Blueprint Medicines (BPMC)

Kristin Hodous Senior Manager, IR
Jeff Albers CEO
Andy Boral CMO
Marion Dorsch CSO
Mike Landsittel VP, Finance
Terence Flynn Goldman Sachs
Andrew Berens Leerink Partners
Joseph Thorne Cowen & Company
Laura Chico Raymond James
Arlinda Lee Canaccord
Konstantinos Aprilakis JMP Securities
Eun Yang Jefferies
Call transcript
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Good day, ladies and gentlemen, and welcome to the Blueprint Medicines Third Quarter 2018 Financial and Operating Results Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions].

As a reminder, this conference is being recorded. to host like Kristin today's now Hodous. would introduce for your conference, I begin. you Ma'am, may

Kristin Hodous

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

Officer, our be the access as Andy on Jeff the for will third we foundational discuss results. of will preclinical well will a Investors our Boral, Today Mike press Officer, Medical to data regulatory for slides Dr. Blueprint business going BLU-XXX; BLU-XXX; will our Finance, as Dorsch, update reviewing at release the Marion XXXX by our of and You Albers, can Executive today Vice President provide www.blueprintmedicines.com. quarter financial Scientific discuss highlights; Dr. our Medicines' website Landsittel, Section Officer, review our XXXX Chief Chief third that our call, quarter Chief will our

then open the will your for questions. call We

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

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

is Albers. our CEO, here Jeff Now,

Jeff Albers

success. Blueprint we Medicines, share of transformation rapidly updates an diseases. the cancer reproducibly our Thanks, with and to At integrated development Ultimately, good efforts deliver highly on of themes. to cancers, delivering precision patients patients medicine This and updates platform, take design our growing increased closer portfolio breadth and strength number biopharmaceutical clinical rare capable to focus morning, to to aim this Across with including is will precision across scientific of to share and and aim immunotherapy. updates medicines our of everyone. to plan We key demonstrates and rare we genomically we'll safety four of our potency company on kinase expertise therapies worldwide. with business. probability morning, to improved areas important our diseases, a an Kristin, By leveraging these build Today vision. key core cancer us that focus we selective defined mission

multiple approvals quickly patient in and advancing we're towards BLU-XXX avapritinib First, for potential population.

registration enrolment announced, This previously of for that year. we this Advanced As and earlier completed for we cohorts patients with and called study patient advanced also morning, indolent have our designation pivotal systemic and reinforce FDA SM and the in expedite feedback that Systemic to Mastocytosis, initiated mastocytosis. of announced designation treatment FDA breakthrough study PDGFRα We PATHFINDER. our and received GIST of advanced we for the both in avapritinib development therapy GIST have believe avapritinib screening breakthrough fourth-line we plans

now for path BLU-XXX on believe, the in NDA on for plan in to we ongoing half detail morning BLU-XXX submit RET-altered have Andy ARROW these discuss and in an call. also clear FDA populations a expedited feedback based study more will of development on multiple in first the cancers. written We this strategy XXXX. we our plans our received later We registration proposed announced we've patient that for

five to by additional have the in of expect the ongoing for GIST study, to together enabling we patient completed and the year. addition populations fourth-line all PDGFRα end studies Taken registration

Second, served we're of efforts. effort. Unit most for Sanofi an and was In spearhead the XX medicine Yesterday Chief Commercial commercial to as building North delivering on has including Genzyme, team our Officer will to in lead years building Blueprint joined appointment America. recently the portfolio where announce from of focus Christy the global executive Rossi as commercial enterprise patients. She will a join Multiple leaders of Christy from Business Sclerosis great organization. role, this our who a scratch experience Medicines with of in commercial biopharmaceuticals and new commercial specialty all leading Head expertise integrated a two key she

In innovative Paul Beresford, Shire years diseases. where President Paul Manager, addition, access based rare launching lead Strategy and for European He Lead to has experience operations and products, market oncology of build he commercial and is Vice was including from operations. specialty Paul and international the international Product efforts out Switzerland appointment we as our in experience. International. and will General XX of announced joined us Global

the is ossificans realization underlying we of potently next to walk foundational clinical theme novel In third nine under cancer to of immunotherapy bone mutant which design we cause XXXX phase plans progressiva, research programs that candidate. that is therapeutic data become few and In these with initiate a research data have Roche, the preclinical development the By and BLU-XXX disease. Our programs for collaboration devastating expect our moments, through fourth up inhibits a end of the our will our including we BLU-XXX show year. expect five nicely. clinical our which Marion of with vision to These therapies. data rare progressing September, out to soon rapid of BLU-XXX the fibrodysplasia reproducible presented continued targeted to

our look to portfolio next As sharing forward mature, continue plan. these the programs of phase to we

important throughout of XXXX. Finally, as today, a fourth expect continuing end the to have theme we XXXX, and catalysts before

patients fourth-line robust Next PDGFRα report the GIST Tissue sharing in with on Meeting. month we GIST anticipate from the data update Connective and Annual will Oncology study we advanced at NAVIGATOR updated driven Society There a GIST.

as of population end will duration these U.S. response potential the these points focus accelerated the rates we for in we'll Importantly be approval basis anticipate a on response in and

patients for share second-line In a GIST. third-line with and data addition preliminary we'll

conference the responses limit on to GIST-related and the proximity the Investor call Call, questions corresponding we'll to today. any Given

to to the lead Overall, all efforts. building critical our a to on across We BLU-XXX. update programs these also registration we're abstract next-generation particular, now presentation we that advanced accelerating towards operationalizing three December. and was for as to ASH focus the we to focused we to authorities for is year enter sharing provide XXXX, clinical all avapritinib submitted the medicine Pharmaceuticals, share to collaboration long-term more including our more Andy clinical program. CStone in the value call continuing BLU-XXX And advance programs. we're look on five additional commercial health data publicly across with in SM our infrastructure believe collaboration precision of IND enabling NAV our on of progress our therapies. continue across BLU-XXX oral showed recently portfolio. for to In an to on forward Chinese I'll data we prepare stage clinical detail We to we patients, Andy? deliver and building Under recently at abstract available. studies, executing in our the accepted turn the In once learned expect information over significant that

Andy Boral

therapy advanced an RET cancer. on targeting in potential on mutations. Thanks, including RET-fusions recent for precision including Activating thyroid XX% update medullary our XX% FDA registration BLU-XXX Jeff, about been feedback of BLU-XXX program, papillary and potent oncogenic resistance RET XX% X% thyroid good to with lung excited identified have is of to and morning strategies. alterations cell of share designed seen non-small everyone. I'm to been about mutations to X% up and patients cancer. cancer, of have selective

XX% rate were dose focus the milligram XX% all response data were frequencies Association the were of a types. These with patients medullary response that as Phase with month, the wide RET-fusion of with In XX% patients cancer. initial data thyroid range and at of The and cancer thyroid was it occur data at ARROW was this dose for we XX cell dose RET activity the positive escalation from clinical in XXX non-small recommended American who medullary patients for rate was study Earlier dose low and cancer in tumor results more least patients. levels. in of a we across published from RET cancer patients XX% dose have treated reported shrinkage in cancer across weeks. thyroid the April, strengthening tumor BLU-XXX of In treated patients papillary alterations These addition, at with for a groups BLU-XXX. the portion X reported XXX have new and shown once other Thyroid the data longer thyroid lung on showed overall daily trial Meeting Annual durations.

with recommended remains rapidly In of of date further addition, these plan With the to in results, with enabling data enrolling goal first ARROW on half registration the treatment datasets patients FDA strengthening consistent XXXX. all we by cutoff. see with all goal recent dose generating the at this data, treated responders Supported with ARROW the data Consistent are we sites updated we of study X these of as mind, expect from trial worldwide the time. a and clinical feedbacks. over the the to Phase in report of

Based We we BLU-XXX ongoing on for on is last including from cancer, will indications, papillary with positive NDA anticipate believe approvals feedback, this that following on for therapy. the with first we RET-fusion and today a include thyroid are response studies. regulatory plans cancer, the rate XXXX. additional of duration several we and systemic FDA. authorities cell RET-fusion consistent thyroid for months, the the prior received With regulatory cancer, progressions the precedent feedback will announcing Over RET-fusion pathways have of overall single-arm to expedited in study, application include with several all the oncology key written and drug medullary positive engaged new sufficient potential which follow-up, lung submit filing ARROW based patients endpoints half from data registration we with response non-small clinical with BLU-XXX

population include to agnostic a all opportunities with we cancers, to cancer there and These Importantly, intend of of RET-altered broad think lung for RET-altered RET-fusion. development significant BLU-XXX. may treatment-resistant and long-term outcomes thyroid acquired therapy and explore Altogether patients treatment also tumor across an cancer cancer, RET-altered cancers. frontline we patients lung with are with longer-term BLU-XXX EGFR-mutant across combination a the opportunities for medullary improve short for indications

over BLU-XXX. the for turn call to will the Marion preclinical updated review I to Marion? data Now

Marion Dorsch

Thank you, Andy.

I Before update let discussing by overall our on strategy. research BLU-XXX, an share start me

product we We provide our strategic our that as identification leverage at to our to Key are clinical with drivers points coverage. is the for highly Chemist Medicinal assigned design and into know, was expertise selective Blueprint core kinase library therapeutic candidates. kinam lead library vision. success Medicine, candidates of either starting discovered we resource in nine evidenced the mutant immunotherapy therapies. which precision interest. FOP. our muscle by Ossificans skeletal our the drug our function, of were very of points of collaboration. of Roche we or five by severely us childhood, or our and using to have rapid to as worked the by ALKX, disease, scientific rare extensive same progressive ligaments that optimize Blueprint has year, cancer then strategy tap and you Beginning we characterized identify internally or to ultra based applied all platform and This patients genetic disabling a loss to disorder the experience our kinase-X in As Chemistry the of to bone, Upon joint capabilities our of starting our including result premature FOP underlying developed and ongoing concept to target of the kinase by death. is proof approach for ultimately this well structure spontaneously up achieved tissue proprietary under a end designed tendons abnormal on of of the Fibrodysplasia trauma. expect physical research Building This activin-like programs Progressiva cause to expertise, disability, into is transformation connective early BLU-XXX to programs into up

challenges. recognized long underlying FOP, technical has has a the been to efforts failed inhibitor as of ALKX persistent prior due develop selective mutant ALKX While cause to

it off-target to the target selectively without Specifically, other proven drive has members difficult ALK the which ALKX inhibiting has family toxicity. potential to of

XXX vitro preclinical compared allowed disease. In kinases library have at compound some family in these pleased guided ASBMR the us to the challenges including more top-line show that to and included for ALKX, were tremendous proprietary BLU-XXX of foundational we ALKX, this ALKX than Slide the was ASBMR. of Our almost and the to X,XXX overcome historic we in ALKX. studies selective ALK present potential Chemistry to On BLU-XXX in expertise other and Medicinal data this showed at structure BLU-XXX presentation to fold data in speak

vitro a I band a inhibited BMPX. genetically the in ligand, A, Additional In ALKX data mouse showed studies BLU-XXX including vivo showed potently of inhibits Activin regardless model. B, activating bone FOP that to video accurate This injury the in BLU-XXX activity muscle your draw in formation treatment or upon Ossification BLU-XXX. particular models surgery. Heterotopic to showed In would shown in of attention addition was that and of bone clearly like with

strongly initiating pleased BLU-XXX. repair as these the think incredibly ALKX support potentially data are therapeutic injury. We of and development and because bone inhibition a were see for multi the also of tissue validate We clinical to important they FOP encouraging strategy fracture following

goal we flare. to in trial a approach regimen following dosing Right acute associated pursue a Subject now, up the be may disease submit IND to of we are of advantages continuous flare the first to Ossification quarter preventing FDA, clinical initiate think by plan volunteers over the not plan we X a of offers intervention year-end. by always IND the a particularly healthy in because an approval for Phase application progression. in XXXX. with Heterotopic with We working BLU-XXX patients Importantly, to disease this

plans In of trial in addition, global accelerating with goal initiate of we the potential are opportunities with Phase FOP to X expedite patients development. identifying BLU-XXX to a

other progress, emerging our results to our over the across turn updates BLU-XXX Mike third financial make stage to I research review call portfolio. programs providing will continue on now look we and to forward As quarter. the to we for

Mike Landsittel

Thanks, Marion.

in of million us which as investing with our to resources We continue quarter decrease continue in third million cash activities commercialization to connection clinical received million pipeline, ended compared infrastructure while strength both achieved The operating $XXX.X allows financial primarily potential XXXX. entering related $XX the collaboration, collaboration, the We partially of in XX, fair candidates. building in occurred position milestone operate million CStone under December product and in of from $XX XXXX. $XXX.X cash the support payment payment the used the the was a of Roche offset June by also of to to which our with cash the upfront into of growing to

of this that the into the necessary We capital fund XXXX. to operations continue to believe us provides our second half

XXXX. G&A revenues to Turning million XXXX. we in the $X.X in recognized primarily This of decrease XXXX the termination to agreement P&L, of third the collaboration to compared million quarter incurred due of the third year. prior With million R&D the for $X.X the Alexion increased million of the of R&D expenses and avapritinib further trials of with were personnel-related largely personnel-related exciting XXXX. clinical pre-commercial or third compared expenses G&A increased have $XX expense expenses. quarter increased expenses was of for clinical for the as XXXX increased associated professional planned Blueprint studies. to in is and quarter planning as million well including This the activities. for $X.X five registrational fees now advancing to as manufacturing XXXX third $XX.X expenses, quarter period expense primarily to was was we $XX.X respect time same for BLU-XXX we by increase in in due to an and compared the through ongoing in This This million driven in increase operating quarter third

the preparing we first the are We half NDA the and for of our commercial a are both actively U.S. in groundwork filing laying and for first infrastructure in XXXX Europe.

fully activities call operator biopharmaceutical for now in will expenses to will that expect before, guided and to as that, additional With the we devote the these increase transition integrated future to prepare resources I becoming company. quarters turn our important over continue Operator? we the we've a As for towards operating questions.


Instructions]. [Operator you. Thank

Our first Flynn Terence Goldman comes from with question Sachs.

Your line now open. is

Terence Flynn

to and CTOS, question. the was I avapritinib and taking coming X any line Phase trial and the more you with announced for trial to the that's GIST or this that the just thanks enrolled that's on respect GIST Jeff at can the just data wanted on second-line progress. CTOS the Hi, point All-Comers there second Congrats that Thanks. will third share that confirm design? wondering present then Andy assuming at details

Jeff Albers

I’ll question. take Jeff. that is this So

third the and will prepared focus that Call any remarks mentioned while we're second updated around the line as the opening Investor detailed questions So data I've CTOS. in at at

We exclude CTOS. at call or separately. will the point say well will we update and we when second likely in terms assuming out out them on provide third you then, And All-Comers mean as you that PDGFRα, line Investor and as will Call development that I'm plans, PDGFRα of KIT-driven


Partners. comes Thank you. Andrew from our question Leerink next And with Berens

Your line is now open.

Andrew Berens

on guys. and Thanks the congrats progress

I the in after the empty but Loxo XXX and regulatory question glass indication. on update and an has discussions the breakthrough on you what's is -- a had say a guess us to I Just honestly hate designation given same half

give for just you on that can So XXX? some us potential color

Jeff Albers

Sure. I This with that. will is Jeff. start

information as start with most avapritinib, with important the be piece accelerated we from of perspective an we approval. always to establishing it path our So have

So start then path. therapy and view as designation that tool to we and ensure the regulatory with we conversation feedback around those breakthrough consistent

plan. potential the with help now have that ensure therapy we something on to from execution that the breakthrough effective feedback we FDA designation could So explore accelerated a be would path, written of announcement that

and whether well. as think we consistent glass very take we in that half works found we done is So what we've glass programs with it full this it a empty, or half other

Andrew Berens

you’ve what I then means give activity of insights guess that just Mutation, seen? some as Gatekeeper that the to this mentioned has for can is XXX us you question And what and a Okay. you've Andy,

Andy Boral


fusions, as the that point unique this against potent the across the is of one mutations And activin has hope about -- our well as the activity is translate expected prolonged durations XXX Gatekeeper into several truly of equally is So of that mutations think the will at the resistance very range now various is variance. we things response.

So we other cause multi-kinase inhibitor, the should resistance, first durations pan inhibiting and by come we clinically. we that Mutations RET that longer could off think well so as will of have -- this of think apply out up to they show could to the but will broader primary of that with Gatekeeper response be that, range to quickly, inhibitors that --

Andrew Berens

XXX sequentially you inhibitor? the it use another Would also develops patient allow potentially as the resistance RET to to

Andy Boral

perspective very potent No, is used think inhibitor RET best on a selective I that first that I the that mean inhibitor. is as XXX our and be RET


Thank comes you. question Thorne & Company. from And Joseph Cowen our with next

Your line is now open.

Joseph Thorne

there Hi and question congrats you the taking and on thank for progress. my

And regulatory you for on Just that for in additional is can when how follow-up there. to waiting study additional or from what simply before that there for we ongoing the that's to Phase patient in and thought plan and in is you look what file X -- ex-U.S. will or results application, to filings need the information GIST subsets from the go be fourth-line a needed order Europe? PDGFRα yourselves just the done ex-U.S. I guess couple on AVA remain these there file studies process

Jeff Albers

Jeff. is can this question and will add necessary. that color take as I So Andy

for what part filing our the on an remains GIST. in the question be PDGFRα So first fourth-line first plan and of NDA for of half U.S. of the done to

and is that sufficient follow-up any response and with of approval, this rates around announced and so the the year components data of we of half in we as accelerated fully enrolled As duration first previously response. one

together step then the accumulating the all then pulling next for filing. that that it’s is -- but process, a just it's then So

within all in give importantly then cutoff of our -- I then a will will soon always NDA plans, of from as what in sense Europe. filing. few previously file a or data the is plan announced of that of thereafter plan all recent first we're and that the a at presentation we're but will the it's that cutoff we’ve international at And CTOS to be weeks recent component of that better we’re you a data think certainly of then what why an so MAA midst file looking and second again we will include been So the basis the the


you. comes question Thank James. our Raymond next Laura And from with Chico

Your is now open. line

Laura Chico

the just XXX. taking down today you if now like with the have I the to regard I little for will Good primary you kind see see requiring just on And Andy you're secondarily is so elaborate key on a morning, mentioned bit I Jeff, differentiation progress? the thanks for terms refer submission update follow-up, gating we as approval you’ll think last or And and be sounds what other one question. regulatory then use emerging question on are before guess to annoyingly that there XXX the we point with of heading of necessary similar factor for what a you as the it could to path, elements similar complete? BLU-XXX, accelerated there to and you that physician XXX

Jeff Albers

why you All that? Andy right. don't take

Andy Boral

So for question. thanks Laura the

compounds for study really it tolerated the set, and then dose follow-up possible main the in we two enrolling to any accelerated regulatory I rapidly of approval focus and study, there with of will other shown recommended a are the X our are the so is build do the on evolving and is accelerated that is differences, focusing enroll the think or patients are happy to that see far. data the past, we've meeting dose and we patients BLU-XXX in that We as small as out have in in inhibitor they path very data approval selective at on Phase this So very time. be around initiated if potent ACR and continuing limitations as rate needed terms time at what aren't really think similar maximum conduct is specific, there we the the

Laura Chico

route the just be moved one you. you? second program, rapid thank FOP exist well And recently in administration half on could be also one about FOP? for of Okay, quick will of should they the sneak path thinking then, Clementia I And we guess would of the therapies that a submission for move towards how I NDA market to also in towards moving potential XXXX. suggest if an

Andy Boral

be So deserves developed yes, is Vera taking we that that for actually the a is as will other multiple see likely and credit an I Clementia so clearly and think activity it's good it think compound team disease just will ahead approach it important some we're Heterotopic such be oral us as that would -- rare be of we've we're it's agents about we always about the I agents important one -- developed. actually being helping various important think define progression learn understood we is FOP targeting say BLU-XXX FOP. goal for could see role developing think in continuous that compound that I and has important Aloe the an as on treatment we particularly playing that selectively paths treatment do is potent think think thing not -- being of is a differentiate of because and disease, think -- BLU-XXX BLU-XXX for cause the with from very flares with for Ossification focused oral some clinical for really defining progresses in offer the regulatory to we disease, of as flares. disease very do we between which out underlying we that but by the potential to to an preventing

We that be think important I important to flares distinction. with will treat setting chronically and an it's acutely think, so think only. we not in


Thank you. Instructions]. [Operator

from Michael Guggenheim next question Securities. with Schmidt Our comes

open. Your now is line

Unidentified Analyst

[indiscernible]. for I in on got [ph] calling Etzer couple Michael. Hi Darout mastocytosis a questions

to First what look to points? and would within [ph] in to type improvements be clinically your constellation, reality a delivery mastocytosis significant begin like what in lent would of the specifically view end smoldering process be symptom in particular sufficient

Andy Boral

back. step I'll maybe so Yes,

So to contrast course avapritinib have systemic mastocytosis rate indolent be And patient In tool advanced of developing the which primary case patient -- a that that end similar are mean I IWG that’s approval advanced to indolent outcome primary in reported that developed have disease in have -- criteria. driver both we will important point to primary we in outcome be primary be tool and enabling using developed -- to specific and year we of we do an registration the off systemic point but but be study. approval disease mastocytosis that kicking endpoint validated in indolent the for tool reported is mastocytosis. will slightly is be where latter study of in that will don’t we population. and specific different the again systemic a we're the expect response the indolent indolent the disease in we a anticipate the part study the in secondary we end

placebo haven’t we Here would really to we think will we would be we comparison details targeting and expect approval. differences. would the differences or gone into be publically what for be about needed But significant

Sorry, did I miss the second part?

Unidentified Analyst

product in existed systemic thinking within how well now an would potential the [indiscernible] frontline the tumors what treatment with let's as No, [indiscernible]? mentioned RET-altered that entering that's in the you with say you helpful. And other also about [indiscernible] that BLU-XXX, evaluate regarding now a as

Andy Boral

drivers -- clinical agents like quickly think that’s to the I RET as from highly move perspective, therapies course about into dominant Yes, so and I as think against the possible. want potent of frontline of development as we think we

care. the initial available are fastest of standard generally and that Now is that plan as the generally in in standards based the received of -- data path registration there in established accelerated oncology cancers and cancer certainly on lung [indiscernible] thyroid single-arm who patients quickest the medullary is care an have is and approval

much get most certainly a to then that would very we first So patients developing development in who frontline approval into frontline and the can. but be and expect no path that in we as setting we rapid quickly have regulatory path standards received those us

Jeff Albers

occur that as in non-small second tumor the part of with of at cancer mentioned continues but for which for Andy our frequency to question those currently types on agnostic and data the various will enrolling patients of we’re of cancer then part are cancers be those evaluate continue a RET and the starting remarks was focus emerging approach thyroid ongoing to alterations study. and our -- for types prepared them lung in And study that a we that in low we the cell and very which

Unidentified Analyst

GIST one And don't on one then if mind. Great. you last

Just curious how a much will you information this have GIST development order by do clinical think data decision you CTOS? within do obviously make you in and will think to second-line plan need

Jeff Albers


enrolling made intentions we you think decision, second-line we'll in comprehensive I forward started trial that more at we our that the Investor move forward and at have provide a regorafenib randomized on update ongoing versus trial path year GIST. of So disclosed have third-line end know with the the quarter an CTOS. this Obviously GIST those in we of second Call as to plans


And our from next question Arlinda you. Lee Canaccord. Thank comes with

is Your open. now line

Arlinda Lee

Hi the guys, for questions. thanks taking

the maybe surveyor separated the know to are care? fourth-line you I dataset you that little might want for the On didn’t data, bit free And prompted you lastly -- additional about evolve, survival on SMP, but the of going of started just we a what [ph] see on if designation FDA I lines ago? voice too and can much see how a progression second-line dataset, provide the out that guess study to then we about disclose the breakthrough information CTOS on [indiscernible] some from year PDGFR considering

Jeff Albers

take have will will Jeff. breakthrough question So, designation. therapy and answer I then this I around is question first the the Andy you

of approval. what an plan fourth-line treating, don’t opening into multiple that's go update the from that as said we duration think that. the and meaningful be in we’ve GIST third-line providing this of will a want PDGFRα to development be lot being rate remarks for terms will a so frontline currently time still in fourth-line cuts regardless regulatory therapy will and will of comprehensive data We’ve that highlighting update. be that about we got a over this will be different any we for Obviously, highlighted second-line where some are So from but Andy as robust X,XXX those because patients a response avapritinib. response currently pulled them on There patients most GIST of early receive be that and is of that evaluation focus with will at get around there FDA of be this line, looking PDGFRα, showing it very opportunity GIST of for to primary lines from the GIST, all together, on we to update, see

really So we'll the the additional to cornerstone that of as provide valuable but look presentation information be will possible.

Specifically more know early to preliminary think look I of is dataset, but you to will that different your so the PFS at obviously be a that variety data. we’ll this I that said question in cut it that

Andy Boral

on based so that terms of dataset. fundamental for it showed is cares at Mastocytosis lot the what at update on is was there really a more the EHA Andy, was It’s data incremental course that that's on of we FDA datasets detail. which was document the the an PGD; the But ASH,


comes JMP question next our Aprilakis Securities. from And you. Thank with Konstantinos

Your line is now open.

Konstantinos Aprilakis

Hi guys, taking for my thanks questions.

Andy strengthening in presented mentioned cancer ATA. thyroid BLU-XXX So at you of signal the efficacy

dose patients with to So driven and follow-ups. to see we updated to got go expect see should cancer data expect forward more we would you I XXX, see two have lung, when phenomenon same lung in when the in

Andy Boral


rate see important of was think the at the as the few response patient cancer increase patients at look really with call pleased the to and on last the real the update at AACR -- the that population call, lung the higher on that way, and very no follow-up on study cancer response escalation part -- So I data doses think presented similar -- were continued all was for the based on a NTD. lung population when our in call with we which we of it's in the is and very we to thyroid

update So in plan population. lung and cancer we thyroid -- We on possibility over to continued that lung in smaller rate first time so our a coming of we to including to updates at improvement the response update for in meetings the hopeful are out. comprehensive the year. disease opportunity specific half and We will always keep on eyes see keep open the study

Konstantinos Aprilakis

smouldering or indolent And regard wondering if to then ASH were on with just data two those patients SMs, follow-up we data and the SM? EHA so updated from any piece I’m patients patients avapritinib with two new EXPLORE Okay, when glass great. of out with there

Andy Boral

avapritinib SM, are of having in have and be Yes, be are other with on however have information give additional an that follow-up interest study a what we an -- patients it that so to at before still excited oral we that ASH we the we certainly update complete the of update update but about will so incremental at out of thinking and we’re reported bring pieces we can presentation.

Konstantinos Aprilakis

related XX one if answers maybe confirm I us it GIST could for when just limit know is opens and to the questions I the quick for there, I enrolment was GIST remind second-line right? you Okay, Jeff. wondering that was but then believe target you cohort said patients one

Jeff Albers

think Yes. enrolment That quarter. that late is I first and started was correct


from Jefferies. you. our with comes And Thank question Eun Yang next

now line Your open. is

Eun Yang

Thank than you. would rate, than and third second anything general a for good XX% question current GIST, of on better line response regorafenib, give therapies so I and less so have be avapritinib that avapritinib. in they

when However cost you give of response of therapy? mind significant one a current there remains over constant provide will challenge stat single-arm in we always compare a study value your so avapritinib comparison, magnitude would

Jeff Albers

Eun, is Jeff. Yes, this

think I data commentary all the types precisely of we falls that the of provide to want hand. So with into in that

and so can been it’s on the the year of we update type mentioned through Investor CTOS. we a that since Call on provided at walk As detail study

Eun Yang

of toward [ph] And for of XXXX you. so look time second half kind you the GIST. file is in first first what year half XXX And product the can Thank launch in by Okay. the so on on commercial of the like end on track the the approval comment time XXXX, next of early XXXX? or your question commercial to probably your side, now for you’re the of first then

Jeff Albers


see of over in vision so is let and array the really we that to several and end expect a guidance on the that ongoing day five with Blueprint therapeutics build with a of with enabling, indications study one by so could Christy have So enabling several this fully commercialize we've potential piece, her vision going got think of out on plans notably FDA always on I additional two important registration potentially accelerated the been the coming and that to path. most has those a we’re to wide hit the you Medicines year, we that But registration of into enrolled, studies months. fact focus be and job, weeks coming increasingly,

And attractive will so when you very is array get it one hand indication opportunity on that and add complexity. into that of that a also

a oncology if and in that the therapies in in Blueprint rare back of vision there you go notion a in genetic companies targeted was to and small build-out Medicines, so diseases, on efficient effectively and identifying founding manner focuses And the maintain infrastructure how longer-term thoughtful in the which commercial focus indications multiple step-wise principles so be as out really building will the educating to treatment those team on then move a fashion as we're patients guiding and forward. can physicians


Thank like you. over for would This today’s to turn closing concludes the I Q&A call now remarks. back session. Jeff Albers to

Jeff Albers

coming further and new forward the Operator, and on systemic taking and updating you support good thanks clinical to your everyone morning continued for GIST We and lot a us and for look for bye. week we time Thanks, avapritinib in Blueprint the Thanks join call to mastocytosis. unveil this data as Medicines.


for conference. you Ladies in conclude program. This does today's and the gentlemen, thank participating

day. disconnect. all great have may You a Everyone