Blueprint Medicines Corp (BPMC) 2016 Q4 法說會逐字稿

Good morning and welcome to the Blueprint Medicines fourth quarter and yearend 2016 financial and operating results conference call. (Operator Instructions) Please be advised that this call is being recorded at the company's request.

At this time, I'd like to turn it over to Kristin Hodous of Blueprint Medicines. Please proceed.

Thank you, operator. Good morning. This is Kristin Hodous of Blueprint Medicines, and welcome to Blueprint Medicines' fourth quarter and yearend 2016 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 in the Investors section of our website at www.blueprintmedicines.com

Today on our call, Jeff Albers, our CEO, will discuss Blueprint Medicines' fourth quarter and 2016 business highlights. Dr. Andy Boral, our Chief Medical Officer, will discuss recent clinical progress. Marion Dorsch, our Chief Scientific Officer, will discuss our upcoming scientific presentation. And Mike Landsittel, our Vice President of Finance, will review our full year 2016 financial results. Then we will open the call for your questions.

Before we get started, I would like to remind everyone that statements we will 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 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 dates. We specifically disclaim any obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

Now, here is our CEO, Jeff Albers.

Thanks, Kristin, and good morning, everyone. Looking back, it's clear 2016 really was a transformative year for Blueprint Medicines. And in 2017, we are poised to build on that momentum with a number of event-driven milestone.

2016 was a year in which we accomplished all of our corporate goals, culminating in the presentation of preliminary data from our three clinical studies for BLU-285 and BLU-554, and in demonstrating proof-of-concept for these drug candidates in four distinct patient populations. We also filed an IND for our RET investigational medicine, BLU-667, which was accepted by FDA and signed a strategic collaboration with Roche, which allows us to accelerate our cancer immunotherapy pipeline.

[And finally] vision of Blueprint Medicines came into clear focus in 2016. We're building the company for the long-term with a diversified portfolio grounded in our unique scientific platform with a clear goal to bring new therapies to patients who have no therapeutic options or would need new options.

We believe encouraging early indications of clinical activity and safety presented at the end of 2016 for our clinical trials for BLU-285 in advanced gastrointestinal stromal tumors or GIST and advanced systemic mastocytosis, as well as our data for BLU-554 in hepatocellular carcinoma or HCC, have earned us the right to do more in 2017 with a focus on four prime areas.

The first area of focus is on the progression of our three Phase 1 clinical trials, BLU-285 and BLU-554. As the data sets mature, we anticipate establishing a rhythm of disclosure at major medical meetings throughout the year. We also expect to provide updated results from each of the ongoing studies this year including any available data from the dose expansion portion of these studies. As previously announced, we are currently enrolling patients in the expansion portion of our HCC study. And importantly, as we announced this morning, we also recently initiated the expansion portion of our GIST file, and we expect to initiate the expansion portion of our systemic mastocytosis trial in the near future.

So our second area of focus is to further define the clinical and regulatory path forward for BLU-285 and BLU-554. This includes potential opportunities for expedited clinical development through interactions with global regulatory authority. We expect to work closely with U.S. and EU regulators and look forward to these anticipated interactions which Andy will expand upon momentarily.

Our third area of focus is to continue to maximize the value of our platform. At Blueprint Medicines, we place an emphasis on our therapeutic discovery engine, and we're committed to maintaining our research productivity. To that end, we recently received FDA approval to initiate a Phase 1 study for BLU-667 in patients with non-small cell lung cancer, medullary thyroid cancer, and other solid tumors with RET alterations. And we expect to initiate this Phase 1 trial very soon.

We're excited about this program as RET kinase fusions and mutations have recently emerged as important drivers in a variety of cancers. As we described before, BLU-667 is designed [exopotent] against wild-type [recommendations] and fusions as well as predicted resistant mutations. And we believe this design allows BLU-667 to potentially offer patients profound durable responses of therapy.

Finally our fourth area of focus is to evaluate business development opportunity. We are extremely pleased with the progress we've made with our existing partners, Alexion and Roche, and we will continue to evaluate opportunities to work with partners that could add value to one of our more advanced programs assuming they contribute two important factors. First is that they will help us treat a broader range of patients or help us expand our global reach. And secondly, we'll look for a partner that can help move our programs more quickly towards approval and into the hands of physicians and patients worldwide.

So at the beginning of this call, I talked about how the progress we made in 2016 has earned us the right to do more in 2017 and what I just outlined means an event-filled year for Blueprint Medicines. In 2017, we anticipate up to a dozen potential milestones, including additional data readouts for BLU-285 and BLU-554.

So what I'd like to do now is hand it over to Andy Boral, our Chief Medical Officer, to provide a bit more detail on what we're thinking about in terms of next steps for ongoing clinical trial and provide his thoughts on when we believe these plans will crystallize over the course of the year. Andy?

Thanks, Jeff. As Jeff mentioned, we are very happy with the progress that we have made on our clinical trials to date. Let's begin with our BLU-285 study in GIST. We determined the recommended Part 2 dose, and we have initiated the expansion part of the study. Based on the encouraging early data that we presented at the triple meeting in December, we are also in the process of increasing the cohort sizes in the study to more fully evaluate the single-agent potential of BLU-285 in GIST.

The clinical data we presented in patients with PDGFR alpha-driven GIST beyond our expectation, we believe, may enable an accelerated path to approval in this population based on expansion in the ongoing single-arm clinical study. Similarly, the clinical activity in safety demonstrated and heavily pre-treated refractory KIT-driven GIST patients at higher doses are very encouraging. In this group, the optimal registration path will informed as data emerges as a 400-milligram MTD or maximum tolerated dose in the expansion part of the study that's ongoing now and based on the discussions with health authorities over the first half of this year.

Assuming we continue to see mounting evidence of strong single-agent activity in this highly refractory population, we will pursue an accelerated approval based on the expansion of the ongoing single-arm study and a -- with a randomized Phase 3 confirmatory study running in parallel. Alternatively, the randomized Phase 3 study will become the initial registrational study.

The data we presented at the ASH meeting in December for BLU-285 in advanced systemic mastocytosis showing marked decreases in objective measures of mast cell burden and improvements in patients symptoms beginning at the lowest dose levels were also beyond our expectations. These data demonstrate that [partnering with] D816V genetics 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 its underlying cost.

We continue to dose escalate to maximize clinical activity. And in the near future, we expect (technical difficulty) to determine the MTD or a lower recommended dose that will be used in the expansion part of the study.

During expansion, we have more fully characterized the monotherapy potential of BLU-285 in the various sub types of advanced systemic mastocytosis. Importantly, seeing substantial clinical activity at the lowest dose levels in patients with advanced systemic mastocytosis, combined with the favorable safety profile, opens the opportunity for BLU-285 in patients with indolent systemic mastocytosis or ISM. As we've learned more about ISM, we have come to realize that for many ISM patients the term indolent is misnomer, and they're in desperate need for therapies that will adequately control disabling symptom.

Just this past week, on Rare Disease Day, an ISM patient and his caregiver visited our offices, and reminded us how hard it is to live with this disease. We learned how the disease disrupts a patient's life on a daily basis, making it impossible to work and socialize outside the home. Even a strong emotion can trigger a flare. These patients need new treatment options, and we are working with urgency to define the fastest clinical path for BLU-285 in ISM.

Now, let me move onto BLU-554 in liver cancer. We've made significant progress in all the expansion part of the study which we initiated earlier this year -- excuse me, which we initiated last year. The expansion part of the study includes regroups of hepatocellular carcinoma patients that are defined by the status of the biomarker FGF19. These include patients who are biomarker negative, patients who are biomarker positive without gene amplification and patients who are biomarker positive with gene amplification.

The clinical data we presented last year clearly demonstrates that BLU-554 is preferentially active in a biomarker positive patient. Given rapid enrollment thus far, we are in the process of increasing the cohort sizes in the biomarker positive groups to more fully evaluate single-agent potential of BLU-554 in HCC.

Based on the early signs of clinical activity that we presented last year, we're evaluating two potential registration pathways in the biomarker positive population. The first is to further expand the biomarker positive groups in the ongoing single-arm study with the goal of accelerated approval in patients who have progressed on sorafenib. And the second is the randomized Phase 3 study comparing BLU-554 to sorafenib as first-line therapy. So similar to KIT-driven GIST, clinical data from early in the dose expansion part of the HCC study and regulatory guidance will inform our decisions as to the fastest and most appropriate path forward with BLU-554.

Hi, everyone, this is Jeff again. So pulling this altogether, we expect to provide updated results from each of these ongoing studies for BLU-285 and BLU-554 later this year, including any data from the dose expansion portions of these studies that Andy just walked through. As we said before, our goal is to provide these updates at major medical meeting.

Given the encouraging data we've seen thus far and the promise from our ongoing collaborations, it's important that we're also thinking to stay focused on the quality of our team and our financial discipline. We have ambitious goals for 2017, and I'm confident that we have the right team in place, and as Mike will highlight shortly, the financial sprint to execute on them.

In November, we announced the appointment of Marion Dorsch as our Chief Scientific Officer. Marion's experience -- Marion is an experienced leader with deep expertise in human biology, and she also has a wealth of experience identifying novel targets for cancer and rare genetic diseases. And importantly, this includes significant experience in cancer immunology. And as we continue to mature the company, Marion's experience driving therapeutic discovery across both internal and partnered program in cancer and rare genetic diseases makes her a perfect fit for us at Blueprint Medicines, and we are thrilled to have her on the team.

And in tandem with Marion's appointment, Christoph Lengauer transitioned to the role of Executive Vice President for he is responsible for driving an integrated approach to progress our portfolio of investigational therapy. Initially his primary focus is on supporting our accelerating efforts for BLU-285 in GIST and systemic mastocytosis.

I'll hand it over now to Marion to talk about a few of our upcoming data presentations. Marion?

Thanks, Jeff. First, I want to say how excited I am to be part of this Blueprint team. Over the past three months, I have gotten to know the group of passionate scientists here at Blueprint and I have become embedded in the impressive set of discovery program as well as the strategic efforts to identify important new cancer targets. Our proprietary discovery platform has proven to be prolific, and I was very happy to be here as we announced the proof-of-concept data from our first clinical program end of last year, which further validated the power of Blueprint's scientific platform.

We have three upcoming presentations in the early spring that I am pleased to announce. The first presentation is at the American Association for Cancer Research or AACR on Sunday, April 2, and we will present the story of the discovery and development path of BLU-285 in a session called, New Drugs on the Horizon 2. This will be an opportunity for us to talk about how BLU-285 came to be from the initial promising hypothesis through discovery and now into clinical trial. At that presentation, we plan to discuss more about the spectrum of activity against KIT mutations for BLU-285 in preclinical models.

Three days later at the American Chemical Society Meeting on Wednesday, April 5th, we represent both the BLU-554 and BLU-285 medicine and chemistry stories in the morning and afternoon sessions titled, First Time Disclosures. Those compounds have very interesting chemical exposures. BLU-285 was a direct hit from our proprietary compound library, and BLU-554 came out of our rational design approach. We look forward to sharing how our discovery approach took two different paths to produce our two lead compounds, which are important Blueprint Medicines programs.

Now, here's Mike to go over our financial results.

Thanks, Marion. Today, we're continuing to operate from a strong financial position to support the activities that Jeff outlined earlier, including advancing our lead clinical programs in multiple Phase 1 trials and maintaining our investments in our early discovery platforms.

As we reported in this morning's press release, we ended 2016 with cash, cash equivalents, and investments of $268.2 million compared to $162.7 million for 2015. This increase was primarily driven by net proceeds of $134.5 million from our December 2016 follow-on offering and the $45 million upfront cash payment from our collaboration with Roche, which we entered into last March. And the above were partially offset by cash use in operating activities throughout the year. Based on our current plans we expected our existing cash, cash equivalents, and investments, excluding any potential occupancies and milestone payments under our existing collaboration, will be sufficient to enable us to fund our operating expenses and capital expenditure requirements into at least late 2018.

Turning to the full year P&L, collaboration revenues were $27.8 million in 2016 compared to $11.4 million for the prior year. This increase was primarily driven by increased activity in our collaboration with Alexion as well as entering in for our collaboration with Roche.

During 2016, we incurred $81.1 million in R&D expenses, compared to $48.6 million for 2015. The increase in R&D expense is primarily driven by increased manufacturing and clinical expenses associated with advancing BLU-285 and BLU-554 into clinical trials. Increased personnel-related expenses are continuing to build our recovery platforms and advance our pipeline.

G&A expenses were $19.2 million for 2016 compared to $14.5 million in the prior year. This increase was primarily due to increased personnel-related expenses and increased professional fees. And finally, for the year ended 2016, we reported a net loss of $72.5 million or $2.64 per share compared to a net loss of $52.8 million for 2015 or $3.07 per share.

So as we've highlighted on the call, we're very encouraged by the progress that we have made during the past year, and we're really looking forward to the opportunities ahead of us in 2017. As Jeff mentioned, we anticipate more than a dozen key milestones over the course of this year, including additional data readout from our ongoing Phase I study. Each of these milestones has the potential to further the vision we have at Blueprint Medicines to build a sustainable company with a broad and diversified portfolio to help bring new and unique therapies to patients.

Now, let's open the call for your questions. Operator?

(Operator Instructions) Our first question comes from Eric Schmidt with Cowen & Company. Your line is open.

Good morning. Thanks for taking my questions and congrats on the steady progress. Maybe the first one is for Jeff. In the past, you've alluded to having a lot on your plate for a small company and maybe thinking about using business development to potentially out-license candidates. Wondering where you are in the discussions there?

Thank you, Eric. So, as I mentioned, when we think about business development, we hold the criteria where a partner will add value to the programs and the two obvious places given the breadth of our portfolio currently. It's a partner that can expand our reach for specific program, and that could be a geographic reach, that could be expertise in a specific disease state, it could be therapeutic in a portfolio, it could be logical combinations with one of our programs. Or secondly, needs to be the case in every case, is a partner that can help speed our progression of our programs as we've increased our confidence in BLU-554 and BLU-285. We think it's incredibly important to keep that focus on how quickly we can bring these investigational medicines to patients.

So, we're exploring a variety of different discussions on that front, but we'll only -- we won't do a partnership just to do a partnership. It will have to be one that meets those [type], that stringent criteria from our perspective.

Okay. And then question for Andy on BLU-285 in GIST and the potential for approval in the single-arm PDGFR mutant subpopulation. Have you had any FDA feedback yet on it, potential tolerated strategy at the FDA in a sense of what the hurdle rate might be in terms of response rate and patient sizes?

Yes. Thanks for the question. So, we do have an open dialogue with FDA. We're really -- we'll have formal discussions with them hitting exactly those questions over the first half of this year sort of as the necessary data mature from our ongoing expansion. So we don't have anything specific to offer along those lines quite yet.

Okay. Have you thought about breakthrough designation?

For sure, yes. And I think that -- when I talk about accelerated approval, to me, that breakthrough designation is something that often goes hand-in-hand. And I think in the PDGFR-alpha subset, the data we have in hand so far, I think, is very well may support breakthrough designation along with accelerated approval. And, of course, we have to have approval of the FDA.

Great. Thanks very much.

Our next question comes from Arlinda Lee with Canaccord. Your line is open.

Hi, guys. Thanks for taking the question and congrats on the progress. I also had a question on BLU-285. You guys mentioned that you guys have reached MTD at 400 mgs daily. Can you maybe talk about what the dose limiting toxicity was? The last update we saw didn't really -- you hadn't reached it yet. So, any additional color would be appreciated. Thanks.

Arlinda, it's Andy. I'll take that. So, as Jeff mentioned, we're planning to update data on all three ongoing studies including the BLU-285 GIST study over the course of the year, and we will present those data in detail at that time. I'd say that we didn't see anything unexpected there, the kinds of typical things you would see with kinase inhibitors. But we think it's important to do that in the setting of presenting the full data set in the medical community, where we can talk about the efficacy and the safety and next step.

Okay. And then, well, maybe can you update us on proportion of patients that are continuing on -- in those ongoing studies?

Yes. I guess we are going to stick to presenting all new data from the ongoing clinical trials at an upcoming medical conference. We just think it's really important to put it together in a whole story in that setting.

Okay. And then, I guess on your newest program to enter the clinic, BLU-667. Can you maybe talk about what other advanced solid tumors besides lung and thyroid you guys might be interested in? Thanks.

Yes, sure. So as you already pointed out, the RET has been most commonly identified in non-small cell lung cancer and in especially medullary thyroid cancer. Interestingly, there's growing data that at RET alteration fusions, as well as -- and sometimes activating point mutations drive the disease in actually a very broad range other solid tumors. Some of the things that we've highlighted -- me and others actually highlighted it as colorectal cancer, breast cancer. But I mean if you looked at some data recently suggesting that you might find it in the 30/40 different tumors at various low-frequencies.

And I should add that the -- in the expansion part of the study where we have a basket group, we will accept any RET activated tumor, and we're very actively looking for additional sites before opening the expansion part of the study since we do very broad genomic screening to see if we find that broad range of patient.

Thank you very much.

Our next question comes from Carmen Augustine with Jefferies. Your line is open.

Hi there. Thanks for taking the questions. So, for BLU-285 in GIST, could you provide any more granularity on what time of year or which medical meeting we could expect to see the dose escalation data from the 400-milligram dose cohort? And also could you look at any dose that's higher than 400?

Hi, Carmen, this is Jeff. I'll take that. So what we'll look to do is, as I setup upfront is creative rhythm of data disclosures over the course of this year given that we now have reached the recommended Part 2 dose. You could expect to see data at a major medical conference in the second or third quarter. And what we'd also -- what I'd also guide you to is that in that same time frame, you'd expect to see data from our BLU-554 study in HCC.

And then in terms of what dose we went to, I think that will be part of our conference update. But as you think about classical Phase 1 dose expansion, they often go up and may come back down a bit, and we want to share all of that information at a conference.

Okay, great. And then for BLU-285 in mastocytosis, what would be the next step on determining a path forward in indolent patients?

This is Andy. I'll take that one. So, as I mentioned when I was speaking before, the activity and very nice tolerability of the low dose really does make us think that there is a path forward, a pretty straightforward and maybe very important path forward for BLU-285 in the indolent patients. Of course, our ongoing study is limited to advanced patients. And so, we really are in the process now of talking with external experts who treat these indolent patients and designing an initial study for these patients.

So, really, the first step is to put together a clinical trial specifically for patients with indolent mastocytosis, start understanding appropriate dosing, frequency of dosing, length of dosing, and the key endpoints in that patient population which would be a bit different than in the advanced population. So, maybe in a nutshell, we're reaching out to experts in the field and we'll also initiate discussions with regulatory authorities.

Okay, great. Thanks for the color. And then one more if I could. On BLU-554, what would you want to see from the dose escalation trials to make that decision on whether to pursue accelerated approval in patients who progress on sorafenib or a Phase 3 in the frontline setting? Thanks.

Yes, this is Andy. I'll pick that one up as well. So the escalation part of the study is finished, and we did start the expansion part of the study last year and are continuing that now and it's been rolling quite well at the maximum tolerated dose of 600 milligrams daily. And what we're looking for is just to have a really good understanding of the response rate and durability of response with BLU-554 as a single agent in this sorafenib relapsed/refractory population that we're enrolling on the current study.

And then, really, the question is, if the response rate in duration looked strong and I think preliminary data look like strong, but if they continue to as we enroll more patients, then we think it is a disease that is pretty classic opportunity for an accelerated approval. It's a very high unmet need. There are no effective therapies, really, that -- following sorafenib. And if for some reason the response rates don't meet the hurdle that we think would be required, then it might -- we might be better off pursuing a head-to-head study with sorafenib in the frontline.

I think I should add that, in either case, we do need to do the Phase 3 study because even if we do the accelerated approval, we need a confirmatory study: so in the first option, when we do the accelerated approval with the confirmatory Phase 3; and the second, which is -- in the second situation, this confirmatory Phase 3 will become the primary registration study. I think all of this will be part of -- will be of -- come out of the data that we observe as well as discussions with regulatory authorities and understanding where we think the highest probability of success is for the drug.

Okay, great. Thanks so much.

(Operator Instructions) Our next question comes from Terence Flynn with Goldman Sachs. Your line is open.

Hi. Good morning. Thanks for taking the question. Just was wondering for BLU-285 in GIST, if you can remind us the design of the two different expansion cohorts, in particular the dose that you guys selected there. And then on the on the Phase 3 trial that you mentioned in the KIT population, have you already made a decision to advance into a Phase 3 trial at some point? Or is that still depending on your discussions with regulators and how the data emerges? Thanks.

Hi, it's Andy. I'll take that. So, on the -- and the first, the design of the expansion. Yes, so that is really continuation of the study as previously designed. So the way the study is designed after the escalation part of the study that we just completed at the now MTD of 400 milligrams, we're enrolling two distinct populations. One are patients with PDGFR-alpha D842 mutant GIST, and those are currently identified by the local investigators that we are developing a campaigning diagnostic to with QIAGEN to implement in the expansion part of that study. That's one group. And we have also recently amended a study, the increased number of patients in that group, to a size that we think if the data are strong to support an accelerated approvable.

The second group is effectively a KIT-driven GIST population. But as we've done the first part of the study we define that patients who have had imatinib and at least one other -- at least one other prior KIT-directed TKI and have progressed and who don't have a PDGFR alpha mutation.

The reason that, that's important is we are in the third line and -- third line plus population defined clinically. So we do not require a companion diagnostics to identify that group. Essentially that finds a group of KIT-driven patients with a very high probability of the exon 17 KIT mutation.

And we've also, in the recent amendment, expanded the size of the KIT GIST, the KIT-driven group with the intention of having enough data that support accelerated approval if the data are -- if the response rates are high enough. Actually, I have lost track of your second -- remind me the second half of your question.

Sure. So, amendment dose, have you, guys -- have, guys, disclosed the doses for both of those expansions --

Yes.

-- in the Phase 3 on the KIT side? Just -- have you made that decision already?

Yes. So the escalation part of the study was done in a combined population of KIT and PDGFR-alpha driven patients, and the 400-milligram expansion dose applied to both populations. And we do think it's important to use the maximum dose in both groups, including the PDGFR-alpha. Even though we saw activity very -- at the first dose level in that group, we do think that by maximizing the dose level we'll have the highest cycle preventing resistance mutations and giving us the longest durability of response. So we're using 400 milligrams in both.

So, in terms of the Phase 3 study, yes, we know that in the long run, a Phase 3 study will be required in GIST whether it's the -- whether it becomes the initial approval study in KIT-driven patients or it becomes confirmatory study for an accelerated approval, which could cover both PDGFR-alpha and KIT-driven patients. In either case, we know we need to that study. And really, now, it's a matter of really thinking what are the best designs, is it a third line study, is it a fourth line study, what are the comparators, what are the key regulatory endpoints, that kind of thing. But we are -- we have decided to proceed with the Phase 3.

Okay. Thank you very much.

And we have a follow-up question from Arlinda Lee with Canaccord. Your line is open.

Hi, guys. Thanks for taking the follow-up. I guess one of other people asking question raised an issue of BLU-554 in HCC. You guys had commented that as the response rate in duration looks strong, you might be able to go into a post-sorafenib single-agent study. And I guess considering that the response rates in HCC are so low, what do you think a reasonable bogey would be on that? Thanks.

Arlinda, hi, it's Andy. So, of course, the final arbiter of that are the health authorities, who we would submit this data to, not us. But I think that some -- I would look at some examples of accelerated approval for the solid tumors as the guidepost for that. It is true that the response rate to sorafenib and actually now first line and regorafenib actually second line in advanced HCC is very low, below 5%. But nevertheless, to have an accelerated approval based on a single-arm dataset that tends to be relatively small, I think, the FDA typically wanted to see response rate in excess of 20%. So I guess I think of that as maybe a barge just based on regulatory precedents. That certainly would depend a bit on -- or not bit, a lot on durability and safety profile, and we would -- as the data emerged and we have a whole story, we'll put in front of them and see what they think.

Okay. Thanks very much.

(Operator Instructions) Our next question comes from Chris Raymond with Raymond James. Your line is open.

Hey, guys. Thanks for to letting me in here at the end here. Just a quick question on the biomarker work that Andy I think was talking about. I think you were mentioning that QIAGEN is working on the BLU-285, KIT PDGFR marker. Just curious, is the plan to have that in place before you start the registration trial, or would this be something incorporated afterwards? Just with some explanation of the timing would be great.

Yes, it's Andy. So where we will be implementing that study and during the expansion part of the ongoing Phase 1 study, we think that's actually pretty important to nail down the [CDX] in that setting so that it would be ready for submission in parallel with any NDA filing. (Multiple speakers) are on time for that.

Sorry, so the objective then will be to validate that in a parallel path with the trial. Is that correct?

Yes, it could be validated in the same ongoing study. Just the way we're looking at it, I think that would be pretty typical for an accelerated approval situation.

Okay. Thank you.

Thank you. And I'm showing no further questions at this time. I would like to turn the conference back over to Jeff Albers for closing remarks.

Thank you, operator. So taking a step back, we realize that these are early days for Blueprint Medicines and that we're just getting started, and beginning to realize the vision that we have for Blueprint. The early data from our three clinical studies for BLU-285 and BLU-554 have started to demonstrate the potential impact and promise of our scientific platform, and we'll continue to invest in that platform as we look to find new, safe, and effective treatments that will address a number of different medical needs in both oncology and rare diseases. So, no doubt, 2016 was a transformative year for Blueprint Medicines, and I want to thank all of you for your continued support and interest, and hope everyone has a great day. Bye.

Ladies and gentlemen, thank you for your participation in today's conference. This concludes the program and you may now disconnect. Everyone, have a great day.