Blueprint Medicines (BPMC)

Kristin Hodous Senior Manager, Investor Relations
Jeff Albers Chief Executive Officer
Dr. Andy Boral Chief Medical Officer
Christy Rossi Chief Commercial Officer
Mike Landsittel Chief Financial Officer
Salveen Richter Goldman Sachs
Dane Leone Raymond James
Joe Thome Cowen and Company
Michael Schmidt Guggenheim Security
Eun Yang Jefferies
Andrew Berens SVB Leerink
Reni Benjamin JMP Securities
Arlinda Lee Canaccord
George Farmer BMO Capital Markets
Chris Raymond Piper Sandler
Call transcript
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Ladies and gentlemen, thank you for standing by. And welcome to the fourth quarter 2019 Blueprint Medicines Conference Call. At this time, all participants are in a listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. [Operator Instructions]

like Kristin the Hodous. now your over would today, speaker I to conference hand to And Thank and you, please go ahead, ma'am.

Kristin Hodous

Good And Medicines Operator. of operating results year you, morning, is and This Thank call. Kristin XXXX Blueprint financial Medicines. quarter welcome fourth Blueprint conference to and full everyone. Hodous

release, issued topics outlines that we morning, we which today. This plan a press the to discuss

our be access website reviewing well that as can the Investors as section www.blueprintmedicines.com. the the at release, of by today we'll press going to You slides

launch Today, full our trials; discuss clinical will will on of Financial and our our Chief Boral, Chief Executive our Blueprint Medicines financial year Officer Landsittel, review AYVAKIT; call, Commercial Jeff recent our Albers, Christy business provide Rossi, Andy Officer, fourth year an initial Dr. Chief Medical our Mike will quarter Officer, quarter highlights; will Chief results. XXXX and Officer, and fourth our full update the on review XXXX

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

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

Now Albers. here's our Jeff CEO,

Jeff Albers

drivers topline cancer on share in we in for morning RET fusion-positive in AYVAKIT. launch XXXX, insights addition discuss results This pralsetinib XXXX, Thanks Medicines will initial early with value highlight key Blueprint financial experience for our lung and Kristin. to

transformation Medicines, three Blueprint for themes. key year biopharmaceutical This company. into integrated our pivotal evolution we as be a is defined will a fully complete global XXXX by

under obviously were GIST. of pralsetinib. avapritinib, year the name off on patients the excited an approval brand the commercializing increasing mutant now We first Exon avapritinib with focus is for the is XX to AYVAKIT The of and PDGFRalpha FDA kick with which treatment

AYVAKIT GIST building for patients foundation and effective With for AYVAKIT PDGFRalpha for this highly geographies. the launches treatment with and precision we're multiple of additional across mutation. and is the the launch, and plan first medicine only indications momentum pralsetinib

strategic The focus cell and is on KIT mastocytosis expanded other second an driven systemic theme disorders. mass

As in we includes mastocytosis a highlighted large recent need systemic tremendous which our across major there's population, markets. believe patients XX,XXX to day, a R&D we medical up

address KIT, towards avapritinib we XXXX mastocytosis. of co-innovation need to uniquely systemic indolent primary majority DXXXV regulatory advanced of XXXX. vast half with in for This filings avapritinib the positioned through of the second patients FM mutant will then SM continue genetic year this in We're in which and driver is the to

cell will more indolent with as in is of first reaching we SM, of kit this X even a inhibitor, goal year which mass the KIT half Additionally, initiate the other disorders. BLU-XXX, next generation patients Phase healthy our well volunteer driven as with for trial

to innovation we in thing cell non-small strengthening EGFR a cancer. to third of platform candidates mutant In pipeline. first continued EGFR in program The lung first mutant our the the highlighting nominate program is The This development mutant product positive kinase, for we January, type wild to now is the EGFR combined over the to prosecute three profile. research of target exceptionally triple selectivity triple early-stage XXXX. challenging treatment plan strength candidate new of with our class potent resistant up scientific development triple just made of

over as aim on a a we the as have the recent capital quick to and half. cadence on next raise, launches heels we and execute therapeutic our of per sheet balance and potential begin revenue. for of runway we providers. now The And strategy healthcare these importantly, realize of product our create meaningful be commercial quarter value with strength areas substantial focus, year with could So to as patients one the to

we highlight several the will we've achieved strong milestones a off setting XXXX already us are morning, path This of early forward. on that

will reviewing cancer, Andy So start data the topline positive of processing RET-fusion Andy? by lung and

Dr. Andy Boral

cell take everyone. on of data topline we Jeff, a X/X pralsetinib FDA. Thanks, encouraging data. RET trial in the fusion-positive In announced and and highly the share morning, initiated patients to moments submission to we a few ARROW This our perspective additional I want rolling Phase non-small good to January, lung from cancer with NDA early morning, topline

me position EMA pralsetinib First, standard strong by and potential were on topline The we this a radiologists, think centrally background some so is which and to why reviewed with blinded let the data is FDA required provide inhibitor. independent approval. pralsetinib is by best-in-class dataset support

once daily. both This demonstrate the data In at using safety because of important dose commercial addition, were indicated all to efficacy patients is milligram setting. a treated at dose XXX expected the be proposed and the

a treated the continued of rate was response objective longer platinum-based we've durations. patients In XX%. with was rate XX%. for broader the Topline naive response In chemotherapy, treated showed as results objective treatment strengthening, population patients, patients previously

the response XX% In measurable of of even patients showed striking. targets achieve a also bar that tumors The deepening by and treatment XX% complete X.X response achieve complete higher of naïve data resist resolution was [ph].

potent the with of is uniquely of consistent milligram patients highlighted prior median treated evidence response not preclinical prolonged durability the with of in dose pralsetinib data long In was reached is regardless XXX equal duration against This we've therapy. showing addition,

resistance. predicted of permutations to likely drivers most KIT be RET the

new signals with no previously Importantly, and consistent were were reported safety safety observed. data the results

sense as which of these further instilled in deliver to We're urgency possible. a data us excited have to as see patients to pralsetinib quickly

on have horizon. we development important forward Looking the several milestones clinical

Immunology Allergy, On the March data of with Academy PIONEER or and mastocytosis will we Annual American X in Part Asthma patients Meeting. systemic XXth the the the trial avapritinib from updated AAAAI indolent at of

reported to cell mast trial. measures of including safety plan X burden robust we quantitative which Part outcomes, presentation, the registration and guide use this to In of share will patient dataset, a the enabling

for decision the in on FDA fourth avapritinib quarter. the NDA our GIST that, we line from anticipate the second Following for

received PDUFA last As a expected, week. three the month from FDA date we extension

will to allow action in fourth the provide GIST by the topline FDA X from to XX NDA and May VOYAGER on take date. data us second the PDUFA the early Phase to trial quarter the This enable agency line the

on turn over commercial With Christy? Christy the that, call I of in to launch PDGFRalpha an AYVAKIT share will update GIST. to

Christy Rossi

XX a morning the PDGFRalpha on Good It's GIST. Andy. this pleasure Thanks, to share morning, launch mutant initial with to insights AYVAKIT of for Exon you everyone. all be of

occurred Of on close any QX the today's of we that not be our after approval reporting course, call. will sales results

that on Instead, as payers to the approval. highlighted first urgency some team, from as weeks well of the few the we'll strong focus launch of providers the initial reception our early from and accomplishments healthcare

FDA of GIST to we receive our first approval, ahead the dialogue AYVAKIT well communities. the launch, the we having been thrilled with On were PDUFA we've were weeks January initiate and ongoing to our of commercial the prepared Xth, XXth continuing approval February At five time date. engagement

GIST ability shared, of over XX an last balancing a specific a course previously strategy, of patients over on needs of designed to mutant we've focus with the scale time and year, go-to-market we PDGFRalpha additional As the with number Exon approvals.

as experts. and sales key managers, with as country. teams integrated we including approach, and and trained groups an actively area providers engaging are access about market focus business team, healthcare around XX the well precision this With of medicine fully These accounts built

Blueprint the products marketing [inaudible] patient hours materials Within Patient Support available, were and of website provider and the inquiries. to receive healthcare open approval, initial program was our and

our approval, approval the pleased and to the interests hands very prescribing of our see we pharmacies first prescriptions. of had and specialty healthcare week investigators. with on product are We’ve encouraged the one trial beyond Within receptivity have initial payable been providers the to processed

and In for meeting addition, in discussions team coverage and broad for payers will secure AYVAKIT. we're access field gone coverage early market appropriate commercial AYVAKIT. out been confident These government to the have achieve well has our with

clinical As a tissue were last as for practice line fourth to with has include XX reported pleased the The treatment added Cancer mutant well Comprehensive soft patients AYVAKIT its for frontline guidelines GIST, PDGFRalpha Network Exon for we lines GIST. or sarcoma. now data a treatment with learn week, AYVAKIT NCCN, as as that updated we to patients National

recommended appropriate among of a is For when category both is peer experts a clinical a a forward AYVAKIT in AYVAKIT well support as to is the indications treatment, consensus adoption from committee for intervention than as leading very step practice, of and positions centers. which additional uniform coverage. This indicates provides as positive cancer U.S.

XXXX, course to of look the more experience we on as gain Over color launch sharing additional forward and insights. we the

our review the call to for I'll now to the quarter. over turn financial results Mike

Mike Landsittel

Thanks Christy.

and quarter full release. So reported results morning earlier our XXXX fourth and press we detailed this year financial

few to I'm For financial quarter well highlights from the a today's going the call, year. as just on touch as

a financial across portfolio, strong in position, critical XXXX to enables strategy entering including very milestones we're which of on planned execution of multiple focus launches. the our ahead our commercial First, us

in in sufficient We plans, proceeds we closed offering million public we fund XXXX. operations current and capital believe in half ended we our net on million. to cash $XXX of January the XXXX $XXX XXXX have approximately with resulting the of Based into second our

to into which mentioned extends as commercial expect meaningful Jeff in Importantly, earlier, revenues. achieve timeframe we runway this a

third a under to of payment, to $XX.X $XX in Ipsen that of subsidiary revenues revenue the as BLU-XXX progressiva. recognized for agreement $XX development commercialization includes XXXX. for quarter is collaboration, an million due Pharmaceuticals, fourth a Second the our and as quarter increased upfront million fibrodysplasia in of Clementia license million, with primarily payment the revenue well The recognized additional due ossificans

global of activities, investments an clinical AcceleRET the for due and Third, as well front-line operating of the the infrastructure compared launches Phase of in lung pralsetinib AYVAKIT preparation in quarter cancer. quarter, prior as expenses as and of to the in development acceleration potential X total increase XXXX there the for was prepared pralsetinib, our to fourth including we commercial

rate seen see expect consistent growth We to over we've quarter-over-quarter that several a quarters. moderate expenses, with past the long-term increases continued in

the I'll that, turn to questions. Operator for over With now call the Operator?


line our comes Sachs. the from And with Instructions] [Operator you. Thank Richter question Goldman Salveen of first

now line Your is open.

Salveen Richter

morning. taking Thanks Good for question. my

prescribing So think forward you see can Part upcoming be X pricing meeting, And to of the what study? have secondly inclusion that to And from is move feedback in about will learnings you. for to initial and just line meaningful fourth what physicians? and you the AAAAI we launch think then the AYVAKIT guidelines understand we and How as to AYVAKIT should pioneers then with also NTCN of indication? data GIST. Thank ongoing [ph] drugs the about

Jeff Albers

Salveen. Thanks,

is have launch the take color NTCN this the I'll and of Jeff. first I'll Andy necessary don't So question. question, maybe if add you why some then take part the Christy

off, to address given So data first we're the going not questions. upcoming AAAAI’s specific

X is enabling Part guide Part of But purpose a to the as reminder, the X. registration

So of like and inform what we'll study to second -- looking be the dose the duration for sample for trial. size will be selection, part things dose the

that then watch seen that components the SM with think the we'll highlighted consistent of then avapritinib we've are for we'll And with repeating. of patients EXPLORER results previously treated that trial. I And those from some indolent Andy [inaudible]

Dr. Andy Boral

Yeah. patient at things ASH, that but Maybe we'll study. tryptase, looking objective just burden. the of to before from think, are little via be of that interim all mass of reduction at tying a should mass the cell be results outcome those cells, like with bone DXXX the course, then, are together that course, we burden. to bit We've of marrow shown that showed that mass say part we we'll And report measures little

Christy Rossi

about And line. launch the I can thinking how fourth and we're comment on

years. So, with how relationship strong the over going with the had GIST weeks. last I'm been a built very community have We many things over very pleased several

the agent the real for having this AYVAKIT. years that's to amongst effective, I first in launch, innovative just highly community sense been support of and development was a excitement many these time think And a to the treat pride certainly, of helping There patients. about available

very and to I've that I investigators personally were starting experienced, been But see. most the prescribing and excited. that think see for excited AYVAKIT outside the the investigators to we're already think, breadth our interest for that of they thing are certainly of is I prescribing me,

gratifying. really been that's So

significant. the In terms population transformational line, as price we of thinking targeted I raise, a initial said, about thinking in GIST. about when benefit I patient we and when set the fourth we were are think approval need there will effective therapies about as fourth that very where, and hold on we continue approved the again, strategy that anticipate line an no currently unmet is to

So second we're FDA to action quarter. forward looking in the

Salveen Richter

Thank you. Okay.


Leone Thank question you. from our the And of James. next comes line with Dane Raymond

line open. Your is now

Dane Leone

update. Hi. congrats taking Thank question you for and on the the

of study volunteer where highlight you end cadence of in to the get terms this hope in you program to XXX year? a year. or the in have the you be further So on the maybe will that it first healthy any that the half Do of thoughts by of

Dr. Andy Boral


we'll starting on appropriate we've -- and mastocytosis as speculated systemic study. starting said have we timing that's identify the study in that a again, haven't the be indolent I of study. again, and mastocytosis volunteer So, healthy patients dose to subsequent we that patients, as this importantly systemic in year It particular described, really

think get I as update. we we’ll started

Jeff Albers

build is This just opportunity we Yeah. a that, is area. leadership fortified frequently about Jeff. we as And we position time. our I within see when our want over about cornerstone mastocytosis commercial as add, systemic talked disease the of think this we

where severe BLU-XXX indolent to strong avapritinib we us of will be systemic on mastocytosis, in mastocytosis advanced nature maybe patients over initially more the those long-term. systemic then patients to starting with will align and mastocytosis really put focused complementary with moderate with systemic a indolent, mild moderate the think position we So

Dane Leone

one on maybe RET. And follow up the Great.

think pleased data I really probably of and data been thyroid expect the of see without we matured that's filing give quarter a that everyone's Where to with -- you of around RET the scale can the kind is sense is us data. lung scope here? second the and a

Dr. Andy Boral

the incredibly that yeah, data ARROW the the from presented. we're the – data thrilled lung So we evolution with recently study,

a coming the know, sufficient it on accelerated data. file robust will application approval, -- registration It So thyroid be and data to support track an a data together quarter. and data meeting we is will set an -- we that at we're study medical certainly, will is to as from in for second the present those we

Dane Leone

you. Thank Great. Okay.


from our Joe Thank comes line Thome next the Cowen of you. And and with question Company.

now Your is line open.

Joe Thome

order stand you and you non-small what additional patients, for been complete on patient? or up the submission question. cleaning just the line the had taking is of Thank to [inaudible] you. Maybe filing, event pralsetinib line third waiting on thank Hi the with last avapritinib data follow on you're now, in study and there? lung And on have you waiting my third cancer just cell one rolling for there from are still remaining then

Jeff Albers

want you RET. start do Andy, with to

Dr. Andy Boral


Jeff Albers


Dr. Andy Boral

small – that So lung yeah, submission pralsetinib cancer. we cell the for so announced driven we rolling as initiated – RET non in

completed complete to as completely materials quarter and we’re We But track it's complete we and there's -- the available. submission. become are the on to on no, track this that filed they become full

Jeff Albers

still see date. by in And we're to then track on VOYAGER, still able turn waiting provide on to be have that. XXth data early FDA to PUDFA take around quickly the data second and topline and on to to action it that events quarter We’ll time May

Joe Thome

you. Thank Great.


Schmidt Thank Security. you. the Michael And next Guggenheim question from our line comes of with

now is Your line open.

Charles Zhu

SM, Good text information XXth clarification, on conference? would the the on there Zhu the indolent differences released Charles for morning, weak again of be Michael data up one just from Thanks on guys. This the the potential congrats and is and Schmidt. between the the presentation abstract on on One the of quick versus the follow actual questions quarter. AAAAI taking quick

Jeff Albers

Yeah. Certainly.

after given to And that strive – at more present always in abstract. on the in we certainly so of we the be then time occurred this current providing case, and the most the So data of perhaps the abstract unblinding will more conference the the information even is update the abstract.

Dr. Andy Boral

Very good point.

Charles Zhu

higher over between I or Makes over independent clarification. review of the versus Got time complete a true Is if RET, responses new occurring drivers another response that up on any Thanks you rates these sensible sense. wondering time? guess, particular could response patients? differences color provide it. it, And on follow for around the

Dr. Andy Boral


of really lung pralsetinib, responses cancer. think – Andy. not with So It's robust lung thrilled particular, been and to for cancer see these in the is this We've evolution with patients lung that in deep are unique just really the data cancer. seen we very

that's tissue itself, it's to of certainly effective shown but evolution is over hard see a tumor very the know as I So it shown. probably And achieving concentrations way was what time, the just in that. access tumor and think, it's think drives good – of this to But usually patients. always I due we we've don't something I the deepening we we measuring highly the of -- by have typically tissue to medicine in think responses

Charles Zhu

Understood. Thanks again. congrats the for questions. the and for color Thanks taking


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

now is open. line Your

unmute If you please line mute it. is on

Eun Yang

couple a I on questions course of you. avapritinib. Thank have No. of

or So, phases first, on how primary of cell you with cancer [inaudible] And [ph]. PSX power Can the that to study that end also to with an just non-small started. first that on the Phase cell line Thank trial? compared us update for cancer. combo lung lung Can prembro the comment as X doublet the trial point mentioned give without previously, second you. you you study non-small

Dr. Andy Boral

Andy Yeah. This is again.

X So front-line study lung study. the accelerate call what the Phase we cancer

checkpoint platinum physician. or So to are inhibitor comparing that's pralsetinib treating there double therapy with without to up a the we

is well based with but cancer benefit of endpoint this survival. study on controlled based there's -- the in available achieve data well and inhibitors The study data checkpoint design powered We our progression triplet a primary powered lung seen free I we with study that data doublet plenty well have with the in -- from frontline of to of frontline. we've think those show on to and

have of we've and the kicked in off on working combination of combination, the specific project. best that deciding continue so literature here osimertinib We disease to that recorded terms to that due yet. its not part like who have really the the do relapses with find mechanism RET time work on have some to actually still that where there do actually study mutant we are pralsetinib to to fusion a In study patients understanding and EGFR we a that and cases how

Eun Yang

Thank you.


from David Morgan with Thank you. question And Stanley. line comes Lebowitz our next of a

Your now line open. is

your If of our unmute next Berens Leerink. And line it. is the SVB with mute, on line Andrew question comes please from

is now Your line open.

Andrew Berens

I Thanks the questions. a have you Appreciate from taking couple Andy also lot guys. pipe. a the for

do wanted guess, pralsetinib to profile what do are then Larkson your the data that on for liability program? Lilly then, [ph] one published the you and And thoughts seeing have for EGFR the you I think that I get primary drug recently and what resistance on initial how Just think primarily also and could differ pralsetinib? for they

Dr. Andy Boral


So, guess, recently us based of a that we the what on Lilly compound potential mechanisms know resistance I [inaudible] yeah, solvent about wasn't to so mechanisms surprising reports published the for of showing of RET kinase. couple as that resistance I be a front – case would to mutations the think and

to pralsetinib, mechanisms we carefully continue tumor over resistance course certainly to are treatment. of We measuring baseline at with look in DNA the of at

pleased been really we've with far resistance on seen, we've has relatively been mechanisms what So seen target rare. we've RET

seen haven't mechanism. We yet resistance a gatekeeper mediated

We and this major at also second mechanism sense this have I mutation, of point, have to be understood. would be have actually What resistance we expected, half? a the it's think but solvent seen still which don't that a to front that's

Andrew Berens


Dr. Andy Boral

Oh! Talk yeah. about EGFR,

Andrew Berens

I out a there the And just probably idea behind Yeah. a just be EGFR drugs? days, having of it's rationale I I little can program. mutation have either confusion a potentially seems rationale drug of and percentage triple also, double any to these if I the failures the About there. what know for bit mutations if you be early guess – could gross drastically through of us walk then but

Jeff Albers

and therapy terms in of many line remains globally very. second cancer It parts in it in two developing started so in in know lung very a is but of standard that line the rationale, next second world. the of world line EGFR step around use really actually line the -- programs rich we a in that – it’s U.S., the as new many this inhibitor of the well, U.S. into and second front moving the of a combination is portfolio. [inaudible] think kind We So, yes, our

setting CXXXS at to the And of we kinase which plus program the mutation is the mutation, in is two of a first the what the [inaudible] programs inhibits driver we second TXXXM our line looking in the mutation see expect the call mutation, of asthma triple lead the as resistance agent. setting which plus EGFR the

something refers -- mechanism osimertinib. like for to think avapritinib get that globally. So patients important We group you and get resistance the be continue CXXXS you a of as then TXXXM to will very of many from sorts

complimentary We the shift with now that's – therapy, again, working the of frontline longer rationale raises osimertinib possibility that of that's take whole CXXXS on U.S., to mutation and of then I the for much the as a opportunity of think, very in see and to program move the osimertinib absence start earlier elsewhere. second that a front the we're seeing that as course going line but TXXXM the to to the

Dr. Andy Boral

of Dark Jeff, to maybe this higher in BLU-XXX find complimentary of a programs. doing to complimentary where that about a the I often mutation think more use the with would line. We really it, remain franchise work just broadly most they're level, add incredibly to avapritinib is frequent is if see that triple that is treatment terms doing should we pralsetinib with paradigms if mutation EGFR you globally blend occur. we're and mastocytosis still molecule out systemic target as with here what second that on osimertinib much building at we're and that

U.S. And paradigm first many in and where double mutations countries Europe, be shifts, most resistance that are on the target would would in likely in emerge. mutant osimertinib particularly that the as line

patients within think have disease. is with what it's an to evolving pralsetinib will we complementary serve to and with we driven it EGFR that going be what need So

Andrew Berens

any percentage to the those mutation? rest predict Is get of to Okay. of the XXX epidemiology there the

Dr. Andy Boral

the common be mutation. think on it We most target Yes. should

evolving. the are actual epidemiology think data I

Andrew Berens

Thanks Okay. a lot.


the our comes from line And Securities. Benjamin question you. Thank of next Reni JMP with

is line now open. Your

Reni Benjamin

do the you a off working of they know sequencing course? And work-line, have you in the questions it's – talk the as to you sort the for us month only in I on but are sense with bit taking any quarter. of Maybe Christy. and for little been used Thanks just whether, primarily campaign? of sort from trends morning. can being starting a any a coming Good well scripts that's just congratulations about or provide frontline Hi. Can the

Christy Rossi

just Sure about being answering I'm you of right PDGFRalpha in campaign, therapy line and mutant way of say are for AYVAKIT what testing and When the question the sort you the we're driving mutational talking seeing used way. sort sequencing of patients?

Reni Benjamin

Correct, correct.

Christy Rossi

Okay. Right. Yes.

So, we've trying we this national medicine, really backgrounds really do by experts at where from will level. of variety leverage to have have a focused the precision we a team influence diagnostic a and account behavior most of been come area is have focused who in get think

there KIT sure in example, cases. therapeutic making as can reported is reflex PDGFRalpha flagged intervention negative, appropriate a for So the that those time in of that are as we is lot do that making sure terms mutations every is are results AYVAKIT

days of being community, therapeutic meaningful well already. to a had we know receiving they're early And that discussions than now see patients been it. them. XX% even certainly intervention they in we've in But wins, reason are that. early for prescribers with the have good started a that And less We to having testing mutational there's those success, because is of lot and

AYVAKIT haven't other see had in But time some think take to until will this because now. have options time, on just, expect we they a we'll start see therapy. so, being therapies used Certainly, lines we'll evolve. been numerous over PDGFRalpha who early of patients they to more and more I other And

Reni Benjamin

And I and in it. get VOYAGER patients a stratified you on just PDGFRalpha, Andy the the study are regarding the to, being mutations trying guess to sense one Got based tumors as then sequencing now XX? outline maybe patient's Exon our

Dr. Andy Boral

if baseline DNA to – we mutation or stratify mutations a circulating hard But KIT and evolution status patients more PDGFRalpha get do study assessment based the on study, does tissue. have follow the on their study and especially tumor, sequencing, actually archival ctDNA large tumor based So, mutations it's in tumor by randomization. for they of pretty we

Jeff Albers

And right on an is to with we to benefit data that's it give really therapy right about patient going likely for incredibly avapritinib. it will over which data that think be be mutational the treated are that very And that optimal are be time sets most this a And patients broadly, because status. us think based going from source. important to view more our rich where see rich to the patients will

Reni Benjamin

Got it Is to the line the FDA crossover for they the looking regarding a review now that that what fourth on it now indication and fourth patients? review PDUFA? then that technical getting for. set Is FDA goes just on XX might are and to line and have and AYVAKIT exactly pralsetinib be May moved arm prior it data that

Jeff Albers


III assessment competitor than part data. the simpler versus regorafenib I [ph] ava looking way Phase They're primary topline it's the efficacy think of that. the just and the topline study PFS of primary from primarily as the for I think data meaning

also study really I know that is they going that think course, they positive? data, the see the want just Of to be summary safety to

Reni Benjamin

very you much. Thank Great. luck. Good


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

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Arlinda Lee

Sorry and SM you expect SSM meaningful start And expansion increase cohort you cancer mutations RET terms. what you consider the year? about [ph] Thanks. then planning data the Phase about SM, a clinically II still would lung patients data their the avapritinib at scope AAAAI this what of additional are on the to or on the provide that. to was data gatekeeper clinical set. Can particularly curious on I see we detail with should

Jeff Albers

start a robust will a update. at meeting. our I'll be can be you those maybe add So medical And presented so RET that with color lung will cancer Andy data in set obviously

that the be will So that FDA. central full reviewed we're set submitting data to

there'll of impactful. will we variety think a break and information that be of And down safety, subsets efficacy certainly so, are

details we're going various the a the unblinded the data together into AAAAI the that it but conference, beyond doses. will the that to For too Andy's will what again, pull already highlighted, of proximity at not given get be meaningful update

still much with efficacy of to blend purpose is part on safety. to X very and and forward feedback dose whole on based part that the on select will from move of be is and – the based to looking with a inform And X track. That the so investigators

Arlinda Lee

you. Thank


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George Farmer

[Technical guys. morning Good Difficulty]

Jeff Albers


there So I questions. three is think

I I’m you a think were up The first little breaking so so bit. certainly sorry one,

I on is question think VOYAGER. it's the first

Dr. Andy Boral

a line fourth I guess? Carving

Jeff Albers

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and think one. I important the assessment of high about third safety really course, your So, importantly, total PFS and actually a line question approval level is assessment

So is that line FDA in submitted. we've the May assessment no, submission fourth the XX already make PUDFA of date order an to NDA for

you that score. I submission I think score – a the wasn’t symptoms. that a a of symptom have by year stratifying quite and approval, study and review. positive review heterogeneity I studies the the a of there for be PRO the I broke asking you're by X on such to NDA complete based the designed little assuming eligible would of on part address the bit and achieve minimum we, question full yes, be PIONEER symptom to line third think if The doing on subsequent in sure is – And is patients complete up later

will and of X but very we design data part carefully don't we those optimize an help see the – issue to the So part data at that we will X. at use them same as look


question our line last And you. the Chris with Thank Raymond comes Piper from of Sandler.

is open. line now Your

Chris Raymond

Hey thanks, just a couple ones.

ranging think posted. lower like study. – So GIST. you than dose was it noticed think I the dose U.S. I in on noticed was last clin that This I starting Again actually a approved those it's looks it at in guys a avapritinib work I CStone know – bridging have with China, talked But that just trials doing we week, that about and

if the run approval a was you U.S. have any to break regulators full need ranging I in color from curious why given a this approval? with Just a dose or Chinese what's study given guess, way there, the requirement

Dr. Andy Boral

is that and case PK in population to at that require Chinese This exposure higher statistically the be patients start This than patients. and in small need lower in Yes. activity a group CDE is Yes. standard safety is a a reestablished just Chinese And in Chinese Western in you level. dose they the from the significant as Andy. requirement not

Chris Raymond

scenario? the on accelerated lot that just sort is of have about talk line a an if granted my some I drug is? a Can in is ability broader accelerated has fine. the question then question that of before broader been approval setting when fourth asked avapritinib FDA gotten approval avapritinib But And think could makes there from Okay. get to a one you have consider we've I fourth might happens line lot I call maybe that be from on understanding guys your there. another know approval some what say questions you investors

Dr. Andy Boral


approval is accelerated where corrected there multiple multiple should effect. have are So that's examples it so that zero not there

Chris Raymond

Great, you. thank


concludes to Thank call this Jeff session. I for turn you. to And back the closing and remarks. like Albert's now would today's answer question

Jeff Albers

as I update time AAAAI Thanks lot. continued Blueprint thanks data support bye. of Thanks, us few we weeks it's forward SM indolent taking year your of an importantly, for look of Medicines. and everyone to a you time the for Operator. And seeing And on Bye, all a join we a to at the provide in again set. tomorrow. know busy


Ladies you for conference Thank may disconnect. today's you call. now concludes participating, gentlemen, this and