BPMC - Earnings call transcript Q1 2021 | Blueprint Medicines Corporation

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Blueprint Medicines Conference Call. At this time, all participants are in a listen-only mode. [Operator Instructions] I would now like to hand the conference over to your speaker today, Ms. Kristin Hodous, with Blueprint Medicines. Thank you. ma'am. ahead, go Please

Kristin Hodous

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

on Christy reviewing Albers, our Mike Rossi, Officer, update; press will Officer, our to will the going Chief Officer, Landsittel, our the access as website highlight can will Medicines first by www.blueprintmedicines.com. provide as Medical well discuss Becker clinical Jeff our a the across first business progress and Chief XXXX section today Financial of our commercial Chief our our financial slides Executive Officer, Commercial You quarter results. review release upcoming review Chief that our highlights; at Today, we'll and call, be Blueprint Hewes, quarter pipeline; our milestones Investors will growing XXXX

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

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

our here's Now, Jeff Albers. CEO,

Jeff Albers

to across an start XXXX portfolio. is our update and morning, on good I'm to off and progress everyone. a Kristin, growing recent productive provide pleased Thanks,

on efforts our call, As strategic three centered are pillars. we XXXX around our outlined year-end

and pipeline. of precision advancing clinical AYVAKIT further second, First, next and therapeutic of to concept; of our therapy GAVRETO; a research proof candidates wave global third, adoption accelerating the expanding

progress significant these already months year, goals. few the into against Just each we've made a of

will GAVRETO. discuss advanced a we in of for continue AYVAKIT the systemic moment, parallel, and to are In approval Christy preparing advance of in anticipated As we AYVAKIT mastocytosis. commercial launches the

our over we're with the near registrational with data our and Phase AACR provide with for month, rapidly with forward advanced variation presented and urgency PATHFINDER response which US, review XX% EMA, deepen a as the MAA occurring of At X overall disease. II we as PDUFA in under moving therapeutic possible trial meeting earlier annual a to As a patients date many fighting this demonstrated the option responses we time. this SM, confirmed in rate to continuing from Type

with patient-reported and patients new improved significantly that systemic reinforce for addition, life. fundamentally the We potential change AYVAKIT these to the symptoms treatment disease outlook In reduced believe with results AYVAKIT of quality showed of mastocytosis. data advanced

our data AACR BLU-XXX, our wave medicines new our shared therapeutic candidates, ability and investigational of targeting candidates, MAPXKX. portfolio BLU-XXX expand programs at next-generation programs have basis. from several to next EGFR to potential BLU-XXX, reach on DXXXV targeting two the also inhibitor, patients in our CDKX, these global Collectively, BLU-XXX, and KIT our We that our comprise including a development and newest dramatically BLU-XXX,

sense areas us size this new these of the underscoring of over forecast these more productivity each fact, we've ago, order by double development bring discovered than of next founding XX and discovery, to positioning programs initiate ahead, future. innovation for several opportunity is to intend a the the pipeline candidates, years to worked multiple in in Becker and with trials of to the significant successfully efficiency. of our months generate five We've commercialization targeted having that clinical additional describe which our urgency. In already the minutes, medical developed to past In our as in we markets same track integrate near and hard To in approved few demonstrated efforts year maximize all us alone, the development on Since advance significant approach, expanded medicines. a years, half and innovation need. to in two we've end, remain proof-of-concept with five to we will the data to and sets

strength, to we make efforts. our the promise Christy position to of we operate pipeline commercial research cancer in hematologic the as for efforts, possible. therapy I'll programs Importantly, our the over as precision resources disorders continue of and and from call turn with our many aim commercial a to tremendous invest to our Christy? with financial real people that, now discuss to as With engine,

Christy Rossi

share good of we including $X.X GAVRETO. and million pleased to as revenue generated sales AYVAKIT progress million Jeff, and quarter In product continue our QX, million, the of morning, everyone. sales launches we results $X.X I'm GAVRETO. first AYVAKIT, $X net and in of of to Thanks, in

start me GAVRETO. with Let

are QX on for we at with shared focused on priorities I call, Genentech launch. partners key As our two the our

first percent The and patients selective new RET who first the is of inhibitor, on are a starting positive good progress against second our are priority. identified therapy. who of seen the our treated growing is patients with We've and RET share targeted increasing the

over March, highest see community inhibitor for the to on is continue driver increasing of and data. we our started grow, our by GAVRETO based among a starting quarter. prescribing of to course on duration in RET had Our treatment, the to time. growth QX, selective of first we a starts and uptake we whom The future of are number the physicians XX% from of An the number of patients many expect prescribers in favorable stronger new number launch. of new setting, clinical share of revenue the end new is of patients the continuing starting And therapy GAVRETO patient exceeding increase key since

to with in community once-daily which We then it to opportunity the continue overall, see to great inhibitor use and RET durable deep market and to well We positive are with makes safety for half be than dosing the treated of feedback GAVRETO, given receive therapy. further manageable patients potential setting. and the RET suited being as predictable fewer profile a the RET targeted being on tested grow responses, and broad

are continued working acted increase partners multi-disciplinary education our We to and this upon. appropriately Genentech impact biomarker to that with is through testing comprehensive at ensure it

in by of second seen the by of therapy other US we winning by efforts COVID effective targeted year. with availability we've the As driven highly the therapy growth expect ongoing and launches, impact half approach identified these number of patients the in of the we as the

lung bring about world. A GAVRETO the an our weeks patients to in making selective the are Finally, fusion-positive around non-small for approved inhibitor China we ago, excited GAVRETO with to the we've RET progress RET first Pharmaceuticals it few received CStone made in approval cancer, cell the in partners NPA market.

partner the and by are launch in our looking approval to GAVRETO We also anticipated of Europe Roche. forward

We drivers to be expect these global of launches in growth revenue additional the future.

Now, GIST, we've let's patient first the alpha in progressing to consistent quarter-over-quarter AYVAKIT. turn Germany. our are US, seen In EU PDGFR and starts in new launch

Our alpha growth first additional patients with in seeing time precision NPA We partner GIST approved secured XX in of PDGFR exon launches. country incremental the making AYVAKIT, it approval anticipate China. CStone mutants with therapy revenue for an GIST also over recently

quarter. in Of and the continues mastocytosis for US advanced primary this with focus in AYVAKIT course, to preparing systemic be our our launch anticipated approval

our of XX% the to initially efforts described SM many about or on X% educational As X,XXX SM the about before, are I US. of US, patients in of patients advanced of overall all We the population majority whom half centers to major that focus to about the at SM represents XX hematology-oncology centers. in X,XXX treat about advanced treated plan

Our identification and in footprint our these prioritize the to increase patient commercial centers medical us while community allows over existing using to affairs infrastructure, portfolio key time.

to in with impact we bring the begin AYVAKIT that realize we to can three As focused key SM, are tremendous patients areas.

current driven prevalent or patient currently patterns identification and be and referral will see already includes diagnosis, who by increasing diagnosed newly that utilization believe which patients, midostaurin, agents. initial driving diagnosed cytoreductive may First, appropriate we of with other time, understanding We AYVAKIT to patients. from tyrosine inhibitors centers. kinase treated key upon Over approval, to on utilization expect be treatment

available widely the will highly DXXXV commercially are sensitive over in and and blood-based XX% mutation in SM remove US, fourth US. believe we for to advanced patients SM grow the non-advanced now quarter KIT availability also diagnosis testing help expect both that, We the barriers call, about driver blood-based testing of our for test to In KIT of DXXXV, that the for patients. making XXXX, cover course will available we SM that announced

number access are disease an this, confident that efforts diagnosed identified into are translate We have combined of education will treatment. and to increased SM patients who other and with

transformative differentiated and a SM. educating will data and its lives. believe meaningfully AYVAKIT their value change the And health is fully ensure use the precision clinical way that We improve providers, are patients healthcare upon we on is profoundly priority AYVAKIT will work other quickly second providers supporting and therapy proposition patients on care educated are approval. to stakeholders a treated solution of and compelling Our

importantly, of of well Blueprint meeting financial and providing best-in-class GIST to therapy most Consistent Your through assistance are providing and and needs access AVYAKIT access program. patients, with the we Finally committed and on our support support and to the as to focus we as will launch GAVRETO, approach facilitating our patient SM for patients. unique best-in-class support

extending unique to the as to and cornerstone launch on year. impact to study we role play have progress initiate the with advanced that, goal continue look excited the patients. development on development I we With now forms build position Becker AYVAKIT with of SM, clinical to can review would like our our ahead of the to to meeting the of will the SM are of we AVYAKIT portfolio. all As the the this in further and leadership disease, HARBOR the to look SM a BLU-XXX, and non-advanced the over as key call expanding forward turn we of of needs portfolio, we and our

Becker Hewes

morning, good Christy, Thanks, everyone. and

of ago we're having next in As the new BLU-XXX development those months. development a nominated couple our programs, on BLU-XXX candidates of in the five we research AACR few call, Two past begin productivity, clinical a of in of XX the midst anticipated discussed weeks months. to period are robust and

of impact novel trial potential to lower total the will symptom the central criteria, dose-dependent Based in a with patients non-advanced range with as healthy doses, safety. eligibility for of followed KIT the DXXXV continuing at data at AVYAKIT. with tolerated a in AACR me including potency to enrolling determine patients with doses extend population randomized franchise. our Part endpoint. showed volunteers, systemic primary start groundbreaking trial SM, score Part the a ongoing and One Let We we placebo-controlled the HARBOR with inhibitor X/X data, a multiple-dose will Phase symptom goal trial similar broader plan AYVAKIT symptoms PIONEER baseline to was aspect tested the and later non-brain Christy's optimal to on broader initiate score. include reduction X KIT a supporting Phase X mastocytosis, pharmacokinetics a penetrant BLU-XXX Similar BLU-XXX once-daily all quantifying evaluate of dosing. the this will patients total the with linear X year. presented to discussion BLU-XXX that the for disease half-life of BLU-XXX HARBOR non-advanced is these well dose provides This a we to a of and This our be in reach on by evaluating program.

addition, In mutation. activation the monoclonal syndrome, plan in mast to the driven also trial exploratory cell by of Part include patients DXXXV we for with an cohort KIT X

Turning programs. our now EGFR to

unmet comprehensive foundational outlined inhibitors medical our strategies combination the brain potent profiles, During we we metastases. EGFR and treat address optimized selectivity our activating needs mutations. providing data on-target brain our coverage BLU-XXX, how CNS important designed double-mutant resistance are AACR, by have or for time, for to call, presented AACR preclinical activity, wildtype EGFR enabling high of to selective inhibitor. prevent At and first penetrant They

BLU-XXX, for inhibitor. EGFR presented also data our additional preclinical We triple-mutant

of patient needs monotherapies these have and address all candidates lines potential combination therapy. designed across therapies, the investigational As rationally to

in and we with profiles. cohorts year, mutation patients to expansions portion followed who this The cancer, BLU-XXX. This previously trial BLU-XXX patients quarter, specific at a initiate the non-small we harboring in inhibitor, for of initiate dose-escalation later tumors BLU-XXX, least will will cell EGFR-targeted prior kinase have received EGFR-mutant tyrosine of trial clinical include similar plan trial by a lung with one

goal. strategy our therapies, activity, will a other advance combinations be plan focus shots rapidly we of these While targeted EGFR of to development quickly multiple initial demonstrating and other on that offers agents both with single-agent into each

With lung ahead. of in we portfolio, patients tumors, for data ARROW by ASCO these in for with we RET clinical quarters will present cell from non-small well GAVRETO across the as numerous oncogene. the in At cancer, initiation are trials updated June, driven well-positioned milestones in solid our clinical with patients trial the as the

and to BLU-XXX, providing it programs a further inhibitor BLU-XXX the We to collaboration E-aberrant into I'd high potential look also the development In and We financial forward CDKX inhibitor, plan year. clinic, over for BLU-XXX BLU-XXX anticipate our in of two data presenting the with immunotherapy to MAPXKX as targeting to move turn under the second our they and now developed to candidates updates. to these preclinical clinic. updates combination discuss like selective bring newest best-in-class cyclin on XXXX, half potent Mike a Roche. cancers, we

Mike Landsittel

morning, our this Earlier Becker. release. first detailed results reported quarter press financial in we Thanks,

call, revenues for sales, million quarter. in $XX.X from first mentioned, were as today's with from included our touch revenue, Christy million. few I'll a quarter in agreements This Total Roche on collaboration the CStone. primarily net and and the $XX.X For highlights $X product million

compared fourth pipeline our early-stage of associated on GAVRETO. by the driven towards the quarter clinic, advancing to XXXX, operating by offset expenses increased our total for Roche costs Our primarily cost-sharing with agreement with moderately

in As resources continued quarters, clinic promising increases they have wholly-owned in the in enter sufficient that billion growth. programs, to over pipeline we first to investment We expense invest $X.X quarter-over-quarter the cash, ended operating candidates. our of the few our next expand with we ensuring in drug growing we quarter as expect see early-stage

the projected and launch AYVAKIT GAVRETO of product across in are collaborations. uniquely US, growth direct milestone revenue on and various on to AYVAKIT these revenue half geographies, forward look Based to positioned both GAVRETO a in sales outside growth and variety SM, multiple planned advanced by we XXXX, second As sources, of from we our AYVAKIT the profit drive our driven in largely US, royalties sources, sharing the through sales including of global payments on of the now XXXX This for expansion with expectations the of to we self-sustaining to anticipated fourth current the to growth both continue revenue help that on Operator? the becoming we quarters, are in I company. will build our for indications in questions. of the for financial be and total turn to the biopharmaceutical milestone AYVAKIT strong and GAVRETO, approximately and million. today and a over revenue solidify would position path payments like comfortable call third operator $XXX

Operator

Richter Instructions] [Operator Salveen now Q&A Goldman will we Thank you. And start the with Sachs. with

Salveen Richter

for about Good talk patient last how was us thinking about moving I you've quarter. regard in morning. question. sense my some you trends And maybe how just Thanks with and given think taking tracking, forward? you're that and identification diagnostics, XQ, the to of cadence the RET Could

Christy Rossi

Sure.

on. is are So as focused really we levers I there said, that key two

number if this identified of and treated. and increasing you one So I share about the selective our like but RET patients, something as be in point. who is the patients well XX% testing, opportunity spoke that maybe inhibitor, QX as that are look treated the you And said the RET is then this having second, with then at of mentioned, at market, to opportunity as a are of

to is we So see. growth. quarter-over-quarter I like as as say, I'd for seen much I still will think growth haven't room there substantial

had other primary and of forward We I identification, well. that which as an But this there, testing, catalyst. think grow, of in I see and with advent of go from that's the has ALK, COVID, cetera. that and partially saw think impact Roche, with driven I'm effective half into we et that then we've by patient we second saw we optimistic the seen go as the a is that know impact year we therapies really will itself, that targeted treatment we'll it with certainly, see on here

care, interest with happen. so look the make number Genentech of that really best that actively our grow patient to partners we'll So to and in clearly, that's at we're working see

Operator

comes the Your Company. line with Marc Frahm from and next of question Cowen

Marc Frahm

taking for my Thanks question. Hi.

in there the in between the you detail and and can ASM of numbers trials, on Christy, can then And ISM -- we've be very access strict gain splits able is gray. being when your you're you're your drug clinical And definitions give [ph] you detail once definition with like, patient along patient guidance? your the those on actually seen how with the expect the the as you're the lines, Speaking guidance a to patients making that to in would ISM you those population are of those in ASM? in approved to some guidance

Christy Rossi

Yes.

clinical seen studies. we've in on, that right our you're So

over I It certainly, even accurately given and The struggle typing most challenging any patient subtype is gray I see patients advanced to between of that's think may practice. clinical may see leading or time to patients. more of course a KOLs patients Where their have the be can and think severe. disease. world's we potential at SM we sort it evolve in in can ISM deal with patient ASM, who or aggressive overlap

subtyping seen cetera the you so, estimates know, in tend market, -- better that's think that diagnosis, et we'll to [ph] that we that how I a a X,XXX Fundamentally, of think X,XXX get approximately, that patients. space that those And providers are sense healthcare closely. to as codes, into of uncertainty, we've I know, it's have And the this overlapping patient right? you manage payers I the are not disease. gave reflect

for will access will have that some. care have provider are really assumption they proposition to we optimistic whom that the believes be will patients that for in given is health that utilization compelling we those have patient AYVAKIT the that good value adopt And our our population. initial patients So driven by

Operator

Raymond from Dane Leone the of with comes next The James. line question

Dane Leone

questions, for taking population the what for the Could maybe for thanks congrats the think and in it and crossover questions you perhaps with May provide would of then the be would think you Thank AstraZeneca on to of development And beyond an me, currently SM the guidance the second guidance. study? about start on terms of thinking question mido? with into terms adaptive in to be therapies docs avapritinib and And of in be Orchard you approved? to treated for other opportunity interesting patient you. you switch Do once are many be year, launch. mido of patients one add two be the how color BLU-XXX. would Thank and terms that partner would more willing arm

Jeff Albers

Hi.

So, Christy, the don't the you question and take SM take then BLU-XXX why advanced question, combinations? around Becker

Christy Rossi

sure. Yes,

they health midostaurin, mentioned in perfect have would treated. The it's we that expect that. And patients utilization on. are hard I the TKIs see patients have driver that remarks, be patients diagnosed probably providers maybe time I suggest as we And SM may [indiscernible]. be of staying utilization assume So XX% than who are will midostaurin. on prepared don't difference adjusted treated approval, we less who treated of we with the know patients of is to midostaurin, those data would other label report The with currently insight initial that where with can upon and off care into that treated being there think a would a be

many of patients. be midostaurin can less a unfortunately in sub-optimal, year is is therapy in with duration what so we and well And SM advanced hear that than

confident disease there of to have and value amongst are uptake the and that KOLs certainly expect sort the would AYVAKIT this we will comfortable so see managing proposition they very people understand quickly. who feeling are that SM, And

to sort and growth, and know, time, and would into longer-term community identification KOLs that grow the you with would happen have diagnosed again get than which expect However, treatment patient be over newly see along of of the that go of patients, driver over we'll broader time.

Becker Hewes

be a makes closely. window over look EGFR, program, with is it as very within a the within And the point. combination number our little of great high we're inhibitors, which the combinations a that osimertinib at drugs so different agents. you a remember going of BLU-XXX. partner understand take for this drugs the looking to our to inhibition look studies and it preliminary resistance We'll may of our like to of on like And of BLU-XXX is an BLU-XXX future, of MET combinations for the trials even respect activity to mechanisms address strategy wild-type wild initially combinations at compound tolerability extremely with types at in and different really

Operator

Your Michael line the comes with Schmidt from Guggenheim. next question of

Michael Schmidt

potentially on question my pursue [indiscernible]. avapritinib in Hey, for morning, versus taking -- one thinking GIST? guys, a would And brand label you good potentially or is questions. then will for SM my And had considerations labeling first thanks I different approval labeling might in I would guess [ph] SM? doses two further given factors upon that sub-types in differ and different other of side different potential advanced with potentially affect SM effects the or used given dosing, that how those

Jeff Albers

Christy, take do to that? you want

Christy Rossi

Yes, sure.

are So make we advanced and to obviously, the first AYVAKIT available finish forms urgently actively then working PIONEER. are and of disease non-advanced to working SM in enrollment and and of eagerly

We are the AYVA in non-advanced of profile of about forms disease. the excited

a indication look are So different. is in therapies anticipate like we that doses available. many other different going the benefit-risk don't the AYVAKIT with brand for different to proved We many just -- of that know other different making

to so XX-milligram many makes with of at feel us Part to at the impact really that disease. non-advanced SM, benefit-risk very patients dose And profile we've and for we're the a non-advanced allow X of seen confident a the forms us have data going favorable PIONEER preliminary

potentially know, we're KIT about you could we Of we next-generation the that that even non-advanced further also Becker anticipate number inhibitor. can excited said, as patients impact. of course, And broaden our that BLU-XXX, as

there's of different again differentiate dose dosing, as what to and and the precedent in be different of profiles. as like, to indications both safety profile well plenty benefit-risk labeling a across look able terms in So efficacy

Operator

David comes line of Lebowitz question Stanley. the next Your with from Morgan

David Lebowitz

on GAVRETO. It's David. Good first morning, the us; two from questions

of may specific physicians other? Are lean is say, in the as Retevmo? to RET launch inhibitor used the early So from the question patients be therapy initial types And first second the on there feedback how is our what one versus AYVAKIT. on stage,

the How by registration on death So to clinical the we the the safety regards in of given should to and patient where Thanks. review questions been look have trials? in the focused? the label the expect FDA,

Jeff Albers

have I'll talk from differentiation Maybe physician I'll question the the then about with components the with Christy start second perspective first and GAVRETO.

And that obviously, progress to middle active the the our I we'll back that very I the of the we're we can So making, understanding in tell and you that the in change we're comment of as dig going and quarter that given day-to-day. is the and last the terms mean highlighted on engaged not we're questions going But wouldn't data review, detail. timing through agency given not making into progress nice and and we're level -- and of of forth, disease. I specifics,

Christy Rossi

durable and well has at that about then I and the community not to dosing. once-daily setting. as GAVRETO And deep become types, across think and QT really of mentioned an even point, beginning And as patient I having you hearing, the it's consistently differences safety, market. What used efficacy the been differentiation. been administration regarding breadth into overall before, and that of of as important, compelling are something hear both. then that and we're think have bigger as how I more and there are in feedback there Certainly are picture the some points important with GAVRETO certainly, The Retevmo, well is that's developing physicians this still get prolongation can GAVRETO. we stages important, And are that who as as course, we of we is though responses physicians the certainly seeing there this that are

of to are continuing inhibitor we're first market for getting drugs. what a And that seeing the of here are new patients. prescribers prescribers on know that so the them really add RET there's we to new ton patients go majority out really the that RET opportunity is identify GAVRETO and me prescribers, so tells one the that RET are is prescribing and still of time. opportunities the do these they're prescribers a potential the new to either educate new majority And And we of not and to because for help

Operator

question Raymond, from next the Piper comes line of Sandler. Chris Your

Chris Raymond

thanks. Hey,

on Just molecules. got question the a EGF

with So guys just able have lower dose therapeutically BLU-XXX at BLU-XXX. on you're these, published going through the preclinical doses reach it data you than to like much a active looks

but curious there's differences around the efforts in translating of obviously, sort to about underlying of a kind do into clinic you and So index? mutations, think just how find the therapeutic that here

Jeff Albers

Yes. to take Becker, want do you that?

Becker Hewes

Sure.

pre-clinical to going is with how animals as look where we're There to metabolism. stick a study to taking So are really guide differences exposure data preclinical humans in obviously to between I dosing. on respect at data and

dose BLU-XXX solid a got we've working So BLU-XXX. we're and for already towards that for

of wild-type. amenable them, We to very EGFR then have huge full we them, combine have some both dosing. a to combine be predictions to easily we as expect about where they we molecule both will we expect for over the the see coverage day, of activity them and And get once-daily because full window

as the combination molecules in the about mentioned such and look I of in potentially at The And great separated in we'll we two So really because BLU-XXX combination time are and also we BLU-XXX, more so not clinic get have timeframe. we can about up same is get for earlier, specifics single-agent other to with which expect osimertinib. to as dosing substantially, the do the catch that. dose MTDs to programs as get really real-time then escalate, combination into with

Operator

Securities. of the Benjamin from Reni JMP comes question with next Your line

Reni Benjamin

the taking if patients what to will types the activation. cell that have it just the be of Or there with discussed split? Can be solely lead non-advanced Hey, an the enrolling already it data? good And just mast SM FDA morning, in this that that on I based BLU-XXX. questions. registrational ultimately And on various guys, study the you is exploratory you for you part? are in mentioned Maybe purposes? it will could evenly been registrational is arm the thanks in here Can think you -- remind monoclonal us

Jeff Albers

of patients score program foundation or AYVAKIT in a symptom is in the our the program higher in score. So with TSS is patients

study. plan us with cohort in lower so is both we to study. is one registrational reason registrational optimally program with be symptom on And set Part this now patients effect to our allow score. being to that at right a of have X that looking in patients part the we symptom the The of those X going that up the The at Part groups of our the look two-part obvious have higher We're also study, in to score.

an as refine be the we opportunity the move study will to there forward. registrational of So portion

we're had design program have how the the going general the ultimately study. registrational of We the But our of data we design drive to let the really agency. with discussions part about

Operator

line question from Nierengarten next of comes Wedbush David with Our Securities. the

David Nierengarten

Hey, for aspect us you a one I currently test. mastocytosis require, just confirm quick had patients, a question. on you remind to diagnosing as mentioned the taking marrow I blood a recall, Can just bone testing in diagnosis? thanks So little this confirmatory Thanks. bit still testing? see be the replace you Maybe a a walk or through that? us how future? Or more becoming would other just patient-friendly in patients buying for you

Christy Rossi

Sure.

patient. bone for of right. biopsy, marrow require you're yes, recommended that's criteria every diagnosis a SM The does SM per So

treated getting the a a know going we hurdle, by is on marrow hurdle. at suspicion for that a bone are can of that versus patients know through biopsy. However, And process certainly, non-advanced times, patients, being for actually that are patients based some that and treated you allergists, some be

patients, and be For bone marrow a that advanced continues very would a biopsy important management part expect SM diagnosis to of over time. certainly we

of simpler more testing by identifying For a non-advanced allergists can managed broadly. can that much be that really important a in think patients, patients I and become way easier way blood-based potential

And available. really sensitive highly the that is to blood-based making so key testing

data know KIT that's miss and in we the there So now, you're from not approach sensitivity testing but you patients available DXXXV specificity. can is of our own right if that terms using

enabling pillars to very will to the the really more years is their still a are to to further work who that's up patients our disease access the quickly. lever I may once of require very blood-based more marrow to -- of testing few pathology. think tested. bone But So our next one a patient diagnosis test right patients understand that the key be they get increase and number critical over positive, Certainly,

Operator

comes line Eun the Jefferies. Yang Your question of with from next

Unidentified Analyst

for on had I questions. [ph] couple just Sarah is a This Eun. of

that think BLU-XXX the First cohort, one think Is potential run combination US osimertinib? to to in differentiate a and And cell there that then, syndrome on trial? of would Thank BLU-XXX there mast you currently? And EGFR think versus when that do do trial just expect you you first the line Where program. quick diagnosis a [indiscernible] start on the you. would the activation many patients then exploratory partnership? do in are with how you the

Jeff Albers

Becker?

Becker Hewes

Sarah, I'll about question BLU-XXX start with and the BLU-XXX.

mutations. driver driver in they with patients they remembering mutation driver beginning. the line, important anti-TXXXM of also at treated osimertinib osimertinib dose Osimertinib first the that have that They -- have the in present emergence the and high has Just of potency how driver. is a over why don't TXXXM to activity very prevent

So the additional plus combination those BLU-XXX things BLU-XXX activity. of do and

So the than BLU-XXX nervous compound that in any penetration of better we're or available aware of. development central has system

and in but the often both is initially central disease prevent -- disease patients nervous less existing system central serve system, system nervous so treating the also with mutation, periphery from and which in small nervous nervous system central the disease, to blood than well, penetrated well is. treat driver going central any will the non-measurable And the to

TXXXM. In of to get BLU-XXX we that coverage would be prevent the So amazing throughout emergence BLU-XXX. present context, the

treat regimen that got the that would know of can treat an we entire resistant and that we've here mutations the emerge. So all all-oral body

expect patient our the treatment of longer doing different front populations a we And a front-line clearly, during respect look study, number the resistant the survival. and progression-free to studies And other and first CNS what as line then as well at of two with parallel there is we in disease determine studies that with it in develop to develop mutations. in there we're will enhanced look activity to patients an so would

Operator

question from Peter line of with the comes Barclays. next Your Lawson

Peter Lawson

the taking for Thanks questions.

we kind guess EGFR-mutant agents, you And additional when a essentially? as is areas think They that it, think low-lying in are ways whether fruit about do see of of market, osimertinib combination around kind meets the of Or -- initial to into or I data of in with to clinical kind need be etcetera? follow-up the break you [ph] Just could just patients? we there it's EGFR. outs then

Jeff Albers

the first talk I'll Becker Maybe about then part the take and can that Sure. of development plan.

So agent in AYVAKIT optimal potential in -- our understand various of to across see GAVRETO arms and I dose the terms both starting similar the up with when of arm through best as data, of design with done expanding then trials getting is what to we single and a just mean, and/or as exploratory Becker out combination. these we've molecule, a activity finding laid

up But be AYVAKIT guide data. and similar we would or So the to GAVRETO timeframe to think safe we'd time how from I be to we we those data running get assumption shared and sharing want a before trials when when

Becker Hewes

to showing to the into then three different breaking described I'd osimertinib is in you way as And think market, activity with respect like the and about this buckets.

patients first-generation patients who another on those who with osimertinib, in and on-target which triple-mutant through will a an like and mutation will have CXXXS have moved with been mechanism to mutation MET Iressa then is, Tarceva, often first combination and either the inhibitor TXXXM The resistance like then driver will the patients develop the be and and driver develop mutation, or amplification.

need in to EGFR. So you cases, both still inhibit

in a population. patients look for kinase then to in by combinations patient is really have we a look may mutation eventually, disease single mechanism that modalities their so inhibitors driven with of address EGFR. And other -- other the tyrosine -- agent as other need types those third BLU-XXX subsequent And activity as to, additional are will resistance of line program, our that it in or in at you mentioned,

to very into population. either pressure on our that is point larger proprietary really to the continue the to including mirror mutations. case, longer allow an own to that the in cancer is think keep breaking treatment following at existing lines osimertinib to and respect to EGFR-mutant survival And agents, kind from landscape, osimertinib I treatment molecule with the drugs BLU-XXX combine to progression-free resistant way looking with of combinations EGFR prevent then expect beta or bucket; emerging a into of really that other our disease and it's the marched of in The subsequent lung inhibitor is with an osimertinib we that the the of part mutation, progressing Again early that other patients to adding patients be then frontline may show or rationale there on failing osimertinib, activity all of forms whose

Operator

next Your Brad Credit line Suisse. of from question comes the Canino with

Brad Canino

Good morning.

X. clarity some the comments Just you on in BLU-XXX made about Phase

to dose patients the will You you're are planning include But you. I TXXXM And with and mutations. reason that you in if the could any so, And why other mutations? that mentioned be assume will CXXXS. specific that? expansion you doing Thank enroll outline

Jeff Albers

other to further during ability we to respect mutations, And with about we're asking -- of preclinically we're expand they the So full EGFR profiling have the to which escalation, both mutations of spectrum if the escalating. currently you're against other mutations, active. the compounds while are identify

to of is MET expected later cohorts to of mutation early to would X fit a in plus resistant profiles. inhibition then that pathway that these that be would active other the like determine another the could we the compound part Phase mutations respect inhibiting of where can we compounds So with, tailor about at our where modality expansion amplification, With need so optimal and information the combine look we to EGFR we a combination

Operator

are now the back closing this over will time. remarks. further at no turn for call to There I Jeff questions Albers

Jeff Albers

operator. Thank you,

I'll So with we started. wrap up where

even to date we today, ourselves strategic strong really the these then the this new targeted walked of we're the efforts under as leadership really Christy focus with on second becoming to further highlighted exceptional clear increasingly at candidates the advanced the to going our we and around of talking when as really wave we be the the our probably And with end-user we in profit and I it's our development targets opportunities bit through further but into to sales at the it, in of next PDUFA together, medicines take our patients, all our quarter walked on AYVAKIT well through in. well that potential around Fouad year, ensuring the position, on as focus call should thinking directly [ph] candidates, on new look really we're then a in I about our us And tend that as about Namouni, a us financials. financials, pipeline our with of strong position the half show commercial that's share, time and GAAP the us The of royalty President but five the position financial acceleration systemic these focusing Given quarterly and prepared imperatives, to our various you with Genentech in see of this And walked Mike us portfolio. all that potentially anchor through potential as through and Genentech, that to sales by broadly to anchored more growth way it's recent also continue that GIST milestones. of therapeutic of find CStone, how GAVRETO, patients And Mike case mid-June. and spent over the lot AYVAKIT to as expand bring early collaborations book, as both we bring expand in in we identify research of Becker R&D, portfolio mastocytosis, underscored promise. a time.

we to first year, pandemic. the optimistic a the together in with a of we time lot And certainly in It's doing over for we we're we have nice know, from for call come to actually about so room US, to accelerate that, the this be and as this our continue office to backside so business do. a of in the work ability

to taking look that, With continuing Bye-bye. we forward near the again you so update in for of future. you the to all thank time. And calls in

Operator

you conference for and gentlemen, today's disconnect. your We now concludes and participation, call. Ladies thank you this may

Blueprint Medicines (BPMC) 29 Apr 212021 Q1 Earnings call transcript