Blueprint Medicines (BPMC)

Jeff Albers CEO
Andy Boral Chief Medical Officer
Mike Landsittel VP of Finance
Marion Dorsch Chief Scientific Officer
Kristin Hodous IR
Terence Flynn Goldman Sachs
Laura Chico Raymond James
Marc Frahm Cowen and Company
Konstantinos Aprilakis JMP Securities
Arlinda Lee Canaccord Genuity
Eun Yang Jefferies
Call transcript
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Good day, ladies and gentlemen, and welcome to the Second Quarter 2018 Blueprint Medicines Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions].

As a reminder, this conference call may be recorded. to host like Ms. today’s now would introduce for your conference, I Hodous Blueprint Kristin Medicines. of begin. may you Ma’am,

Kristin Hodous

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

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

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

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

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

is Albers. our CEO, here Jeff Now,

Jeff Albers

we multiple to portfolio Thanks, and good progress programs. with strengthen across important quarter, build everyone. second differentiated the and morning, continued Kristin, In our

encouraging to objective and the very disclosed new with were strategic you our in in with Pharmaceuticals call, initial our patients data which for we mastocytosis at RET-altered avapritinib advanced During is highlighted on cancers update China. CStone systemic on morning, our last presented This in BLU-XXX data partnership recently AACR.

wanted Blueprint to of with half clinical our and includes first to submit defined our rare diseases. we cancers, developing proof growing potent cancer year. focus First, the up genomically highly leveraging portfolio capable of strength aim of medicines with a to however, rapidly globally. compelling of perspective focused on sustainable an research platform, our We business. provide the immunotherapy. for for concept delivering and on diseases scientific company to build multiple Medicines, expanding I candidates and avapritinib additional we selective kinase therapeutic our patients At patients By to in differentiated on Today, defined and stage NDA genomically including three nine programs first plan pipeline next therapies

the believe medicines be beginning constellation strategy that we broader to maintaining we’ll a afford the scalability from infrastructure our and commercial portfolio-driven the our single and for of measured towards will build leverage transformative physicians advancing a and create by patients us peers value our while view pipeline. that by opportunity won’t separates We’re organization but our us. Ultimately, and

since with than results, presented portfolio Phase we Over ramping XX Hematology the a exciting vision now advanced centerpiece make patients responses Congress which the at showing with advanced study with In already These and serve patients durable in program initiated European month. data up making is to initiation strategy. data reality. June, last mastocytosis. combined with quarter, this avapritinib rapidly our we the in towards to pivotal systemic confidence progress the GIST, activated in Association our for last GIST, tremendous strengthen as compelling continued VOYAGER deep more the avapritinib new we June, X in third-line global of sites In

strategy. to of third-line potential registration pillar the support With approval in represents an GIST, trial avapritinib our this important

announced first for As X NDA submit the last to in the the next ongoing of data based first we GIST half avapritinib our PDGFRα quarter, remarkable from for plan year study. NAVIGATOR on Phase we

SM Importantly, pivotal this quarter, the the have underway given data By defined to trials expedite approval potential to-date GIST we to in within of to patient submission. end populations. include GIST avapritinib the expect four of also fourth-line plan we in NDA the and with

populations first in Overall, XXXX. impact XX,XXX and patients opportunity in patient on in the significant. GIST Across care populations In data addition, this to design study we’re at approximately on inform second-line we’re avapritinib estimate a GIST will is year initiated GIST from be patient later focused NAVIGATOR EUX we new trial the to These the second-line of multiple look to track the clinical mastocytosis systemic including for there avapritinib, data a and report updated Japan. patients. the are U.S.,

avapritinib, progress to continue Beyond multiple we exciting other across programs. make

in working and patients half clinical regulatory continued I and enrolling ongoing initial tolerated to in as additional activate been the our months in safety from the robust execution well program sites shows very year, high move Overall, to we’ve expanded encouraged the ARROW registration focus this forward BLU-XXX anti-tumor with globally RET-altered the cancers. patients the earlier, lung at this with data rapidly. Based dose of of early XX data, we sites As on rapidly escalation for as feedback have dose. Phase we’re of rates we ARROW compelling plans hard patients response maximum obtaining In to X noted investigators by BLU-XXX favorable on ahead. we’ll study strategy. activity these study and than evaluate Today, the a thyroid second our enthusiasm in more

towards fibrodysplasia also disease for clinic progressiva. the We’re the program rare the advancing ALKX ossificans BLU-XXX bone

in American healthy submit to a will volunteers. trial abstract the preclinical BLU-XXX the clinical at of presentation enable Recently, and plan. end Bone excited by meeting program that track the BLU-XXX us share year as we Phase on that as to a Research oral Society well were September supporting clinical We’re initiate for was on At an annual XX. X the to data IND Mineral the learn our which for initial meeting, we’ll accepted foundational

addition, we research Last a earlier achieved to a our flourish. ongoing And In targeting pipeline wholly-owned initiated under month, undisclosed genetic resulting program rare immunotherapy $XX a significant research disease we Roche in the milestone milestone new continues with payment. driver. cancer this an year, collaboration million

to with CStone for important this want collaboration Now, a take in partnership We reasons. I June. is our to believe highlight moment announced which we strategic Pharmaceuticals several

enables recent emerging an focus potentially to in innovation of reforms regulatory partnership us First, reach advantage a patient take and un-addressable on the to population. China previously

China ranging for with the sales. patients Medicines milestones ongoing liver to inhibitor our enroll the anti-PD-X has in of Second, in annual on percentage accelerate future an Ultimately, focused This will enable to view sites million us new the end XXXX. Worldwide, programs, on million upfront rapidly checkpoint this the includes cancers financial tiered the our year, naïve half monotherapy clinical opportunity trial plan clinical potential China. to for submit to terms each the us than trial BLU-XXX. Phase XXX the to term. in of more shared finally, partnership the TKI Along we particularly significant development clear will payment, agreement reflect of for in a active $XX studies. from in mid-teens gross X collaboration in By the the combination these occur low-XXs in year, health IND to long potential BLU-XXX royalties investment over a Blueprint further to CStone we company’s so our and the China and authorities. and global there’s China believe Then clinical initiate opportunity cases collaboration’s net fuel at with all

systemic Now in mastocytosis. the Boral his provide I’ll data ask Andy clinical avapritinib perspective on Andy? to advanced

Andy Boral

Jeff, good Thanks, morning, everyone. and

progress we our described, clinical stage As programs. Jeff across tremendous made

working Congress We’re patients. ongoing our an portfolio new to to the mastocytosis Phase of to advanced quickly expand important with activities we EHA in ongoing Stockholm. at X were hard update delivering our in medicines month, share the trial goal Last the trials on with across systemic EXPLORER clinical and advance patients pleased

in as at enrolled we dose long-term patients year, ASH with the the the part expansion meeting. and enrolled the so escalation two of the results part expansion the initial the results results well annual the initiated trial and of trial the dose reported patients the as Last in for at EHA part As completed shared parts. a we trial of Subsequently, reminder, includes on far. we escalation durability for trial focus

we presented the a response response durable profound We’re ASH. show overall based criteria. of very on an data rate represents XX% encouraged earlier response with from the dataset activity clinical deepening that the IWG rigorous of This at and

the the reported data durable only response be EHA preliminary on durability months. response XX% better avapritinib for criteria. results rate the of also comparable on based than midostaurin, a is FDA responses poster view In to medicine to approved we very SM our IWG advanced the Importantly, ongoing of initial appear XX The up which had positive. from

study length view been are in patients. almost addition, patients potential to and remain these after used the two our the of years avapritinib on In XX% a time data an half be start, consistent in is These hematological have malignancy. impressive advanced which has treatment that of patients with chronically

and tolerated. only Most is adverse In patients discontinued three to addition, adverse therapy the moderate events data were treatment-related avapritinib events. showed to well due mild that generally

designed Looking with PRO systemic forward, a outcome patients patient survey reported we specifically are or for implementing mastocytosis.

of further avapritinib also life. quality that the to patient’s benefit the expect provides characterize We to

cells, These Importantly, has a that all in KIT show mutant to mast data DXXXV allele burden. tryptase, in give the fraction. marrow cell improvements potential serum patients population. spleen clinical objective in confidence approved be data profound The bone very and showed robust broad reductions mast volume us avapritinib

XX% that’s been an resolution other marrow, cells bone with of exciting therapies. complete neoplastic patients In before outcome in addition, seen of mast their showed never

in Overall, mast is of multiple trial. and burden. patients the show avapritinib’s selectivity advanced encouraging to common smoldering mastocytosis. see mutant, exquisite more disease patients with measures systemic robust and driver data We cell in which an disease enrolled were the nearly mastocytosis the patients patients the patients the on demonstrate two form also for intermediate improvements the these unique across both DXXXV data spectrum all with EXPLORER these Similar KIT in of of disease, systemic clinical

reported FDA to next PATHFINDER data patients with clear the patients XX have This mastocytosis support with including required single-arm primary clinical will Phase enroll The We outcomes, criteria. progression-free to and and in evaluable endpoint naïve patients. be up patient X or key some trial safety. both favorable approval compelling previously IWG quarter. SM, and endpoints So advanced survival steps. finding be to the overall and will initiate open-label, feedback, will have clinical for advanced plan IWG and third survival, per an systemic will with C to Patients in the we treated treatment trial secondary finding designed include response

clinical across dosing, the Importantly, index investigators believe benefit avapritinib demonstrated tested to and clinical maximize we all strong those will levels has optimize tolerability. wide enable and activity therapeutic to

support open-label X selection, dose selection. intend We including efficacy second extension. Phase randomized PIONEER, will patients will study, SM. trial seamless so sequentially trial a to smoldering a a dose intended escalation indolent do in study, – not dose the The The [ph] design and escalate be to and in me, assessment with placebo-controlled follow approval excuse

smoldering patients have receive trial will for The reported outcome Importantly, be with mastocytosis. the [indiscernible]. indolent patient primary who the in a developed enroll will survey avapritinib and to specifically all opportunity systemic endpoint patients

and activity Now how for and advanced devastation home many with improved to SM. I where both inhibitor avapritinib highlighting the the of program our We’ve with strong by at treatment stories of in Xtrial I that potentially encouraging avapritinib. demonstrated This Blueprint in path rapid driver updated Phase highly the strategy data to identified want heard patients by investigators a medicine, selective a personally disease bring and approval. from clinical excited the early possibility this am close Medicines matched genomic embodies disease we’ve kinase

serious other this program patients as as candidates is help advance that therapeutic goal can with quickly cancer Our additional diseases. now and well to

over the Mike our second Mike? quarter. to I’ll the turn to Now, for financial results call review

Mike Landsittel

Andy. Thanks,

key X million expenses increase So trial advanced operating growth million as I’ll recognition from expenses, study $XX was Phase quarter SM pre-commercial of we for QX upon agreement. $XX.X operating represents quarter, were received well personnel our run prior in of our we Phase initiation portfolio payment the late-stage million in expenses the with X increased G&A Phase The million the of to with professional year. With start CStone of year the the by expansion the the X BLU-XXX, execution increase to the costs our million from $X.X third-line performance personnel-related rate were and avapritinib, in the was across increase preparations and and manufacturing quarter an in reviewing as the increase in of recognized of revenues incurred study This XXXX. $XX.X then which primarily of increase the collaboration expenses an and of to fees, GIST, due continue drivers in R&D from The of our second investments infrastructure. second due guidance. $XX.X our for the prior respect of increased cover upfront including the that development the to million revenue planning activities. as build cash position our quarter I’ll XXXX. we second $XX.X in We

our to balance sheet. now Turning

in which financial equivalents to as We We from quarter XXXX. as with portfolio. to second and $XXX.X the compared ended to believe to the continue decrease payment milestone CStone cash was strong the XXXX. to from position million second upfront by were execution investments of us related received the in capital December both payment our operate of fund cash $XX which us cash cash, XX, the The position our entire provides million Roche, used million focus million $XX allows We on into current $XXX.X of a across and from operations operations partially of quarter. our second half that necessary offset the

progress year-to-date months and our made further we expanding development ahead. activities look pre-commercial the We and have we efforts pleased to by very forward in are clinical the

will bring into the expect cancers new rare for of operator other expenses we the questions. such, I As With over will diseases. we meaningful increase that continue call the our with to our as turn genomically operating hands that, to therapies efforts and Operator? patients accelerate now to defined


you. Thank [Operator Instructions].

first question Terence comes Our from Goldman Flynn Sachs. with

open. now is line Your

Terence Flynn

KIT-driven first, next NAVIGATOR Thanks FDA you that us front? was that did Jeff, Thanks. solely that you remarks some avapritinib see in second-line the then And Hi. trial fourth-line during that be you you’re or Maybe dialogue as Can what on for mentioned to well. GIST the question. mentioned taking filing on if tell for based be a also your the Great. XXXX? have want just data on going you to this decision a avapritinib, with And into guys you would do for in to data GIST, there study? the advance that pivotal

Jeff Albers

onto the Thanks, one and the then first Terence. I’ll question take I’ll pass Andy. second

an regulatory of So as with filing any it’s both data accumulation and feedback.

as And on so there and currently obviously and we general GIST position makes GIST on clear we we data medical fourth-line which second to-date, year. no and and data clinical of would around you view and in sense the provide line feedback still based to updates concrete evaluate patients data when are is like. where have more one share it we’ll to that in hand gather this later We have that with path need we’ll treatments, the we’ll study available continue are look PDGFRα in And the was And then filing. that into a our the second that. blend the know question fourth-line forward what more

Andy Boral

Thanks, Terence.

study this genetic patients on focus with before said plan trial year. potential line on decision subset on two look like or where to degree as this update in against [ph] either later initiate the very in a XXXX something later data So high be could in a arm have to the would be we second-line a at study study will continue a focus on when we’ve we and in on that activity we that data-driven is a year. well – there could of see and approach. CTEN based it all to as enroll arm that The results and avapritinib disclose randomized comers the We that we of general selection NAVIGATOR does we really prompt second plan the will the

Jeff Albers

be either a Yes, case registration -- but would that in

Andy Boral

agree. I


you. Thank

Chico Our from with James. comes next question Raymond Laura

line now open. Your is

Laura Chico

little Rydapt, have about I Xx and data. If around I focus think question. Hi. look and pertinent you did still there. the help guess that me Good it at thanks bit and for Thanks a I you the PRs it’s SM. on I’m rate, can EHA for two Rydapt comments morning. looks just context into wondering put the label taking like Andy, versus on against I if CRs a higher. just response the

basis? that going estimating CRH I then but should CR/PR missed you’re that and on it that PATHFINDER the mentioned follow that be the how you’re range. I kind we am PATHFINDER. I responses you got exploring wanted in thinking on know potential dosing be using well in? one how understand Thanks. I as So But there. lower to might we clarity much study? planned And And doses on the yet just PIONEER curious, up about I just correctly don’t fits think have of to in a

Jeff Albers

Laura. Thanks,

I the It’s It’s imagine was the And there XX%. funny; excess that in an should cut. question. coming there the either is number. CRH your remember based low So said important about that’s XX% SM CR/PR data, we comparable response in and CI, at terms systemic on the XX% rate again in of when mastocytosis The to in a U.S. EXPLORER CR/PR. rate emerging, I where question about look the data that’s response XXs. of label see the so and is ours XX% exact midostaurin. from and we in I is as in and midostaurin from that’s was It’s

And CRH always make One, because CRH, points. I them call CRs. a So it of the of they we not, either or CR/PR whether together doesn’t guess are I want CR/PR. they’re include – you to PRs will out couple are CRH incorporating CRH or into

really that’s the strong affect field completely leukemia acute patients has blood recovered. not cell have or that CR/PR recommendation the platelet complete hemoglobin they count doesn’t count their but the and mast initial that’s it And total of from now So their The case with recovery or complete commonly CRH CRH is of cells that mast in in counts, of their of the investigators of their other burden where lack resolution leukemia. rate. taken our if resolution used have a

that’s do systemic an So think in we and as advanced well. endpoint mastocytosis important relevant

we we far of chronic reported recently avapritinib both once to SM decided milligrams an as activity the we ASH study, use in proceed across PATHFINDER optimize day to So very have support avapritinib as in We’ve advanced in with a range potent anticipate these as with used EHA of a wide the dose XXX and patients. in order at us and be seen gives to luxury this to at doses dosing that PATHFINDER.

So a be will XXX it milligrams day.


Thank you.

comes question from and next Company. Cowen Our Frahm Marc with

now is line open. Your

Marc Frahm

of just that kind to up Maybe identifying efficacy at of lower surprising burdensome your that match does lower the better the more the section the you’re certainly have as and about making you first these the up for to Yes. retain dose lower going their signals efficacy patients doses tolerability been dosing. going and a on give indolent see at what to should there where for particularly us of started those some parts disease PROs, doses robust to to I maybe something But setting. truly confidence chronic that of of you’ve patients? to kind with follow are understand most smoldering in when kind question is you’ve last through disease,

Jeff Albers

and question the on blend a the – the the talk tool then decision little about do together. obviously take and part around XXX they milligrams I’ll they PRO bit of Maybe first question Andy

the XXXX, when then in XXX day a we’re that starting per shared evidence clinical to up, escalation, we we at first doses saw as the moving low as reminder and of dose at range. milligram the up XX all Just data doing milligrams as ASH benefits

are in into and PATHFINDER advanced but response that where rigid you is. virtually look with you that so is that about you a when And some clinical where And the get optimal can’t what a have efficacy rate luxury the confirming push you when what’s situation used at higher afford benefit Andy word setting, we patients a very a we than of all to dose sit. XX% criteria, does flexibility think striking

investigators view our driven this to is our with to that that milligrams. were benefit we and maximal really get starting XXX work by seeing So closer was

will do feels So it that’s a deescalate evaluate you’re to talking to like go about flexibility just therapy want to clinicians continue higher, and important. the always chronic want when But be do right step. they going they to to treatment, want

systemic have specific bit And piece So both across I what DXXXV a think mastocytosis. we was and that of on. about the potent is then inhibitor of the very we’re more the PRO the clearly span blends a to little focused which the driver second of

Andy Boral

think also about Marc, maybe just was correct the I me and lower it but particular in dose. ISM piece

SM So just looking – advanced dosing. than everyone remind the bit to actually at I quite the a I ISM we’re think dosing different want

So lower active if PIONEER Really there first will understand those active, patients the be a in life could is patients drug for normal on exploring we have therapy doses part expectancy that we be decades. the in the study. and an these substantially of

outcome – active actually is the tool is allow we’ll of updated this dose prolonged study very X going dosing when patient data detail of to data there, we more year. at EXPLORER and a look present lowest actually the range in XX to will course, criteria it’s critical just pretty ongoing The finding we criteria, to reported that describe – PRO – on So later potentially.

closely is two things. just similar maybe we’re and So SM I’ll give I’m We’ll criteria. The advanced the a on kinds Maybe ISM bit PRO. at actually related looks see of – symptoms fair mixing PRO detail the our far from that improving that data we of EXPLORER, relevant that we the are pretty confident so from just PRO that say we’re developing. to

Marc Frahm

XXX? kind with lower. you up no there’s milligrams lower is with you blend? indolent pattern at matching are design the of PRO guess real sarcastic is that disease or efficacy those that setting [ph] is and see my question symptoms I which right, advanced XX But do of it of we much the see patients, the at data and XX have, the kind milligrams improvement improving to – aspects signs Is sound things as in disease they of as than more you what for for see your you’ve more

Andy Boral

GI It’s with you very related around we which want indolent of see in disease. line would I is are dramatic the is to mast we important bone compartments; Patients of think in to thing burden really the see fatigue and that that shows burden reductions due what the in where for whole blood be and spleen, is variety all symptoms have the and the issues the total patients the to spleen, drives enlarged often really. the – And marrow. body body who cell splenomegaly that counts. DXXXV what And in might systems burden allele in

in driver reduction and the what disease burden range see filtration and we improving. of the the the broad So overall of is that’s systemic the symptoms

Marc Frahm

of or something be great. had there treatment HCC wait or are have thought next still think until And and – updates I also program from we XXXX? FGFRX that months is the to six naïve that the then then planned going RET Okay, to also would in that’s possibly just the data they so, cohort

Jeff Albers

So range is there a updates. of

combination or update time; the really BLU-XXX, inhibitor more today a to find year mastocytosis. year one But do providing to systemic well BLU-XXX given on a this in later GIST we’ve and accelerator year. in be data and presentation. an past look and as an I we’ll view BLU-XXX avapritinib later the a indicated provide of population next lung a the clinical update We’ll collaboration, for we and in trial. cancer. on provide as an update patient the a we on an highlight well discussed later further as both avapritinib there medullary – for And with that year there’s variety RET we the importantly ongoing what will TKI SM preclinical ongoing then indicated the CStone as provide we at strategy as Andy we’ll regulatory later in both of discussions them We’ve landscape an as we’ll September opportunity the as strategy this that thyroid for highlighted, we’ve on FGFRX oral evolving BLU-XXX that both the GIST year update updates naïve BLU-XXX data early have this within provide update So additional and XX, ongoing indicated We’ll this HCC. in have broadly we is

never So as definitive. we have definitive –

mastocytosis. evolve ongoing BLU-XXX feedback As to terms important, that the we update trials, pivotal systemic get or as through new Andy we’ll in our we also there. trial. trials And of in view then amend we’ll look provide And two then updates walked an provide we as

Marc Frahm

Okay, Thanks. great.


Thank you.

Securities. JMP with Aprilakis Konstantinos from comes question next Our

Your Mr. un-mute. if Aprilakis, is now line muted your line open. is please

Konstantinos Aprilakis


that. about Sorry

me RET to see but is X? and cohort do need move cohorts So expansion covered, there of it’s either we been the this in walk for to the could to I’m phase. I of mention rationale if the what sorry through sort program, Phase think four first BLU-XXX, on your from these you

Jeff Albers

Andy, do you take want to that?

Andy Boral


Konstantinos. thanks, So

also X our studies minute potentially initial study So in for current other seamless with expansion and of were BLU-XXX, a it’s I’ll designed, Phase think path. X be to and then will we to as effectively a the get Phase registration a expansion further MTD a

recommended now and So at reported ACR escalated the to as we’ve we’re year dose. MTD the this at expanding we

more anticipation more had for cohorts There cancer. Those of we’ve then all of paths. TKI cohorts so to where And the that of two And patients increasing RET-altered basket and to sites. four medullary sites or are the have will think Europe we that XX lung and non-small substantially whether several of cell not. as increasing opened a registration number sites the cancer the are to prior data potential a Asia already in in cancer we’re at looking then We lung now study the solid actively a going think cohort are enrolling and thyroid tumors. RET-altered We seen there. go according the than both patients U.S., divide those already study have in continue I number We the robust well, offer expanding we up. on very the footprint

pleased really the point across RET seen and activity we’re that across clinical with mutations. So consistent we’ve fusions

mutations are thyroid actually current approval emerge alteration the baseline, that can and could could approaches expansion could specific agnostic offers for medullary there the for that point or think be focus ongoing both of we from be opportunities approach. agnostic both. those to cancer be tumor opportunity cancer tumor a So study. all But focused with lung with fusions, potentially And the also

plans our with as discussions emerge data FDA. update will and the based We on

Konstantinos Aprilakis

target enrollment you per cohort? guided the that What’s Have yet?

Andy Boral

The increasing per where and XX be current has that will study be ClinicalTrials.gov. that out in we’ll coming cohort

Konstantinos Aprilakis

Okay. very much. Thanks


Our Lee comes with Canaccord. from next Arlinda question

line is open. now Your

Arlinda Lee

questions. second-line datasets Hi, guys. I to up my wanted that Thanks year. avapritinib see expecting we’re taking for the to later follow this on

to to kind on the burden or on but allowed based receive patients since dose Will will patients dose patients the trial, subsets XXXX efficacy going [ph] from going other kind going in for as mastocytosis talked this you’re duration these the on on later see Will drugs of patients, to starting start be association year trial. or is doing of need another to And You treatment be the update fixed whatever? to leeway SM allowed or there know Thanks. – we titrating what treatment get update a the is about based year be us disease? dose of in terms are the a you’re there see and component the an why in in be disease? XXXX, to compartments to of are and I indolent the you’re in going other for doing be you then going update? some later And of this

Jeff Albers

Sure. the and can the about systemic later I’ll Thanks, Andy question year Arlinda. take around mastocytosis. first components different talk the data this update maybe of then you

will much Andy obviously that time, trial RET has be Phase So in the the the from overall data trial trial over trial evolved over the update evolve that can talked just time. that and way ongoing about

to decision know updated the because the what in forward good will indicator we our a indicator a early update ongoing VOYAGER at fourth-line then of of [indiscernible]. move And with to second-line to with the we will basis be arm trial – that It or registration head-to-head pretty be on an filing data that as to The is patients And dataset. guide forward which it a so patient that plus our from will optimal haven’t PDGFRα second-line include study what updated as guide to a that’s that our and we’d trial help the that move include of will path latest you add that patients, population. it think include earlier protocol third-line had to in our we it that was important that what time. amendment a amendment was see will decision to expect will so avapritinib we

the have understand clear prior think to me be and biopsy an So for enough all data understanding we our different I on totality. we to of disease. in blend take circulating this at the share think that’s for have, hard of impact understanding moment. answer – avapritinib’s forward, it that the all We’ll going for it’s on DNA handle therapies, with we and all a your package getting will we’re you data path progression increasingly and that. to tumor That’s of we clear you the that benefit have of To registration question stage gestations total data that how disease, the need

like a is with move been our or lines to exactly is intrigued And talked in we’ve line, of seeing a understanding mutations clearly seeing seeing we’re clear so rationale that And the real What that the of have strategy matters. there’s and avapritinib is by later range the the benefit what we’re earlier. that to by avapritinib calls showing that core several matters. what about on consistency breadth with last molecule activity

in second-line based totality GIST also decision peer we’re seeing various across be And the so will patients or lines. based second-line on is the patients our of but both activity it’s

a avapritinib overall these can data benefit array based that such think around will we’ve strategy have understanding of you is piece what broad clear need the I So our will on and of why offer -- to GIST the speculate patients. is a decided to-date we because there that go on within at population, a and Then that update to clear second patient clear across

Andy Boral

the at in to that we dose placebo-controlled assessment which be to will very patient regarding is study part use dose do efficacy population. take of is part reduce to dose period that be PIONEER patients expect so Certainly the all start the reported a study same time. the is Arlinda, and the for the a to of We Then in study again necessary for at to as that’s the the well ISM will SM, again plan tool. randomized able forward robustly tool the efficacy the Yes, part the prolonged study, outcome a patients using tolerated selection in PRO of portion dose first that optimal implementing will randomized effects. if side access the dose determine

group be of affect other would kind able the targeting itself. still of of certainly the it course to symptomatic There the are approved nothing specifically that well. symptomatic going would thing. give drugs, as standard terms therapy are take drugs placebo therapy cromolyn patients of on care be so disease, be In The standard will will there backbone that disease no that care antihistamines and patients get and underlying of else and be to the to of

Arlinda Lee

million hear until really Roche, milestone to then curious. maybe or on about we hits Are XX these going The just Okay. question. not last anything clinic? programs Thank And guess a I something the you.

Jeff Albers

some Roche. advance set progress along make. indicative of enter Yes, the is opportunities the to with the a these nice that on, find were would of I hope milestone’s that And we obviously we at before we will But team decision the look programs make certainly as clinic. to continue that earlier to updates we we’ll provide

Arlinda Lee

great. Thank you. Okay,


Thank you.

comes question Jefferies. with Eun Yang next Our from

Your now line is open.

Eun Yang

GIST. couple second-line A questions of you. Thank on

about GIST. So second-line response a a rate competitor inhibitor in over XX% KIT showed

estimate thinking on [ph] that mutation? hover the a comment patients also as be based can set XX I bar patients, KITs kind second-line you number patients, and we genotype this of small on you the percent what should of So know but about in given of you And can GIST the if you don’t patients. XX overall

Jeff Albers

can I’ll add color and to Sure. start Maybe Andy or that. then Marion can

we regulatory want I to provide what in bar an So we of bar share or GIST, a in feedback. get as second-line regulatory update terms think data actual is or a

a proof at that think whether importantly or not seeing which XX%, benefit. shown response too I has or still guess rate. would XX% plus XX% you’re early. X% mean a [indiscernible] it’s The therapy only I X% is are

move So the it in see genetic as think forms line activity the to as of But right – is. there’s we understanding classic reason underlying determining therapy. we that intersection greater range avapritinib what activity most GIST that is is believe bar and matters drivers cancer well of as of expect different to no That’s across we to would in there. and up of mutations

we’ve patient just actual specific terms not – whether piece mutations of what the the an not only what been with point specific second patient by but on who that avapritinib. shared of XX GLEEVEC. treated One important better less it’s is the for ways. understanding in mutations based But is In that’s maybe occur, to-date mutations data think Exon a of answer I are matters I’ll two within a question the or for has Exon

update KIT-driven for So provide lines update across broader both well when an activity year. look this to understanding seen a across they as range we as and what the much seeing they are what GIST of where are what mutations so clinical on we’re we’re we’ll matters seeing a but activity later provide avapritinib we’ve specific

Eun Yang

because with advanced you you pre-commercial move are drug and drug after are thinking going high And SM, priced there multiple particularly doing of a registrational as into a and is helpful. activities, indications dosing avapritinib trials there. how different you the start precedent That’s and out preclinical, about with pricing

So you about the can think about what you you’re pricing on strategy? thinking comment – how

Jeff Albers

Thank you.

the this So where have before hand I start a But we and activity think with very have finalize seen patient early talking give quite regulatory feedback KIT-driven robust there about data in populations is I or is patient refractory we we’ll a to SM specific in clear the highly molecule want that price. or we hand GIST different specifics all and population. think in you’re to in need, several it’s whether where advanced a PDGFRα

be as life is new clear in need doubt there we expectancy consistent at that think much a piece our pricing mastocytosis. actually patients also expanding we these in and move that mastocytosis, systemic likely with need that As where will we but have areas indolent believe I those we’ll into dose systemic short of in important with patients a such about the that a forward think think how no would indolent greatest lower there’s

pricing So symptom we think make But that patients. we’ll pricing do potentially to burden a in have some flexibility population. those high is clear. lower There want I in

seen that a what very I those time symptom high think patients. meaningfully more indolent so over mastocytosis anything sometimes if learned percentage patients in systemic is of mastocytosis we’ve there than burden, some systemic we’ve having the are about advanced of

of clear so. the or very come into when year series high need, clear a you pricing a – blend over with that active play will next And a diseases we with that, relatively so think molecule strategy

Eun Yang

you. Thank

no yes Just one and really question. it’s quick question, a

Roche So in booked XX to going once [ph] quarter? milestone, it million be is third

Mike Landsittel

is This Mike.

we’ll later to more payment, more price actually over the details the research XX-Q the we’ll when essentially as a of have we’ll accounted into So for is know on how we’ve the our based accounting proportional And But today. have agreement. the term filing overall today, But we effectively similar method that details have in upfront transaction spread the that – milestone file accounting. Roche later deal. be XX-Q the will on for of you folded

Eun Yang

Thank you.


you. Thank

next comes question Nierengarten Securities. from Our Wedbush David with

questions please line I to for this am Your at call now is turn Mr. Albers Nierengarten, closing further like would line over un-mute. back Jeff I your time. muted, is if open. the showing any not remarks. now to

Jeff Albers

bad feel about David. I

joining and So again, is of the thanks all for a thank and everyone taking I operator day busy you know it for for time. you.

a building vision in as of of year on first year. for of our and closer underway drug By to our fully enabling half this realizing to these up move we the days the trials are new track integrated we’ll us at As five be business. next talked application earlier, Blueprint registration we have about to end first we’ll exciting clinical Medicines submit

a we exciting our plan and medicines and work every we have and it a on with So updates patients further of you. great look really moment. lot forward day. our think all delivering this to sharing Thanks to goal day progress achieve of is an on We to


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

You great may all disconnect. Everyone, day. have a