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Blueprint Medicines (BPMC)

Participants
Kristin Hodous Senior Manager, Investor Relations
Jeff Albers Chief Executive Officer
Christy Rossi Chief Commercial Officer
Mike Landsittel Chief Finance Officer
Andy Boral Chief Medical Officer
Marion Dorsch Chief Scientific Officer
Terence Flynn Goldman Sachs
Joseph Thome Cowen and Company
Charles Zhu Guggenheim Securities
David Nierengarten Wedbush Securities
Andrew Berens SVB Leerink
Benedict Shim Canaccord
David Leibowitz Morgan Stanley
Eun Yang Jefferies
Call transcript
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Operator

Good morning, and welcome to the Blueprint Medicines First Quarter 2019 Financial and Operating Results Conference Call. My name is Carla, I'll be your conference operator today. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question-and-answer session. [Operator Instructions] Thank you. to Medicines. call like Hodous to would Blueprint of I turn over the Kristin And please. ahead Go

Kristin Hodous

Thank you, first 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

website Chief results. reviewing be Investors will XXXX the as answer Dorsch Financial our first during Albers, quarter the update quarter we'll Dr. Blueprint Rossi, Andy Officer; our Officer Dr. be our Commercial will Christy Officer well also available section on the call. press strategy; the an provide our slides our portfolio-based review financial our Medicines highlights; on to as our today Officer www.blueprintmedicines.com. will Chief Scientific release Marion access commercial going call, can the and our You of Officer to by Today Chief Chief first at of Boral, will our Q&A Medical Chief portion business that questions Landsittel, and Jeff your XXXX Executive discuss Mike

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

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

Albers. Now, CEO, Jeff here's our

Jeff Albers

financial morning results first good Welcome call. to everyone. quarter our Kristin, Thanks, and

a Officer, our least our progress; Rossi based time position. on the Commercial Blueprint and We will multiple solidified patients months. Blueprint of XX planned programs fully end the achievements our global BLU-XXX Chief foundation two marketing This this closed them. first key precision build Over transforming biopharmaceutical need we'll we company. multiple U.S. and focus urgency pending for applications at last innovative and accelerated in continued with commercial BLU-XXX, year, two XXXX, for to offering our commercial a the strategy to the strategy, marketed clinical into hires, global BLU-XXX announced follow-on view company a Medicines programs, regulatory and four with Christy have commitment months therapies first the avapritinib, financial deliver core to we second that Earlier who strengthens business. towards several integrated for launch and These recent the review therapies next our we applications XXXX themes: marketing highlight morning, BLU-XXX acceleration our of Europe. I'll or and By to underscore over avapritinib on expect to the the

GIST, patients our vision. this this clinical therapies. two have towards progress track our to currently Most In that for importantly, no to on an made groups PDGFRα expect submit and NDA with expand we and also important approved patient pipelines. first We quarter, first achieving avapritinib for further we're the discovery-stage quarter fourth-line

an Medicines to quarter. to addition, we in European In Agency announced the plan third MAA submit the

approval. X confidence combined be the results from we PATHFINDER accelerate was announced support plans EXPLORER mastocytosis, submission X from the preliminary data that by with the quarter could trial avapritinib to in an program first Phase trial NDA driven into decision of increased Phase XXXX. systemic For This sufficient our the to the

additional and we'll FDA advance continue to to path quickest trials data forward. with both work define become available, the the As

for ARROW Based the reached X on enrollment achievement previously for of our the to the target submission patients early in non-small an cancer cancer cancer. we've of first BLU-XXX, enrollment cohort announced Phase registration-enabling quarter targets For treated XXXX. lung cohort, with for RET-mutant thyroid NDA lung we RET-fusion into and the accelerate trial medullary cell plans

excludes In platinum-based BLU-XXX lung for cell progressed any addition, Designation for and PD chemotherapy. or PD-LX prior we're line second strategy. Breakthrough therapy for This has aligns time positive with pleased to broad have that initial following non-small Therapy received our cancer second this RET-fusion indication our registration requirement

under collaboration trial. trial our in program cleared the we the us hepatocellular patient in In BLU-XXX HCC IND ongoing patients first Pharmaceuticals. China. CStone the been Chinese We dosed for quarter, our X the authorities TKI-naive enabling morning, with our announced This this to also advance to with Phase Chinese has first to bring carcinoma continued health

XXXX. updates pipeline. fourth with further stage the represent program follow-on trial these programs in expand for BLU-XXX completed offering strengthened the the experts on clinical a in from and quarter fibrodysplasia ossificans acceleration of advance plans our trial of these pull X And into of All Phase our ongoing X portfolio. to April, our we volunteers Finally, healthy forward in financial to patients advance and initiation together, nicely. to with progress clinical the announced Phase progressiva the input foundation continues Based research FOP we've our

trial annual NDA the cancer near-term and to early and June, to important in planned plan milestones. PDGFRα thyroid lung detailed MAA avapritinib present data of ASCO registration the addition we data submissions medullary In and and BLU-XXX for results GIST avapritinib, we meeting several cancer. and in fourth-line for RET-altered from At our ARROW anticipate updated

to each for European in plan the at avapritinib Hematology in updates updated systemic response plan clinical Congress efficacy, data share mid-June. trial including duration In advanced of of rate our mastocytosis to the is safety. present of addition, we EXPLORER datasets we which these Society on and important and Across

call put Since cylinders, building also turn all is in developing a over capabilities. and guide forward. fire continue the a clear place path to to our team to on commercial rapidly leadership has clinical more and Christy we're details. share commercial our strategy I'll to As Christy onboard regulatory teams now coming

Christy Rossi

which great to be to this Medicines providers Medicines towards step Blueprint share on organization One It's I six approach journey our is operations these are program to broader About directly example experience Blueprint how morning patients joined therapies approach initial to One needs. who exciting to in Medicines the Biogen patients the the clinical other the in efforts the behalf the healthcare you to will Genzyme team on our among three deeply to all key with important perspective with thinking better created Jeff. patients community, and Genzyme, to building field crowded on customer-centric focused unique medicines priorities. aspects this my by worked waiting healthcare bringing commercial franchise launched multiple next at introduced and October, leader with of and ground understand Genzyme, specific of tirelessly Ultimately, me around this I've all months needs who successfully partnering their Since lead importance day. Sanofi led others ago, healthcare support address North their and A and reimbursement their business them. and engaging in Thanks, for sclerosis as and Blueprint this the franchise I At the a Sanofi the the up provide Sanofi MS MS and unit drives company Previously, two joining oversaw and portfolio. where and designed patients from our providers. American of at across I therapies. in with a taught providers healthcare we MS so and at multiple patient our of each superior providers This outcomes.

our and strategy. talent stakeholders to and our portfolio clinical teams; externally internally a and establishing with First, assemble and top second, diverse scientific initial third, go-to-market leadership with engaging key strong team; recruiting

BLU-XXX a impressed with integrated rigor and augment the hires on I've launch evolution I in Europe. am we in the up leaders the our team market across now of Medicines' plan the serve, we've of marketing biopharmaceutical to affairs step thrilled medical corporate marketing place, with science, and leadership In this patient areas. been sales, As core and with team, roles effectively patients. speed, access, strategy, Blueprint prior submit oncology, of strong population. specialty medical we're Under engagement to deep we impact all building strong our therapeutic our and With our clinical communities announced in to working disease of with of leading are U.S. depth rare excelled and provide team range experience commercial avapritinib the deliver to strategies applications for which key highly experienced and I've have bring a and commercial company. for implementing the and to assembling. to who capabilities of a our Individuals this foundation in biopharmaceutical focused companies Blueprint and XXXX onboard medicines months, research recent we've into next our speed multiple across existing got fully gotten are in

a focus areas. across a portfolio the multiple therapies of in to therapy commercial disease precision Our launching extends single indication building foundation beyond a single for

plan with Over months, effectively, geographies. we and XX scale. to indications to innovative balance launch do we XX spanning To focus to multiple the need two this next therapies,

providers in portfolio also needs effectively to and while address Our our healthcare customers. unique more our will approach reach the of patients specific leveraging indications,

organization seek will in optimize growth. focus to capabilities to avapritinib foundation this organization build launches long-term we balance are the for the commercial initial our using between field the globally BLU-XXX while example planned U.S., strategy to Our core of and delivering and scale. create an is The

important providers better With also clinically in cancer. has field and for oncology healthcare cancer This and commercially, focusing analyzed to this we enable different us GIST considered including mastocytosis. on target both Scientifically, focus insights. single accounts team overlap therapeutic analyses approach, PDGFRalpha in define treating of and and cancer. to foundation create a GIST, resulted understand populations are we've of have will As to with One and Avapritinib GIST core support about accounts. These targeted lung the strategy behavior. areas, lung cancer build and to footprint insight XX% on healthcare of efforts fourth-line our lung a launch to several success systemic lung the GIST data example prescribing testing available provider approaches, our informed we've that to is and

to system and the As development Avapritinib targeted to clinical advance awareness we and we identification Similarly, patient will we work the in of improve using Avapritinib patient launches we insights, generate areas, supporting scientific to as new to BLU-XXX are and and patient we critical initially strategy. populations. facilitate and optimize across these experience continuously setting, gaining data bring our through we more are these continue commercial outcomes with plan and which the to BLU-XXX expand clinical understanding testing effective drive of our stakeholders across and Through healthcare access. first therapies, increased gain

and these genomically core the now the expect a our and a risk will our discussion serious access. value Avapritinib with to organization. of term to testing other insights from and therapeutic These stakeholders potential that our to rare that and on of landscape. BLU-XXX payers provide research therapies efforts and system, new full providers, populations innovations the approach. of underway, deliver creating our and aim the transformative patient we indications principles Fundamentally, importance work provide realize are today's the about will over We scientific context we within commercial medicines and range obtaining us this enable engaging and reduce dialogue the We believe for with the diseases productive across improve across around focus long defined further build patients, to areas. on foundational We evolving could healthcare recognize facilitate healthcare value investigational care especially as stakeholders these

care, to look make I confident journey. step Blueprint excited quarter. ahead, next our this financial turn first call the results impact over for incredibly I'll can patient about as the Mike we Medicines to I'm now the in take our As and on review

Mike Landsittel

highlights touch on we in press detailed This today, few morning XXXX quarter the financial I'll reported Earlier quarter. from a Christy. just release. results financial first Thanks, our

end acceleration have commercial offering financial the position anticipated First, portfolio. investments that public closed $XXX.X in of very in strong follow-on and were the of our $XXX.X cash key approximately and to the April, net a proceeds and at million our raised support with is pleased million to the Combined quarter, regulatory first ahead expansion very we of in milestones. continued

that into our meaningful our investments, net be indications operations will begin to offering, our We from expect putting XXXX. cash proceeds recognizing could a across and potential milestone in position that Avapritinib potentially us fund to excluding our but geographies, recent of and estimated be sufficient By the time middle payments any existing including revenues. BLU-XXX under collaborations, and multiple approved

Second, quarter, in to increased I've continue of operating In XXXX. approximately increase highlighted expenses. we our quarter quarter-over-quarter calls, as operating to total expenses compared an $X.X on million see past incremental by the the first fourth

a end the we cancer We In clinical we and initiate of continued of expect XXXX, Avapritinib plan acceleration expense this to commercial BLU-XXX expand clinical in activities organization. half of fibrodysplasia progressiva. new as our in second build through year, in first-line second-line GIST, quarter-over-quarter growth ossificans trials for development BLU-XXX lung and the

field-based the In to support continue hiring manufacturing building we'll addition, affairs commercial launch, supply. the and our capabilities Avapritinib of drug including of and commercial teams planned sales medical

continue The who to our offering. proceeds further position, gratitude participated the shareholders medicines want express bringing operator to move patients. Operator? solidified all over long-term our before for the Call our Finally, efforts I company Q&A, the affording we of new public our in to to to build important offering and April the the us on focus for financial flexibility to to on questions.

Operator

of first Instructions] Terence you, is the of Flynn Our from Sachs. question Goldman Thank [Operator sir. line

question Your please.

Terence Flynn

me. taking questions and for Maybe on for progress. Thanks congrats the all the Hi. two

on just the First, data. XXX and ASCO

well? you help but an think Just the reasonable and match generally force talked that about scale. inhibitors the that kind there, by wondering then then it balancing SM. And going side, assume require you footprint us guys you'd about you. sales Avapritinib are that think about type the Would commercial considering. is help incremental bit that to RET fusion? the the with as Maybe for EGFR of durability solid on tumors one differ of durability us little mentioned if the will ALK of And RET is that the size build for a to us focus overlap Or of inhibitors. possible Thank and of it's

Jeff Albers

then start Hi and maybe Terence, in I'll and to Jeff. Andy turn over one it to I'll your this question. chime then the first with is Christy take well ask to second as

how EHA dotcom mastocytosis, only first durability. at from is make various ASCO of data of data we're the any -- for sit I ASCO the speculate would blink broadly ASCO one comment and currently. BLU-XXX just or weeks be say we're and being is I worked not we presentation couple given will are actually going that excited on these around at more would a and on to a though what of away say, systemic where variety very So that then presentations presented reasons. or that today, we for

of time; regulatory seems data various only have First to the continues across ever-expanding program increasing secondly, the be continue global our we very three, is, mature within paths of enthusiasm passage the nicely; investigator group and to productive nice forward. with the very to engagement

to the ASCO systemic in of in really why sharing then think with Christy we mastocytosis few And time. today tumors? how forward looking on weeks' we're that fits so second the solid details you and to don't one, we're size a more Breakthrough And terms Designation of Therapy excited see and how footprint and about at

Christy Rossi

thinking Sure. about We're of size at point about it. talk but bit to the more guide a a in how not to to specific happy going sales I'm this we're of little terms force, footprint the

bringing therapy number of really indication, As different we're patients XX we in in the patients just across XX U.S. I benefit one bringing two next that therapies months not potentially one our patient a a therapy and said, the have populations. but Blueprint to fortunate first to is of position, the to bringing over and

build create our way. informs about overlap plan different are thinking commercial that really think a populations that prescribers focus natural field in between across a see to the oncology us of patient enables who to And these an taking of that so do the And team advantage settings. variety treating we

time will think are of enable and team that uptake those and we sure centers many sure where In treated focused with we that's because envision will excellence these will for initially is as terms will reaching team, practices type to how potential we engaging really focus community at But this which have of we're and and us that to structure also going want having Avapritinib providers that patients do being that. much centers make well. and community be we over happening benefit we're we be know both to very oncology BLU-XXX. important of that be to from We therapies both make patients that they to of oncology initial want the on

in holistic down learned thinking that of proceed more in we advanced really about of that in that previous we're therapies I'm compelling a will sort has In I've allow and flexible as SM be SM, development of rare opportunity again the our as is some terms this excited patient about the mastocytosis way need I've in there. incredibly near the us the and more systemic term my ISM a to reminiscent population about both of in build and in way and just lives. with in it road which worked chronic incorporate

some Our SM little not facilitating will There rare are significant like patient The focus incorporate does But a where a quite allow see going It as probably that structure centers. in the more about on there's aspects bit we to our a to and to bit be around diagnosis. as different. feel SM. be overlap of there be as little disease us focus -- going we identification certainly will is will think be oncology.

on like it. we will are the what specifically this the overall the it's has look And to so building but certainly at that comment flexibility incorporate there, field to organization premature point structure

Terence Flynn

Great. lot. Thanks a

Operator

Thome of from and Company. Cowen the is Joseph of line question next Your

Your question please.

Joseph Thome

tell you first premature? there questions us Or we primary data? say given to Hi follow-up for patients of will an of that submission, And they FDA the been PATHFINDER and taking and follow-up. terms bit that how degree congratulations going from be I too and just many year. started progress on with early some little SM. advanced EHA, of At a of quarter. one the need Maybe PATHFINDER get that direction on on thank in XX-month for kind be the are the a the on my FDA essentially maybe to about in Avapritinib endpoint you look saw this at it's able the period you've that last

primarily it Or is So active some you as this FDA you a do safety database? need larger meeting? well need before

Jeff Albers

why don't you that Andy, take question? So

Andy Boral

Yes.

anticipated at So the presentation I'll with EHA. start

that of on that with that at data is -- we've I on and and last study emphasis study. deepening really durability So have the the on in update the we to continue focus the will reported emphasis been indolent responses. ASH responses additional with And Phase patients that really an year with will from study EXPLORER which on an enrolled SM

think together We pulling the I look EHA. we're -- presentation at data are to and -- forward those

path data as terms with see forward very with a advanced this regulatory then maturing year. our patients response about far advanced as ongoing We're ASH data. we FDA Designation XX% really using at is really very rate with interactions criteria In that in in remarkable rigorous regular We gives of the in very the response nicely study IWG Breakthrough and the of showed disease. SM seeing last -- us that with the Therapy and

based preliminary So Phase an this NDA us has together complete submit data the from I can the study, we given this study. with EXPLORER -- data along confidence that from PATHFINDER on is

coming we is that the out next on we with details map are over and year. filing track but planned ongoing a conversation the months, matter course that our will for Of of FDA

Going back you those at not mature any, PATHFINDER for about would you EHA ask did data not be EHA. with

Joseph Thome

All right. so Great. much. Thank you

Operator

from is question next of Guggenheim The Securities. Michael Schmidt

please. question Your

Charles Zhu

and Thanks within the Zhu for morning on is questions on Michael taking Good a the for few the congrats systemic question This Schmidt. quarter. guys. have I mastocytosis. Charles

-- and being, remind you SM of data provide how to just patients any there color SM that in at advanced sources number? many very were patients first used the what you've U.S. one The arrive could us quickly

Christy Rossi

take I'll Sure. that.

So approximately previously filings our we EUX Japan. there SM believe as major the advanced and are as in and X,XXX in patients U.S. markets includes that well that we've communicated

your imprecise. areas, estimates As the you've while around populations seen in anytime are disease these experiences probably through working patient you're new

So number in that a or at sources number where data facilitates diagnosis and therapies be we the dynamic when we to we've often find patients community. is expect have what of see been certainly not looked there patients bring patients similar arrive conclusion, And therapies that could at actual the to we available good forward. which is more but and are effective treatment we a less that a may brought increased thing new for markets Avapritinib of in if

So make market as to patients. more like what about we'll we bring available it and Avapritinib this learn looks forward

Jeff Albers

markings This and is effective points all imprecision to comes rare you fact data two Jeff. Maybe patients being start the disease I'll more that just add systemic of the see to the around as forward, identified. therapy mastocytosis has a broadly raised that where then Christy of the the

really best. certainly component advanced that even systemic world well just a systemic learn, was symptom that And is so cases in indolent advanced mastocytosis at indolent mastocytosis That mastocytosis included numerous many patient our experts is by third in the patient more very amongst or only burden a there more in ASH we're are systemic study mastocytosis a in patients we presentation, that disease high imprecise that a the fact has an characterized a subtypes. where study. systemic these patient, distinguishing who or of distinct rather Maybe an continuum SM And between is the error smoldering bar. that SM convinced the broadens than

those XX,XXX major that whole Christy already. around So look we at markets huge patients broadly the error that with more highlighted group bar as in

Charles Zhu

patient are you of And for patient and then That it. guys non-mast an of Avapritinib mastocytosis bit types associated example clinical have potential therapy vice versa? For with identification with implications makes or a duration but specific around question. thinking the malignancy patients sense. severe more cell malignancy? Got diagnosis a you a SM of How about systemic cell if non-mast with indolent different these a

Jeff Albers

why Andy that? take you So don't

Andy Boral

Yes.

have So important covers is were off indolent mean Jeff very setting, that to spectrum progression be systemic of a indolent be it across what their broad today actually the EXPLORER would definition ASMs, alluding certainly from aggressive disease those aggressive patients very, associated with can of mast leukemias. mast cell of as associated the complex of and neoplasm they importantly mastocytosis we're non-mast profound associated very another cell -- I systemic is ASMs cell It advanced. these it the we've patients, neoplasm. In as neoplasm? evolve just heme mastocytosis the study, to way and associated the a Actually mastocytosis, you as seeing hematologic carrying I all pure think shown disease. saying, really advanced not that by activity within advanced that can subtypes

that many which mechanisms these through avapritinib well in there are actually KIT we far, really there see associated as a the can doing may evidence -- clonal evidence other in mechanism active be with the so the and is distinction might associated be cases It be disorder other that that's is patients mastocytosis. that that and on by data mutation -- don't increasing based So well. in found

Charles Zhu

for high-risk AML eligible is I hematologic Are patients makes trial. That more you. It MDS malignancies the those you and question Thank one Phase Okay. have Philadelphia last Phase EXPLORER chromosome-positive II to looks if you've I from malignancies. housekeeping specific to associated excluded don't And PATHFINDER? this And sense. like mind.

Andy Boral

Yes.

and therapies. those really have are an So excluded or EXPLORER as will from for because they acute be well need for patients from other – PATHFINDER

or So chemotherapy. cytotoxic need AML their just treat urgently you to MDS with high-risk their

once but that's under for control they the be treated. the studies is potentially aggressive candidates So would until not malignancy

Charles Zhu

Thanks it. questions. the taking for Got

Operator

Thank you.

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

question please. Your

David Nierengarten

Hey. question. couple. Thanks taking I for my a have

profile. is FOP there XXX, quick inhibitors to differentiated bit then maybe like studies are there? bit when with First one strategy strategy PDGFRA if little Are you likely if that discuss your could being are combination any for palovarotene launches a market a Thanks. lessons your from on differentiated leading or second to commercialization on off about And planning little you if a other approved?

Jeff Albers

Okay.

take for have the you take the question I'll around strategy second strategy combination So Andy for question. BLU-XXX one. Christy FOP that that then first commercial I'll for And have

Christy Rossi

Thanks.

a the great So commercialization from options I where a patients. that additional perspective start market multiple thing options if you in there's are for think therapy about

So we this I having if think welcome at multiple category analogs. within look therapeutic you of options number also a available.

You mentioned palovarotene.

You multiple options precision of really helps adoption think look well. ALK the at as I overall. drive Having to strategies

having we'll here good treatment to so thing for and for And that expect the options many therapy is going be patients. us a of

one products forward. specialty in personally where ends thing as space, And that other differentiating ninth I've is bring learned For brought I've market. what it forward having me you over time immediately product when in multiple is therapeutic to there obvious this often worked I commented markets eight, a are many, up many first options and not available

urgency health important incredibly all a these in you in and world be company care the bringing responsiveness a experience real providers what of term. So happens real world engagement over of of can with with sense how very forward engage direct to the way and evidence terms things long patients

So when opportunity. this BLU-XXX. the the that is excited I I'm incredibly At supporting data look at about point,

best-in-class product and think have and the a market. evolve we'll think as forward it how we in experience we gain here bring things potentially I I see

Andy Boral

it's David, Andy.

ALKX FOP. or developing we're in inhibitors the turning So the BLU-XXX that to

which is And driver daily single inhibits selectively progressiva. we BLU-XXX thing critical think is is -- of therapy. agent that ALKX as medication. the once BLU-XXX disease. developing of the and only out think the It's of fibrodysplasia I make will there the It's are for We ossificans that backbone a oral aspect it really therapy it critical the potently fundamental distinguishing

which multiple are certainly to future FOP combinations. patients think And have the we'd there could as for things explore. in for is be think a with great thing drugs those I developed I are the potential opportunities

actual the you Ideally, and is. it lot the approved I BLU-XXX can and will indication think BLU-XXX someone they taking I to a if flares on palovarotene. flare patients a might if with imagine -- though it want prevent where when has situations combine depends what place. palovarotene first is the are But in might

David Nierengarten

you Thank for Okay. that.

Operator

is question the Leerink. SVB Andrew next Berens of from Our line of

please. question Your

Andrew Berens

Thanks. Congrats progress. guys on the

my I the practice ASCO know you're me run question not saying going data. by Let to

profile this more is of a the about So XXX. question of

I that as doses cell orphan think XXX higher activity of thing concerned profile the designation of should is in JAKX/X XXX versus cancer. small think a guess and may agent tolerability the in about regard? a the XXX are that that I of some the an people about that How come last at we little from

Jeff Albers

take I'll Andy. question. It's that

So a potent any RET inhibits far of the targets. other selective potential BLU-XXX highly excess in and is clearly and inhibitor of RET

very So fusions, two as unique I durations with various small now across equipotent cancer activity want rates showing think the the all permutation and drivers we or cancer. mutation the really the that lead similar of response see the of see different previously really fusions also permutations of thyroid see lung to of response. settings We're issue. that to that it's -- very various permutations the And happy including prolonged a of could again you medullary fusions which we remind being being we're BLU-XXX of the with cell is in the lung gatekeeper molecular major forms XXX, in about that very concern we treated XX% to both and cancer, thyroid in the data previously don't in evolving treated cancer

but long and certainly a studies compound. be have is data best-in-class the been going ASCO, update the confident have are that data safety course that determined. what on could to be Of activity we We'll and that we at

Andrew Berens

And trial. cancer with of ones necessary why approvals Okay. approval. have And randomized seen I -- one some lung subgroups just to you would ROSX? frontline I other needing be approval in just versus be does of was to as the both pointed single-arm on guess wondering it We've prevalence to Why and the in RET like seem LOXO tumors think a and driven full it RET, non-small some then RET. more similar cell frontline not a a trial, I with accelerated

Andy Boral

Yes.

an I'll path frontline. with will Yes we And that. Andy. in circumstance cancer, most had Crizotinib I forward forward specific approval to NDA. a a accelerated to think FDA. take cancer at It's has and possible that actively are the certainly, so Lung continue was The rapid in the for is an aggressive in discuss there lung approval be already approval might we conceivably and very So ROSX where opportunity an frontline. path looking supplementary

So is different. every a situation little

that a in we what we Europe in the even frontline cancer is think single-arm a were likelihood in very or lung to available know very other care. parts if the there's than show a I is and valuable U.S. high better -- of countries have want your medicine your would better approval can randomized study also standards to compound of is you world that it's that that

So with various comparative we the for proceed reasons will Phase study. X

Andrew Berens

Okay. patients Have trial naïve there been in already? enrolled the XXX

Andy Boral

have patients. some We do Yeah. naïve

Andrew Berens

Thank Okay. you.

Operator

next from the ahead is please. Canaccord. question Our Arlinda of of Go line Lee

Benedict Shim

question. for Thank Arlinda. you my is This taking for Ben

for but will at before, asked line been you and has PDGFR? and data between this out ASCO, the breaking if upcoming GIST Sorry response fourth be survival dataset

Andy Boral

that. I'll Andy. It's like

will really the on from registration data presentation focus So GIST for the NAVIGATOR study. the ASCO

the is we potentially about about the to explain potential focus So in two PDGFRα response a that the in assuming to safety. will is to -- and be of is on that data our therapy to which And because community And on compound, more expect lines more XX a important want groups really update beyond -- GIST and the filing we'll avapritinib population. duration. across an successful all we think activity a population, fourth opportunity which line label the Exon we it

And much to ASCO. at focus very we the forward look so be of updating the will And that presentation. really

Jeff Albers

Jeff. is This

apart. clarification, will Just broken be as they a

You'll as see to well. them be able separately

Benedict Shim

much. Awesome. Thanks so

follow-up quick you provide how XXX. go-forward Lung a dose? you Just many the think on of durability patients -- will the will And be presented? do patients,

Jeff Albers

this Jeff. is So

a go any opening I but across being plan. into there'll in the As board, clinical comments, a going data set wide across to It's robust we set and the details. away guiding criteria presented of the of aspects to said development each weeks -- our will be a terms EHA range think data of only of or of at the few ASCO those we're in of specifics not presentations

Benedict Shim

much. so Thanks right. All

Operator

is The from of Stanley. David Morgan line next question of Leibowitz the

Your question please.

David Leibowitz

data I forward Could advanced tumors? we question. you see between my path the taking there? smoldering And for much indolent? characterize very when challenge you latter and do guess, Thank for And the mastocytosis what systematic is

Jeff Albers

start maybe is Jeff. then as well. Thanks, chime David. and I'll This in I'll have Andy

this but setting. systemic as through So, we moments various subtypes that mastocytosis, ago a sounded continuum on view disease. her of regular But Andy in organs. they're -- a think who really the again, indolent her it enough and the mean, was few we use high is it to patient life would continue if all are the I and anything, organ specific on a time advanced incredibly again. our walked and week see variety life I a impact from of different patient was mass disease. clearly a I interview indolent there earlier way cell And patient as describes that But time this characterize program is journey woman like a mastocytosis. one burden, function has her infiltration fortunate systemic symptom there of compassionate And panel any and she impacting I unique. children, to

have And symptom burden. indolent high you with an patient very sometimes

systemic his common been has advanced burden. is with is DXXXV for Sometimes you the have what activity of in mastocytosis patient all And inhibit Andy of which as for low when patients. you compelling mutation summary symptom of strikingly consistency an us those highlighted the data

of the on a systemic groups get patients year. better will over we And is of that a continuum. of which studies. we've so, patients. -- this what is earlier appropriate better on in obviously dose this which of we'll data like But that as a sense mastocytosis so for get right end within studying this and time avapritinib patient our are which doses, therapy study initiated indolent first We part multiple guided ongoing play And study by the we gotten out suspect year sharing from to focus we've handle we're particular different also

So, Andy, anything you ...

Andy Boral

I'd allergists than to these they patients. multiple that and as with good a we or investigators dermatologists echoing uniformly are a add bit really are and all Maybe that These XX% really tell that is candidates of with think the world we're -- all of No. bit clinical to Jeff's potentially for trial more that a advanced. And us they avapritinib. sort gastroenterologists, the group around disease our that of and different misnomer see it’s -- we're talking their patients working is we've and pointing a for of Yeah. now indolent

that there's met real need think we is not with available So, therapies. a

David Leibowitz

plans by for additional you in you submission guess Thank On a probably design the with happens quarter? third question. that. they're considering And how in, what BLU-XXX, trial were one have the going when I palovarotene your quarter, get affected beginning fourth a to does in

Andy Boral

Andy. It's that. like I'll Yes.

were They've even our course, think, our XXX for of So, initiated we with plans. while. we program. palovarotene their line aware of been expected. progress, And in a trial really out I doesn't what is we before there It clinical impact

Jeff Albers

combinations of highly Medicines for perspective. nicely therapies And opens the is road. Jeff. is add there, of opportunities commercial because selective foundational I'll a one the you it this this think create But highlighted elements down from Blueprint Christy if up I

our think road. no impact down backbone And potentially as and of that execution so XXX could We the see look near-term therapy we'll said the explore clinical to Andy the trials. on for as we potential of further then serve combinations

David Leibowitz

that. for Thanks

Operator

Jefferies. is Eun from of Our Instructions] question [Operator next Yang

question, please. Your

Eun Yang

Questions you. Thank XXX. on

patients from So QD response dosing, rate optimal the the the escalation. as nicely at you dose XXX enroll increased more

So be as XX%? that reasonable rate in the to have more would QD, milligram patients XXX response be expect you would to that close enrolled approaching the

Jeff Albers

one. Jeff. Hi, is I'll This that Eun. take

going on Obviously, As to it on in that how share not we'll or said questions, answers hand cuts. we're data. we've to based in ASCO speculate got given to what earlier we've now proximity data presentations the the guide or

that forward So you of to seeing all Chicago. in few we'll in together weeks a pull look and really

Eun Yang

response are mutations. with strong at second therapies then And we have targeted develop quite eventually Okay. question as but XX% rates is, tumors seen obviously, secondary range,

seen mutation product? develop it identified this is Have sites perhaps second-generation guide to who pretty have some So but you to been that question in premature with common help could treated patients or ask you secondary XXX.

Jeff Albers

take So, that. I'm Andy to going have

So the question is XXX. around

I could and think far just also resistant twofold this would patients second-line that evolve. the speculate then maybe rate is so the how we've we response seen and predominantly and highlight that

Andy Boral

the you mutations concept lot as of – of So, for developing path at And addresses a therapies as we're We that are – the see over of that how the populations. a you'll how think targeted already very we Blueprint with is to said, avapritinib that about resistant developed time take more really see data happy of to basic can you'll we've therapies. targeted patient evolution ASCO. details compound and create – other

that's area So in. an we're that very interested

what We well information compounds think to to patients, positioned over the they tumor circulating haven't are it's develop but new those at samples mechanisms course on understand and mechanisms to disclosed We any of the we doing the and – very blood our all baseline of sequencing DNA from of about resistance. both address very therapy we resistance, important might are be yet. of

Eun Yang

RET-fusion for cancer? lung What's question. Phase X timing starting the last non-small you. Thank first-line The Okay. in

Jeff Albers

Jeff. is This

the we've in half to guided So of that this trial second starting year.

Eun Yang

call I to And over like the Thank Operator: Albers. to back now would you. Thank you. turn Okay. Jeff

Jeff Albers

Excellent.

Blueprint taking operator ASCO a mentioned, us and seeing short look all you thank to just for forward and everyone in I thanks And time your in So for weeks. as we Thank continued today Bye-bye. you Medicines. you. of few join support to

Operator

conference concludes attending. you today's This Thank for call. all

You may now disconnect.