Jeff Albers - CEO Andy Boral - CMO Mike Landsittel - VP, Finance Marion Dorsch - Chief Scientific Officer.
Eric Schmidt - Cowen & Company Arlinda Lee - Canaccord Carmen Augustine - Jefferies Terence Flynn - Goldman Sachs Chris Raymond - Raymond James..
Good morning, and welcome to the Blueprint Medicines Fourth Quarter and Year End 2016 Financial and Operating Results Conference Call. At this time, all participants are in a listen-only mode. Following the formal remarks, we will open the call up for your questions. 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 President Kristen Hollis [ph] of Blueprint Medicine. Please proceed..
Thank you, operator. Good morning, this is Kristen Hollis [ph] of Blueprint and welcome to Blueprint Medicine’s fourth quarter and year-end 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 on the investor 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 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 obligations 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, Kristen and good morning everyone. Looking back at clear 2016 really was a transformative year for Blueprint medicine 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 age population.
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 are 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 patient who have no therapeutic options or would need new options.
We believe the 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 that major medical meeting 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 password for BLU-285 and BLU-554. This includes potential opportunities for expedited clinical development through interaction 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 were 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 filed very soon.
We are 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 active [ph] potent against wild-type presentations and fusions as well as predicted resistance mutations.
And we believe this design allows BLU-667 to potentially offer patients profound durable responses for 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 will look for a partner that can help move our programs move 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 be more in 2017 and what I just outlined in an event filled year for Blueprint Medicine. In 2017, we anticipate upto 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 are thinking about in terms of next step for ongoing clinical trial and provide his thoughts on when we believe the 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. To determine the recommended part two dose, 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 [Indiscernible] are also driven just but beyond our expectation and we believe may enable an accelerated path of approval in this population based on expansion in the ongoing single arm clinical study.
Similarly the clinical activity in safety demonstrated and heavily treated refractory hit driven GIST patients at higher doses are very encouraging. In this group, the optimal registration path of view formed as data merges the 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 focussed 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 with a randomized Phase 3 confirmatory study running in parallel.
Alternatively, the randomized Phase 3 study will become the initial registration 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 -- 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 o maximize clinical activity and in the near future we expect to determine MTD with a lower recommended dose that will be used in the expansion part of the study. During expansion, we will more fully characterise the monotherapy potential of BLU-285 in the various sub types of advance systemic mastocytosis.
Importantly, seeing substantial clinical activity at the lowest dose levels in patients with advanced systemic mastocytosis combined with a favourable safety profile opens the opportunity for BLU-285 in patients with indolent systemic mastocytosis or ISM.
As we have learnt more about ISM we have come to realize that for many ISM patients the term indolent is a bit newer [ph] and there is a desperate need for therapies that will adequately controlled disabling symptom.
Just this past week on rare disease day, an ISM patient in first ever visited our offices and reminded us how far do we live with this disease. We learnt how the disease disrupts a patient life on a daily basis, making it impossible to work and socialize outside the home. Even a strong emotion can trigger a flare.
These patient’s needs 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 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 of biomarker negative, patients with biomarker positive with alchemia [ph] implification and patients with a biomarker positive dsychemia [ph] amplification.
The clinical data we presented last year clearly demonstrates that BLU-554 is preferencially 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 evaluating single agent potential of BLU-554 in HCC.
Based on the early signs of clinical activity that we presented last year, we are evaluating two potential registration pathways in the biomarker positive population. The first is to further extend the biomarker positive groups in the ongoing single arm study with the goal of accelerated approval in patients with 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 and the dose expansion part of the HDC study and regulatory guidance – division as for the fastest and most appropriate path forward with BLU-554..
Hi, Boral, this is Jeff again. So following 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 you said before, our goal is to provide these updates at major medical meetings. Given the encouraging data we’ve seen thus far and the promise from our ongoing collaborations, it’s important that we are also thinking to see focus on the quality of our team and on 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 – is a experienced leader with expertise in human biology and she also has a wealth of experience identifying novel targets for cancer like diseases. And importantly, this includes significant experience in cancer immunology.
And as we continue to make sure as a company Marion’s experience driving therapeutic discovery across both internal and partnered program in cancer and rare genetic diseases makes a perfect fit for us at Blueprint Medicine and we are thrilled to have her on the team.
And in tandem with Marion’s appointment, Christoph Lengauer, was transitioned to the role of Executive Vice President. He is responsible for driving an integrated approach to progress our portfolio of investigational therapy.
Initially as primary [Indiscernible] focus has been supporting our accelerating efforts for BLU-285 and 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. I just 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 scientist at Blueprint and I have become embedded in the impressive [Indiscernible] 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 at the end of last year which further validated the power of Blueprint’s scientific platform. We have three upcoming presentations in the early springs 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 a story of the discovery and development path of BLU-285 in a session called [Indiscernible] on the Horizon 2.
This will be an opportunity for us to talk about how BLU-285 came to be from the initial promising head processes through discovery and now into clinical device. At that presentation, we plan to discuss more about the spectrum of activity against pigmentations for BLU-285 in [Indiscernible].
Three days later at the American Pharmacists Society Meeting on Wednesday 5th we represent both the BLU-554 and 285 medicines at [Indiscernible] stories in the morning and afternoon sessions titled stress [ph] time disclosures. Both compounds have very interesting chemical exposures.
BLU-285 was a direct hit from our proprietary compound library and BLU-554 came out of our rationalized assignment forward. We look forward to sharing how our discovery at – took two different paths to produce our two lead compounds which are important Blueprint medicine programs. Now here’s Mike to go over our financial results..
Thanks Marion. Today we are 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 trial 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 collaborations 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, our 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 into 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 advanced our pipeline. G&A expenses were $19.2 million for 2015 compared to $14.5 million in the prior year. This increase is primarily due to increased personnel-related and increased professional fees.
And finally for the year ended 2015 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 are substantial to further division we have at different medicine to build the 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?.
Thank you. [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, just 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 our partner will add value to the programs and that’s two obvious places given the breadth of our portfolio currently is 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 that could be logical combinations with one of our programs.
Or secondly need to be the case in every phase is a partner that can help, lead our progression of our programs as we’ve increase 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. We’ll have to be one that meet those type – that stringent criteria from our perspective..
Okay. And then question for Andy on 285 and just potential for approval in the single-arm PDGFR mutants population.
Have you had any FDA feedback yet on it potential tolerated strategy at the FDA and get 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 to serve as the necessary data mature from our ongoing expansion. So we don’t have any specific to offer on those lines by yet..
Okay.
Have you thought about breakthrough designation?.
For sure, 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 through designation along with tolerated approval and of our course we have FDA..
Great. Thanks very much..
Thank you. Our next question comes from Arlinda Lee with Canaccord. Your line is open..
Hi, guys. Thanks for taking the question. Congrats on the progress. I also had a question on 285. You guys mentioned that you guys have reached MTD of 400 mgs daily. Can you maybe talk about what the dose limiting toxicity was for last update, we saw didn’t really – you hadn’t reached it yet. So, any additional color would be appreciated. Thanks..
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 these kinds of typical things you would see with kinase inhibitors, but we think its 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 safety and next step..
Okay.
And then, 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 the newest program to enter the clinic 667. Can you maybe talk about what other advanced solid tumors as far lung and thyroid you guys might be interested in? Thanks..
Yes, sure. So as you already pointed out, RET has been most commonly identified in the small-cell lung cancer and in, especially medullary thyroid cancer. Interestingly there are growing data that its RET alterations fusions as well as sometimes activating mutations drive the disease in a actually very broad range other solid tumors.
Some of the things that we've highlighted – we and others actually highlighted colorectal cancer, breast cancer, but I mean, we looked at some data recently suggesting that you find in 30/40 different tumors met 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 fights before opening the expansion part of the study who do very broad economic screening who find that broad range of patient..
Thank you very much..
Thank you. Our next question comes from Carmen Augustine with Jefferies. Your line is open..
Hi, guy. Thanks for taking the questions.
So, for 285 in GIST, could you provide any granularity on what time of year or which medical meeting we could expect to see the dose escalation data from this 400 milligram cohort? And also could you look at any dose is higher than 400?.
Hi, Carmen, this is Jeff. I’ll take that. What we’ll look to do is, as I set upfront is creative [ph] wisdom of data disclosure over the course of this year given that we now have reached the recommended Part II dose.
You could expect to see data at a major medical conference in the second or third quarter, and what we’d also – we’d like to also guide you to, is that in that same timeframe you’d expect to see data from our 554 study in HCC. And then in terms of what dose we went to. I think that would be part of our conference update.
But as you think about classical Phase 1 dose expansion it often go up and may come back down a bit and we want to share all of that information at a conference..
Okay.
And then for 285 and mastocytosis what would be the next step on determining of pass forward in interim patients?.
This is Andy. I’ll take that one. So, as I mentioned, when I was speaking before the activity and very nice tolerability at the low does is really does make us think that there is a path forward, a pretty straightforward and maybe very important path forward for BLU-25 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 the regulatory authorities..
Okay, great. Thanks for the color. And then one last I could. On 554 what would you want to see from the dose escalation trial to make that decision on whether [Indiscernible] accelerated approval in patients who progress sorafenib, or a Phase 3 in the frontline setting? Thanks..
This is Andy. I’ll take that up as well. So the escalation phase 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 and with 554 as single agent in this sorafenib relapse refractory population that we’re enrolling on the current study.
And then, really the question is, if the response rate in duration look strong and I think preliminary data looks like strong, but if they continue to as we enrol mutations then we think it is a disease that is pretty classic opportunity for an accelerated approval, it’s a very high unmet needs, there are no effective therapies really that are following sorafenib.
And if for some reason the response rate 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 front line. I think I should add that, in either case we do need to do the Phase 3 study because even if we do accelerated approval we need inflammatory study.
So in the first option we would do the accelerated approval with the Inflammatory Phase 3, and the second we just – in the second situation, Inflammatory Phase 3 would become the primary registration study.
I think all of this will be part of -- will be of come out of the data that we observed 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..
Thank you. [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 285 and 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..
Terrence, it’s Andy. I’ll take that.
So on the first the design of the expansion, yes, so that its 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 and 300 or 400 milligrams, enrolling two distinct populations; one are patients with PDGFR alpha D842 mutants 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 study that increase number of patients in that group to assize that we think if the data are strong to support [Indiscernible].
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 a imatinib and we form one, at least one other prior to KIT-directed TKI and have progressed and who don't have a PDGFR alpha mutation.
And the reason, 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 mutation.
And we’ve also in the recent amendments 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 second half of your question..
Sure.
So, the amendment dose have you guys – have disclose the doses for both of those expansions 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 applies to both population.
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 given us the longest durability of response, the reason 400 milligrams in both.
So in terms of the Phase 3 study, yes, we know that in the long run in Phase 3 study will be require in GIST whether it becomes the initial approval study in KIT-driven patients or it becomes inflammatory 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 design.
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 things. But we are – we have decided to proceed with the Phase 3..
Okay. Thank you very much..
Thank you. 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 questions, raise an issue of 554 and 850 [ph], you guys have 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 rate in HCC are so low what do you think a reasonable boggy would be on that? Thanks..
Hi. It’s Andy. So of course the final arbiter of that are the authorities who we would submit this data, 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 been actually now first line and regorafenib actually second line in advanced HCC is very low, low 5%, but nevertheless to have an accelerated approval based on a single-arm dataset that tends to be relatively small.
I think, FDA had typically wanted to see response rate in excess of 20%, so I guess I think of that as maybe a barge just based on regular 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..
Thank you. [Operator Instructions] Our next question comes from Chris Raymond with Raymond James. Your line is open..
Hey, guys. Thanks for to let me in here. Just a quick question on the biomarker work that Andy, I think was talking about. I think you mentioned QIAGEN is working on the BLU-285, KIT- PDGFR biomarker.
Just curious is the plan to have that in place before you start the registration trial or would this be something incorporate afterwards, just if you put some explanation to the timing would be great..
Yes, 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 CVX [ph] in that setting, so that would be ready for submission in parallel with any NDA filing. We are on time for that..
So the objective there would be to validate that in a parallel path with the trial, is that correct?.
Yes, I think it could be validated in the same ongoing study. It’s the way we are 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. Taking a step back, we realize that these are early days for Blueprint Medicine and that we are 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 in 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, 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..