BPMC - Earnings call transcript Q4 2017 | Blueprint Medicines Corporation

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Operator

Good day, ladies and gentlemen, and welcome to the Fourth Quarter and Full Year 2017 Blueprint Medicines Earnings Conference Call. [Operator Instructions] As a reminder, this conference maybe recorded. I would now like to introduce your host for today's conference, Mr. Kristin Hodous. Ma'am, you may begin.

Kristin Hodous

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

fourth XXXX and release the XXXX Andy Albers, will our Finance, discuss going Landsittel, highlights results. that Today financial by well an Officer key business as Mike the for on areas and of year at Chief www.blueprintmedicines.com. on Investors will section XXXX, Boral, will quarter our review You of we press our the Medicines’ Dr. be clinical update Jeff will Executive Medical and, President focus year stage fourth pipelines can our Officer, Blueprint as quarter Chief Vice access website call, reviewing full to slides full provide of and our

questions. We for open your call will the then

I uncertainties, risks, in Risk recent factors, make that filings section implied with SEC. including results and those everyone make the materially the Before forward-looking events remind or those with we most Form could to conference started, set a Quarterly as statements and of forward-looking forth our XX-Q on other any other any Actual Factors Report filed various include SEC of the we or expressed this would may will from on get statements. like we by differ statements result that call

any We forward-looking our disclaim only should be representing update not statements. In to as our as addition, made and any subsequent today revise specifically any as relied of call date. statements obligation this of views forward-looking any or on upon represent views

CEO, Now, here Jeff Albers. is our

Jeff Albers

the XXXX as was vision medicines and aided from clinical Blueprint an programs moved populations. of good distinct clinical to for a which and transformational development with We gastrointestinal provide patients ultra other our excellent diseases we strong subsets quarter our PDGFRα or avapritinib advanced the of expand highlight BLU-XXX accomplishment pipeline patient which tumors, were the closer and data and range genomically remarkable a XXXX. of avapritinib progressiva, the these patients. foundation in We of have of FOP three a concluded at for year fourth to or clinical with our research capstone of continued, in year Kristin, of treatment two severe ultimately to quarter delivering fourth of in for believe realizing results pipeline studies ossificans targeted stromal with rapid Thanks, pleased our morning systemic mutations. We particular the as which mastocytosis updated in nomination I also Medicines in as [ph] served significant to activity achievements everyone. demonstrated fibrodysplasia candidate development in KIT want disorder. a of presentation a rare patients

we update and on patients. advancing addition, in lung active Reporting preliminary that cancers. was trial cell medullary non-small clinically provided of Phase BLU-XXX In RET-altered I cancer cancer in thyroid BLU-XXX our rapidly

on tumor our year are of an candidates financing we positions follow the rare December. exciting I for continued BLU-XXX of for four development Phase and XXXX, carcinoma, ahead. strengthened types six plus quarter the clinical Finally, fourth By advancement us in hepatocellular expecting in end our therapeutic a in diseases. we of progress with balance our in significantly our Coupled with sheet the trial

key these five of XXXX them a are for focus expand achieve including upto expect areas. the upto portfolio we’ll goals to our recognize research programs end of discovery cancer ambitious size, have and nine efforts We on continue our immuno therapy we’ll obviously so our with company under four programs Additionally, collaboration our by we Roche. stage to that to

registration in the initial to The the we’ll avapritinib the first to we broad potential of avapritinib of To and a today, we provide the is Europe. following PDGFRα or mutations. by on of our observed believe end, Based concentrate consistency this population has efforts have on U.S. with driven toward diseases KIT benefit activity continued advancement patients activities.

mastocytosis ongoing multiple from mastocytosis, systemic enabling II and systemic regulatory additional mastocytosis. Phase our regorafenib task enrol in Phase XXXX. population versus indolent forward patient populations trial trial the for and rapidly multiple trials a these fourth, third, trial on Phase trials, GIST in agencies registration First, third GIST and feedback a and including And present in line II III and mastocytosis across studies provide data seek systemic GIST, within appropriate in second across advanced updates smoldering a initiate systemic global most updated

define is to and Our for focus of second BLU-XXX. clinical BLU-XXX the path forward area

we both inform new regulatory with For programs, development along expect feedback data plans. to our

for For in we data of in from year dose half I BLU-XXX the additional cohort this study ongoing expect I Phase second year. in track expansion RET-altered first our data BLU-XXX our ongoing are initial half to then of the present on TKI-naïve we the escalation Phase study from results including from year the the and cancers

Our robust in research to third drug BLU-XXX clinical a we to area our XXXX, differentiated discovery towards platform. of by focus [Indiscernible]. advance continuing optimize trial plan is to pipeline In expand our

plan programs highly addition, more discovery productive also In collaboration wholly we our with Roche. while owned two atleast advancing to nominate

to opportunities. is to growing on fully integrated, us our about only focus Medicines' enable business medicines area our our a partnership as company transformative All-in-all to reach. continue global and profile investigational company. capabilities, and/or work expand With development are fourth sheet operational of internal another a into event evolve be state to development focussed accelerate commercial to looks XXXX strategic of at that hard the to more balance biopharmaceutical the continue clinical year filled learn strong we programs Blueprint evaluate we and Our

the Now, I’d to to activities. call our turn Medical Boral, Officer clinical discuss Andy over Chief like to our Andy?

Andy Boral

Thanks, Jeff good everyone. morning and

of a trial December, the achieved we’ve pipeline across clinical of great proof our drug candidates. our clinical progress lead this success, three stage we across in across concept This clinical on portfolio avapritinib an preliminary on in rapidly the I’ll activities toward our and plan As expand With investigational Jeff assets to medicines the in goal regulatory described, BLU-XXX for development anticipated developing Built the XXXX XXXX. data morning on portfolio. XXXX milestones we program update with update significantly provide preview approvals. an global made of

avapritinib on update in an First, clinical advanced GIST. activity

expanded call ongoing clinical we KIT in navigator the data now I trial we PDGFRα-driven strong quarter, both fourth which As we the based the trial on GIST. in announced Phase and

showing the cohort third mutations XXX spectrum to line avapritinib cohort we and a patient we a broad KIT that in covers the data Specifically, patients added implicated of sub second increased line today about GIST. Enrolment track very Phase we PDGFRα-driven of with in enrolment progressing trial well. I complete to We’ve by patients on are GIST and mid-year. trial enrolled is XX

data As is today. an the we based remarkable this there for population we approval opportunity an early observed previously on discussed, we’ve in believe

addition, plus fully In cohort the believe now enrolled. populations significant patients we enrolment third the both in need line across highlights XXX for Collectively, of rapid advanced GIST. a therapy is new

free patient compare with of trial the track registration voyager in avapritinib primary half supporting on third-line trial to regorafenib avapritinib in first are endpoint we randomized the survival. the Phase will goal of population. the the of GIST broader year in initiate this of III global progression with This Finally,

called data very also Explorer the We expanding mastocytosis, we Meeting systemic the clinical key Annual strong on presented ASH On at in program are now some of based in this trial the the we December. slide, avapritinib the summarize data.

indolent those at can fall to avapritinib had valuable all from servers [ph] of active form Overall, is clinically patients slide, the doses disease disease. response As gives and high in us XX% an confidence lower objective the water see all tested which you in control. explore

dose to recorded investigators regulatory develop plan there in measures Phase establishing in mastocytosis authority were indolent data in and the our the in work advanced strong systemic with in half patients and mild trial systemic initiate a registration. registration well adverse of discontinuations concept this initiate year first most patients in outcome of additional a to Phase We trial engage provide the with global half XXXX and was of small with goal finding of avapritinib no with mastocytosis II with in first to II pathway proof second data to to at additional half and to framework the meantime, to continue ongoing enabling the adverse a part the by of generating a we the recommended In portion of ongoing expansion of dose. combined Phase to events. spends Importantly, the tolerated due Each year. I the moderate enrol goal the X study with

cadence mentioned, disclosures continued Jeff important we’ll of a throughout XXXX. As data have

potent of inhibitor patients our fusions first highly trial from and data off-target to initial multi on cancers. of BLU-XXX the our selected activity. with is mutations. be of key inhibition with of escalation kinase BLU-XXX milestones the used clinical toxicities I RET fewer Phase in RET currently clinical will pre dose believe, RET-altered more Based has with BLU-XXX potential compared inhibitors One we RET data, to offer

In populations levels. escalation reported activity more addition, picture multiple and patient enabling safety durable presentation, responses. across mutations the upcoming dose of BLU-XXX range designed detailed In specifically trial dose of inhibit the was goal a including clinical predictive in with a to we the portion of of

second cohorts PDGFRα line we report sub plan GIST to and advanced and avapritinib, the with the including from I updated for data cohort. patients data line For from in Phase initial ongoing mature [ph] the trial increasingly data third

As trial mentioned, advanced also mastocytosis data we from of expansion systemic the this previously the year. I later initial present Phase to aim I portion

on feedback regulatory In anticipated registration addition, programs. as we we’ll avapritinib global from share updates path to obtain authorities both across our

take support cohort record of of moment in an ongoing opening I in trial a BLU-XXX expansion also pre driver underlying to I advanced the expect half disease. this based plan development trial results including initial XXXX FOP our and year. TKI-naïve on patients new disclose with new to highlight a to in the our data Finally, the their component were Identifying from want to the hepatocellular overall it’s towards genetic treating key of -- initial data part in patients to we BLU-XXX a clinical clinical For advanced [Indiscernible] strategy. later carcinoma of Phase that development relating second

mutant frequencies. trial for other occur at avapritinib GIST ongoing will portfolio [ph] by established that systemic serve to these driven proof-of-concept Our to includes now additional a initiate and forward Overall, XXXX. basket and and cohort with identifying tremendous with diseases been we in a indications. progress across KIT we year great Similarly, tumors RET-driven at mastocytosis, BLU-XXX trial a lower plan Blueprint PDGFRα has to an that anchor exciting XXXX targeting look beyond the

turn the full I’ll over from Mike? to call review the fourth XXXX. our financial results year quarter and Mike Now to

Mike Landsittel

Thanks, a Medicines position strength. Andy. Blueprint financial of continues operate from to

This million. will for we resulted close us and funding avapritinib in with mentioned, near ended proceeds Jeff stage clinical drivers to mentioned. approximately Andy clinical XXXX continue successful enable which in value registration As enabling research including offering, that long-term follow-on just across net portfolio, $XXX.X trials investments our a and of the of

turning and quarter P&L. the for full Now, termination during our of to the were fourth to to fourth revenues compared This million for compared full of in $X.X of million Revenues Collaboration revenues million prior million quarter due XXXX year $XX.X were of for quarter was Alexion the XXXX. XXXX, agreement the year. $XX.X fourth decrease the the primarily $X.X the XXXX. fourth XXXX quarter for year to

million compared During $XXX.X were expenses by further compared the increased driven he was increase for with for for the as period related XXXX. the $X million well advancing compared R&D expenses R&D $XX.X in million fourth $X.X development for of associated expenses. personnel avapritinib, primarily clinical the into million of $XX.X and expenses incurred BLU-XXX increased same for R&D XXXX. million XXXX, the t $XX.X as and year. manufacturing to in full expenses clinical quarter, XXXX year expenses last to fourth This million full quarter we year were G&A quarter to BLU-XXX

including to to spending both For the market due increased as costs. year. G&A as related research $XX professional million was increased relation and G&A increase $XX.X were well public personnel expenses as full in million This in year, fees expenses largely prior compared the

quarter was per loss to $X.XX $XX of $XX.X or net XXXX. loss net quarter compared million million, or fourth a per the of $X.XX share the for Our share for

of year net to for full net full XXXX, the was XXXX. compared $X.XX loss year $XXX.X share or of the or million $X.XX share loss For of a per per $XX.X million

payments and investments December offerings and proceeds turning compared with follow-on our primarily operations. current cash and offset expenses April will the We in our ended of Now used equivalents collaboration cash we XXXX. December excluding and to mid in balance milestone cash, as based million net XXXX operating our XX, of This enable cash that closed potential that to requirements of find our XXXX. CapEx our expect cash And option sheet. fund any to increase us to million investments equivalents $XXX.X by in our XXXX, due from the Roche on plans, to cash, with under $XXX.X to requirements was partially

to So to now milestones our pre-commercial medicines that and beginning move the Jeff With forward building will the to are Accordingly, will expect well quarters, towards we includes to as operator questions. the as the the expenses on our ramp as This operating over patients. investments. and earlier, our XXXX, we we key up accelerate and to Andy in continue both we of commercial for investment similar call manufacturing our now avapritinib. trend executing turn saw as for to supply provide our foundation XXXX priorities I infrastructure expanding clinical to efforts commercial drug for subsequent we Operator? bring in continue that, accelerate as to outlined

Operator

Our Instructions] Sachs. Goldman Terence comes first [Operator question Flynn with from

Your open. now line is

Jason Jakoby

And provide Thanks. reasonable to for thanks last any there, Jason ASCO. Terence. on Can are you initial data would seeing question. just be that Jakoby for on safety Hey BLU-XXX an or expect This you and your at December? the update it update is efficacy disclosure in since on ACR then

Andy Boral

Andy hi I that Yes, take Jason, its one. can

data, be said, say a point medical more the we’ve done disclose this data we’ve year. update So done enrolment, detailed the to as the will full half But, what’s safety activity update we ongoing study we before, at as we in a It I of about data, a to -- from also etcetera. the -- the event meeting before on as as plan coming. data first at won’t Congress plan

Jason Jakoby

Thanks. All right.

Operator

comes Our Andrew Berens from Stanley. Morgan next question with

now is line open. Your

Andrew Berens

very it, for appreciate thanks and guys, Thanks slides. the helpful

one on Just the question GIST program.

have that that door data? Now possibility you that patients, it broad broad the refractory ask line a Phase label, in Navigator Is like GIST. ahead getting second seems any sort to is going of open could label could III label a for of there

Andy Boral

that as Andrew, well. Andy its pick I’ll one

-- and group BLU the really by broad So we driver trial. we forward the do across think be big study be the in line certainly of kind through was on line, data said, to opening in as that subset navigator line contemplate really agencies cohort for We third but based part of should mutations. are be population, the range know based we a saw we I would that second I randomized based we case part similar I [Indiscernible] think certainly second on, see to should a excited a analysis ongoing the and to a set. compound comparing second-line, of need that or paths subset, what the high two to like in really decisions of do refractory approach that those data as with and the degrees don’t a activity bringing certainly part potential driven in And of if study current for the see big the in a I some second genetic activity would the current in would in line group detailed a a specific highly of data has

Andrew Berens

look success for as at we high should what And the second-line in setting? Okay. the bar

Andy Boral

on of be the it be discussions; responsibility can’t high I speculate good will guess durability. a I will with Yes, regulatory that. certainly ongoing product That

Andrew Berens

thanks appreciate it. right, All Okay.

Operator

Company. Eric from and with Our next question comes Schmidt Cowen

Your now open. line is

Eric Schmidt

the us avapritinib discussions little can a systemic you plans Maybe on another thanks detail bit your the for stored setting. registration-enabling design, mastocytosis, the advanced where ended in give have in similar Phase expect updates. Andy design, and with of just to Morning just on something more we and terms regulatory is one II for trial up the in might trial should that you

Andy Boral

patient ongoing get we in on year expect will we so are not we’re that I of SM will course Advanced study package X are validated X ongoing patient the datasets when the and over getting and part then of single-arm speed together population the discussions update full very are IWG Phase working for criteria. – and launch from discussions integration regulatory specific working our those we the a more the that’s assessment think on provide gaining study. out update that We So two separate expansion are what do of and of essential the the reported We’ve of or broad are study. Phase context regulatory tool a Advance think data as addition Phase patient also a a we the outcomes range the be the of the to -- developed ongoing really ahead expanding currently response reported SM, strong regulatory will data. very But outcome of I the plans, study we will in get cover that for study we the definite strong and SM

that we’re So package. those will we think confident make – regulatory strong two a datasets those together

Eric Schmidt

of there Then what clinical of AML you types on beyond on expansion and Okay. others? the other maybe development GIST comment the Can systemic assume XXX mastocytosis. I looking you tumor and

Andy Boral

Yes.

So solid low – a both are kick-off plans a include – mutations KIT key would U.S. broad tumors, we seen subsets that mutation KIT are – would itself the AML as and goal liquid basket is mutation and a both up they routinely basket open-ended the range. And really excuse us out as study in of of malignancies PDGFRα frequencies at and broader in red finalized KIT-driven. across sites stromal, hem a melanoma, turns in Europe and both their and has me, would hem KIT broad who study fairly across we certainly kick-off the and solid – malignancies. in a solid a and shown to PDGFRα that sequence to it be so range, tumors be and a have there find range but get

Eric Schmidt

much. very Thanks

Operator

with next comes Our Eun from question Jefferies. Yang

is open. now line Your

Eun Yang

although much. XXX, cancer. so cell I patients non-small questions a Thanks rate response in very a number XX% it’s have lung on few small about of --showed

for of inhibitor? that’s kind So selective bar think you efficacy do the RET

Andy Boral

expected Again, what example, its would really one a be Andy. good saw certainly But general. is activity. on I It’s can’t speculate in they

would not to I lung which meeting pull that our present common slightly so together fusions clarifying the KIFXB include expect. data does is us will the We point year, one just as And maybe this maybe that cancer, we within misleading.

Eun Yang

And Okay. study, have such dose cell non-small escalation as then your most in inhibitor current cancer anti-PDX? the patient experienced lung checkpoint

Andy Boral

it becoming therapy. It’s a yes, more just mix, but common

So and or any patients who have we we’ll mix accept had therapies. prior

Eun Yang

then question… And last Okay.

Andy Boral

Maybe we can if when make the know. and I’ll you we prior about therapy, let describe any will that certainly conclusions relevance add just we we’ll the data present

Eun Yang

Okay. the question. last And

multi-kinase inhibitor response the So inhibitor, when at you up other there rates have combine products least with such -- gone as with nicely.

that any So, combinations of future? the the in I’m just wondering planning study you’re XXX kind potential with

Andy Boral

will potential study Yes. pure those is agent will decisions data. more single evolve XXX The combinations, development board the current and plan of we include that accumulate

Eun Yang

you. Thank

Operator

with question next Our Lee from Genuity. comes Canaccord Arlinda

now is open. line Your

Arlinda Lee

and for kind indolent, that this information what hold complete systemic secondly for questions. have rate to When your to you’re you looking guys PDGRF, Thanks them. few data? of on later the response And the you continue initial to we Hi. Thanks out of data you at see end mastocytosis, having then I of a year? One trial for taking my planning that cohort start given or in terms mid-year. enrolment durability guide are

Andy Boral

Andy. It’s Hi.

that ongoing that in second-line So on plan the as we initial and we’ll on year it would PDGFRα as on study data data this in both time. fact the study do for later include KIT-driven have update update GIST general, to cohort, and and that well PDGFRα that

coming complete a study the will to look months. forward and that we So update that sharing in

Arlinda Lee

Okay. Thanks.

Andy Boral

one advanced is in in this starting in they level, systemic indolent DeAngelo And study yes, a second dose lot to population at activity as was fact are For the spleen – excites year. And the half the of which size of -- bone mastocytosis study, XX [Ph] dose the we’ve in includes us systemic the reductions, tryptase avapritinib on study indolent about that seen are bringing kick-off planning the based described that lowest barrow, at mastocytosis things the ASH. Dr. milligrams. activity that in we

dose that lower developing mind, later we with at will in the a disease we indolent in study anticipate this start be study. two-part drug the and So year levels

study in finding The first a several explore part MTD. doesn’t be already sequential It since will parallel. dose to escalation defined be need where we've a dose we’ll levels

based valuable efficacy to should dose placebo part-two of response finding on part-one would tools since expect more So randomized identify primary the into forward endpoint are the patient dose outcome be a an that take controlled for by the which will the we definitive study parallel and study be that looking optimal patients at go as actually to not study IWG.

Arlinda Lee

Okay, much. great. Thanks very

Operator

question next from Leone with comes BTIG. Our Dane

Your open. line now is

Dane Leone

you Thank for my taking Hi. questions.

and vein the time? guess see comment how think in clinical your comment like to being a pivoting start us started programs you broader development how could potentially I of bit strategy and just this clinical you're I you’re program traditional Can mutation of your the some maybe I just the took of working changes avapritinib, in with more way up. When least at one if to broader. your in lines on in clinical entrectinib and that going later going seems more how programs program over path give a those and more So, trials specifically like the of the and label-based with It guess evolving by RET a RET approach on FDA that shaped a low that's of you you developing [ph]. you you

Andy Boral

there it’s start-off so like. and But yes, chime you you think can and several we – about Jeff Andy. lot – think that’s I’ll Dane, I that if to question. Yes. in that are pieces

systemic going with kinds in path-forward the can is targeted the to well-defined very address clear avapritinib and GI beginning different regorafenib. advanced tumors of from of there’s that there’s imatinib BLU-XXX just population a a think avapritinib stromal to I in from [Indiscernible] least progression actually the tumors starters kind bit mastocytosis. a For GI At in stromal you then drug

not are be accelerated therapy maybe randomized, lung but chemotherapy certainly not ahead different, is that inhibitors some I starting – setting and because kinase will pick – cancer particular indications approach, would the it and a a most to things BLU-XXX medullary then approved. population. nicely it therapy. and the the large are nice I the keep more that so but really there large, to GIST, to a lung be I would the traditional quickest in paradigm a there’s medicine up best think in drug, target approval in a The established to are We early get thyroid and in in is So of path approval target the ways move picked cancer. But soon. early we on for the targeted that expedited anchor of expectation think with approach, little the be single-arm. the the different. so into There available the knew paradigm the to think really rapid well, randomized just approval its well way we no therapy, approach bit and broadest – number pose patient populations selected of RET an in They

So different than it’s GIST.

most among both expand the had we of have agent. like to want therapy lines So move population. even there’s quickly to we opportunity cases first study there But patient standard with earlier think genetically-based prior targeted basket a chemotherapy. into of the In nice broad to more

we’re I that So think thinking similarly about both. for

RET Just seems broadly for more tumors. already who people patient straightforward to were add, put initially there the -- opportunity where sequencing

Jeff Albers

a just question. I’ll maybe And as a take up you back step philosophical further as opened even and in jump

For every -- and this is Jeff.

that at program to target Then we guide is a is follow every one so second That subsequent the we development when least in the in for the always very data and breakthrough discovery, this we profile the product the early indication. ought there tenant develop therapy designation of a that next path will potential a be is one let the nominate indication. mandates to rapid and us will data. For approach to program, a internally

environment So portfolio that signals in of we the it And essence a program changes we on take we third that based that see, as competitive medicine be emerge, events regulatory program a that can would tone in areas that any then we've in account think component understand earns within is that, interest into vacuum. don’t whether landscape we are the develop growth course about continued macro and/or investigational expand. will opportunity these

starting constantly we always pretend Andy debate to clear it the pressure optimal And have start said, a exactly when test so we program, know and and out as don't we play path-forward we to a how path. will want but

Dane Leone

Great. And indolent I with maybe up could follow one the on smoldering plans. systemic mastocytosis

it more ask trial are colleagues to and the across apalutamide the we also think survival where using that type setting it my directly that where would metastases SPARTAN a by something endpoints was are like were so voyager, the a out a comparison, about free it's going but response indication regulatory you more generally get end-point looking at think what question, party be in of because number something versus in saw indolent explorer, rate --that to little with this we you're of fell bit a a the I endpoint. was navigator doing clinical be trying which the thinking progression pathway when

Andy Boral

here? systemic want you indolent clarify, I about mastocytosis talking Dane, to are just

Dane Leone

will a different. more of as? to that you’re progression into people will trying terms Right. what to little and be disease in defined bit weigh be the going Yes, a aggressive prevent to treat Clinically because the it’s endpoints clinical obviously form of

Andy Boral

Now don't is at response different there indolent for at problematic least systemic measure There sure, bone And Mean, of the currently and a burden, and least – doesn’t the evaluated traditional mash that’s well nausea, and actually more way disease fact. blood activity. bronchospasm, mastocytosis no than holes there. have. makes to it the patients the the response disease array in or in that any the amount no and tryptase is will constitutional been they isn’t the to correlate symptoms really other the of be are vomiting, rash don't actually sense pruritus, well the GI, to disease marrow broader malignancy of defined. in what correlate and in amount and

actually this I measures outcome the as be so and of a about – well in of ready-for-use primary that patient way year. tool a study this to which these in validated be developing [ph] will validated mastocytosis tool impact in start – we that corporation a will incorporated indolent the developed describe think of it this study patient will we’re the the half symptomatic and in talked reported on be second endpoint our started I systemic PRO when on a having And the are in planning, the we’re specific disease way disease we

and So practical the its so it’s actually or patient to not progression endpoint, from is progression rate probably that’s disease it symptom. offense, only not even indolent the around really – by about slow of pretty to quite improving time advancing that doesn’t all happen

Dane Leone

squeeze Great. one one in ALKX If on last could I program.

about a – question scheme recycle going is there asking specifically? you’re forward we to nominate program clearly I’m So you program program. different how wasn't just opportunities how ask, other -- back, this at That first you I think Is is FOP, in broader moving took essentially obviously programs. isn’t that the should but with that of ALKX that the philosophically this

think through clinically right put something You to say, test it of this broad going methodology Is yes, we’re it? way opportunity to actually because move vetting the to set. with at is your about forward we a see the back

Jeff Albers

about evaluated Maybe on and path. question how we So, can development weigh in I’ll Andy part second that this the of is the Jeff. clinical then take

capture you was think cut well. business back When from rights retain transition. we first I Alexion, that order the complete So, the of that to

and if piece was bar, the at expectations time. weren’t We data but those highlighted profile hit that Marion rights second agree a Dorsch, The this that to. preclinical evaluate sure not we’ll back that our product later that we the target candidate development and that’s did look only testing would year. that had our When last call. we molecules to we Alexion we’re Subsequently up share with on as to end that the treated we CSO came for

success. what’s that was of when felt Andy What’s program vis-à-vis an opportunities, then hand other treatment promising funded we maybe the with like path most that forward. program do years all also looked you the evolving programs, bit underlying clinical our the out into was we said, we initiated we evaluate And about a same development with first frankly like it the and data out of all development on we path? promising driver wanted with two it of potential on opportunity, programs, has back. one in was that landscape opportunities, felt quite approach to thought a the and And of terms very we accelerated There so the is fully the this at think put that that approach. a which to in weigh with eye since development couple of targeting a you to disease we the we the BLU-XXX probability how do and And process, And mutated going available path.

Andy Boral

Yes.

of impact think only rare the are And said, Jeff can fundamental the ones we that’s that. to that as we we we the with mutation point selective targeting inhibitor likely this do a the certainly the is most have way a that that highly least at So genetic on know driver disease really think disease. huge

of the way efficient now with study. that effective the measure about understand the hard to to most the the develop So to the development fields to in think most in path setting effective – the disease, ways best working develop are experts most to we variety

about we’re excited all FOP. really in the opportunity I think

Dane Leone

very you Thank much.

Operator

JMP question next of Michael from comes Our King Securities.

line is Your now open.

Michael King

say I questions Hey, of anything guys. all had about gotten this a few just dose the sort number point of about questions. XXX squeezing you’ve me – I'm on to-date? obviously we’re Thanks know through for the if excited that congrats of guys very in can at I progress just lot cleanup levels it. you of don't I couple have throughout XXXX. and of and

Andy Boral

do going of second It’s and quarter year will just present is until up coming you past, we’re have Yes. the It we’ll pull Andy. Mike, be [Indiscernible] if together to It’s the to data. everything what done say like the in this then we’ve -- really presentation we'd anything just we not as

Michael King

chemo you this that Andy, everybody But, there In to were or questions, the any on in there words, at for it? either – patients chemotherapy? Okay. answers say naïve based refused ineligible other round previous are can in just safe least your gotten have its that to of its study will one prior

Andy Boral

Again, we’ll as get there. that out we’ll – get we lay

normal expect to non-small have lung that have cancer. have You’ll they type cell essentially chemotherapy

Michael King

a the techniques the what on the then to understand – I landscape. for with are the Thoracic tissue. a try in variant Oncology. that J guess are used RET based positivity. something RET would work And company the landscape, a recent was you article fusion about come would standardized with They KIT patients, patients? was me diagnostic these interesting archival that or And IHC to various that there in like to frontline confirm was patients to talk now Okay. either would study RET And up have diagnostic But test QPCR to

Jeff Albers

Mike, Yes. sites have we so there are all all, testing, on of robust first that based the allow going their -- use assumption local RET we now patients Our testing.

are there you As it. approaches to said, few

for at be regulatory the development, or other will but We of field a change also partners down going not. and tumor needed road. rapidly regulatory in to we’re look even its an Whether just systems what planning are that evolving whether from perspective at looking CDx we’ve whole done all sort had a to in a CDx patients as studies we DNA a opportunity,

we’re in approach how that going changes. as very certainly but point CDx, we inflexible need Our at the this assumption terms is that we’ll landscape of the

Michael King

Fair enough. Okay.

Jeff Albers

one ready be We’ll if have to certainly needed.

Michael King

Got to other have you be it. fulsome And present rate data that wondering, when more XXX, would overall be data talk then TKI or would a about endpoints it Would naïve can data you if again in population, you what points just present. on the response ready second and later? we could spend you just like PFS

Jeff Albers

Yes.

be progression-free again it relatively now new I the data that will treatment would were the patients at naïve number – that we’ve talk we cohort to to on describe what of reported be be be things It a mixture. would will in the so can see it other of than like the the So saw for it can at think we to we update that naïve similar survival duration do available But is time. the it point of the durability The focused we rate study, is will similar done TKI that in I time. response on and we as small before. than mature less an what TKI what and more and mean, degree duration that -- cohorts, so

Michael King

for, right the about if you that talk that going just far out And a be maybe to molecules? us little Okay. could about I first of tangible I a going about you we a mentioned This clue might Are more is Are Jeff. tangible, talk have maybe Anything closing results able come give we time you having else question Can to tangible see publicly we now? that. to might agreement targets? say what evidence guess as Roche as guys or collaboration. of

Jeff Albers

Sure.

right approval the and wanted disclosure add now consistent moving approval, that. of Roche’s along of is makes complexity. we level before. to so That collaboration so there highlight feedback is a the So, we so that made two unveil progression to any the now require nice a with well substantial we’ll up that that talked but with signing when obviously to programs quite guide of It But agree have the context within – that we collaboration were early a moved about portion and what those both efforts, our and of not programs be. we collaboration. to of might initiated the time time we’re our Roche discovery Those the at had very find

Operator

from question next comes Chico James. with your Laura And Raymond

Your line is now open.

Laura Chico

for question. thanks guys taking Appreciate it. Hey, my

encountered have I And you’re patient agent envision these just about about we these any you I guess guess back another us I’m and you as a I if I could follow-up. sequentially XXX you those be could guess switch lines wondering, one in that And which data more a curious sequencing I’m wanted of guess DCC-XXXX, in I’m of I how cross might I be have guess agent resistant to up I potential would along about thinking speaking use progressing should development I remind in the guess or agents? other agents then And over treatment, earlier of lines So to therapies? specifically on scenarios profiles GIST. in

Andy Boral

questions. with that Laura, I’ll thanks the off start for It’s Andy. one.

step KIT, and different molecules a a the binds is and selective take while can very less PDGFRα the to while to very the few is inhibitor, KIT, far really want binds remind as everyone of DCC X the maybe other compound very we inhibiting on binds PDGFRα much active and the form molecules, that as the selective tell Kinases So, form. so we and much this and KIT Type inhibitor to to avapritinib inactive really I have approaches back to DCC minute focused that just to hit

the in both I it's where like just I actually to resistant. line that population if from the late perspective, think unlikely two So think that that GIST that basic cross strongly probably have seeing, the I expect were rate are percentage results but a patients. percentage, in at seeing compounds reported not response they same activity probably -- not were similar would you

help is on by that after would particular two the for would we that I can I data that just would avapritinib, the drug than in I other, true. that actually patients that So one hope have expect think this itself either together be and would -- drugs more work

at this activation DXXXV totally is is characteristic covering KIT the in So rest a as distinguishing on as well probably the active mutation real team. DXXXV avapritinib PDGFRα at KIT in of

really DCC not active So, which these quite from is drug distinct the mutations. I think activation on

opportunity if an offers for resistant cover would mutations away an they there right wouldn’t. that that we activation is think I distinction arises and that So that

PDGFRα Another extend DXXXV have it sideline an GIST subset patients opportunity mutation. probably that think not in almost gives also I mastocytosis to really to avapritinib compound. available the of that And to DCC to where all that is that’s

them in activation PDGFRα [ph] have potent would I there. mastocytosis activation – systemic would and these see So expect and you I won’t don’t GIST inhibit we activity if mutation you if you like expect both inhibit activity

So two they and profiles will I different think have compounds.

Jeff Albers

Jeff. currently, or Andy treatment molecules better this will our paradigm And that’s early resistant us these it is the would maybe of room level then is term in for I And for are comment, available paradigm. so in observation with I both think perhaps therapies certainly speculate Andy’s change. hope too higher we to no. agree of sunitinib after later And take piggybacking line matnib, the the than improvement up cross – that treatment A I’ll hope the of and initially regorafenib think and from leave set,

earlier, think but let said we I in treatment paradigm. for data of As I guide the opportunity there, us significant there’s change

Laura Chico

That’s also Okay. then, helpful of follow-up related XXX setting. in Thank perspective. you. kind the my question And GIST guess I

be guess, of I have I and You in therapy we enrolment mentioning little registration probably mentioned breakthrough concluding there. activities if year-end. update get commercialization about more can kind by the expecting what the an XXXX wondering designation we’ll you you ask before say here. a preparation And mid bit should And I’m

for through career the run not for I’m on at commercial this just a openings; related seeing many brief point. functions think listings website I the

kind of I right up where and I'm wondering So might guess of seeing now? just you more when build we anticipate a at

Jeff Albers

This that. is Jeff. I’ll take Sure.

opportunity this by update later year perspective. one our we’ll an plans more fulsome on from a So, find a provide commercial is to

be these a our pre-mature to If I But are a position start it’s PDGFRα, there are a both you positions. funneled in sites. is Europe lot U.S. academic indication a think I bit don’t and first of that a We much think lot the with on. generally of too that to seen will would early in patients guide that notion regard. haven’t

we manner, about of actually our that targeted therapies, that the in commercial type can as -- as molecules can so that think should very And mimic build nature. the any a build occur much it's targeted precision in be

provide but are for moment. laying that team. have commercial extensive Kate Kate going the Haviland has initial hires experience. --- it we her Business subsequent of folks So, are that the of board over some groundwork be there commercial this will officer year under greater both this at plan. Chief and details small not commercial our we And certainly and Currently at there And to looking myself on on course

Laura Chico

Okay, thanks guys.

Jeff Albers

excited though. it are We about

Operator

from comes question with next [Indiscernible] Citi. Our

line now Your open. is

Unidentified Analyst

All right, thanks.

of extends Just give extent of your in thanks. both patients beyond us be Could on cancer, a as in the the thought, RET-market. RET-market scale the that and market quick you just question a lung terms to that thinking prevalence patience to sense on interested latest and I’d non-small your incident, the and cell thyroid

Jeff Albers

Hey this. I’ll sure, take

in analysis it evolve of and have occurred see those in and close of look provide line of yet medullary you around some to cancer, around so range across mutations at of that of So RET-fusions occur that overall as major obviously reason is , it’s and In but primarily are if broader Japan, in to roughly at XX% lines more. a haven’t you which if see treatment, patients there non-small are go, the to upwards clear done [Indiscernible] guidance the so some there it’s estimates any as that before mutations of done thyroid on is if XXXX from non-small at at cell it’s specific much estimates EUX there we basket, are you cut little a depends provided we’ve going a the perspective, markets rate, but lung higher we extend in a X% you total. occurs but from XX,XXX wide well, occurrence mutations the to it a defined cancer that frequency we to a as solid we [Indiscernible] or type patient major we you X% anywhere population a XX,XXX guidance we if to data smaller then And that tumors XXX patients occurring others our patients you markets bit take estimate that U.S. in terms how front it there estimate cancer number. that cell XXXX of the look range that we lung

Jeff Albers

the it prospect case the some RET represented panels fusions for can regarding are on the that there detect is foundation Great. and the panels are thermal mutations in still [Indiscernible]. panels, some, major RET And the the the what meaning currently there gaps of gene are the alumina or

Jeff Albers

by they testings available Yes, while the the RET certainly fusion that’s done covered be And can by and individual well can by panels. be mutations are – they tested. specific the available

Jeff Albers

All thank right, you. Great.

Operator

over to further At questions. back I’d this Albers Jeff time, I’m remarks. call Mr. showing the to like for closing no turn

Jeff Albers

operator. you Thank

So obviously critical if we continued and you so for we in evolution time interest. well moment back, rounded set a medicines. questions, Really all had taking believe a appreciate very you your a of of Blueprint the take continued the step

platform capabilities. robust with proven a have We

a differentiated research and committed and stage have at and We deeply a have hard is working that we clinical improving patients. portfolio, team of the broad lives

with currently, of ultimate strong combined our biopharmaceutical patients. transformative, integrated targeted medicines And so fully delivering and achieving a clear which position a this to our goal, financial genomically foundation gives path company us becoming is

your that, continued with conclude thank So and bye support you interest. we’ll Bye, for the and call we

Operator

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

Blueprint Medicines (BPMC) 21 Feb 182017 Q4 Earnings call transcript