Blueprint Medicines (BPMC)

Kristin Hodous Investor Relations
Jeff Albers Chief Executive Officer
Andy Boral Chief Medical Officer
Mike Landsittel Vice President of Finance
Marion Dorsch Chief Scientific Officer
Terence Flynn Goldman Sachs
Eric Schmidt Cowen and Company
Arlinda Lee Canaccord
Laura Chico Raymond James
Konstantinos Aprilakis JMP Securities
Eun Yang Jefferies
Dane Leone BTIG
Call transcript
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Good day, ladies and gentlemen, and welcome to the Blueprint Medicines First Quarter 2018 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 call maybe recorded. I would now like to introduce your host for today’s conference, Ms. Hodous. Kristin begin. may you Ma’am,

Kristin Hodous

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

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

the our available Marion Officer We is your questions. will Dr. on and call for then Chief Scientific open Q&A. call Dorsch, will the also for be

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

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

our here Albers. Jeff Now, is CEO,

Jeff Albers

selective of strategy focus morning, defined foundation everyone. in the our cornerstone of our patients medicines potent We clear. craft potentially good focus our transformative and is Thanks, genomically diseases. Medicines, Blueprint This medicines. is growing kinase pipeline At highly for and Kristin, with the experimental

building class library a home in with we of and expanding approved medicine is Our owned first have proprietary to concept, early powered assembling sight a wholly of to and portfolio proof and rapidly team candidates kinase clinical grown investments line delivering our research compelling our progress. three Today, patients. R&D first

differentiated We’re its business accomplishments goals. over key proud long-term. committed of we’ll these pharmaceutical we’re morning, building by achievement progress continued company and sustainable vision to XXXX demonstrated our a This a on and towards focus of and the two updates

Cancer we’re American the presented highlight that clinical Annual the we’ll BLU-XXX Association at AACR recently Meeting data Chicago. First for Research in or

Second feedback we’ll on avapritinib the planned share and an mastocytosis. regulatory encouraging program clinical systemic including registration a on update

trial the data. context the results, Before these BLU-XXX and take step for I a back broader frame want we discuss to

As you presentation months. set presented this can that the and BLU-XXX data major AACR represents avapritinib XX proof viewed believe see past at this at BLU-XXX slide, compelling concept for clinical scientific data we the over on we the trials. are meeting that and to together When a of impacted power platform. show profoundly these existing in more medicines scientific the X We medicines of experimental kinase and orders potential even approaches the than selective Phase demonstrate our BLU-XXX have our of data early magnitude design potent clinical these

targeting biology. to and to both quickly highly addition novel ability reproducibly these our develop drug and demonstrate results candidates In difficult active

scale the Overall, similar Medicines build a company. and to combination execution nominated end owned on medicines unyielding portfolio that advance in opens leading of we great we we differentiated and another early kinase opportunity a program and manner. that focus hope into Blueprint science the believe discoveries to sound wholly research Our selective recently to

with share Andy AACR. from Medical RET-altered to I Now key to to ago, ask in patients This data few we our these Officer I weeks morning initial for points Two cancers perspective. focus his on thrilled at clinical Boral, were Chief before BLU-XXX. highlight BLU-XXX let’s want results a share

to-date our of it’s Blueprint with is BLU-XXX scientifically think stressing and driven progress approach our truly we vision First, of Medicines. representative worth how

mutations. established Here to we the a design resistant during with we be in rapidly with hope match study. target equipotent include clinical to starting predicted predicted RET,with including range selective initiated we X to and Phase profile mutation RET achieve a to inhibitors. clinical We RET, target the our of proof then a driver highly these compounds the ability selective resistant proprietary that for of selected library, extending response. We against population to mutations concept candidate therapeutic well dose these our optimize of difficult or disease fusion potentially kinase testing identify expanded durability Using of points, escalation first

development Second, obviously rates cancer stage preliminary at multiple of the thrilled lung outcomes we’re cell non-small thyroid to and Over clinical consistency we’re patients with very see clinical this early high pleased BLU-XXX, response across patient of variables. and with cancer. in medullary

We’re of prior in regardless activity response therapy. or seeing type alteration RET anti-tumor tumor

against combined part the evidence trial. exposure BLU-XXX showing early with RET as we into inhibition, pathway early and of pharmacokinetic clinical of and When dependent seeing activity brain the dose think we activity also encouraging advance metastases. highly data the as expansion We’re that’s pharmacodynamic

safety profile. has demonstrated a favorable BLU-XXX Third, very

safety. reminder, escalation is of a the As dose primary goal

of mild adverse of a out to events of adverse majority report patients XX treatment. was adverse event. one to there The association and events discontinuation treatment related we one due So great only were regardless or

where over we in period X hope BLU-XXX and bring and time evaluate they see across primary RET-altered dose maximum criteria, Finally, goals extended support a population we’ll at expansion, tolerability remind want this need. from medicine stage to very the I in and signal directly that clear of development. optimize everyone we strongly safety be us activity to By a this position outset, ultimately were clinical into these our dataset dose but demonstrate are data to a these initial where the are right want this broad and a from of of population good cancers. escalation a investigational From of in dose that to data to compelling also a advancing larger Phase an of patients identify early portion trial, tolerated

Now additional to program. BLU-XXX I’d detail on to like Andy highlight over Andy? avapritinib turn provide and the recent to with the to data call our progress

Andy Boral

and morning the to good Jeff, Thanks, everyone call. on

of treatment these of Me that wonderful ROS offer So promising soon as mentioned, data. the will from the Blueprint target encouraged patients results thoracic very hope by with to patients benefit EGFR, initial BLU-XXX for driven RET-driven all Jeff share have as us such Medicines data. and therapies are at personally oncologist, a that’s cancers opportunity already cancer. transformed It’s early ALK revolution to in lung the

on to moments results. So this to want dose I escalation some a the from take details morning few focus

XX% patients prior First, or alteration therapy. and with measurable tumor of regardless disease RET type reductions, tumor of

showed doses As evaluated started was Three escalate Patients from across disease multiple permitted will to one escalation Jeff variables. XXX dosing remarkable these to once-daily at milligrams. ranging regimens noted, safe. dose deemed consistency higher level who to lower we XX milligrams dose

context overtime. levels to continued milligrams at these encouraged were milligrams Often all dose a to ranging once see and XX in very treatment from responses of responses day. the patients observed XXX in starting escalation dose We’re

for AACR a note were the response the yet. in first that patients XXX worthwhile at patients evaluable the XXX only the However, the and the cohort milligram milligram hadn’t time measurements presentation, in actually cohort been of of tumor few done to because none

and Importantly, patients XXX on all remain the XXX today. cohorts treatment in milligram

we’ve optimized and patients across treatment. have today, All response. duration Overall, we’ll these a on dose the patients more and an daily, evaluate And In trial, optimal we on milligrams at with all the responders remain responses milligrams addition, larger time. of XXX in initial range AACR, addition XX to these are of based of passage XX data most seen cancers of exciting dose favorable profile focus expansion far. XX or of think higher. RET-driven seen confirmed totality receiving once patients data reported of date a remained are are on we believe This which generated the now is treatment where receiving maybe that consistent very of XXX and safety we XXX for promise dose rate the the we’ve on BLU-XXX. the part multiple the population to so At with with in

early which we’re data, trial, So Phase we’re based ARROW now enrolling the actively promising on the of calling X our clinical the trial. global part expansion

at support working rapid we sites patient patients As footprint To we as additional the accumulated expand discussed the hard list AACR, meet and escalation. during trial expect enrollment we’re waiting near-term, steady in globally. demand enrollment, a with on to dose of

look we’ll potential activity evolve later data In to expansion increase this pathways enrollment authorities in approval. to addition, target plan we to discuss and regulatory the year. initiate the safety for to continue clinical positive cohorts a interactions with And fashion, finally, if expedited to

clinical program and provide toward Now systemic I’d gears shift our development to and avapritinib an in progress potential mastocytosis. GIST like on approvals and update the

you our As selective KIT and therapeutic PDGFR-alpha is and avapritinib lead inhibitor a know, candidate.

total, establish separate in systemic course than KIT-driven start third with updates toward In to the we PDGFR-alpha advanced trials the expansion showing these X more and completed plus patients line enrollment to pathways important our registration share Over X trial. updated two GIST. XXX third activity announce I’m to XXXX, initial Phase with data from line advance than GIST In clinical of we’ve excited that pleased with patients we’ve disclosed patients enrolled GIST, XX both in clear and of clinical PDGFR-alpha more on strong and recent on data, we progress. mastocytosis. we’re cohorts the NAVIGATOR GIST Phase advanced in indications. Based just several driven plus

GIST, approval PDGFR-alpha, As that from patients received feedback the possible avapritinib X NAVIGATOR year trial. DXXXV expedited of treatment last based for preliminary Phase FDA a of we the reminder data driven with an on maybe

to anticipate XXXX. and position we’ve the an a in required completed first of we initial half that drug being application Now submit up enrollment, generating in data new

third Phase throughout global initiating In and dosing geographies addition, compares first quarter in our clinical We trial avapritinib the which GIST. sites recently site for in to regorafenib anticipate X additional XXXX. activating activated second we VOYAGER patient rapidly line which, across

case potential approval this requires KIT base in driven for refractory study Our GIST.

ongoing plan based X data we expedite evaluate authorities. to registration regulatory However, to to opportunities population, with continue dialogue additional on do and as Phase

at registration and the progress preliminary plans path arm may significant interactions productive presentation We with to for our mastocytosis. feedback update on made in in offer FDA in approval. single quarter. development a American hematology Society in our recently FDA systemic to which Annual the a and our registration dada received we’ve Finally from systemic and indolent the the for FDA based plan advanced both expedited study is potential importantly, on support indicated advanced mastocytosis, following a systemic December in mastocytosis the of the advancing first Meeting of plan in Most

development which In for the and is smoldering exciting given precedent regulatory also this issue. FDA that addition, systemic indicated no support mastocytosis, in plan particular in is indolent there

systemic with show advanced we finding manner our or in single arm PATHFINDER could trial registration patients XX of an to clinical we systemic emerge FDA criteria. data in C here advanced should date, with will trial the consistent the believe call a mastocytosis finding of approval have X the IWG clinical on mastocytosis. be Based PATHFINDER avapritinib the required patients for data the Phase Patients in enabling of mastocytosis, will Now an plan to enroll expedited we to the The with systemic approximately evaluable basis date. feedback which design advanced trial form trial.

patients, previously both overall required. so endpoints not survival IWG, progression free enroll survival, to and endpoint response treated outcomes, reported plan naïve is and prior for treatment overall primary we safety. will be include might secondary treatment key patient addition, The storm In

mid-XXXX. We’re on track to in trial initiate a

the smoldering received in on registration from In positive indolent FDA systemic our enabling, mastocytosis. X with addition, we and Phase feedback trial planned patients

open We end of patients for systemic plan dose assessment initiating parts, will placebo selection, our goal include reported primary trial to outcomes trial with with randomized X is clinical work to of efficacy mastocytosis. expect indolent advisors the an and the by to year. refine We Patient label extension. controlled multiple our with a Phase FDA be developed the continue questionnaire this plan a including and the specifically to endpoint

clinical So expand to in a the – variety of of program fronts. the development program summary, on we’re and breadth depth avapritinib excited clinical program

our the to Mike? Mike turn or President review the I’ll quarter. for Vice over results Now, Finance call Landsittel, our first financial to

Mike Landsittel

support mind commercial operations continuing development further to Medicines the of potential a Blueprint of platform financial position. to assets to strong discovery to continues across tumor our maintain efforts. types. growth clinical fund we us Andy. a and for By build enable will of pipeline beginning our Thanks, next as seek to infrastructure of range become our the stage expansion company, while a This to our of a

for under quarter quarter decrease collaboration of of quarter $X.X were Turning Alexion to due fourth XXXX, was result the quarter $XXX,XXX revenue as to which our effective as recognized XXXX, compared Standards XXXX. with the of of the revenue first on our of adoption for termination a revenues P&L, agreement the XXXX. primarily X, XXX collaboration impact This in on Roche well as first to the January recognition, the million Accounting first was the Codification

the first incurred quarter, BLU-XXX to G&A the primarily This personnel $X.X compared XXXX. due avapritinib, same and increase This clinical largely million clinical expense expenses year. During last driven associated increase related related in by advancing in $XX and $X.X million for R&D costs increased with G&A in quarter, further of BLU-XXX the trials, million compared expense manufacturing to personnel quarter $XX.X professional pre-commercial we R&D first through for period increased and million fees to as was expense planning including as well increased was activities. were the for expenses. expenses

quarter Finally, net or million per of first to quarter net $X.XX per share or $X.XX year. compared first loss a we loss $XX for the for $XX.X million reported of share the last

middle our be investments our cash This was we balance cash with that of current collaboration the under decrease XX, as excluding fund equivalents to compared will $XXX.X to $XXX.X to and investments our used expect in XXXX. activities. our and our on million cash potential into any related plans, fees cash, ended expenditure capital option and cash, the equivalents Roche payments to Turning quarter enable of and milestone expenses sufficient continue operating operating to primarily us in of with XXXX. we million sheet, to requirements December Based cash

into that plan and our are expenses increase our questions. pre-commercial activities. call made Operator? we efforts turn year to continue to planning progress so efforts operating that accelerate our now over operator to hands patients. will about the and, will of the medicines excited keep I to we’ve the expect development continue We To far new to as our broadening clinical we we With that, this expanding the and for bring


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

now is line Your open.

Terence Flynn

first? minimum avapritinib success kind frame the just trial, some thanks embedded for Hi, point end taking the respect help bar in on the then can to for development to there is setting, as Maybe And need you PIONEER the that questions. on PATHFINDER to in be indolent on you duration? us front, with the trial? do of the validate Or in Thanks. the going PRP

Jeff Albers

on want Andy, first. that to take PATHFINDER

Andy Boral

sorry. I’m Yes.

question to So the question PATHFINDER the… about – about specific the your trial,

Marion Dorsch

success. Minimum bar for

Andy Boral

success, for bar yes. Minimum

advanced we’re yes, middle year PATHFINDER kicking which the is So patients study Phase in the that off our of trial X this with mastocytosis. for systemic

what current have study distinguishes ongoing study single-arm patients Phase background, – that and our have that’s IWG mastocytosis, X – So distinguishes again advanced from is study, just it remind includes Phase in – a everyone X who systemic must finding. specifically with what patients to our

be in point know at numbers of rate response. We that a is response. eligible We endpoint must durability. the And specific would this of point on in potential yes, that discussing and with at the that’s it have time path that – path substantial approval the for we’re So reasonable time. require don’t accelerated And evaluation in response with the acceptable the FDA. middle accelerated – primary

with we systemic the response PIONEER in the smoldering system at Andy, current rate ongoing mastocytosis, a the end CR, response. do at PR for the will we is the on advanced response avapritinib bar study rate this findings see that we so high mastocytosis. expect high – to expect And ASH we’re Angelo X that outcome Phase to indolent improvement year in for our clinical showed systemic ongoing in the as of do planning XX% X, reported the the a be of looking discussions. and and study with think pretty Phase start I of study, terms Based study,

There study study. outcome are based of tested back actually having discussions. for where forth is in first has and Its to meetings multiple with have initial primary both time already but for of validation a the of the systemic patient some the earlier is been the had happened validation development apply for been steps final performed. one of We’ve and complete agencies which systemic for it is the said endpoint much That many I mastocytosis. part in as mastocytosis, tool steps actually we that regulatory patients specifically ready and during on actually So patients in reported tool. with indolent we the validation are

Terence Flynn

dose, that maybe end be finding like part spot there up work guys then versus a to And that for to indolent setting a for of the in PIONEER. you’re that’s going is dose smoldering on possibility could is same? Do the it dose Okay. just you advanced a some sounds there dose or different expect of likely still be

Jeff Albers

definitely We’ll lower indolent in smoldering and dose a population. end Yes. at up the

first of faster. actually a sequential but we’ll The dose of doses. makes look just part can study the quite MTD, at we finding of Phase a know we want clarify bit that finding piece simultaneously, which a the range study, is study. not study dose multiple the X since it’s And look – Well at go it classic I to doses a

the dose starting at dose below patients. pinned advanced but quite likely it we’re would be haven’t in even We milligram highest that yet, down XXX the

Terence Flynn

Thanks lot. a


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

line open. now is Your

Eric Schmidt

just can’t on we’ve systemic Maybe mastocytosis. this hope maybe from question to very don’t or from response you Terence’s quarter. you get Well, as regulatory seen. thanks, hurdle follow-up next guess the over question and X the be, Phase I and that already the progress all advanced what given might PATHFINDER a a study already current congrats the in last on get on have high do

Andy Boral

a complicated. Eric, that we’ve question and it’s talked yes, a that’s about lot

is FDA key an I two several – from the of start are ongoing a PATHFINDER and will think, they discussions mastocytosis X the with we’ve couple with study, a get findings. had I will – now concept approach. patients IWG EU things at that in are; key things all least, are on the agencies a so one study There there that and C the our not board Phase the in – PATHFINDER two have from evaluable weren’t study. It’s are really embedded think, that of

if actually response differences, the that majority so will from Dr. from the remember pathology you marrow so for pivotal entry. have of this a we patients in in for when from expected case DeAngelo’s the evaluability be key for were we it’s the responsibly evaluable From only case a large subset start – bone are the evaluable the And had study. for the response. the at early perspective, presentation of patients review central regulatory And ASH other will study, of required actually, of

actually ongoing one hard to review implementing retrospectively. able with but systemic actually study, apply the the the mastocytosis. retrospectively, of in to that it’s we’ll off are pretty actually case, in experts We’re pathology it we world’s have by In get essential this to won’t because samples everybody be

total point thing, either just patients quick robust. pretty we study yes, to the X number just ongoing add actually set one together do expanded in had of Maybe, I probably much whether is a to just So the study be want we other Phase would new that case. similar, patients current study, out, I to more think, will the data – the or

patients affecting probability we of adding probably is really this many timeline success regulatory actually not more and gives really the So without higher much. think a

Eric Schmidt

didn’t track today, that Thank Andy. Okay, much patients going? thanks in data hear for about BLU-XXX TKI-naïve on is another how are we you. update half just that for including on We program or still second

Jeff Albers

Jeff. is this So

still very track. on much We’re

and as before on as Phase trial focused second checkpoint the arm of TKI-naive, we’re X BLU-XXX, with that we ongoing first inhibitor. a you highlighted a forward; is one one in paths combination initiation of trial to two So primary with is our allude the

Phase then much And on had this various to those, data, much this combination very look discussed update trial we – to year that the so a didn’t what later share initiate because from before. we’ve on the line it’s X year. look study we that in And cohorts in with and later

Eric Schmidt

Jeff. you, Thank


comes The Arlinda Lee next question Canaccord. with from

open. now is line Your

Arlinda Lee

One, a Hi guys, over more color taking any had questions. and for on to additional seen those provide can thanks responses. a question on few BLU-XXX you that my landscape. I you alluded time RET you the responses

you would KIT on wondering oncologist, BLU-XXX, this discontinuations you’ve and other and how either theoracic also been something another discontinued And due how couple with background, curious, is any I’m color of on prior a provide case your can with an these any of if inhibitors on you can among this fusion overlap I’m – in seeing Thanks their a you KOLs. much. effects patients or suddenly baseline on reports be of or recurrent And given did haven’t – you the neuro Secondly, provide any the expect Loxo talk patients very XG given you a you just kind you’re [indiscernible] often this to the in treatment. there’s patients as the RET any therapy, those they Andy, seen one selective had BLU-XXX? may have see episode trials on to treatment do and report but depressive in your expect neither I’m patients, response that seen point that thirdly, curious then, I on whether would you had about And guess, and RET the of information then, before. or

Jeff Albers

over is I first questions the had comment second and on Andy, I’ll three to. is one, have, this There to take maybe responses Jeff. the to So it on

those ongoing longest enroll over but to are lowest do is an for up time. we front, patients period Phase are at on doses So continuing time mentioned and this patients I obviously, X the know study of the Andy as

with our we’ve the the a pointed surprisingly Andy responses time, earlier about not we’ve that responses think cuts, confirmed, time, of passage month since is I responses of – that it’s so, emerge, I data newest blend become month or continued to had new which been see think don’t passage a with And out both, surprise.

perspective continuing on, The a those to benefit. patients and is staying see are important our from piece

So, obviously, a questions, But by we’ll over have time, to then, conference. that the – of a today, yes, provide will these. to a fulsome context evolve and an emerge. find and you opportunity as third we your update And continue at more again, Andy, the of medical data second they in encouraged I’ll be very take as

inhibitor The one a highlighted selective we RET And one the treatment, know patients I first that depression? have is the any second prior is, of any had inhibitors. indications multikinase RET

Andy Boral

so Arlinda, thanks. Yes. Yes,

who with is inhibitor. [indiscernible] aware came we’ve I’m on of half They patients and therapies, of with at progressed – that that would for inhibitor course, in to well RET I had disease tolerating course, with RET about do inhibitors, a look prior and the with At treatment patients and of BLU-XXX, in inhibitors – inhibitor other from had doing prior hope hope I and forward our showed range was one either we hope compound. study, loss that opportunity than seeing, of reason RET treated on a conversely, there they we don’t inhibitors selective don’t not And multiple of recently. so study point, how previously with RET BLU-XXX progressing out sure much terms patient is broad and think, some, actually on continuing who who study have of patients least well have they’re there relevantly on do for evolve can a RET enrolled a inhibitors, some prior this tolerate with or Phase a and, prior I very a of, patients room know with – very in or the after situation. this the with either to RET they multikinase patients the the treatment I’m to one chemo, course, seen I says both So have other X another, the will of that therapy who immunotherapy patients advanced patients disease do have have other well prior a BLU-XXX well

near area where get multiple treatments. they need we So

up the LOXO-XXX. In you with cancer of terms received patient with KIFXb publication the KIF department of brought in the that case lung that

effect in of patients So brain terms the and doctor maintain off both my in moment not own – Taking better a hat, patients, treatment. patient lung – of in in when surprise my just other brain we are the this bit. I those study. reductions our patient advanced our description improvement can and to a experience after related do and they treating study, of CNS maths, published at the responding with the putting effects, whatever or CNS my of a in seen having metastatic usually cancer, is to feel does really to maths BLU-XXX and treatment, we be me have thought that depression point, that a and Blueprint on oncologist case

So details report, I it did – a it’s case don’t surprise me. that but I of have the course, but think, aspect

Jeff Albers

This studies Jeff. this I there times, is and obviously, on are I said mean, many stringent. a marathon I early is this a multiple going not think, so that

on taking to any would much caution time. case I But going we’re at I lot. a guess, learn too think, this I into So, studies

early really least, is effective for of that, is multiple patient therapies, meet activity Andy effective the now that there patients to think there encouraging that to these is what piece that at population, help be these a in order this And, is appears of population. one many a made can preliminary is more need. important understand this in in therapies could evidence I that showing need patients on, there is

Arlinda Lee

maybe, maybe you Can on you. can about you’ve seen talk thank you question, a – Okay, last discontinuation Thanks. the just BLU-XXX?

Jeff Albers

milligram with have these case [indiscernible] we metastatese, with disease, a metastatese a cancer enzymes. a widely presented. as you adverse So level complex who very that – It’s on to XXX had of one with, metastatic patient as patient discontinue liver a the BLU-XXX an lung due studies, on had dose had elevation often liver had actually the who dose a events patient as ALTA, it’s

think, enzyme that drug thought – innovation the the due this In to BLU-XXX complex case, and used. contributed I was if physician possible case, that disease, related the due to are considered that to for in the therapy potentially discontinued those to being always thus effects of it’s determine it’s liver kind it in reason.


with next Our question comes James. Laura from Raymond Chico

is now line open. Your

Laura Chico

for question. wondering, up I the estimated taking a on you terms bit thoughts guys landmarks BLU-XXX, breadth what the have NSCLC on of thanks I could think, are good provided for in opine of kind agnostic your the opportunity of I’m of size category? guys guess, Hey, that little tumor roughly in some patient, XX,XXX. you following opportunity? And and just if terms the

Jeff Albers

this is Jeff. Well

that tumor done study. agnostic As patients that of continue seen solid actually, haven’t range that In major occur round we major at We variable this we’ve a an until we us, the fusions provides at moving RET not which identification do when broad and Japan. we provided we is further to of And already to to and we those landscape, XX,XXX of characterize the you it medullary same do of across markets, first number to on in as be U.S., a with a And it as patients. we’ve in then, arm X,XXX frequency some hand, expansion, intentionally those have cancer, because low land describe that thyroid that. data mentioned, EUX know, the patient opportunity patient end, – some of acceleration what is range fairly of potential guided look the tumors. estimate number from number around for enough markets. that them, And goes

next And effective look clinical therapy what the can in So on to to is start of provide update, But and know look sit set could we like for more encouraged, population. need provide time patients data we over today know we’ll that a there take there have very then, level we’ll time. some we that I that an where you a fulsome maybe those guidance a that a we we’re at. are tell opportunity

Laura Chico

Okay that’s helpful.

Jeff Albers



comes question Our next with Konstantinos Aprilakis Securities. JMP from

is open. line now Your

Konstantinos Aprilakis

question. Good my morning taking and thanks for

PIONEER. FDA As Phase prior SM. the it dose now PoC in indolent what was the a Thanks. I a tool for in on components that primary And trial, agency post smoldering it for those were was share you that favorable a also appears outcome you’ve adopt can guidance the feedback you light endpoint slated such and of patient provide And settings. color green caused stance? registration wondering some could escalation trial and enabling used with got and per report and X if to

Andy Boral

about is in Phase we’re X that this and So the planning systemic mastocytosis. that indolent study

a the One, disease also systemic broad components in is impressive spectrum And in been I of that is point single few in advance think together disease. to across that driver in So think really a that that our from that highly it. has think mastocytosis the it and advance I and there’s And driver I this agreed demonstrates which important a very smoldering preliminary those through indolent is seen a is active severities, essentially we’ve the together data advances. another this indolent disease perspective avapritinib with represents FDA DXXXV mutation, connecting population.

we but also read don’t in indolent. so we’re there we’re you their that mean my And put some I from been the seeing impression they data is in to in mouth, agreed data through do advance the seeing I potential want has that and to words

other, think, has several the we’re I proposing parts. study that The piece discrete is

variety of And so a questions. answer I’ll

course efficacy Of that on to and answer we question. think part in study a multipart seamlessly an then I can question move of is a critical, dose but the

the was use across severely there multiple patients to the general think that’s affected that regulatory are actually in agencies and with agreement is population there smoldering no The can you response IWG. measurement by that I symptoms. very indolent really analogous

of so provided is And so following expecting. are a that reported they’re tool the terms as PRO far development most tool guidance patient that the of outcome measure the appropriate in we

approaching of and And of input in structure not that. discussions we terms exactly that the third with having going which population. part to safety the more the the what’s they from way terms Righteously the PRO so part of crossover exact important that gives a then the different in were of be of I think And they opportunity – data had are say us not we outcome. allow very study, with to we’re And follow-on to in how avapritinib to there comfortable can an an expected are some have placebo provide in safety will question.

the will despite that the unmet group very really agencies at think encompasses with disease from promising with patients a severe advanced and address endpoints life look their indolent a matter systemic And avapritinib that is so a it’s that understanding normal questions. having a the data of through quite of this that preliminary single the needs I expectancy mastocytosis

In think – definitive well, when and proceeds, have disclosed we on about the details to we as not terms questions agreement the the of we study peer structure the talk DRO, outcome it. exactly and gotten have we the of haven’t decided specifically I – of more would

Konstantinos Aprilakis

Great, thanks, Andy.


from comes your Jefferies. open. question now Yang is next Our with Eun Eun, line

If on unmute mute, Hello? phone it. you please is

Jeff Albers

Hi, Eun.

Eun Yang

thank two GIST. and have for as mentioned very questions. accelerated pushing for as I much. approval line previously you later well you Hi, PDGFRa are fourth One that avapritinib, in you

to the So fourth NDA line was are in submit expect the GIST initial first and half? submission later data? When you

Jeff Albers

about Okay, question this avapritinib The is Jeff. in GIST. is data

our that With highlighted. and out So driven to that on plan that of have already we’re head plus to syndicate as study regorafenib had to as to discussions that any dataset. submit require that ramping that up that continues a versus Andy process on be in PDGFRa to the lead care, say we third-line refractory line head that focused respect third and around

are to is I evaluate we XXX approval alluding piece patients, that accelerated think an plus the we in in continuing possible. manner mature you believe are as and that the a such they if to data that

that that in We we conjunction file with will – PDGFRa filing. would

approval head of to line third head with either the an seek or now PDGFRa starting. So happens off it simultaneously will study that Jeff is

Eun Yang

thanks. is Okay, product. question on the And second FOP

expecting differentiations in show the or difference? any sometime data, you agreed products product are to developments we this other to preclinical compared data are a year. think preclinical present do you see to we When it’s So some

Jeff Albers

Marion, that So, take question.

Marion Dorsch

targeting I about Yeah, to going kinase. after excited be the are four that actual mean the – present year. scientific is we the at to later And preclinical planning a genetic continue actual to conference this very we disease data driver as our of programs you alluded

molecule initiated and soon really very package are this BLU-XXX, of year highly it’s program. we earlier our as [indiscernible] in think But most – patients on selective have likely looking early the focused because and Jeff for and the de-enabling the are forward in to as be to will we potent they BLU-XXX as possible. ALKX studies characteristics to that I alluded for the inhibitor earlier however

Eun Yang

Thank you.


comes [Operator BTIG. Instructions] Our Dane next with from Leone question

now is line Your open.

Dane Leone

eligibility thank on press more thoughts and some of I Hi, and for programs. and indolent criteria your background terms question a you taking I effort, of questions couple wanted update for sin of on guess PRO the therapy. around to specifically little details bit the the smoldering and/or a

PRO corticosteroids then you guess the about from for what serum know be patients? How of in these of a main specifically for separate you do the kind and tryptase and PRO I and those baseline levels drivers sensitivity around thoughts poorly combining these So controlled your think patients, will generally sensitivity biomarker histamines are PRO on together. the

Andy Boral

take that. will I Andy. it’s Dane,

So a couple things. of

have we stop patients symptoms very can’t difficult these course many their of background certainly and disease So have therapy.

study decide, TKI would avapritinib go should on mastocytosis the systemic therapies. with to study We that how – one should addition ongoing background a of you indolent to big study question evaluation is patients with standard it And now do how society quality in be evaluating is you go it in which an So the of scores alluded particular part on development of with in PRO to early just the terms life other the of correlating measures. mastocytosis doing what in behave with such understand we’re is – collaboration avapritinib. actually is of mastocytosis PRO with patients in actually as a it

with can, a not we well controlled available word, that this on – severe Indolent that define essentially are So important criteria we a the score eligibility where is point, Mastocytosis, be indicates the will is of this study. at piece Systemic therapies an it severe but inventing

we’ll reduce that are supportive actually – well, So frequency actually looking measures opportunity real Those patients like will and the to who antihistamine will a steroids to score our of be off not their continue steroids, ability a with as gives correlating allowed vein other things things number And patients pick endpoint PRO reactive us kind and of based the steroids actually other controlled. be patients therapies. well to are on of and because a at identifies of or like cutoff ongoing and secondary therapies.

excuse me like marrow more and do. a piece more or measurable it I agencies – mast bone – improvements, number disease very don’t will have the other very Another numbers to in marrow mast is bone patients regulatory maybe with to tryptase, to laboratory-based that don’t we patients think objective improvements evidence But important or cells. of cell all be like correlate that than link have of PRO is as think well, indolent the reasonable important patients show

real think of and those we DXXXV in mast changes burden patients marrow potential correlates. in cells objective changes another actually in So bone is

on we cell like PRO burden, high are correlate of with established is with endpoint a score objective a So – And course important a to we our that of CRO based other proposing quality mast then, on on PRO already tryptase. primary burden, do think based the in with the for be what we’ve show excuse reasonably measures. entry minimum – PRO correlates based of the also needs required correlating me, more level life will improvements DXXXV findings other the over study, it that

thinking kind it. So we about this of how are is

Dane Leone

is, clear to a be there be just findings lab the could to something it I or that out significant If like Great. way you’re would tryptase. serum the demonstratively like laying square

Jeff Albers

tryptase, bone I that’s like no held – we data, level I and of that things it with that actually mast in marrow there’s as improvement. to will not bone disease, be be really There’s disease don’t overstatement. that – a marrow and lot mast cells will an correlating symptomatic data, because So indolent in smoldering think

say. linear with PRO But to yes, think synchronization I So want we of be with think is available a directional not an we’d measures. our some objecting, the relationship indolent expectation of between I the improvement been traditionally in the a patients We’d clear – need and one it’s directional some more predictive bar one But correlation. see want the some I don’t other maybe see of PRO to would disease”. would of measures of symptoms or reasons in “objective consistency

Dane Leone

not ask enrollment, and enrollment how Great. – rate those advance with with – to them Is how mytosterone, one PIs in more to of you there of side. guidance pretty specifically, could just them that of a the the beat therapy Relative do the that clinicians, avoid may feedback those And mytosterone your lot that from healthy what’s prior think also study? if that I or majority able terms patients the having that is a step, on titrated. to accelerates does – could affect do the be discontinuation have being that of something for given

Jeff Albers

patients don’t preference may prior mytosterone Patients have received mytosterone. have any may or for I not prior treatment. maybe wasn’t So We clear earlier.

and are yet, the many very actually study So not makes patients it or not and approved, think, easy – will maybe that reimbursed some are are not actually much. countries believers there very just enroll. mytosterone coming There and it’s the places, sites where either mytosterone there have to we used from being we and

So a ongoing studies. studies, little actually ongoing there of for the the evolution, was mix we very when coming we started, an off course, on been expect except interesting patients and it’s

patients seeing of it though it’s Europe, both approved are patients bulk U.S. by is the a and we even more now any in means. but with Now few prior mytosterone a not

Dane Leone

very much. Thank Great. you


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

Jeff Albers

to continued thanks operator and and in for everyone taking the for your Thanks, us Great. Medicines. time interest today join Blueprint

goals have this achieved particularly continue proud across first the quarter that progress And be we we the As the year. feel we achieving that key we portfolio XXXX. we’ve discussed of in momentum morning, towards this to for

our forward sharing updates So further lot. a to on we progress. look Bye-bye. Thanks


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