Q2 2020 Blueprint Medicines Corp Earnings Call

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Operator: Ladies and gentlemen, thank you for standing by, and welcome to the Blueprint Medicine second quarter 2020 financial and operating results conference call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question and answer session. To ask a question during the session, you will need to press star one on your telephone keypad. If you require any further assistance, please press star then zero.

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Operator: I would now like to hand the conference over to your speaker today, Kristen Fotis of Blueprint Medicines. Thank you. Please go ahead.

Thank you for standing by and welcome to the Blueprint Medicine second quarter, Twentytwenty financial and operating results conference call.

Kristen Fotis: Thank you, Operator. Good morning, everyone, and welcome to Blueprint Medicine's second quarter 2020 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. Today on our call, Jeff Albers, our Chief Executive Officer, will discuss Blueprint Medicine's second quarter 2020 business highlights. Christy Rossi, our Chief Commercial Officer, will provide a commercial update, and Mike Landsittel, our Chief Financial Officer, will review our financial results. Dr. Andy Burrell, our Chief Medical Officer, is also on the call and will be available for Q&A.

At this time, all participants are any listen only mode.

After the speaker presentation, there will be a question and answer session to ask a question. During this session. You want me to press Star one on your telephone keypad.

If you require any other assistance. Please press star then see rail.

Now I'd like to hand, the conference over to your speaker today Kristen soda.

Different medicine. Thank you. Please go ahead.

Thank you operator.

Good morning, everyone and welcome to sleep Medicine second quarter, 2020 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 a slice it will be reviewing today by going to the Investor section of our website at Www Dot blueprints medicines dotcom.

Kristen Fotis: Before we get started, I would like to remind everyone that the statements we make on this conference call will include forward-looking statements. Actual events or results could differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties, and other factors, including those set forth in the risk factors section of our most recent quarterly report on Form 10-Q filed with the SEC and any other filings that we may make with the SEC. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements. Now, here's our CEO, Jeff Albers.

Today on our call, Jeff Albert Our Chief Executive Officer will discuss blueprint medicine second quarter 2020 business highlights.

Christy Rafi, our chief commercial officer will provide a commercial update.

And my claims at all our Chief Financial Officer will review our financial results.

Dr and to grow our Chief Medical Officer is also on the call and will be available for Q1.

Before we get started I would like to remind everyone that statements. We make on this conference call will include forward looking statements actual events or results could differ materially from those expressed or implied by any forward looking statements. As a result of various risks uncertainties and other factors, including those set forth in the risk factor section of our most recent quarterly report on form 10-K.

You filed with the FCC and any other filings that we may make with Youll see.

In addition, any forward looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date.

Jeff Albers: Thanks, Kristin, and good morning, everyone. I'm happy to provide an update on what has been a productive quarter for Blueprint Medicines. At the beginning of the year, we talked about how 2020 was shaping up to be transformational for Blueprint Medicine as we evolved into a fully integrated precision medicine company. And it certainly has been, given the achievements we've made against our goals over the first half of the year.

We specifically disclaim any obligation to update or revise any forward looking statements.

Now here's our CEO Jeff hours.

Thanks, Chris and good morning, everyone.

I'm happy to write an update on what has been a productive quarter probably permit.

At the beginning of year, we talk about how 2020 was shaping up to be transformational [laughter].

We evolved into a fully integrated precision medicine company.

And it certainly has given the achievements we've made against our goals over the first half the year.

[noise], let me highlight how this progress sets us up going into the second half the year across our three areas of strategic focus.

Jeff Albers: Let me highlight how this progress sets us up going into the second half of the year across our three areas of strategic focus: First, establishing our commercial foundation with AvaKit, which we will now harness for ProsthetMiv. Second, prioritizing systemic mastocytosis given the significant medical needs, and third, leveraging our discovery platform to advance future pipeline opportunities. Let's start off with a strong U.S. AvaKit launch for the treatment of patients with the PDGFR-alpha-exon-18 mutant gene. As Christy will discuss in a moment, we've gained critical commercial experience and have built a nimble and highly effective team, along with infrastructure, to deliver our products to patients.

First establishing our commercial foundation with even care, which we will now harnessed for profit.

Second prioritizing kinda mastocytosis, given the significant medical me.

And third leveraging our discovery platform to advance future pipeline opportunities.

[laughter] strong U.S., even kit launch for the treatment of patients with PDGF, our Elfa excellent 18, Jeff.

As Christie will discuss the moment, we gain critical commercial experience an adult a nimble in highly effective team along with infrastructure to deliver our products to patients.

Jeff Albers: Our early experience with AvaKit gives us a strong foundation for our planned launch of Prowl Setinib in the coming months, which will now be amplified as we integrate the capabilities of our new partners at Genentech in Rome. We're also capitalizing on our AvaKit experience outside of the U.S. Just a few days ago, we received a positive CHMP opinion for Ava Pritniv for the This achievement sets up a final decision on our marketing authorization application by the end of this.

Early experience with any of the Kid gives us a strong foundation for our planned launch process in the coming up.

We'll now be amplified as we integrate the capabilities are new partners at Genentech and Roche.

Well the capitalizing on our even kit experience outside of the U.S.

Just a few days ago, we received a positive C.H.M.P. opinion Braver Britain NIM.

For the treatment of patients with PDGF or alpha di for to be needs and yes.

This achievement sets up a final decision on our marketing authorization application by the end of this quarter.

Jeff Albers: Assuming Avifritinib is approved, we expect to initiate our first commercial launch in Germany, with additional European countries to follow. Additionally, as in the US, we expect this initial launch will lay the foundation for future systemic mastocytosis commercial efforts as well. Building from commercial readiness, our second strategic area of focus is the great progress within our systemic masses.

Assuming a buffer and it is approved we expect to initiate our first commercial launch in Germany with additional European countries default.

Additionally, as in the U.S.. We expect this initial launch will lay the foundation for future systemic mastocytosis commercial efforts as well.

[noise] doing from commercial readiness of our second strategic area of focus is a great progress within our systemic mastocytosis program.

Jeff Albers: We believe SM represents the single largest opportunity across our clinical stage portfolio. The significant medical need, combined with our differentiated approach to potently targeting the FM disease driver, opens an opportunity to advance a new treatment paradigm. Earlier this year, we reported very encouraging data from part one of our pioneer trial of avipritinib in patients with indolent systemic mastocytosis. Based on these data, we selected 25 milligrams once per day as the recommended dose and finalized the design of the registration enabling part two with input from regulatory authorities. And today, we're pleased to announce that we've initiated Part 2 of... Later this quarter, we plan to report top-line data from the EXPLORE and PATHFINDER trials of avipritinib in patients with advanced systemic mastocytes.

We believe S. M represents the single largest opportunity across our clinical stage portfolio.

The significant medical need combined with our differentiated approach potently targeting the SM disease driver opened an opportunity advancing new treatment paradigm.

Earlier this year, we reported very encouraging data from part one of our pioneer trial of either Britain and.

In patients with indolent systemic mastocytosis.

Based on these data we feel like a 25 milligrams once per day as the recommended dose and finalized the design of the registration, enabling her to with input from regulatory authorities.

And today, we're pleased to announce that we've initiated part two of this trial.

[noise] later this quarter, we plan to report topline data from the exploring Pathfinder trials of either Britain <unk> in patients with advanced systemic mastocytosis.

Jeff Albers: The combined data set, which will support the submission of a supplemental new drug application to the FDA in the fourth quarter, will include response assessments for approximately 50 patients starting at the recommended 200 milligrams once daily. Additional data supporting the submission will include response kinetics, including the time to response and the duration of response, objective measure readouts such as serum triptase and mass cell burden, and updated safety results across the broader trial population. Finally, a robust discovery platform continues to provide broad opportunities for the future. Earlier this year, we nominated a first-in-class development candidate, Blue 945, for triple mutant EGFR positive non-small cell lines. This program combines potent inhibition of treatment-resistant triple mutant EGFR with selectivity over the wild-type, highlighting the ability of our scientific platform to continue to address challenging target product profiles.

The combined data set.

To support the submission of a supplemental new drug application to the FDA in the fourth quarter.

Well good response assessments for approximately 50 patients starting out the recommended 200 milligram once daily dose.

[noise] additional data supporting submission will include response kinetics, including the time to response in the duration of response.

Objective measure read out such as serum trip Tayfun MFL burden and updated safety results across the broader trial population.

[noise] finally, a robust discovery platform continues to provide brought opportunities for future growth.

Earlier this year, we nominated a first in class development candidate Blue Nile, four or five for Triple meeting, Egypt bar positive non small cell lung cancer.

This program combined potent inhibition of treatment resistant triple mutinied yet bar.

With selectivity over the wild type King.

Hi, what do the ability of our scientific platform to continue to address challenging target product profile.

At the virtual ESMO Congress in September we plan to present, new preclinical data blue knife or five.

Jeff Albers: At the Virtual Esmo Congress in September, we plan to present new preclinical data for Blue 945, as well as share an update on our plans to bring this therapy into the clinic early next year. Importantly, the Triple Mutant EGFR program is just the first of up to three new development candidates we hope to nominate by the end of the year. Our progress across these areas lays the foundation for significant growth for Blueprint Medicines as we continue on our path to become the world's leading precision medicine company. We look forward to updating you on these critical data and regulatory milestones over the coming months and throughout the second half of the year. And with that, I'll now turn the call over to Christy to provide an update on our commercial. Christy

As well as share an update on our plans to bring this therapy into the clinic early next year.

Importantly, the Triple mute Egypt Bar program is that just Oh first of up to three new development candidates, we hope to nominate by the end of the year.

Our progress across these areas lays the foundation for significant growth for Bluebird medicines as we continue on our path to become the world's leading precision medicine company.

We look forward to updating you on these critical data and regulatory milestones and over the coming month and into the second half or throughout the second half of the year.

And with that I'll now turn the call over to Christy to provide an update on our commercial efforts Christie.

Christina Rossi: Great. Thanks, Jeff.

Great. Thanks, Yes, and good morning, everyone.

Christina Rossi: And good morning, everyone. I'm happy to share an update on our commercial progress, including our first full quarter of AvaKit sales in the PDGFR Alpha Exxon 18 Mutant Jet and our ongoing preparations for the planned launch of PROSET through the first half of 2020. We have been focused on establishing the foundation that will support multiple anticipated global launches in the coming years, with potential near-term approvals of Pralsetnib in the U.S. and Avipritnib in Europe. We are truly excited to be on the precipice of delivering a portfolio of precision therapies that address significant patient needs. Of course, the successful launch of AvaKit has been the essential first step on our journey toward realizing this goal. In the second quarter, we generated $5.7 million in net sales, or $9.1 million since launch, driven by strong execution against our launch strategy.

I'm happy to share an update on our commercial progress, including our first full quarter of either kit sales NPD Jaffer Alpha eggs on 18 <unk> Jess.

And our ongoing preparation for the plans to launch a process.

The first half of 2020, we have been focused on establishing the foundation that will support multiple anticipated global launches in the coming.

With potential near term approvals of how sad and Mad Binney, U.S. and Eva <unk> in Europe.

We are truly excited he beyond the process.

I've delivering a portfolio of precision therapies that address significant patient needs.

Of course, the successful launch it.

It's been the essential first step on our journey toward realizing this school.

In the second quarter, we generated $5.7 million in that sell.

Or 9.1 million since launch.

Driven by strong execution against our watch strategies.

We quickly established blueprint with key oncology and hematology centers of excellence.

Christina Rossi: We quickly established Blueprint with key oncology and hematology centers of excellence and other stakeholders that were critical not just for our initial launch in JIF but for Proust-Zett-Nibb and systemic mastocytosis. We achieved broad access for AvaKit rapidly, and we continue to see strong patient access in line with or better than our label by focusing on clinical education and individualized patient support. We have ensured that prescribed patients can start treatment quickly and also remain on treatment as long as it is clinically appropriate. I've been particularly pleased to see that refill rates have been high, suggesting that our real-world durations may exceed those seen in our clinical studies. Finally, last quarter, I noted that I was encouraged to see strong breadth of prescribing on LARP.

And other stakeholders that are critical not just for our initial launch and yet.

<unk> for profit that Ned and systemic mastocytosis.

We achieved broad access for either kit rapidly.

And we continue to see strong patient access in line with or better than our label.

By focusing on clinical education, and individualized patient support.

We have ensured that prescribes patients can start on treatment quickly.

And also remain on treatment as long as it is clinically appropriate.

And I couldn't particularly pleased to see that refill rate has been high.

Adjusting that are real world duration may exceed those seen and our clinical studies.

Finally last quarter I noted that I was encouraged to see strong Brasil prescribing watch.

Christina Rossi: This trend has continued in Q2, fueled by the efforts of our seasoned field, and we continue to add new prescribers, with approximately half now coming from the community setting. I've noted before the significant overlap between GIST and lung prescribers in the community. And this has enabled us to advance our profiling and disease awareness efforts as we prepare for the next chapter in our commercial evolution, our anticipated launch across that note. As I've shared before, we plan to deliver a best-in-class RET inhibitor to patients, focusing on our differentiated clinical profile, including deep and durable responses, a predictable and manageable safety profile, and once daily dosing.

This trend has continued in Q2 fueled by the efforts of our season to feel team.

And they continue to add new prescribers with approximately half now coming from the community setting.

I've noted before the significant overlap between jets and lung prescribers in the community.

And this has enabled us to advance our profiling and disease awareness efforts.

As we prepare for the next chapter in our commercial evolution.

Our anticipated launch across that net.

And that's shared before we plan to deliver a best in class Ret inhibitor patients.

Focusing on our differentiated clinical profile, including eat and durable responses.

He predictable and manageable safety profile and once daily dosing.

We will be launching with a patient and health care provider centric centered approach that recognizes the importance of community oncology centers.

Christina Rossi: We will be launching with a patient and healthcare provider-centered approach that recognizes the importance of community oncology centers and a highly experienced team for whom this is the top priority. I was confident in our ability to launch before we entered into our transformative partnership with Genentech. And as we started to engage with our colleagues there to implement this collaboration, I could not be more excited about the power we will bring across our two organizations. While Blueprint will be driving launch efforts, we are already working to integrate with Genentech and identify near-term opportunities for them to amplify and extend our efforts. For example, by leveraging their substantial diagnostic and data capabilities, in addition to their experience, As we prepare for the launch of our second medicine in a single year, we are well-positioned to maximize the opportunity for Pralsat Nibs, with a differentiated product profile.

And a highly experienced team for homes. This is the top priority.

I was confident in our ability to launch before we entered into our transformative partnership with genetic.

And actually started drinking age with our colleagues there to implement this collaboration.

I could not be more excited about the power, we will bring across our organization.

Well blueprint will be driving lunch at that we are already working to integrate listen on pack and identify near term opportunities for them to amplify and extend our efforts.

For example by leveraging their substantial diagnostic data capability.

In addition to the experience of their team.

As we prepare for the launch of our second medicine in a single here.

We are well positioned to maximize the opportunity for process.

With a differentiated product profile.

Christina Rossi: Focus, and a Nimble Team that is

Okay, and nimble team that is amplified by a strong partner.

Christina Rossi: Focus.

Christina Rossi: and a foundational infrastructure from our experience with AvaKit.

And a foundational infrastructure for my experience with either kit.

Christina Rossi: We are through.

Christina Rossi: We are thrilled with our commercial preparation and execution thus far. We are well on our way to building a best-in-class commercial organization to effectively deliver multiple precision therapies to patients. I'd now like to turn it over to Mike to discuss financial...

We are thrilled with our commercial preparation and execution best spar.

We are well on our way to building a best in class commercial organization.

To effectively deliver multiple precision therapies to patients.

I'd now like to turn it over to Mike That's financial assay.

Thanks Christy.

Michael Landsittel: Thanks, Christina. Earlier this morning, we reported detailed second quarter 2020 financial results in our press release. For today's call, I'll touch on a few financial highlights from the quarter. As Christy mentioned, we were pleased to record $5.7 million of AvaKit net product sales in the second quarter. The cost of sales remained low as we continued to work off inventory of AvaKit that we expensed pre-approval.

Earlier. This morning, we reported detailed second quarter 2020 financial results in our press release.

For today's call I'll touch on a few financial highlights from the quarter.

First you mentioned, we were pleased to record $5.7 million Eva <unk> net product sales in the second quarter.

Cost of sales remain though as we continued to work off inventory of advocate that we expense pre approval.

Our total operating expenses increased slightly compared to the prior quarter.

Michael Landsittel: Our total operating expenses increased slightly compared to the prior quarter, driven in part by an increase in stock-based compensation. Looking forward, in light of our global collaboration with Roche, we expect to see quarter-over-quarter expense growth stabilize. For the second half of 2020, as savings through the cost and profit sharing arrangement with Carl Suttonibs offset planned increases in R&D investment in systemic mastocytosis and our discovery portfolio. However, we do anticipate continued increases in non-cash stock-based consultation expense for the foreseeable future.

Isn't in part by an increase in stock based compensation expense.

Looking forward in light of our global collaboration with Roche.

We expect to see quarter over quarter expense growth stabilize.

For the second half of 20 point.

Savings through the costs in profit sharing arrangement with cross Sutton.

Offset planned increases in R&D investments and systemic mastocytosis and our discovery portfolio.

We do anticipate continued increases in noncash stock based compensation expense for the foreseeable future.

With over $1.4 billion in cash after including the upfront payments from a risk collaboration we are in the strongest financial position that we have ever band as a company.

Operator: With over $1.4 billion in cash after including the upfront payments from our Roche collaboration, we are in the strongest financial position that we have ever been as a company, excluding the upfront payment from Roast. We anticipate the potential for additional revenues of up to $80 million in the second half of 2020 from a combination of milestones from our multiple collaborations and product sales. We also received an additional $20 million in cash in July from our collaboration with Clementia for Blue 782 that was previously recognized as revenue in Q4 2019. Including our collaborations and future anticipated product revenue. We now have a clear pathway to financial independence and enhanced flexibility to invest more in multiple high-value opportunities across our portfolio. We are in a unique position for a company of our size and age and are looking forward to sharing further updates with you as we continue to make progress across our portfolio. With that, I would now like to turn the call over to our operator for questions. Operator?

Excluding the upfront payment from Roche, we anticipate the potential for additional revenues of $80 million in the second half is 2020 from a combination of milestones from our multiple collaborations and product sales.

We also received an additional $20 million in cash in July from our collaboration with dementia, probably 72.

That was previously recognized as revenue in Q4 29.

Between our collaboration and future anticipated product revenues, we now have a career pathway to financial independence.

And enhanced flexibility to invest more in multiple high value opportunities across our portfolio.

We are in a unique position for a company of our size an age and are looking forward to sharing further updates with you as we continue to make progress across our portfolio.

With that I would now like to turn the call over operator for questions operator.

At this time, if you would like to asking questions. Please press Star then the number one on your telephone keypad. Your first question comes from the line of South and [noise] with Goldman Sachs.

Operator: At this time, if you would like to ask a question, please press star, then the number one on your telephone keypad. Your first question comes from the line of Salveen Richter with Goldman Sachs.

Hi, everyone. This is andrew or on for Saudi and thanks for taking my question My.

Andrea: Hi everyone, this is Andrea from Salveen. Thanks for taking the questions. My first question is probably for Jeff. Can you just remind us the expectations for patient screening enrollment for Part 2 of the Pioneer Study and when we might see data? And then I have a follow-up question.

My first time, maybe for Jack can you just remind us the expectations on timing for patient screening enrollment for part two of the pioneer study and when we might see data.

And then I've a follow up question.

Jeff Albers: Sure. So this is Jeff.

Sure.

Jeff Albers: On Pioneer, we talked about this at the last quarterly call that we've now initiated the study and continue to see a lot of enthusiasm from both sites that were involved with Part 1 as well as some new sites coming on that are going through the screening process. We have not guided to the timing of readout. As a reminder, we plan to enroll 200 patients, but we want to see how the site initiation and impact of COVID plays out, not just in the next few weeks, but also over the coming months. So to better handle the consistency with which sites are staying open, we're going to hold off on providing that guidance.

Turning to Jeff.

And here and we talked about this at the last quarterly call that we've now initiated the study and they continue to see a lot of enthusiasm from both sides that were involved with part one is well with some new sites coming on that are going through there's the screening process, we have not guided to timing of readout.

As a reminder, we plan to enrolled 200 patients.

But we want to see how is that this site initiation and impact of kobe's plays out not just in the next few weeks, but also over the coming on so doing better handle on the consistency with what you say theres a staying open we're we're going to hold off on providing that guidance.

Got it and then maybe just a second question for Christie, how should we think about the cadence of hiring for the either <unk> launch both in the U.S. and an expected in the you and then can you speak to the progress of how you guys have been incorporating the Roche team in preparation for processing, just maybe some connections that if any.

Jeff Albers: Got it, and then maybe just a second question for Christy. How should we think about the cadence of hiring for the AvaKit launch both in the U.S. and then expected in the EU? And then can you speak to the progress of how you guys have been incorporating the Roche team in preparation for Proc. NIV? Just maybe some actions that have been taking place there.

Taking place there.

Christina Rossi: Sure, so the first question was around AvaKit in the U.S. and EU. The U.S. team, as I said before, for AvaKit is in place, has been in place since launch, and in fact, that team was really built with a portfolio focus in mind, so that team will be driving the Procetnib launch and really all

Sure. So the first question was around eight I cant in the U.S. and you as you know the U.S.P. and as I said before her in a cat is a is in place at has been in place since launch and in fact, you know that team is really felt with a portfolio focus and mine. So ah that team will be driving that process.

And have lunch.

Christina Rossi: And really, all along, our primary priority with that team has been...

Really all along I primary priority with that has been very much focused on preparing for.

Christina Rossi: and launching PralSETnib. And so we're going to see that team's primary focus really shift, currently it's disease education and profiling, and then upon launch, we'll be focused on driving promotion for PralSETnib. For Europe, we have a small focused NIMBLE team in place in the countries that we anticipate near-term launches. The focus of that team will be on driving the initial AvaKit launch. And because that market is very focused on a number of treatment centers with whom we already have relationships, we believe that we can target that market effectively with a small NIMBLE team. And then that team will have the foundational infrastructure that will prepare for an eventual SM approval.

Oh that net add and so we're going to see that teams a primary focus really chef currently it is disease education or profiling.

We'll be focused on driving promotion or for profit that's for Europe.

We have a small focus nimble team in place.

Countries that we anticipate Trump watch as a you know that the focus of that team well be on dry they'd be initial a they kept lunch and because that market is very focused at a number of treatment centers with him. We already had relationships and we believe that we can try to that market effectively with a smaller nibble team and then that team.

Well have the foundational infrastructure that will prepare for an eventual at Democrats on yes.

Christina Rossi: And Christie, the second part was integra-

[noise] and critically the second part what integration with Roche Oh, sorry, yes. So.

Christina Rossi: The Roche team, as I said, has been very involved in these initial weeks since we announced the collaboration. Due to the proximity of what we anticipate to be an imminent approval for Prospect Nib, certainly Blueprint will be driving many of the launch efforts, but we've already identified ways for Genentech in the United States to come in and really amplify our efforts, particularly around patient identification, leveraging data, helping to drive testing, as an example, and then clearly just leveraging the power of their portfolio of targeted therapies into which Prospect Nib fits really nicely.

The Roche scheme as I said has.

Very involved.

<unk> steel wheel.

Collaboration at either due to the proximity of what we anticipate to be.

Approval for four across that Deb, I, certainly blueprint well be driving many of the lunch efforts that we've already identified ways for to that pack in the United States.

It's really amplify amplifier effort, a particularly around patient identification leveraging data helping to drive testing as an example, and then clearly just leveraging the power of their their portfolio of targeted therapies.

So you know we're still working through specific I somebody tells on roles and responsibilities that have identified ways, where they can come in and really help us out of the <unk>.

Christina Rossi: We have identified a number of ways where they think they can come in and really help us.

Christina Rossi: Great, thanks so much. Your next question comes from the line of Peter Lawson with Barclays.

Great. Thanks, so much.

Your next question comes from the line of Peter Lawson with Barclays.

Peter Richard Lawson: Hi, thanks for taking my questions. Just on the RET space, just wondering what you're hearing from physicians regarding the better CRE, like a QT prolongation versus the loxose molecule, and I know we're kind of here in mixed meth.

Hi, Thanks, Thanks for taking my questions just on the rent space, just wonder what you're hearing from decisions regarding.

The CR rate.

Like is Qt prolongation, but it's will cause some older children. When they were kind of hearing mixed messages. So it's great to support your here.

Jeff Albers: So it's great to understand what you're hearing.

Jeff Albers: I'll start and then Andy or Christy can weigh in. We continue to be very encouraged by the data that we put out most recently at ASCO and the response to that. As you know, the consistency of activity we're seeing, the deep and durable responses, the notable CR rates, which, you know, for many physicians are something that they're very encouraged by, and they'll want to see how that translates into our ability to respond over the longer term. And then the predictable safety profile that Andy noted in his opening comments that, you know, particularly if you push out the community, what we hear is that physicians want, are So there are obviously differences when we look at the academic sites versus the community sites, but overall, we think we have a very promising profile with PralSetna.

Sure. This is Jeff I'll start and Andy Christy and then can weigh in with Katie I'm very encouraged by the the data that we put out most recently at ASCO in response to that if he knows the consistency of activity we're seeing even.

Trouble responses.

A notable.

I see our Raceway, which you know for many physicians or somebody that they're very encouraged by and don't want to see how does that translate into durability of response over the longer term and then the predictable safety profile that you noted in his opening comments that you, particularly push on the community.

What we hear his position.

One are much more comfortable with.

[laughter] side effects that they manage historically aired are consistent with other therapies.

And the more unusual adverse events the more they can have an impact on what they're going to how they're going to treat so there's obviously differences when we look at the academic sites versus the median overall.

Overall, we think we have a very promising profile with profit.

Andrew Scott Berens: Maybe I'll just add that we've talked to a lot of both our investigators and other kinds of community docs now, and the CRH, I think, repeatedly comes back as a distinguishing characteristic. And a bit more on the QT, I think the hardest thing about things like QT prolongations is that they restrict the use of other medications that patients often need just for general supportive care, antibiotics, antibiotics, and so I think just not having to worry about

[laughter] true.

Yes, Thanks, Yeah, I mean does and that and you know we've talked to a lot of other investigators and other come community Docs. How is your rate I think repeatedly comes back as a distinguishing characteristic.

During the Q cheap I think the hersey about things like easy prolongation that restricts the use of other medications that the patients often need just for general supportive care antibiotics and amenities. So I think should not have you worry about that is a real advantage.

Andrew Scott Berens: [inaudible]

Great. Thank you and then just come to the launched on them in <unk>, which we look out is that no other rent roseland or how can what are the best Brooks is here and what does because of the puts and takes this isn't launches.

Christina Rossi: Great, thank you. And then just on the launch dynamics, what should we look at? Is it, you know, other RATs, RAS1 or ALK, and what are the best proxies here, and what are the kind of puts and takes versus those other launches?

Christina Rossi: Chris, did you want to take that?

Chris you want to take that.

Sure. So you know I think all all of those analogs are helpful.

Christina Rossi: Sure. So, you know, I think all of those analogs are helpful. But none of them are perfect, right?

Christina Rossi: So, you know, I think certainly, you know, a driver that many are often interested in is just sort of the acceleration of testing and patient identification. So, you know, if you look historically at other targeted therapy launches in lung cancer, you tend to see, you know, a ramp of testing practices that is driven by the availability of effective therapy. And I think that ramp has become more accelerated with recent launches as the space has really shifted towards the utilization of testing more broadly. You know, we'd expect a similar dynamic with FRET and certainly having, you know, potentially two agents on the market is very helpful in that regard, right, to really drive testing practices. But obviously, you know, each space is slightly different in terms of the frequency of the mutation, the number of players in the space, et cetera. So, you know, we are, as Jeff said, really excited.

Perfect right. So I think certainly as a driver I think that many are often interested and it's just started the acceleration of of passing uptake.

So you know if you look historically at other targeted therapy lunches and line you tend to see a you know a ramp of testing practices that is driven by the availability of effective therapy and I think says that ramp has become more accelerated with recent recent launches base is really shifted.

Utilization of attaching more broadly Oh, yeah, we'd expect to similar dynamic with with threat and certainly having you know potentially to agents on the market is it's very helpful. In that regard right. So really it's really drive.

But obviously you know those each station slightly different.

The frequency of education and the number of players in this space et cetera. So you know we are as Jeff said, you know really excited and did the ethic about getting out there with that with process.

Christina Rossi: I am enthusiastic about getting out there with Prospect Nibs.

Unknown Attendee: https://www.cdc.gov.au

Looking forward to you hopefully having that available.

Great. Thanks.

Christina Rossi: Great. Thanks.

Your next question comes from the line of market frame with Cowen and company.

Marc Alan Frahm: Your next question comes from the line of Marc Frahm with Cow Hunting Company.

Hi, Thanks for taking my questions.

Marc Alan Frahm: Thanks for taking my questions. Let me just follow up on an earlier question about the ISM trial. Jeff and Andy, in the past, you kind of talked about a backlog of patients, you know, as you were waiting to select a dose and things like that. As you've activated sites, have you seen kind of a bolus of patients coming in from the sites as they come online as you would have expected? Or are you already kind of seeing a little bit of reluctance from patients to come into the clinic?

Maybe just follow up on an earlier question about the Isom trial I'm guessing has definitely in the past you talked about a backlog of patients as you were waiting to selected dose and things like that as Youve activated sites have you seen kind of a bolus of patients coming in from the sites as they come online like you would have expected or are you argue.

Seeing those a little bit of reluctance from patients to become another clinic given the pandemic.

Jeff Albers: So this is Jeff Maybell starting. Andy Kalea, I think that's too early to say.

Yeah, I'm jumping on certain anyway, I think that's too early to say I mean every site is unique you know I find that I often paint in broad strokes of the U.S. versus your up and then I'm reminded that Massachusetts is different than Nebraska.

Jeff Albers: is unique.

Jeff Albers: You know, I find that I often paint broad strokes of the U.S. versus Europe, and then I'm reminded that, you know, Massachusetts is different than Nebraska, so what we're seeing is there is clearly demand. And that really underlies our continued push that systemic mastocytosis is the biggest opportunity that we're focused on and provides really meaningful upside as you think about patients not only being treated and identified but then staying on for So I think that your thesis is completely intact.

So what we're seeing is as you there is clearly demand.

And then that that really underlies our continued.

Push that systemic mastocytosis is the biggest opportunity that we're focused on and and provide really meaningful upside as you think about patients not only being treated and identified but it's staying on for a long period time, when a targeted therapy like gave a pretty them. So I think that.

Jeff Albers: As we see that then play out site by site, I think there's going to be some variability. We have sites that definitely have backlogs, and how they're going to manage that and how often patients come in, it varies by the investigational site we're seeing. So we just need to work through that, and that's why we feel like it's inappropriate to provide specific guidance.

That thesis is completely attack as we see that didn't play out site by site I think there's gonna be some variability and we we have they said definitely have backlogs and how they're going to manage that and what how often patients come in is it varies by a investigational site work we're seeing.

So we just need to work through that that's why we feel like inappropriate to provide specific guidance. We know notwithstanding a pandemic. The demand is there and you will continue to see these patients enrolled we're seeing patients already lining up to come in so.

Jeff Albers: We know that, notwithstanding a pandemic, the demand is there, and we'll continue to see these patients enrolled. We're seeing patients already lining up to come in. So even with a pandemic, the medical need is high enough that we're going to enroll these patients. But how that will exactly play out when you think about 20 to 30 sites, I think.

Even with a pandemic that there's a medical need is high enough that we're going to you to enroll these patients, but that's how that will exactly play out when you think about 20 to 30 sites I think there's going be variability.

Andrew Scott Berens: I think you framed it well, Jeff. As we talked to our investigators, they really do have a list of patients that they're looking at. And as Jeff said, it really is a matter of being in Florida now versus Massachusetts versus somewhere else in the country. It's a bit of a moving target. When people in different places feel comfortable coming into health care centers in the setting of them not having an immediate life-threatening disease, we think it's going to move quickly.

And in <unk>.

Thank you you framed it well Jeff.

The degree as we've talked to our investigators theirs. They really do you have to this patient.

And it's just there's really as a matter.

You know in Florida, now versus Massachusetts versus you know somewhere else in the country. It's just it's been a moving target when people in place we feel comfortable coming into the health care centers in the study Im not have eased my friend and immediately like friends.

<unk>.

Okay.

Marc Alan Frahm: Okay. Okay. Great. And then maybe a quick one on AvaKit for Christy. Just in the past, you've mentioned seeing demand both in PDGFR as well as outside of PDGFR. You know, following the Voyager trial results, have you kind of noticed a change in the new patient start trajectory as maybe demand outside of PDGFR shrank?

Great and then maybe a quick one on the on the because for Christy just in the past you've mentioned seeing demand within PDGF ours was outside of PDGF are you're following the Voyager travelers will have you noticed a dip a change in the new patient starts trajectory as maybe demand outside if you did you have our Uh huh.

Christina Rossi: Sure. So, you know, as we said, we see utilization, you know, across. It's difficult to, you know, say precisely what the patient breakdown looks like between PDGFR-alpha and other GIST patients. You know, mutation status is not something that you can kind of pull consistently, and so you're sort of relying on what you just may learn organically about these patients. You know, I can say that we're continuing to see both prescribers added as well as new patient starts, and I, you know, we're seeing them across. So, we're seeing them in PDGFR-alpha as well as in kit-driven GIST, although clearly our focus is on PDGFR-alpha, which is where the drug is labeled. You know, I think going forward, certainly, you've got a number of dynamics at play right now. You've got, you know, COVID. You have repregnant now been approved.

[noise] sorry, so you know as as he said, we see we see utilization crosscut, it's difficult to say precisely what the p. shit breakdown looks like it's been pretty Jeffrey Alpha and other gestation seeing indications that says not something that you can kind of pull consistently so.

You are sort of relying on what you just me learn organically about these patients.

Yeah, I can say that we're continuing to see a close prescribers added as well as new patient starts and I. You know, we're seeing them across that we're seeing that I can you get Ralph as well as a picture of ingest, although clearly our focus is exactly.

Which is which is where the drivers label I think going forward certainly if that number of dynamics at play right.

Kobe.

Now a prayer so yeah I think all of this will factor and but I would continue to expect to see you patient demand both in PDF, Ralph a bolus outside of that I mean, we know that those patients that very significant need that.

Christina Rossi: So, you know, I think all of those will factor in, but I would continue to expect to see new patient demand both in PDGFR-alpha as well as outside of it. I mean, we know that those patients have a very significant need there, and, you know, current therapies, even with the approval of RepRetNib, that need remains. So, I think we can expect to see that on an ongoing basis. Of course, for our team, as I said, the focus for the second half of the year will really be primarily from a demand generation perspective focused on PropRetNib, and then, of course, you know, starting to prepare for what we hope will be a launch in advance of that.

You know current therapy, even with the approval out for Pat.

That that need remain so I think we can expect to see that on an ongoing basis of course for our team you know the focus as I said to the second half of the air will really be primarily from a demand generation perspective, I'm focused on crop that NAV.

Of course, that's repair for what we hope will be a lunch.

[noise], okay, great. Thanks.

Marc Alan Frahm: Okay, great, thanks.

Operator: Operator, your next question comes from the line of Diane Leone with Raymond James.

Your next question comes with a line of Diana Leon with Raymond James.

Dane Vincent Leone: Hi, this is Dane filling in for Diane today. Hi. We'll be back tomorrow. Congratulations on the update. Two for me.

Hi, This is Dan filling in for Diane Oh [laughter].

All right well be back tomorrow.

Congrats on the update.

Two for me firstly on the advance that's.

Dane Vincent Leone: Firstly, on the advanced SM, how do you think about having the pivotal data set in hand at the 200 mg QD dose and how that could update the label for avopretinib? The specifics to that question are that right now you have a label that was all-encompassing for Avokit to be approved based on the data you had from a safety perspective across both SM and GIST. But you're going to have a more specific update for safety at the 200 mg dose, which I think a lot of us expect to have a lower event rate and more dynamics around how to manage some of the thrombocytopenia clinical sequelae. So, what are your thoughts on how we could see an FDA label update that would help inform clinicians and actually better characterize the actual dose that you think should be used?

How do you think about.

Having that pivotal data in hand at the 200, Meg QD dose.

And how that could update the label for Eva Prednisone. The specifics that question is right. Now you have the label that was all encompassing for either could be a crew of the data you had from a safety perspective across the us and just but you're gonna have more specific updates.

For.

Safety out to 200 make dose, which I think a lot of us expect to have a lower event rate and have more dynamics around how to manage some of the thrombocytopenia or clinical supplies. So what are your thoughts of how we could see a few label update that would help inform clinicians and actually.

That are characterized the actual dose that you.

Andrew Scott Berens: It's Andy. Yeah, Dane, I'll take that.

I think should be used for it so.

Andrew Scott Berens: Um, so, so yeah, no, I think that it will be laid out, um, at the 200 mg dose. You know, very typically, in both US and EU labels, when new indications get added, there's a safety section on the study that supported that, that, indication. And I would fully expect that we would have a description of safety at the 200 mg dose. And there may, you know, I mean,

It's Andy I do think that so she had no I think that it will be laid out to me because she's very typically in in both U.S. and you labels when new indications get added there's a safety section on the study that supported that that Tom indication and I would unfold.

We expect that we would have a description of the safety of the 200 make dose and their me I mean, obviously, we are forward looking here and spend a little bit on radio kids thinking, but they could my they might also have some sort of integrated analysis that covers both.

Andrew Scott Berens: We're forward-looking here and depend a little bit on where the FDA is thinking, but they might also have some sort of...

Andrew Scott Berens: Okay, and would there be additional language you think on, you know, platelet count and management of symptoms or any guidance included in the label from that perspective that would be updated?

Okay and would there be additional language you think on.

Platelet count and management of symptoms or.

Any like guidance included in the label from that perspective that would be up.

Andrew Scott Berens: Yeah, no, I think...

Yeah, I think there there will there should be guidance on on platelet counts in the label I mean that we would actually want because we think it's really important Tom.

Andrew Scott Berens: No, I think they're...

Andrew Scott Berens: There should be guidance on platelet counts in the label, which we would actually want because we think it's really important to have patients with severe thrombocytopenia have that dealt with before they get AvaKit when they have systemic mastocytosis, so I'd expect there would be a separate description of guidance for patients with advanced SSI.

You have patients with severe thrombocytopenia out that dealt with before they get Eva image at and they have systemic business I. Just started expect there'd be a separate description of guidance for patients with advanced system.

Dane Vincent Leone: Great, and the second one for me is, can you give a status update on Blue 263?

Great and the second one for me is can you give a status update on blue to six flourish.

Andrew Scott Berens: Yeah, no, as we said previously, the study, the first human health volunteer study, has started, and it's moving along. You know, it's an ongoing study, so we don't have any specific information to provide at this time, but it's enrolling as expected. Actually, I'd say that's an area we're really pleased with. I think that was an area where I thought that COVID-19 could be a problem, but the site really came together and figured it out and has done so.

Yeah, so that.

As we said previously to study the first human Health Bouncers study has started and it's moving along you know it's an ongoing healthy volunteers. They should we don't have any specific information to provide at this time, but it's enrolling as a as expected actually I'd say, that's an area. We're really pleased I think that was an era.

It is where I thought that the coping 19 could be a problem at the site really came together and figured it out it has kept things on track.

Andrew Scott Berens: things on track.

Andrew Scott Berens: Great. Have you given a timeline of when we will see an update on when that program is going to progress or any initial first-in-human results?

Great and they have you given a timeline of when we did see.

An update on <unk> went out programs got progress around here or initial person human results.

Andrew Scott Berens: Yes, we haven't given any specific guidance on that, and as the study evolves, we'll plan and let you guys know.

Yes, we haven't given a specific guidance on that and as a study volatile will plan like you guys now.

Dane Vincent Leone: Okay, great. Thank you, guys.

Okay, great. Thank you guys.

Operator: Your next question comes from the line of Michael Schmidt with Guggenheim Security.

Your next question comes from the line of Michael Smith.

[noise] good morning, guys as Charles you on for Michael Schmidt, Thanks for taking the questions and congrats on the quarter.

Charles Zuan: Good morning, guys. This is Charles Zuan from Michael Schmidt. Thanks for taking the questions and congrats on the quarter. First, with respect to ongoing AvaKit commercialization and the impending cross-segment launch, it looks like you guys do have a nice emphasis on the community setting, so I was just wondering if you could provide some additional color on not only perhaps the potential synergy between GIST and RETS,

First with respect to ongoing shift commercialization and pending.

Looks like you guys do you have nice emphasis on the community setting. So I'm just wondering if you could provide some additional color on not only perhaps the potential synergy between just and rich, but also I guess you know some from each of around your current presence in the communities studying and what's his Roche order should could potentially bring to the.

Christina Rossi: But also, I guess, you know, some detail around your current presence in the community setting and what this ROADS partnership could potentially bring to that, for example, your exposure to flat iron versus non-flat iron utilizing EMR practices.

For example between your exposure between us versus non Florida.

Christina Rossi: Sure, so you know the community has been a focus of ours out of the gate, and you know we have known for a long time that upwards of maybe 70% of patients are treated in a community setting. I think you know, way back when we talked about sort of the overlap that we see between prescribers for just lung diseases being, you know, 60% plus, and so these are centers that our team has established a presence with and is actively engaging with AvaKit, and then, as I said, is really focused on driving disease awareness for PralSentenib and anticipation of that launch. You know, when I think about our colleagues at Genentech, I think again that they can really help to amplify and extend those efforts.

Yeah Mark.

Chris you want take that.

Sure so.

Thank you maybe had said that focus of ours out of the gate and we know of love that upward. So maybe 70% of patients are treated in the community setting up I think you know way back when we talk about sort of the overlap that we see between prescribers for adjusted lung being a you know.

60% classes. So you know these are centers that our team has established a presence with and is actively engaging for Eva cats that as I said is as really focused on driving disease awareness a for profit that NAV and anticipation of that launch you know what I think about our.

Colleagues at Genentech, I think again, they can really help to you amplifying extend those efforts, yes, certainly the data patient identification as a piece of that but I think it's off the just the breadth of of the teams that they haven't really being able to complement our efforts and getting out of teaching ex the community setting.

Christina Rossi: You know, certainly, patient identification is a piece of that, but I think it's also just the breadth of the team that they have and really being able to complement our efforts and get out and reach into the community setting. And when I talk about an approach that prioritizes the community, for me, it really goes beyond just the engagement that our sales team, for example, may have there. You know, we really believe that making the process of prescribing as seamless as possible for community oncology is going to be really critical. You know, we are launching with a strategy that supports not just prescribers but also community oncology pharmacies and their ability to really maintain the management and care of their patients, which we know is something that's really important to them, and so it's really a 360 approach. I would say that

When I talk about you know what approach that prioritizes community for me it really goes beyond just.

Gauge meant that our sales team for example, they have their you know me really believes that as making the process of prescribing as seamless as possible a sports media college it could be really critical you know we are launching with a strategy that support not just prescribers, but off that's me oncology pharmacies and their ability to.

Really add making the management and care of their patients, which we know it's something that's really important though it's really a threesixty approach I would say that.

<unk>.

Got it thanks for that detail and next question regarding the person is and that leads to six three in a into one.

Charles Zuan: Got it. Thanks for that detail. And my next question regarding Ava Pritnib and BLU-263 and Indolent SM. I was also kind of wondering to what extent current or longer-term safety signals from Ava, Kit, and ISM could be used to either inform or potentially even supplement future efforts and data collection for BLU-263? And also, as a related follow-up, do you see any, I guess, potential differences in patients remaining on therapy given that potentially ISM patients will see that their symptoms are resolving, but maybe healthy volunteers will not, and will that be kind of a less motivating factor to stay on therapy?

Well he will also kind of wondering to what extent could occur in for a longer term safety signals from you, but can I assume be used to either in form or potentially even supplement future efforts and data collection for blue to six three and also as it related follow up do you see any.

I guess potential differences.

Patients remaining on therapy given.

Potentially some patients we'll see that there's sometimes resulting but maybe healthy volunteers will not.

Kind of no less motivating factor to stay on therapy.

Andrew Scott Berens: So maybe I'll start with the last question. So the healthy mental care study is a very time-limited study that really does assess PK safety and establish the best starting dose for the patient. So there's not really an issue there. They'll stay on for the short period of time they need to get those data.

[noise] Andy this is somebody that's sort of electricity be healthy volunteer study is a very time limited.

You know study really to assess.

PK safety and stylish the best starting dose for the.

Page that there's not really the she didn't know stay on the short period of time. They do you get those data I think what are the real opportunities that.

Andrew Scott Berens: I think one of the real opportunities that bringing 263 forward offers is that we do think that it shouldn't have the potential for the cognitive effects that we have seen with avopretinib at higher doses. We are very happy with the data at 25 milligrams with avopretinib where cognitive effects have not been an issue. But I think certainly there is, in theory, the potential that over many years, someone gets avopretinib for five or 10 years, I suppose it's possible that those sorts of things could emerge. And that's the kind of opportunity then that 263 offers to solve, or the kind of problem that 263 would offer to solve. And then again, though we still emphasize more with 263 moving into even less advanced forms of systemic mass psychosis, where it's maybe more with what it solves is the theoretical concern that may not even exist, just because of the data at the higher doses. So I think we are still looking at it as a compound that will develop initially in less advanced forms of SNF.

Turning to the free Board offers is that Tom is and we do think that is that it shouldn't have really [noise].

Inshell for for the causes effect. So we have she was able to breakeven higher doses. We are very happy with the data at 25 milligrams of even for a NIM where.

We're cognizant that has not been issue, but I think certainly there is.

In theory this potential that over many years someone you gave a pretty improved by over 10 years I suppose it's possible that those are things could emerge.

The kind of opportunity in the two sixthree offers to on Saul Oh or that kind of problem at all.

He would offer solve and then again, so we still emphasize more with strategic three movie achieving even on the less advanced forums systemic, Massachusetts, where you maybe more the more with what the fleet what it solved it is the the theoretical concern that may not even just for either just because of the data.

And the hired us so I think we are still looking at it as a as they come and that will develop.

Initially less advanced farms.

Jeff Albers: Yeah, maybe more at a higher level. I know I've talked about this before, but we've made it very clear that systemic mastocytosis is our priority. It fits perfectly with our strategy of going after very targeted patient populations with an exquisitely selective inhibitor, and we've got a monogenic driver of DH16V mutations in this case. It's the intersection of oncology and rare disease, and when we made the decision to move forward with 263, we looked at this as we were operating from a position of strength, and let's double down on that. And as Andy just highlighted, we think it' We continue to be highly encouraged by the emerging data we're seeing with avifritinib, both at the 25 milligram and 200 milligram doses, and we are investing heavily there. We're focused there, and I think that really is what underlies the strength of Blueprint.

And maybe the floor at a higher level.

I know I'm talking about this before but we've made it very clear that systemic mastocytosis is our priority to fit perfectly with our strategy of going after it was very targeted patient populations with in exclusively selective inhibitor and we've got a monogenic driver DHX TV mutation in this case.

It's the energy intersection of oncology and rare disease.

And when we made the decision to move forward to six three we looked at that said, we're operating from a position of strength and lets double down on that and as Andy just highlight we think it's a way to.

Extend that that read our reach and positioning so no. We don't know exactly where it will be needed. We continued to be highly encouraged by the emerging data were seen with Ingram freight NIM I thought that the 25 milligram 200 milligram doses.

And we are investing heavily there were focused there and I think that really is what underlies the strength of blueprint right now.

Very helpful. Thanks for that the children Congrats again on the quarter.

Charles Zuan: Very helpful, thanks for that detail, and congrats again on the quarter.

Your next question comes from the line of David Lebowitz with Morgan Stanley.

Operator: Your next question comes from David Lebowitz, with Morgan Stanley.

David Neil Lebowitz: Thank you very much for your question. With respect to advanced systems, let's run us through data that you sent last year. I recall you had originally intended to submit it then and tried to delay it until after Pathfinder. Have you, I guess, updated...

Thank you.

HM.

[laughter].

[noise] binder.

David Neil Lebowitz: The data that was in the past and what we're looking for.

David Neil Lebowitz: I'm looking for, I guess, incrementally, to prepare.

I guess.

Update.

David Neil Lebowitz: to prepare you for submission.

And what we're looking for.

Jeff Albers: Sure. So you broke up a little bit in the beginning, but I think I captured the gist of it, which is around the emerging ADVANCE-SM data and a reminder of where we were previously. So there's a couple things at play here. First, I think the most important piece is that the data we have shown to date in ADVANCE-SM has been very consistent in terms of high response rates or disease control rates. The objective measure waterfalls show virtually all patients seen a benefit. And the way our trials have emerged is that we've now blended two different trials because of the unusual nature of the IWG response criteria of needing a baseline, which was not built into our part one.

I guess incrementally.

For submission.

Sure.

So you broke up a little bit the beginning of I think I captured the gist of it which is around.

The emerging advantest them data and a reminder of where we were previously.

So there's a couple of things at play here first I think it is the most important pieces that the data we have shown to date in advance that that's been very.

Then.

In terms of high response rates are disease control rate the objective measure waterfalls <unk> show.

Virtually all patients seen a benefit and what the way the our trials have emerges that we've now blended two different trials because of the unusual nature of the I can DG response criteria of needing a baseline which was not built into our part one.

Jeff Albers: We've moved forward with multiple doses where we've pushed up more towards a just like dose of 300 milligrams, but then focused on 200 milligrams, given that we were seeing similar response rates or the same response rate as 300, but it just took a bit longer for that to emerge, but with a cleaner safety profile. So there was a compelling intersection at 200 milligrams where you get to the same point eventually and with fewer adverse events. And when you're thinking about chronic treatment, that's important. And then we changed the exclusion criteria as we identified thrombocytopenia as a risk factor. And so it was really the blend of all of those that went into interactions with regulatory authorities.

We've used we move forward with with multiple doses, where we pushed up more towards the just like dose of 300 milligrams, but then focusing on 200 milligrams given that we were seeing similar response rates are the same response rate and 300, but it just took a bit longer for that to emerge, but with a safer cleaner safety profiles.

So there was a compelling intersection at 200 milligrams that you get to the same point eventually and with fewer adverse events and when you're thinking about chronic treatment that's important.

And then we had changed the exclusion criteria as we identify thrombocytopenic as a risk factor and so it was really the blend of all of those that went in interactions with regulatory authorities. They said.

Jeff Albers: They said it's pretty clear that there's strong efficacy here and there's a manageable safety profile, but we're looking for more homogeneous, if you will, focus groups, thinking about the number of patients who are at 200 milligrams per se across those two trials. And so we agreed to aim for about 50 patients at the 200 milligram starting dose, but obviously, all the data, regardless of the starting dose from the two trials, will be part of our data submission. I think about the second part of your question, which was what we showed before, which is... Andy Mayo, I think it was in the 70 percent, 70 something percent response rate, 77, 77 percent response rate. Yeah, but there's a lot more to the data set. I think I'd probably be out of my skeet if I tried to quote exactly what was shown at ASH previously.

It's a pretty clear cut that or is there a strong efficacy here and there is a manageable safety profile, but we're looking for a more homogenous. If you will you focus groups thinking about the number of patients who are at 200 milligrams per.

Per se across those too.

Two trials and so we had agreed to aim for about 550 patients at the 200 milligram starting dose, but obviously all the data regardless of UBS starting dose from the new trials will be part of of ours data submission I think.

To your second part your question, which is what do we show what did we show before which is.

And you May all I think it would it was in the 70% 70 something percent response, let me have another 77% response rate, yeah, but theres a lot more to that asset I think.

I think I'd probably be out over my skis, if I try to quote exactly what was shown.

Asked previously, but I think then the important factor is is this is really more of a a function of time to have a data center that regulatory agency can focus in on adding starting dose and we view that frankly the passage of time is more as you said.

Jeff Albers: But I think the important factor is this was really more of a function of time to have a data set that a regulatory agency could focus in on at a starting dose. And we view that, frankly, that passage of time is more... sounds pithy, but administrative, that we wanted to get to that point where we had enough patients. But we're very confident in the data that we'll be putting forward. I think it is transformational in nature for these patients. And then again, going back to Pioneer, we think we're just getting started, that this is, you know, an area where there is need, it's clear that a lot of patients are misdiagnosed, and it's clear that it's really a continuum of disease with one driver. These subgroups tend to blend together, and we'll be focused on identifying patients there over the long term, both within our clinical trials and then beyond that, commercially.

Sounds picky, but administer area that we wanted to get to that point were had an appreciation.

But we're very confident in the data that will be putting forward thinking is transformational in nature from participation and then again I'm going back to my here. We think we're just getting started so you know that this is.

An area, where there it's clear that there is need it's clear that a lot of patients are misdiagnosed.

And it's clear that it's really a continual disease with one driver so they shouldn't be subgroups tend to blend together.

And we'll be focused on identifying patients there over the long term both within our clinical trials, but then beyond that commercially.

Jeff Albers: Your next question comes from the line of Ren Benjamin with JMP Securities.

Next question comes from the line of Ren Benjamin with JMP Securities.

Reni John Benjamin: Hey, good morning guys. Thanks for taking the questions and congratulations on the quarter. I guess maybe just starting off, can you talk a little bit or provide some more color regarding the patients that are receiving AvaKit? Are these majority in the...

Hey, good morning, guys. Thanks for taking the questions and congratulations on the quarter.

I guess, maybe just starting off can you talk a little bit will provide more color regarding the patients that are receiving if the kid are these majority in the frontline setting you know after as a diagnostic testing or.

Unknown Attendee: www.microsoft.com. www.microsoft.com

Are they still receiving kind of standard of care and then.

Unknown Attendee: Unknown Attendee, Dr. David Lebowitz, Unknown Attendee, Unknown Attendee,

Primarily a theme this more in the last line setting just any sort of colors to the patients.

Christina Rossi: Christy, do you want to take this?

Christy well take that.

Sure.

Christina Rossi: Sure. So, you know, again, our insight on a patient-by-patient sort of basis is not perfect. So I would start with that caveat.

No.

Again, our insight at a patient by patient sort of basis.

Not perfect. So I would start with that I caveat I think what we saw at lunch with certainly that there were a PDF braava patients started identified who came on to therapy. As you know relatively quickly those patients often had been treated with other therapies prior to receiving either.

Christina Rossi: I think what we saw at launch was certainly that there were PDGF-alpha patients sort of identified who came on to therapy, you know, relatively quickly. Those patients often had been treated with other therapies prior to receiving APHA kits. We're continuing to see new patients come in, and from what we can see, I think that's shifting a bit to patients who are being identified and diagnosed with PDGF-alpha as the mutation and then immediately going on, right? So that's a mix of patients, you know, certainly in academic centers where testing is, you know, more regularly done and is done earlier on when that patient comes into the center. You might see them come on earlier, but often it takes patients' referrals to get there. And so, you know, net-net, I would say that in the U.S., PDGF-alpha patients are predominantly not being identified sort of by the diagnosis. They're still often being treated with, you know, one or more therapies before receiving APHA kits. But again, it's a mix.

Yes.

We're continuing to see new patients come on and I would you go from what we can see I think that shifting a bit to patients who are being identified and diagnosed.

With PDGF Ralph as education.

And that immediately going on right. So that's a mix of patients you know certainly academic centers were testing is a you know more.

Regularly Diane has done earlier on when that patient outcomes compared to the center you might see then come on earlier, but often it takes patient referrals to get there and so you know that that I would say that in the U.S. PDGF Ralph the patients are predominantly not being identified certified at diagnosis, they're still.

Being treated with.

Therapies before it before but I guess, but again it is that.

Got it.

Reni John Benjamin: And then just regarding Prosetnib, has there been, I'm sure before the deal was done, there was an extensive review, but is there a plan now to have an extensive review regarding the Prosetnib development program and kind of what's the current status of that? I think the competition with Osimertnib is still planned. I think you mentioned during a previous call that you're evaluating other combinations. Can you give us a sense as to when we might have a better sense as to how this entire development program moves forward?

And then just regarding costs.

Third been I'm sure before the deal was gone there was an extensive review but is there a planned out to have an extensive review regarding the process development programs and kind of what's the what's the current status of that I think so calm and with those there's still plans.

I think you mentioned during the previous call that you're evaluating other combinations can you give us a sense as to when we might have a better sense as to how this entire development program moves forward.

Jeff Albers: Sure, this is Jeff. I'll start with that. So you're correct in both regards that yes, obviously, there was a comprehensive review of all of the data and looking back as well as all of the plans looking forward. The teams, much as Christy just highlighted how the Genentech and Blueprint Medicines commercial teams are already working together and, frankly, had already started sharing information prior to the signing of the agreement; the clinical teams have been doing the same. We're looking at what that longer-term clinical development plan that both companies would recommend would look like, and then how would that come together. Encouragingly, the forward-looking plans were very similar. And so now, through collaboration, the teams are coming together, and there will no doubt be some changes.

Sure. This is Jeff I'll start with that.

So you're correct in both regarding the yes, obviously there was a comprehensive review of all.

The data at the looking back as well as all of the plans looking forward.

The teams as much as Chris you just highlighted how the Jeanette Tech and blueprint medicines commercial teams are already working together and frankly already started.

I'm sharing information prior to the signing the agreement the clinical teams have been doing the same we're looking at what is that longer term clinical development plan.

That both companies would recommend and then how would that come together encouragingly. The forward looking plans were very similar and so now through through the collaboration the teams are coming together and no no doubt be some changes in one of the ones I'm. Most excited about is that.

Jeff Albers: And one of the ones I'm most excited about is that there will be a condensing of time on many of the things that at Blueprint we likely would have done sequentially, where now there's the opportunity to do several things simultaneously. But we will need to work through those with our partner. And then in terms of when and how we'll update that, that will also flow through the collaboration in terms of how much is disclosed and when that's disclosed. But it is a robust development plan.

There'll be a condensing of time and on many things that at blueprint, we will likely would have done sequentially. We're now there's the opportunity to do several things simultaneously.

But you.

Wouldn't need to work through those with our partner and then in terms of when and how we'll update that you got that will also flow through the collaboration in terms of how much is disclosed and when that disclose but is it a robust development plan.

Jeff Albers: Got it. And then maybe one final question for me. You know, I think the pipeline, the upcoming pipeline gets overlooked a lot, and there's a lot

Got it and then just maybe one final question for me.

I think the pipeline the upcoming pipeline and gets overlooked a lot and.

Reni John Benjamin: There's a lot going on here, could you just...

There's a lot has.

Reni John Benjamin: Remind us, I think, you know, the last set of my notes has eight programs, you know, that are underway, four of which are wholly owned. Can you maybe just help us focus on what are the key ones? Obviously, we have the data coming out for 945, the preclinical data, but what other programs that you think are important that we should be keeping on our radar screen?

Just remind us I think.

Last sort of my notes have like eight programs.

Our underway for which are wholly owned can you maybe just help us focus on on what are the key ones I've. Obviously, we have the data coming out for nine four or five the preclinical data, but the other programs that you think or or employees that we should be keeping on our leaders.

Jeff Albers: Sure. So, I would combine the two EGFR resistance programs that we disclosed at R&D Day last year, 945 being the triple mutant, and then we have a double mutation program that would be one that focused on the on-target resistance that would emerge as Osta-Mertinib moves into a first-line setting. So, those are two. We're guided to having up to three development candidates by the end of the year. So, Blue 945 is one of those.

Sure.

So yeah I would.

Combined the two Egypt bar resistance programs that we disclose a at R&D day last year, nine four or five being the triple amusing and then we have a a double mutation a program that that would be one that.

Focusing on the are on target resistance that would emerge as somerton it moves into first line setting.

Those are two we guided to having up to three development candidate by the end of the year.

Jeff Albers: So, there are two other programs that have made very nice progress, and we're shooting for having development candidates, and as that occurs, we will provide updates thereafter. Another bucket is our cancer immunotherapy collaboration with Roche. So, we have highlighted MAP4K1 as one of the programs in that collaboration that continues to make nice progress, and there are additional programs that remain undisclosed there. And then there's a grouping of additional undisclosed programs at various stages in pre-clinical development.

So blue nine for five is one of those so theres two other programs that have made very nice progress up and that we're shooting for having development candidates and as that occurs we will provide updates thereafter.

Another bucket is our cancer immunotherapy collaboration with Roche I'm. So we had highlighted map for K. One is one of the programs in that collaboration that that continues to make nice progress and there are additional programs to remain undisclosed there and then there's a grouping.

Additional undisclosed programs at various stages, a in preclinical development and so when I took all that together I, we fundamentally agree.

Jeff Albers: And so, if I took all that together, we fundamentally agree that it's an area that's overlooked, and we appreciate why there's a lot of excitement with our lead programs. But when I think about this moment in time, and we talked a bit about this when we announced the collaboration, Hoover Medicine is in the strongest position it's ever been in. I think it's often overlooked how difficult it can be for new biotechs to build a fully integrated business. But we now have that capability.

That is it's an area that's overlooking we I appreciate why that there's a lot of excitement with our lead programs, but it when when I think about this moment in time, and we talked a bit about this when we announced the collaboration with her medicines is the strongest physician it's ever been yeah, I think oftentimes it.

Overlooked how difficult it can be for new biotech to build a fully integrated business. We now have that capability. So it was that Christy leading those commercial efforts and generate a lot of the questions. This morning out on the front end I can take US a team that is chomping at the bad bid to get moving with proud that live in earnest stopping pending.

Jeff Albers: So, we've got Christy leading those commercial efforts that have generated a lot of questions this morning on the front end. I can tell you it's a team that is chomping at the bit to get moving with Pralsetnib in earnest, pending regulatory feedback. We've now strengthened that with a partner that we consider to be one of the best when it comes to targeted therapies, certainly supplementing our efforts in the U.S., but then globally, bringing Pralsetnib to more patients globally in a faster time frame. That collaboration now allows us to focus more of our energy on systemic mastocytosis, which we think is the biggest driver of long-term value for patients and for us. And then it has also freed up resources for us to really focus on what we think is an underappreciated and promising pipeline. You don't get many moments like that where all three of those factors can come together simultaneously and be backed by a strong financial position. So, you know, I think it's up to us now to convince you that there is value in that pipeline. But I think we've got a plan to.

Regulatory feedback we've now strengthen that win with a partner who that we consider to be one of the past when it comes a targeted therapies.

Yeah, certainly supplementing our efforts in the U.S., but then globally, bringing problem that have to more patients globally in emphatic faster timeframe.

That's collaboration that allows us to focus more of our energy on systemic mastocytosis, which we think is the biggest driver in long term value for patients and for for US and then it has also that freed up resources first it really to focus on what we think is eight underappreciated promising pipeline.

You don't get many moments like that with all three of those factors can come together simultaneously.

Backed by a strong financial position. So I know I think it's up to US now to to convince you that there is value in that pipeline.

But I think we've got a plan to methodically roll that out over the coming years and there's a lot to be excited about in that pipeline.

Jeff Albers: Synthetically roll that

Jeff Albers: over the coming years, and there's a lot to be excited about in that pipeline.

Jeff Albers: Great. Thanks for taking the questions and for asking them. Thank you.

Great. Thanks for taking the questions and right.

Thank you.

George Former: Your next question comes from the line of George Former with BMO Capital Markets.

Your next question comes from the line of at Georgia, former with <unk> capital markets.

George Former: Hi, good morning. Thanks for taking my question. Could you guys remind us how much follow-up data you'll have in the advanced SM setting when you ultimately present the data and file with FDA? And also, could you comment on how your patients are doing in Part 1 of the Explorer trial? Have they all moved on to long-term extension? And what sort of data do you need from that in order to secure approvals on both indications?

Hi, good morning, Thanks for taking my question.

Could you remind us how much follow up data you'll have in the advanced.

FM setting when you ultimately presented data and file with the FDA and also could you comment.

On how your patience are doing and part one of the explore trial have they all moved onto long term extension and what sort of data do you need from that in order to secure approvals on both indications.

George Former: So maybe I'll start with that, and then Andy can chime in. So in terms of the additional follow-up, it'll be a blend of a couple of things. So one thing is we provided top-line data late last year, so just as a function of time for patients who had already been enrolled, you'd expect it to be somewhere in the six to nine months of additional follow-up. Of course, there are also new patients that we've not shared data on, so they'll, by definition, have less follow-up. Many of them will have been enrolled late last year. And I can't remember. I think that, from a regulatory perspective, all of those patients had been enrolled when we provided that top line. So they would have all been enrolled as of last year. So let's follow up on an IWG criteria that really matters. Because a part of the definition of response is a component of time, of how long they've been in response. And then we'll blend it together in terms of the second part of the question, which is...

So maybe I'll start with then Andy.

In China so.

In terms of the additional follow up.

So it'll be a blend of a couple of diesel one is we have provided topline data.

Late last year, so just a function of time or patient twin already been enrolled you expected to be somewhere in the six to nine months of additional follow.

Of course, there's also do patients on that Weve not sure data on those so now my definition has less follow up.

Many of them will happen.

Enroll.

Okay late last last year.

And I can't remember that I think they think the enrollment for from a regulatory perspective all of those patients have been enrolled when we provided that top line. So they would have all been enrolled as of last year.

So let's follow up from an eye WG criteria that really matters because a part of the definition of response is a component that uptime of how long they've been in response.

And then well well blended together in terms of the second part of the question which is.

Jeff Albers: Actually, maybe you're talking about Pioneer of the Immune Study there.

My question.

Or maybe we're trying to pay you lose on either so.

Andrew Scott Berens: http://thevenusproject.com Those patients have all gone into what's called Part 3 of the study that we discussed, which is a long-term follow-up, and we haven't given specific data from that part of the study yet, but they continue to do well. Okay.

Hi, pioneer yeah, Delia since it is for that that.

Those patients all go we've had gone into whats called part three of the study that we do test, which is a long term follow on Tonight and there.

Doesn't given specific data from that from that some part of the study yet, but they continue to do well.

George Former: Okay, great. Good. And then I have a question for Mike.

Okay great.

Good and then.

Michael Landsittel: How are you thinking about accounting for the upfront payment from Roche? How should we incorporate that into our models? Yeah, sure. We're working through finalizing the accounting with our auditors, but we expect that we will recognize a majority of the $675 million upfront payment as revenue in the third quarter. And then we also anticipate, if you recall, there was a $100 million equity investment; a majority of that will be recorded within stockholders equity on our balance sheet. And then we'll certainly provide kind of a further update next quarter, but that's what we're anticipating at this point. Okay, great. And can you and can you comment as to whether a milestone will be coming on with approval or filing of the PALSETNIV application?

Question for Mike how are you thinking about accounting for the upfront payment from Roche, how should we incorporate that into our models.

Yes, sure. So we're working through finalizing the accounting with our auditors, but we expect that we will recognize a majority of the 675 million dollar upfront payment as revenue in the third quarter.

And then we also anticipate if you recall there was 100 million dollar equity investment a majority of that.

Recorded within stockholders' equity on our balance sheet and then we'll certainly provide kind of a.

Further update next quarter, but that that's what we're sitting at this point, okay. Great and can you can you comment as to whether a milestone will be coming on on approval or filing of the past setting that aside on approval the process.

Applications.

Michael Landsittel: Yeah, we haven't provided detail on the specifics of the milestones, but I think, if you recall, what we said at the announcement of the deal is that there's a potential for $90 million in near-term marketing and approval related milestones. And so, you know, certainly we're excited about what's to come, and I'll probably just leave it at that. Okay, thanks very much.

Yes, we haven't provided detail on the specifics of the milestone, but I think if you recall, what we said at the announcement at the deal is that there's a potential for $90 million in near term.

Marketing approval related milestones and so.

Certainly we're excited about what's the car middle probably just leave it at that.

Okay. Thanks very much.

[noise] question comes from the line.

Michael Landsittel: Your next question comes from the line of Eun Yang with Jeffreys.

<unk> with Jefferies.

Eun Yang: Hi, this is Sarah on behalf of you, and thanks for taking our question.

Hi, This is there on for years, thanks for taking your questions.

Sarah: I touched on this earlier in the presentation. I was just wondering...

You touched on this earlier in the presentation I was just wondering if.

Sarah: Um, how has the commercialization for advanced...

How the commercialization for advanced Us I'm with what are they mainly treated in the academic setting I. You said there was some overlap with can you provide more color on those and would you need to expand your sales force.

Unknown Attendee: Transcription by Transcription Outsourcing, LLC.

Philina Lee: I'm going to ask you to describe more color on this, and will you need to expand your sales force?

Philina Lee: Sure. Christy?

[noise] sure Christie.

Christina Rossi: Sure. So, you know, there certainly is overlap with particularly advanced SM, which tends to be treated primarily at sort of oncology, hematology centers of excellence, and academic centers. The specifics around the stakeholders involved in treatment are obviously, you know, frequently different than what we see for lung or thyroid.

Sure so.

Hi, there certainly is overlap with particularly advance that down which tends to be treated primarily.

Sort of oncology hematology centers of excellence academic centers.

The specifics around the stakeholders involved in treatment are obviously frequently France, a somewhat we see for for a lot more side writer Jeff.

Christina Rossi: We have not yet decided exactly what the commercial structure will look like for SM. However, you know, we've definitely taken, as I said, a portfolio approach to thinking about our structure. And we certainly have some roles that are already engaging in activities to prepare for an advanced SM launch. In particular, in the U.S., we have a precision medicine team that really plays a role across the portfolio and, in fact, has been primarily focused on RET and SM since the beginning of this year. So, you know, more to come on that. Advanced SM is certainly a very targeted market. We expect to be able to address it nimbly, as we have done to date. And then, you know, we'll look to how we may evolve as we approach end-to-end SM approval down the road.

We have not that it yet you exactly what that commercial structure will look like for however, you know we definitely tigana. They set of portfolio approach to thinking about our structure and we certainly have some roles that rd engaging in activities to prepare for.

Launch and in particular, the U.S. and you have a precision medicine seem that really plays a role across the portfolio and in fact has been primarily focused on.

I'd read.

Since the beginning of this year. So you know what at more to come on that and advance ascent is certainly a very targeted market, we expect to be able to address that nimbly as we have today and then we'll look to highly may evolve as we as we approach.

Approvals on grass.

Philina Lee: Okay, thank you.

Okay. Thank you.

Your next question comes from the line Arlinda Lee with Canaccord.

Operator: Your next question comes from the line of Orlinda Lee with Canaccord. Hi guys, I have a couple of questions. Christy, you talked about being encouraged by retail rates.

Hi, guys.

Question Kristi you talked about.

Philina Lee: That's the real world.

Being encouraged by refill rate.

Unknown Attendee: Transcription by Transcription Outsourcing, LLC, in your clinical trial, but you also said that U.S. may not have, you know, patients tend not to be going on abrupritinib at first diagnosis. So I'm kind of curious if you could provide any additional color on the basis of those statements. And then maybe my second question would be on the additional nominated candidates for the remainder of the year. Would you mind, please, outlining the criteria and prioritization of how you think about your current product in Salesforce and how that impacts your criteria of prioritization for your candidate?

That.

The real world.

Durability.

What we've seen.

And your clinical trials, but you also said that U.S. may not have seen a patients tend not to be.

Going on it represented.

First diagnosis, so I'm kind of curious if you could provide any additional color on the beach.

And then maybe second that's my second question would be on the additional nominee candidate for the remainder of the year.

Would you mind lease outlining the criteria and prioritization of how you think about.

Your current products and Salesforce and how that impacts your criteria.

Certainly.

Okay.

Philina Lee: Thank you. Great.

[noise] Christianity, no I think the second see [laughter] right [laughter].

Jeff Albers: So, as I said, I have been really encouraged to see that patients who have started on AvaKit tend to be staying on therapy and refilling on schedule. And that comment, you know, speaks to a few things. First of all, certainly we're not seeing a negative impact of COVID on those metrics.

No.

I think that we I am I have been very encouraged to see a set piece and you have started on it I can't tend to be staying on therapy, and we feel like on schedule and that comments you know I think speakeasy. Thanks first of all you know certainly we're not seeing negative impact of of coal bed on those metric, we don't see a point have perfect visibility.

Christina Rossi: You know, we don't, to your point, have perfect visibility into, you know, a patient level sort of clinical profile, but based on what we can understand and sort of looking at the mix of patients, we see PDGF-alpha as well as patients who are sort of more late-line kit patients. We've been encouraged to see patients refilling, you know, beyond what we actually expected and I think what I attribute that to is, you know, the real world benefit risk and tolerability profile of AvaKit. Patient management has been, you know, really a key focus of our team and, you know, we see that prescribers are individualizing approaches to dosing with patients and certainly, you know, you know, modifying patient dose to make sure that it's appropriate for the clinical characteristics of the patients that they're treating and we see that that really leads to increased persistence and endurance and so, you know, for me, that's a great sign early on in this launch and I also think it speaks well as we think about the profile of AvaPrint and more broadly as we move forward to potential approvals in NASM.

You know a piece that level sort of a clinical profile that based on what was.

Mr looking at and the mix of patients CCP, Jeff Ralph as well as as patients were sort of more lately kept pace than we've been encouraged to see patients reselling as you know.

What we actually expect that and I think what I attribute that to yell at is you know that the real world benefit risk and Tolerability profile of cats and patient management has been really a key focus of our of our team and we see that prescribers are.

Our individualized they approaches to dosing patients.

Certainly you know.

Modifying patient dose to make sure that is appropriate for the clinical characteristics of the patient that they're treating and we see that that really the Q and increased persistence and addresses though you know for me that's a great sign early on and this last and I also think it speaks well if you think about the profile of they've been pretty broadly asked me the forward keeps constructive.

Christina Rossi: Hi, this is Jeff, and I'm looking at the We're at the bottom of the hour, so it was a broad question that would take more time to address, but it's obviously multifactorial in terms of how we select new targets, but one of them, fundamentally, that's been a cornerstone is targets where we believe there's a monogenic driver and one where we ought to be able to get to clinical proof of concept And what that then has transformed into is a portfolio approach that we highlighted at the R&D day where there are several lung targeted programs with Pralosetamib and EGFR programs, there are hematology opportunities that will grow or build from what we do with systemic mass cytosis, and then it continues to be targeted in rare genetic diseases. So those are all reflected in our early discovery platform just as they are with our more advanced.

Okay.

That's.

Hi, This is Jeff and I'm looking at that we're at the bottom of the hours. So it was a broad question that would take more time to address but it's obviously multifactorial in terms of how we select new targets, but one of them fundamentally that's been a cornerstone is targets, where we believe there's a monogenic driver.

And one where we ought to be able to get to clinical proof of concept relatively quickly based on the chemical matter, we have enough and.

Our library and what that then as Phil trance form into is a portfolio approach that we highlighted the R&D day, where there are several lung targeted type programs with profit in Indonesia Bar program there their team hematology opportunities.

That will grow or build from what we deal with systemic mastocytosis and the continued to be targeted rare genetic diseases. So those are all reflected in our early discovery platform and just the they are without more advanced programs.

Jeff Albers: So with that, I think we need to conclude the call. Operator. Operator

With that I mean, I think we need to to conclude the call.

Operator.

Operator: At this time, I would like to turn the call back over to Mr. Jeff Albert for his closing remarks.

It sounds like to turn the call back over to Mr., Jeff outbreaks for closing remarks.

Jeff Albers: I love that. I turned it over to you to turn it right back to me. So thank you, and thanks to everyone for taking the time and joining us today and for your continued support of Blueprint Medicines. And it's going to be a busy second half of the year, so we look forward to updating you again soon and hope you all stay well. Goodbye.

Well I'll, then turn it over to turn every that can be so thank you and thanks, everyone for taking the tide and joining us today and for your continued support a blueprint medicines.

And it's going to be a busy second half of the air. So we look forward to updating you again soon and hope you all stay well.

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

Today's conference call you may now disconnect.

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