Kristin Hodous – Investor Relations Jeff Albers – Chief Executive Officer Andy Boral – Chief Medical Officer Mike Landsittel – Vice President-Finance Marion Dorsch – Chief Scientific Officer.

Terence Flynn – Goldman Sachs Eric Schmidt – Cowen and Company Chris Raymond – Raymond James Carmen Augustine – Jefferies Mike King – JMP Securities David Nierengarten – Wedbush Arlinda Lee – Canaccord.

Good morning. My name is James and I will be your conference operator today. At this time, I’d like to welcome everyone to the 2017 Second Quarter Blueprint Medicines Corporate Earnings Conference Call. All lines have been placed on mute to prevent any background noise. After the speakers’ remarks, there will be a question-and-answer session.

[Operator Instructions] Thank you. Kristin Hodous, you may begin your conference..

Thank you, operator. Good morning. This is Kristin Hodous of Blueprint Medicines and welcome to Blueprint Medicines second quarter 2017 financial and operating results conference call. This morning we issued a press release which outlines the topics that we plan to discuss today.

The release is available on the Investor Section of our website at www.blueprintmedicines.com. Today on our call, Jeff Albers, Chief Executive Officer, will discuss Blueprint Medicines’ second quarter 2017 business highlights. Andy Boral, Chief Medical Officer will review our clinical updates.

And Mike Landsittel, Vice President of Finance, will review our second quarter financial results. We will then open the call for your questions. 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 factor including those set forth in the Risk Factor 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 dates. We specifically disclaim any obligations to update or revise any forward-looking statements. Now, here is our CEO, Jeff Albers..

Thanks, Kristin and good morning everyone. Blueprint Medicines had a very productive second quarter and we’re pleased to share recent progress with you today.

We took the last several years building a pipeline of highly targeted kinase medicines for patients with genomically defined diseases with the goal of bringing into the clinic highly selective and potent drug candidates with less off-target activity and a higher probability of clinical success.

Through these efforts we generated promising early clinical data for our BLU-285 and BLU-554 programs. We received Breakthrough Therapy Designation an FDA feedback for BLU-285 and PDGFR alpha-driven gastrointestinal stromal tumors or GIST. And we recently initiated a clinical trial of our innovative RET inhibitor BLU-667 in RET ultra cancers.

Today our line of study extends toward potentially delivering an important new medicine to patients with advanced GIST and becoming a commercial space company. We think this is a tremendous achievement for a company that began operations only six years ago.

I often tell our employees that success simply give us the right responsibility to do more and work harder. And that’s exactly what we’re doing as we enter the second half of 2017. Earlier this year we laid out our key goals for 2017.

These included one, advancing BLU-285 and BLU-554 clinical trials towards critical data disclosures; two, defining the clinical and regulatory path forward for these drug candidates; three, maximizing the value of our broad research platform; and four, evaluating strategic business development opportunities.

At mid-year and part of the progress we’ve achieved and feel very good about our momentum going forward. At the ASCO Annual Meeting in June, we presented updated data from our ongoing Phase 1 trial of BLU-285 and patients with advanced GIST.

The results demonstrated compelling clinical activity in two distinct patient populations, patients with PDGFR alpha-driven GIST and patients with KIT-driven GIST.

And today we announced that our second clinical data disclosure will come on September 10 at the 2017 ESMO Congress, where we’ll present updated data from our ongoing Phase 1 trial of BlU-554 in patients with advanced hepatocellular carcinoma or HCC.

We also announced today that we initiated enrollment in the expansion portion of our Phase 1 clinical trial evaluating BLU-285 in patients with advanced systemic mastocytosis. We expect to disclose updated data from this trial by year-end, continuing the cadence of data readouts from our ongoing clinical programs.

Following this successful end of Phase 1 meeting with FDA earlier this year, we were pleased to receive favorable agency feedback on our proposed registration plan for BLU-285 in advanced GIST.

Similarly we plan to engage regulatory authorities on a range of options for further development of BLU-285 and systemic mastocytosis and BLU-554 and HCC in the second half of 2017 and into 2018.

Across all of our programs we aim to define clinical and regulatory strategies to bring new medicines to patients as quickly as possible based on clinical data, feedback from regulatory authorities and input from clinical experts. With respect to our research programs, we continue to maximize the value of our scientific platform.

As I mentioned, earlier this year we advanced our third drug candidate BLU-667 into the clinic. In addition we continue to advance a range of preclinical programs both in collaboration with Roche and on our own. As you know last week Alexion notified us of their decision to exit our research collaboration following a strategic portfolio review.

Alexion indicated that their decision was solely based on refocusing their R&D strategy and certain core therapy to get areas. As a result we have an interesting and unexpected opportunity ahead of us.

Under the collaboration we were evaluating multiple compounds in preclinical development for the treatment of Fibrodysplasia Ossificans Progressiva or FOP. FOP is a severe – also a rare disorder of the connective tissue characterized by the abnormal transformation of skeletal muscle, ligaments and tendons in the bone.

Our research is focused on innovation of mutated forms of the EIF-2 kinase, which is believed to be the underlying cause of the disease. Moving forward we plan to explore opportunities to advance the FOP program on our own based on the extensive discovery in preclinical work completed today.

We also feel an obligation to the FOP patient community to take a very close look at potential option for this program. Our first step in this process will be to coordinate in orderly transition of the program with Alexion over the coming months.

As we evaluate opportunities to advance the FOP program, we’ll stay disciplined in managing our overall clinical and research portfolio. Across our pipeline, we consistently focus on opportunities to transform patient care and pursue expedited development path.

We also recognize strategic business development plays an important role in discipline portfolio management. And we continue to evaluate potential partnership opportunity that may allow us to accelerate our programs or expand our global reach.

Overall we’re extremely pleased with our year-to-date accomplishments and look forward to a busy and productive second half of 2017. With that I’ll now turn the call over to Andy Boral to update you on the progress of the clinical trials..

Thanks, Jeff. We’re very excited by our clinical progress encouraged by the results that continue to emerge from our trials. This morning, I’ll briefly review each of our clinical programs including recent progress.

First, we’re really pleased to have a compelling clinical data and a clear path forward for our FIT and PDGFR alpha inhibitor BLU-285 in advanced GIST.

At the ASCO Annual Meeting in June, we presented new clinical data from the ongoing Phase 1 trial of BLU-285 in two patient populations, patients with PDGFRa D842-driven GIST and patients with KIT-driven GIST.

So across both populations BLU-285 continues to be well tolerated with great adverse events with treatment related observed in only 25% of patients. The clinical activity in patients with PDGFRa D842-driven GIST were remarkable.

The data showed an objective response rate of about 60% by RECIST criteria and a nine month median progression free survival of 87%. Based on these very encouraging data the FDA granted Breakthrough Therapy Designation to BLU-285 for the treatment of unresectable or metastatic PDGFRa D842V-driven GIST.

Moving forward, we plan to pursue expedited approval in this population based on additional data from the Phase 1 trial. We currently expect to complete enrollment of the PDGFRa D842V expansion cohort by the middle of 2018.

In patients with heavily pretreated KIT-driven GIST the clinical data showed very encouraging dose dependent activity with tumor regression and prolonged progression free survival in patients treated with the higher doses of BLU-285.

This was in the clinical setting where few patients respond and the expected progression free survival with available therapies is less than two months. To advance development of BLU-285 in this population, we plan to initiate a global randomized Phase 3 trial of BLU-285 compared to regorafenib in third line GIST in the first half of 2018.

Overall this dual approach it was the opportunity to quickly address an important unmet medical need in PDGFRalpha D842V-driven GIST, while also pursuing approval in the largest population.

We are planning additional regulatory meetings with FDA and EU help authorities over the course of this year to continue to refine the regulatory path of BLU-285 in GIST. We’re also evaluating BLU-285 in advanced systemic mastocytosis.

Preliminary Phase 1 data for BLU-285 in SM were presented at the ASH meeting last December and showed mark decreases and objective measures of mast cell burden and serum tryptase as well as improvements in patient symptoms from the lowest dose levels tested.

These data demonstrated the targeted – the KIT D816V genetic driver with a highly potent and selective inhibitor like BLU-285 has the potential to address not only the symptoms of the disease but also underlying cause.

We’re happy to announce today that we recently initiated the dose expansion part of the trial on enrolling patients to the recommended part two dose of 300 milligrams once-daily.

Based on the activity of BLU-285 in advanced SM even at the lowest dose levels and feedback from our clinical advisors we also see an important opportunity for BLU-285 in patients with indolent systemic mastocytosis. Indolent SM is a non-malignant mast cell disease for patients of normal life expectancy but a plethora of debilitating symptoms.

We’re working closely with our clinical advisors to outline a path forward for BLU-285 in this patient population.

Later this year we plan to share additional data from our ongoing trial BLU-285 in advanced SM including advanced assessment of response rate using the IWG criteria, which is now recognizes a valid regulatory endpoint with the recent approval of Midostaurin.

Our second product candidate BLU-554 is in development as a potential treatment for patients with advanced HCC. As you all know the treatment options for HCC are limited and the prognosis for patients is generally very poor. The only approved therapies for HCC are sorafenib and regorafenib which provide overall response rate of 2% and 7% respectively.

Approximately 30% of HCC cases are thought to be driven by FGFR4 pathway activation. To target this disease driver and avoid off-target effect associated with other members of the FGFR family we design BLU-554 an exclusively selective FGFR4 inhibitor. This approach represents the first biomarker directed treatment strategy in HCC.

Last November at the Triple Meeting, we presented early clinical data to demonstrate it antitumor activity for BLU-554 in biomarker positive patients. Based on these data we increased the size of biomarker positive expansion cohorts in the ongoing Phase 1 trial.

At the upcoming ESMO 2017 Congress, we plan to present updated data from the dose escalation part of the Phase 1 trial as well as expansion data for patients with sufficient follow-up. Our ESMO abstract included results of the data cut-off of April 20, 2017. And consistent with prior presentations we’ll present more recent results at ESMO.

In the ESMO data set we expect to have additional information to help determine of our patient flection strategy for FGFR4 pathway activation is effective and to better understand the clinical activity of BLU-554 in this very severe rapidly progressive disease.

Finally, we continue to make good progress advancing our third product candidate, BLU-667 in the clinic. BLU-667 was specifically designed to selectively inhibit RET mutations and fusions including predicted resistance mutations, with a goal of providing more durable clinical benefit to patients with RET ultra cancers.

Earlier this year, we initiated a Phase 1 trial of BLU-667 in patients with non-small cell lung cancer, medullary thyroid cancer and other advanced solid tumors harboring a RET alteration. In the second quarter, we opened additional clinical sites to expand enrollment of patients in the dose escalation part of the trial.

Overall we’re pleased with our progress to date and enthusiasm of investigators and patients to participate in the trial. We anticipate providing more robust updates on this clinical trial in the first half of 2018.

As you can see, we’re all staying very busy here at Blueprint Medicine and we’ll look forward to updating you on the progress across all of our clinical trials in the future. With that, I’ll turn the call over to Mike to review our financial results for the second quarter..

Thanks Andy. Today we are in a strong financial position to support the significant activity that Jeff and Andy just outlined. Let me start by walking you through the second quarter P&L. Collaboration revenues were $5.9 million for the second quarter of 2017, compared to $7.1 million for the same period in 2016.

This decrease was primarily due to lower revenue recognized under our collaboration with Alexion.

As a result of the discontinuation of our collaboration with Alexion, we won’t be entitled to receive any additional milestone payments and upon effectiveness of the termination of the agreement will no longer be entitled to reimbursement by Alexion for certain R&D expenses related to the program.

Research and development expenses were $33.3 million for the second quarter of 2017, compared to $21.3 million for the same period last year.

The increase in R&D spending was primarily attributable to increased clinical and manufacturing expenses associated with the ongoing trials for BLU-285, BLU-554 and BLU-667, as well as increased personnel related expenses including stock-based compensation expense.

General and administrative expenses were $6.8 million for the second quarter of 2017, compared to $4.7 million for the second quarter of 2016. The increase in G&A spending is primarily due to increased personnel related expenses, which includes stock-based compensation expense.

Net loss for the second quarter of 2017 was $33.4 million or $0.86 per share compared to a net loss of $18.9 million or $0.70 per share for the same period last year. Now turning to the balance sheet and our cash guidance.

We ended the quarter with cash, cash equivalents and investments totaling $421 million, compared to $268.2 million as of December 31, 2016. The increase in cash was primarily due to the net proceeds of $215.6 million from our follow-on offering that closed in April 2017, offset by cash used to fund operations.

Based on our current plans, we continue to expect that our existing cash, cash equivalents and investments excluding any potential option fees and milestone payments under our existing collaboration with Roche, will enable us to fund operating expenses and capital expenditure requirements into the second half of 2019.

As Jeff mentioned earlier on the call, our plans also include evaluating opportunities to advance the pre-clinical program that was the subject of our collaboration with Alexion. Importantly, now that we have defined the regulatory path for BLU-285 in advanced GIST, we are beginning to ramp up clinical development and early pre-commercial activities.

For example, we are beginning to build infrastructure to support the planned global Phase 3 trial in third line GIST and expand CMC activities to provide clinical and commercial drug supply including commercial validation batches for BLU-285.

Longer-term commercial planning is underway and we expect to provide further clarity on expected operating expenses in the future. With that, we would now like to open up the call for your questions.

Operator?.

[Operator Instructions] Your first question comes from the line of Terence Flynn from Goldman Sachs. Go ahead please. Your line is open..

Hi. Thanks taking the question and congrats on the progress this year. I was just wondering what you guys are hoping to see from the 554 Phase 1 expansion trial here to move into the Phase 3 program.

Is it really just a better response rate given – you laid out the 2% to 7% with prior drugs? Or is it something with respect to durability? Maybe you could just help frame for us how to think about that decision to move into Phase 3. Thanks a lot..

Terence thanks. This is Andy, I’ll take the question. So as I said at the beginning on – when I was presenting, the outcome of these patients is very poor with available therapies. As I mentioned just less than 10% of response rate with both sorafenib and the other approved therapy regorafenib. And very short progression free survivals.

What we’re really looking for in this study are two things primarily. One is to fully understand how to best select the patient population. So we are selecting the patients for FGFR4 pathway activation with our immunohistochemistry test for FGF19.

And we think a very important part of this study will be to evaluate on activity primarily response in tumor shrinkage according to degree of FGF19 positivity.

But we’re also interested in looking at degree of prior therapies, the patients in our study have had multiple prior therapies often, geographies, etiology and so that’s one important piece for the next study how would we select those patients to optimize activity.

And the second of course is to understand the degree of activity in this study that would be mostly measured by tumor shrinkage and response. And as we pull all those data together, we’ll make a decision on the best next step for development..

Your next question comes from the line of Eric Schmidt from Cowen and Company. Go ahead please. Your line is open..

Thanks. Congrats on the steady progress. Maybe another question from me for Andy on BLU-554 in HCC. Are we going to get – in terms of the number of patients maybe give us a little bit of color there? But are we also going to get all three dose expansion cohorts and all three kind of nearing full enrollment at 45 patients each..

Thanks, Eric. So I’ll take that as all to Andy. I assume you’re not talking about the ESMO presentation, there the abstract which will come out August 30, you’ll get much more information there. And the presentation will include data on essentially the entire dose escalation part of the study.

And then the dose expansion part for patients whom we have sufficient follow-up. So we’ll be a subset of the expansion part of the patients. The study is enrolling well, but a lot of those patients have come on relatively recently and so that would limit the amount of data from expansion..

And at the time of ESMO, will you guys be in position to kind of give us an update on your development plans for molecule?.

I think, our focus really as I said before is going to be an understanding and describing the effectiveness of the patient selection process, seeing if there are correlations with other predictors of response and then summarizing the data. At this point, I think it may be premature to layout the development program at that time.

We do look forward to having more data from that program over the course of the year before we’re ready to get into the development plan in detail..

Okay. And then maybe just a quick one on the RET inhibitor.

What’s your best guess of when we’ll see the first clinical data there?.

So we’re expecting to – so maybe I’ll start just by saying the study is ongoing, enrolling well, the investigators have been enthusiastic. Our plan though is to present data in the first half of next year at this point. We’d like to have a good – maybe we’d like to have a good story to tell at the time, we present the data..

Thanks, congrats again..

Thanks a lot..

Your next question comes from the line of Chris Raymond from Raymond James. Go ahead please your line is open..

Hey, thanks. Just a question BLU-285 and you’re dosing in GIST. I think, last I heard you guys talk about dose at ASCO, you mention the sweet spots between 300 milligrams and 400 milligrams, with 400 milligrams being the MTD.

But I don’t know that you guys have said that you’ve really hit on the dose but with the KIT Phase 3 starting in the first half I think you said next year and you are obviously also going forward the expansion cohort for PDGFRa.

Can you talk about that in terms of hitting on dose and maybe how those conversations with FDA are going?.

Chris thanks. This is Andy, I’ll take the question. So we did define the MTD of BLU-285 in GIST as 300 milligrams – actually 400 milligrams a day.

Something that we think is a great opportunity for this program where we have a relatively large expansion experience are evolving in GIST in 285 with both our KIT-driven population and PDGFR alpha-driven patient population, each be 50 patients.

As well as the ongoing systemic mastocytosis study, we have an opportunity to really explore dose a bit and figure out what is the best dose for start and to start-off and that will also support long-term therapy with a good balance of maximizing activity and maintaining a good safety profile.

So the MTD is 400 milligrams and we have been – we’re exploring anywhere between 300 milligrams and 400 milligrams. We have decided in the mastocytosis study to proceed with a dose of 300 milligrams because after discussing review of the data that seemed to give us the best balance of activity and long-term tolerability.

We very well may end up at the same dose in GIST the 300 milligrams with the option to explore 400 milligrams in the future..

So you might have titration protocol, I guess that’s great..

Yes. So one thing, imatinib is taken that I think it has been reached pretty successful is to start – if you look at the label that you start at 400 milligrams, if patients tolerate you can actually escalate to 600 milligrams to 800 milligrams. And I think that kind of flexibility has turned out to be very effective.

So we’re considering something along those lines. But the maximum tolerated dose would 400 milligrams. And the question is, we’re in the range of 300 milligrams to 400 milligrams do we start and do we allow interpretation escalation..

Okay. And then on the FOP molecule that you got back from Alexion. Maybe talk a little bit about your appetite. I know you’re taking this forward alone but you have another competitor there with Regeneron starting efforts with their own approach.

So you at least have another player trying to recruit patients et cetera, which is always from a competitive standpoint a challenge. Just maybe talk about your thoughts there, do you need to re-partner this or from your lay of the land is this something that can easily be done internally..

Thanks Chris. This is Jeff. Maybe I’ll start and then I’ll ask Marion Dorsch our key Chief Scientific Officer to talk a little bit about the mechanism. So I think it’s a great question, we were surprised to have Alexion step away from the collaboration given the nice progress.

And so our first order of business is just to ensure a smooth transition of all the information that we’ve collected to date preclinically as well as the work that’s been done within Alexion. So I think that’s regardless of what we think more broadly where our focus is now.

And then as I alluded to from a portfolio perspective what we’ll look at over the course of the remainder of this year is, one where does the science lead us in terms of understanding our molecules, the quality of those, our confidence in a path forward.

Two, reevaluation of the broader opportunity and I think that includes competitive landscape certainly as with all programs that evolves as you move forward through preclinical development and this doesn’t seem to be an exception. That said, it’s a pretty rare opportunity to have a program you’re excited about fall back to you in this manner.

So there’s a – because of the mechanism and the fact that L2 may in fact be a driver of disease. There’s a lot of interest here to figure out what’s the best path to bring this full program to patients. So maybe Marion add a little color just in terms of mechanism and competitive landscape..

Yes. This is Marion. As Jeff mentioned, I think we are very excited to explore the opportunity to potentially advance that on our own. And we feel that the mechanism that we’re targeting and our molecules are designed to inhibit – up to which we believe is the driver for that disease.

So we feel that our mechanism is really going after that underlying heart of that disease and provides really a great opportunity to have – to make a major difference in that very severe rare genetic disease.

So as we move along, I mean obviously – they are as you mentioned Regeneron is out there, their molecule is targeting one of the ligands for October. And so it’s a different approach and I mean hopefully in the end I mean one of those would make a difference for patients.

But we think that going after the mutation and the real generic driver it’s a very, very promising mechanism..

Thanks..

Your next question comes from the line of Carmen Augustine from Jefferies. Go ahead please your line is open..

Hi. Thanks for taking the question. So for BLU-285, we have expansion enrolling now at 300 milligrams in mastocytosis and then you’re looking at somewhere between 300 milligrams to 400 milligrams in GIST.

Could you speak to pricing strategies in each indication in the context of their relative sizes and pricing of approved drugs?.

Sure. So this is Jeff I’ll take that question. I think at this point it’s we’re way too early in our clinical development to speculate on price. I often maintain that. By the time we move these two potential commercialization, the landscape will now likelihood of change in some manner.

But I’m a firm believer that if you have a therapy that is targeting a driver of disease and you have clinical data that supports if you’re having a major impact that type of innovation will support premium pricing.

And I think we’ve been somewhat fortunate when you look at systemic mastocytosis in GIST that active doses have paralleled one another relatively closely.

Maybe taking that one step further, it was the activity into advanced systemic mastocytosis at a very low dose, which really opened our eyes and increased our enthusiasm around moving forward with clinical development in indolent systemic mastocytosis.

So they’re having a differential in dose may provide us with real pricing flexibility as we take one molecule forward in multiple different disease stage. So, the short answer, too early to speculate but two, the clinical data is guiding us in the direction that we think we’re very fortunate..

Okay. Thanks for the color. And then one more on 285 in SM if I could.

Are you expecting that data could be presented at ASH and anecdotally have you noticed any changes in enrollment of the trial since right after approval?.

Sure. Maybe I’ll take the first half – portion of that and Andy I’ll let you talk about anecdotal enrollment. So what we’ve guided to you is that will provide an update by the end of this year and ASH is certainly a very logical venue for us to share data..

This is Andy. In terms of enrollment, this study continues to enroll well. Midostaurin was available through compassionate use in U.S. and Europe already when the study started. So the approval, so far has not really affected the content of the study. And I don’t actually think it well..

Okay. Great, thanks..

Your next question comes from the line of Mike King from JMP Securities. Go ahead please your line is open..

Hey good morning guys. Thanks for taking the questions. A lot have been asked but I just wanted to touch on 285 for the PDGFR alpha population. Just wondering as far as the turnaround for the Phase 3 population, and you say, approximately 50 patients and I thought I read it correctly maybe I’m wrong.

First half of 2018 before that’s fully enrolled is that correct Andy? Or did I – am I mistaken there?.

Mike, this is Andy. We are aiming to complete the enrollment in the first half of 2018….

Just of 50 patients..

Well under 50 patients, exactly..

Okay. Now is that hesitated to jump to commercial potential for that but given that the enrollments been ongoing and I forget exactly what the last reported number of patients was with PDGFR alpha D842V’s.

Is that any kind of an indication of the population size – market potential just as far as the ease of which you are able to recruit these patients?.

So this is Jeff. Maybe I’ll take that. So our estimate is in the major markets in U.S., EU, five in Japan that there is approximately 500 to 700 of these patients with PDGFR alpha-driven disease, make up about 5% of the advanced GIST population.

And what we’re experiencing now and that’s been the case since we started the trial is that most of the patients with the PDGFR alpha limitation do seem to migrate to larger academic sites. And so that’s why early on the rate of enrollment was certainly faster than we would have anticipated.

So there’s nothing that that’s changed from that perspective. One of these we’re doing is of course in a clinical – in the context of a clinical trial, you don’t have all sites that see the patients as part of your study. But we are adding sites currently. So we’d expect to find more patients in other geographies, be at regions of the U.S.

or new countries within Europe..

Okay.

And that 500 to 700 Jeff is an incidence number right?.

Yes..

Okay. And is there any kind of prevalence outside that, I know they have a poor prognosis, but I don’t know if there was any..

No, there is – not at this time. If you look at historical norms the response rate is close to zero percent. I think a survival of around a year. So I think that’s part of what’s so compelling about the data that we shared at ASCO is that – hopefully we could be the type of therapy that helps to create a prevalence number..

Right. Okay, great.

And then for SM – I know that you’ve said that – or you may have said that there are maybe some indolent patients in that mix of the – in expansion part of the study is that correct? And do you think you might have some of that data at ASH as well?.

Mike, it’s Andy, I’ll take that one. So the ongoing study is specifically for patients with advanced systemic mastocytosis. We are doing a central pathology review to confirm diagnosis. And over the course of the study likely we will end up with some patients who might meet the criteria for advanced and so they’ll be indolent patients.

But if so they’ll be very – they’ll be right on the edge. We won’t have enough of those will be calling that out I don’t think specifically at ASH.

But we are very – because of the activity in the current study at the initial dose levels, which are at the low dose levels, which are very well tolerated for a long period of time that’s really what got us so excited about indolent systemic mastocytosis as another opportunity.

And the plan is to – we are actually already meeting with clinical advisors and thinking about – what a study would look like in that population and expected to pursue that next year..

Okay. Great, it’s helpful. And then finally – real quick on FOP.

Is the program clinic ready or are you unwilling to say at the moment when that might be ready for clinic?.

Yes. This is Jeff, I’ll take that. We have not disclosed that previously as I said, our focus now is on the wind down with Alexion and once we’re through that we’ll be looking to provide a more robust update on status of the program and potential plans that we will have either on our own or in some form of collaboration or otherwise..

Great. Thank you. I’ll get back in queue..

[Operator Instructions] Your next question comes from the line of David Nierengarten from Wedbush. Go ahead please your line is open..

Thanks. So I’ll add that pronunciation the growing – most of my questions have been asked.

But we saw of course Agios approval and pricing yesterday which I believe is a record in terms of monthly cost for a disease with roughly similar incidents to or preference to mutated forms of GIST and other rare indications does that provide a new benchmark kind of pricing that you’re looking at.

I know it’s pretty early but in terms of thinking about again prevalence severity et cetera. And then to follow-up on another question does that complicate or change any plans for the 285 and mastocytosis pricing potential. Thanks..

Sure. So this is Jeff. I’ll take that. So first off it was great news to see yesterday from our neighbors Agios and certainly great for patients who they have now.

Marion Dorsch our Chief Scientific Officer, was closely involved in that program, while she was at Agios and I know she takes a lot of personal pride in seeing that program now reach commercialization. So I think I’d start with that.

From a pricing perspective again, the landscape is changing rapidly enough that I don’t think we shift directions based on each subsequent approval.

In fact, if you look at midostaurin in the context of advanced SM I think that the pricing there is more aggressive where you really see that intersection of where does that genetic disease and oncology come together. Our data will lead us to what we think the value is, at the time we’re much further along than we are today.

To the second part of your question, in terms of complicating with indolent I can – I would say we have perhaps – our view is the option that the activity we’re seeing at 300 milligrams and 400 milligrams, in advanced systemic mastocytosis and GIST and the fact that in systemic mastocytosis we saw a clear activity, starting at 30 milligrams has really opened up a pretty meaningful window, where the notion with indolent SM where it’s the same genetic driver, same mutation D816V may give us flexibility from a pricing perspective, rather than restricting our ability to take that program forward, which may – it was probably our view prior to starting the clinical trial.

That was going to be a hard patient population to access. I think we’re much more optimistic at this point..

Great, thanks. We’ll see at ESMO..

See you then..

Your next question comes from the line of Arlinda Lee from Canaccord. Go ahead please your line is open..

Hi guys, thanks for taking my questions. First I guess on 285 in the systemic mastocytosis, you mentioned that you’d be – do you think the IWG criteria, I guess, I am curious, at the time by year end, I guess what portion of patients you think might have biopsies.

And then maybe can you talk a little bit about the key considerations and issues you have with moving into indolent. Thank you..

Arlinda hi. It’s Andy, I’ll take that. So yes, we are using the ECNM IWG criteria as the definitive response criteria for the study. And I think importantly the approval of midostaurin really has validated that, as a regulatory endpoint. So that’s incredibly helpful for us. As we think the next steps forward in advanced systemic mastocytosis.

All patients get biopsy at baseline and the way the study is designed. These pretty typical patients get biopsy then, at two months. But then after that it’s more infrequent. So the number of patients with biopsies and that we have data for really just depend on the timings, since they were enrolled on the study.

And I think it’s premature to give you particular numbers about that certainly that will all be presented – when we present data, we’ll give you – at that point know the details. But there has been no trouble getting biopsies, so we’d expect to have biopsy data on essentially all the patients on the study.

In terms of indolent, maybe just repeat the angle of your question on the indolent..

What are the considerations?.

Consideration for….

Moving forward..

Moving forward. Thanks. Thanks, Jeff. I think the first step is to see how the data are evolving against population and better understand response across the dose levels, and understand where we think will have the most potent activity.

But we’re already actively talking with clinical advisors and thinking about designs for – study designs for indolent patient population. The next step would be to start developing some protocol concepts and we plan to have some actions with regulatory authorities next year on the topic.

So I think it’s an emerging process that will develop over the coming months. But there is nothing – at this point, we think we should proceed indolent, there is an opportunity..

Okay. Great. And then I guess, maybe as a follow up on 285 expansion.

Is there an opportunity for you to enroll second line GIST in the trials that are ongoing or might that be an exploratory other trial that you’d be looking to start?.

Hi, it’s Andy again I’ll take that as well. So as I think we’ve discussed before, we are very interested in looking at BLU-285 in earlier lines of therapy in GIST second, including second line.

We think that our data in the KIT-driven population that we’ve seen so far is actually very encouraging that’s in a population of actually median of fifth line therapy patients with a very heterogeneous mutation profile or 85% actually had prior regorafenib based on other experiences in GIST and the general experience in solid tumors.

We would expect that activity would become more robust as we move earlier in therapy, where patients at less – have been less. Pre-treated have probably simpler genetics.

So the short answer is that yes, we’re very interested in exploring 285 in a dedicated second line population and we are currently talking with advisors and thinking internally about the best approach for making that happen..

Okay. I guess thanks. And then I guess lastly on FOP, at some point you had talked about priorities of your different earlier programs. I’m kind curious now that you’ve got it back, how does this kind of slot into your priorities with fibrolamellar carcinoma program..

Yes. So this is Jeff, I’ll take that. Good question. I take it we maybe didn’t say it very clearly we don’t have room for this program, as we operate right now.

That we’ve got the number of programs we have advancing that we’re constantly looking at – how we prioritize across programs, regardless of stage what our nomination process is for new programs that are either wholly-owned or under the Roche collaboration.

So I think I started off in the call saying that some of our past success has just earned us this opportunity to work harder. That’s what we’re feeling right now, that we’re working hard. But that said, having a molecule that we believe and as much as or a program that we believe in as much as the FOP program.

That will have seat at the table, we’ll have to figure out, what the best option is and that may mean sewing other programs. It doesn’t necessarily mean it will be vis-a-vis another program at the same stage. But that will be part of a comprehensive program review that will do over the course of the remainder of the year.

And whether that had come back to us or not, is always something that we do, and something that we take very seriously that we think the best way to bring medicines to patients is to focus on execution. And we are never satisfied with how we are doing it or how fast we’re moving or how effectively.

And so, I simply look at this as a real opportunity to help a patient population that has devastating disease. And so we take that seriously..

Thanks..

And your next question comes from the line of Mike King from JMP Securities. Go ahead please, your line is open..

Hey guys, thanks for taking the follow-up. One of the conversations I have been having with clients is certainly about competitors in the FGFR4 space, both public and private, I don’t know if you would care to give or want to give a kind of a competitive profile. 554 relative to competition as far as activity binding affinity et cetera.

But I just thought I would ask..

Yes, Mike this is Andy, I’ll take that. And Jeff chiming as well, we definitely focus primarily on our compound and we think that the profile of 554 is an FGFR4 inhibitors pretty – is pretty unique. It’s extremely potent and really exclusively selective, probably the most selective compound that we have in the clinic at this point in time.

For FGFR4 we think that the – well, even from our clinical data, actually, not only do we think we know, that on that has avoided the typical toxicities of the pan FGFR inhibitors things like hyperphosphatemia have been really [indiscernible] for those. It’s not been an issue with our compound.

And so where we are now is that based on the data we presented at the Triple Meeting last year and of course you’ll see update coming up in ESMO. I think established proof of concept that BLU-554 is selectively active in a FGFR4 activated population.

We are confident that we have a selection method that is identifying these patients although as I mentioned in my prepared remarks, we want to further explore this to see if we can optimize things even further. So I have no doubt that we have a terrific molecule.

The main competition I suppose that’s out there is for FGFR4 would be we know that Novartis has a compound out there and they have presented some data. I think all I have access to is there publicly presented data.

And I think it probably is a good molecule, but it’s the – there is still uncertainty in terms of how that patients have been selected, what their level of activity is. And I think that will play out as we both present data as the study has evolved. Earlier, there are a couple of other compounds in that early run….

Yes I’m thinking like Insight and H3..

We don’t know much about those compounds, we know….

Okay..

We know that they’re good companies, they probably make good drugs, but they’re behind us and we don’t really have any specific information..

Okay. Fair enough. Thank you..

And this concludes the Q&A portion of the call. I would like to turn the call back over to Jeff Albers for some closing remarks..

Thank you, operator. So to conclude, I want to reiterate our segment for the second half of this year, as I said earlier we plan to present updated data from our ongoing Phase 1 study of BLU-554 at ESMO in September and we remain on track to announce updated data from our ongoing Phase 1 study of BLU-285 in systemic mastocytosis before year-end.

I want to thank you all for your continued support and interest in the Blueprint story. And I look forward to updating you again soon. Thanks for your time and have a great day. Bye-bye..

This concludes today’s conference call. You may now disconnect..