth in the Advanced FM portion of our business. That's obviously relatively moderated compared to ISM, which is our primary growth driver and now represents the majority of patients on therapy with AvaKit. But, you know, we do see, I think, a potential kind of halo effect that, you know, with the awareness of AvaKit for ISM, as our breadth of prescribing increases, it's happening, you know, not just in allergy but also with a number of new hematology oncology prescribers as well who are activated and interested to get involved through ISM.

Speaker Change: Yeah, Matt So I would say, we do continue to see growth on the advanced S. M portion of our business. That's that's obviously relatively moderated compared to I S. M, which is our primary growth driver and now represents the majority of patients on therapy.

Speaker Change: With with Eva kit, but we do see I think a potential kind of halo effect that you know with the now the awareness of Eva Cat four for ASM.

Speaker Change: As our breadth of prescribing increases it's happening you know not just an allergy, but also with a number of new hematology oncology prescribers as well who are activated and interested to get involved through through either.

Speaker Change: That's great. Thanks.

Operator: Your next question comes from the line of Ami Fadia from Needham. Ami, your line is open.

Operator: That's great. Thanks. Your next question comes from the line of Ami Fadia from Needham. Ami, your line is open. Thanks, and congratulations on the quarter.

Speaker Change: Your next question comes from the line of Amy <unk> from Needham Amy Your line is open.

Amy: Thanks, Congrats on the quarter My question is on Blue Italy.

Amy: What data do you need to generate to understand.

Amy: Golf ball with clinical development plan for indications beyond chronic octavia.

Amy: And how soon can you start to initiate clinical trials in both indications and.

Speaker Change: And separately, if you could provide any additional color on.

Speaker Change: How you're planning to develop at an estimate.

Speaker Change: Any update from Peter Thank you.

Unknown Executive: Ami, thank you for those questions. And Becker, do you want to talk about the data you want to see at 808 and how we're thinking about getting those broad proof of concepts, as well as, you know, the plan for the harbor study with L&S NIB, which, as we've said before, L&S NIB is really positioned to maximize the long-term performance of our SM franchise? And so we certainly are going to be spending more time talking about that in the second half of this year. Yeah, um, Amy, so blue 808 is our

Speaker Change: Thank you for those questions and the fact that do you want to talk about the data you want to say it in a way to how we're thinking about at the end of those broad proof of concepts as well as you know the plan for harvest studying with Alan that's net of which as we've said before I mean, unless it is really positioned to maximize our long term performance of our <unk> franchise and so we certainly are going to be spending more time talking.

Speaker Change: About that in second half this year.

Unknown Executive: Yeah, Amy, so Blue 808 being our exquisitely targeted wild-type kit inhibitor is one that we're taking into healthy volunteers first, and that's obviously to get the initial PK and tolerability, but it also gives us an opportunity to see the ability of the drug to reduce trip days even in normal healthy volunteers, and we believe that that'll give us a good steer of what the therapeutic range may be in patients, and as Kate mentioned earlier, this is a very tunable drug where we expect to be able to use it across a wide range of therapeutic doses, and that may be as a single agent, as we illustrated in our mast cell webcast last week, where the mast cell is really the driver, like chronic urticaria, and then, you know, we've shown in our Blue 222 program, in our EGFR program, that we make highly combinable molecules, and that's going to be the essence of that next wave of investigations where we're looking in more complex diseases where the standard of care is well-established, such as asthma, where the combination is going to be necessary to really forward the field in those indications, and so as we enter into patients next year, that'll give us a sense of what our dose range is for patients who have mast cells that are causing disease, and then we'll be able to look at, as Flood said, in a number of different, more complex indications and understand where that therapeutic range is and get a quick proof of concept in those indications. And then with respect to L-anastinib, as we've said, we are getting more and more data as we study the patients on Pioneer, and we learn more from commercial patients about the really complex nature of systemic mastocytosis and the broad breadth of patients that can benefit in many different ways. And so we look forward to sharing more before the end of the year about our design of Harbor and really our holistic approach to the development of L-anastinib and

Speaker Change: Yeah.

Speaker Change: So bourdeau eight being are exquisitely targeted wild type kit inhibitor is one that we're taking into healthy volunteers first and that's obviously to get the initial PK and tolerability, but it also gives us an opportunity to see the ability of the drug to reduce trip days, even in normal healthy volunteers and we believe.

Speaker Change: That'll give us a good steer of what the therapeutic range may be in patients and as Kate mentioned earlier. This is a very tunable drug where we expect to be able to use it across a wide range of therapeutic doses and that may be in as a single agent as we illustrated in our mass so.

Speaker Change: Webcast last week, where the muscle is really the driver like chronic urticaria and then we've shown in our <unk> program and are Egfr program that we'd make highly combinable molecules and that's going to be the is the essence of that next wave of investigations, where were looking and more complex disease.

Speaker Change: Louis where the standard of care is well established such as asthma, where the combination is going to be necessary to really for this.

Speaker Change: The field and those indications and so as we enter them their patients next year that'll give us a sense of what our dose ranges work for patients who have vessels that are causing disease and then we'll be able to look at as flawed set in a number of different more complex indications and understand where that therapeutic ranges.

Speaker Change: And get a quick proof of concept in those indications.

Speaker Change: And then with respect to two hour enough to know as.

Speaker Change: As we've said we are getting more and more data as we study the patients on pioneer and we learned more from commercial patients about the really complex nature of systemic mastocytosis and the broad breadth of patients that can benefit in many different ways and so we look forward to sharing more about this before the end of the year about our our design of.

Speaker Change: Barbara and really our holistic approach to the development of our bell enough and have enough of them.

Speaker Change: Okay.

Operator: Your next question comes from the line of Colleen Kusy from Baird. Colleen, your line is open. Great, thanks.

Choline QC: Your next question comes from the line of choline QC from Baird. Your.

Choline QC: Your line is open.

Operator: Good morning. Thanks for taking our questions and congratulations on the quarter. Any commentary on how you're measuring the early success of your DTC campaign and what your plans are to invest further in the second half?

Choline QC: Yes.

Choline QC: Great. Thanks, Good morning, Thanks for taking our questions and congrats on the quarter.

Choline QC: Any commentary on how you're measuring the early success of your DTC campaign and what your plans are to invest further in the second half of this year.

Philina Lee: Yeah, so I think, headed to Philina, I think our direct-to-consumer campaign really is evidence of our conviction that we have a tremendous opportunity to help just a vast array of patients with ISM. How do you want to think about those efforts and how we make sure that we understand they're bearing fruit? Yeah.

Speaker Change: Yeah. So I think headed to find out I think our direct to consumer campaign really is evidence of our conviction that we have a tremendous opportunity to help just a vast array of of patients with with I S. M. A C.

Speaker Change: How do you want to think about those efforts and how we make sure that we understand their bankers.

Philina Lee: Yeah, Colleen, one of the most direct things that we look at is the patient's awareness of AvaKit, and we're really encouraged that the efforts to date have already increased the unaided awareness of AvaKit eightfold while continuing to have a lot of headroom to grow, so this is really an area that we will continue to lean into in a highly targeted way. We know where these patients are looking for information on the disease, and we're really targeting both our media campaigns, and you know another thing we've talked about is the monthly opportunities we have for patients to come together with other patients to share their experience on AvaKit.

Speaker Change: Yeah, Colleen one of the most direct things that we look at is the patience.

Colleen: Awareness says of Eva kit and we've been really encouraged that the efforts to date have already increased the unaided awareness of Eva cat eightfold, while continuing to have a lot of headroom to grow. So this is really an area that we will continue to lean into in a highly targeted way we know where these patients are looking for information on the disease and <unk>.

Colleen: We're really targeting them.

Colleen: Both our media campaigns.

Colleen: And then the other thing we've talked about is the <unk>.

Colleen: Monthly sort of opportunities we have for patients to come together with other patients to share their experience on Ava cat that element of patient storytelling is so important in a rare disease, where in many cases in SM patient hasn't yet met another patient so the opportunity to connect here about their experiences.

Philina Lee: That element of patient storytelling is so important in a rare disease where, you know, in many cases, an SM patient hasn't yet met another patient, so the opportunity to connect, hear about their experiences, and what the journey to AvaKit has been like for them, we believe will be really compelling. So we do expect to continue growing on these initiatives through the second half of the year in a highly targeted way.

Colleen: And what Oh.

Colleen: You know what the journey to Ava kit has been like for them, we believe will be really compelling.

Colleen: So we do and we do expect to continue growing on these initiatives through the second half of the year in a highly targeted way.

Philina Lee: And as a follow-up, just in your early market research, what's responding most with patients to keep them on treatment? And what gives you confidence in maintaining this low discontinuation rate in the future?

Speaker Change: That's helpful. Thank you and as a follow up just in your early market research, what's resonating most of the patients to keep them on treatment and what gives you confidence in maintaining this low discontinuation rate in the future.

Philina Lee: Yeah, Colleen, I think that really is a testament to AvaKit's clinical profile. Patients, it's a highly tolerable drug, easy to take.

Speaker Change: Yeah, I mean, I think that really is a testament to advocates clinical profile I mean patients it's a highly tolerable drug.

Philina Lee: I mean, we saw in the Pioneer study that the side effect profile of AvaKit was, you know, superior to standard of care and the kind of cocktail of symptomatic treatments that patients are on. And that's a testament to the activity and the fact that we see clinical impact across a wide range of symptoms in patients. You know, I think it is such a heterogeneous disease that is absolutely critical, and that a patient, regardless of what their most bothersome symptom is, is seeing great impact with AvaKit. You know, I think that that is just a profound kind of equation for people that we see as responding, and patients are staying on. Becker, do you have something to add?

Speaker Change: Easy to tag I mean, we saw in the pioneer study that the side effect profile of advocate was.

Speaker Change:

Speaker Change: Superior to standard of care and the kind of a cocktail of symptomatic treatments that patients are on and then it's a testament to the activity and the fact that we see clinical impact across a wide range of symptoms in patients mm.

Speaker Change: It wasn't such a heterogeneous disease that is absolutely critical and that a patient regardless of what their most bothersome symptom is seeing great impact on Ava Cat you know I think that that is just a a profound kind of equation for people that we see is resonating and patients are staying on drug or do you have something like I think this is a couple of question.

Becker Hewes: Yeah, I think this is, you know, a couple of questions that have picked my interest, the one about drug holidays and then this one about convincing patients to stay on the drug. You know, as Kate said, when they feel better, they don't need to take holidays; they don't need to be convinced to stay on the drug. And so that really speaks to the way that allergists think about treating this disease and having a very tolerable therapy and treating patients to see how they do.

Speaker Change: To have it.

Speaker Change: My interests the one about drug holidays, and then this isn't about convincing patients to stay on the drug as Keith said when they feel better they don't need to take holidays. They don't need to be convinced to stay on the drug and so that really speaks to also the way that allergists think about treating this disease.

Speaker Change: Having a very tolerable therapy, and and treating patients and see how they do and.

Speaker Change: It just speaks to why that there's consistent treatment duration and I also wanted to comment on the advanced SM duration. You know we're in the two years and a leukemia like disease and that really bodes well for the ambulance situations as well.

Becker Hewes: And it just speaks to why there's consistent treatment duration. And I also wanted to comment on the advanced FM duration, you know, we're into two years with a leukemia-like disease, and that really bodes well for the ambulance situation as well. Yeah, Philina, do you want to add?

Philina Lee: Yeah, maybe I would add Colleen. These are patients who are really highly motivated to regain quality of life, and just to share, I think, even just some sound bites from engaging customers across the country who have shared some of their patients' experiences is what we hear is patients getting their energy back, being able to go back to work at the jobs that they had to pull back on previously due to the symptoms of SM, the ability of patients to participate in activities with their kids or friends or loved ones, going So it's really kind of a profound thing, and I think we are really just at the beginning of this journey.

Speaker Change: That's the way to do you want to have yeah, maybe I would add on calling these are patients who are really highly motivated to to regain quality of life and just to share I think even just some soundbites from engaging customers across the country, who have shared some of their patients experiences. What we hear is patients getting their energy back being able.

Speaker Change: To go back to work at the jobs that they had to pull back on previously due to the symptoms of of S. M. The ability of patients to go participate in activities with their with their kids or friends or loved ones I'm going to camp for the first time in really reclaiming and starting to re.

Speaker Change: Remember what normal looks and feels like so it's it's really kind of a profound thing and I think we really are even just at the beginning of this journey.

Operator: Great, thanks for taking our questions. Thank you. Our next question comes from the line of David Lebowitz from Citi. David, your line is open.

Speaker Change: Great. Thanks for taking my questions.

Speaker Change: Yeah.

Speaker Change: Thank you. Our next question comes from the line of David Lebowitz from Citi. David Your line is open.

David Neil Lebowitz: Hi, this is advising on behalf of David.

David Neil Lebowitz: On this on the Sea basin.

Speaker Change: Proportion I wanted to ask that they could never went from 25%, 20% how should we think about patient growth versus actual C code and what proportion of patients will be on <unk> by the end of the year and I have one more follow up.

Operator: So unfortunately, we have to limit to one question just because we're coming on close to time here. But so I believe the question is around, how are we thinking about just the overall dynamic relative to free goods? Again, being very early in the launch of a new therapy and building a new market. Philina, how are you thinking about that as you kind of gave this view of around 20 percent since launch?

Speaker Change: So I Unfortunately have to limit to one question, just because theyre coming on close to time here, but so I believe the question is around how are we thinking about just the overall dynamic relative to free goods again being very early in the launch of a new therapy and building a new market. How are you thinking about that is as you kind of gave this view around 20% since Clinton.

Operator: And how do we think about that as a driver of top line versus some of the other, you know, variables, including compliance, new patient starts, our European launch, all the other things that are adding to top line? Yes, it's your question. I think there are

Speaker Change: Launching and how do we think about that as a driver of top line versus some of the other variables, including compliance new patient starts are European launch all the other things that are adding into topline yeah.

Philina Lee: Yes, it's your question. I think there are really a couple things that factor into our free goods mix, which as we saw, decreased to 20% this quarter. So it's both the payer mix of patients with ISM, which trends more towards commercial. The second piece is that more patients were able to access paid therapy. In terms of what to expect through the rest of the year, the free goods mix is really something we'll need to continue to watch.

Speaker Change: So to your question I think there's really a couple of things that factor into our free goods mix as we saw that decreased to 20%. This quarter. So it's both the payer mix of.

Speaker Change: Ah patients with ice, which trends more and more towards commercial I'm. The second piece is that more patients were able to access a paid therapy. You know in terms of what to expect through the rest of the year of free goods mix is really something that we'll need to continue to watch because as you said more patients will will continue to add on.

Philina Lee: Because, as you said, more patients will continue to add on and be treated with AvaKit. So we're in the early days of the IRA. And so we'll need to watch how that plays out. Our expectation continues to be continued strong and steady growth in the number of patients on therapy, favorable low discontinuation rates, high compliance, we'll watch the free goods mix, and continue to watch our international launch.

Speaker Change: And and be treated with a with Eva kits. So we're in the early days of the IR, a and so we'll need to watch how that plays out our expectation continues to be continued strong and steady growth in the number of patients on therapy favorable low discontinuation rates high compliance, we'll watch the free goods next end and continue to watch our our inter.

Speaker Change: A national launches.

Speaker Change: Okay.

Philina Lee: Thank you. There are no further questions at this time. I will now turn the call back to Kate.

Speaker Change: Thank you there are no further questions at this time I will now turn the call back to Kate.

Speaker Change: Okay.

Kathryn Haviland: Thank you, operator. We feel great about the progress we've made in Q1. And we recognize that there's a lot of work ahead of us this year, but as we move forward into the middle of the year, we do so from a position of strength. We are well positioned to build on the momentum we've achieved here coming out of Q1, and we look forward to updating you on our progress. So, thank you all for taking the time to join us today and for your continued support of Blueprint Medicines.

Kathryn Haviland: Operator, we feel great about the progress we've made in Q1 and we recognize that there's a lot of work ahead of us this year and as we move forward into the middle of the year. We do so from a position of strength, we are well positioned to build on the momentum we've achieved here coming out of Q1, and we look forward to updating you on our progress. So thank you all for taking the time to join us today and for your <unk>.

Kathryn Haviland: Sports Blueprint medicines.

Operator: This concludes today's conference call. You may now disconnect.

Speaker Change: This concludes today's conference call you may now disconnect.

Speaker Change:

Speaker Change: Okay.

Speaker Change: Yeah.

Speaker Change: Yeah.

Speaker Change: Okay.

Speaker Change: Yeah.

Speaker Change: Okay.

Speaker Change: Yeah.