Blueprint Medicines (BPMC)

Jim Baker Vice President, Corporate Affairs
Jeff Albers Chief Executive Officer
Kate Haviland Chief Operating Officer
Andy Boral Chief Medical Officer
Mike Landsittel Chief Finance Officer
Jason Jakoby Goldman Sachs
Marc Frahm Cowen and Company
Arlinda Lee Canaccord
Charles Zhu Guggenheim Partners
Call transcript
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Good day, ladies and gentlemen, and welcome to the Q4 2018 Blueprint Medicines Corporation 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 maybe recorded. I would now like to introduce your host for today's conference, Mr. Medicines. Blueprint of Baker Jim Sir, you begin. may

Jim Baker

which Thank quarter and operator. call. today. morning, press Good to we operating morning. fourth Medicines This release, a issued Blueprint to we the full-year discuss conference topics plan Welcome results you, outlines XXXX and financial

Executive will we the Today, access can Chief highlights. Dr. our Investors our fourth Kate Finance Haviland, Medical and XXXX Albers, update our at our Chief blueprintmedicines.com. full-year our and that and Section our our release, Boral, reviewing quarter clinical call, today our as medicine Operating the Officer, as of GIST. will You Chief financial be Officer well Mike in by recent will provide on results. our an discuss Blueprint Jeff business on Officer, approach press review recent slides the Chief discuss will Officer, going XXXX will personalized to website strategy Andy progress; Landsittel,

call questions. the open then for will your We

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

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

Albers. Now, Jeff our CEO, here is

Jeff Albers

Avapritinib morning, for XXXX good and to our and programs welcome upcoming our well business everyone. we’ll milestones our strategy, Blueprint quarter highlight financial as call. key lead results And Today, BLU-XXX. Jim, as fourth Thanks,

Our years began therapy to the X programs portfolio rapidly robust build clinic focused precision vision been we’ve multiple a under leading Since in ago, and company. a has than advanced platform the into we less invested always operations strategy. scientific

the Now, behind. Avapritinib patients our treating physicians to is their and medicine of BLU-XXX first available we’re and approaching making point close

we In we fact, confident less increasingly be repeatedly the a spark will this patients to remarkable and founding. that vision new them two first and that for we’re cycle sustainable distinct the fortified the develop to than our company could us from across we medicines XX enable successes initial In bring expect in with achievements And patients. these deliver that foundation directly XXXX, innovation years to discover medicines portfolio.

and XXXX. lead in patients BLU-XXX significant our advanced a for and Avapritinib initiated ARROW indolent expedited thyroid into medullary FDA second to of registration both amended concept our advanced mastocytosis. in our for cohorts for advanced expansion. We opportunity rapidly leading global proof and GIST, protocol in FDA therapeutic In allow and second first For Avapritinib in obtained enrollment reported GIST. NDA topline on feedback submission data to for cancer plan to three planned fourth-line of candidates and China. our to we to the compelling we for Hepatocellular with for non-small bring and line efficient strategy trial lung a pathway our a new systemic half collaboration reported Pharmaceuticals, we completed CStone the announced potential BLU-XXX PDGFRA data cancer cell carcinoma approval development mastocytosis, trials systemic submit line of critical an opened and for expedited and registration pathways our Through We

research Finally, expanding clinic our two wholly-owned pipeline research while programs. new the BLU-XXX into with we fourth our advanced therapeutic candidate

inflection point. critical As approaching we enter XXXX, a fast we’re

Under marketed stage EU. Our products this Blueprint two the our least by we expect to pending and a commercial In at announced to XXXX, into U.S. recently have business. four in marketing end we for a launch strategy transformation two-year applications global company. strategy XXXX commercial vision, the milestone, our the preparation of or

addition, steady In our year. pipeline. envision we business quarter, In our invest have stage specifically, new catalyst and and therapeutic into to expect candidates a development More we research of up eight our continue programs submit for six this we for GIST. plan to PDGFRA Avapritinib to the candidates clinic and first as clinical in near-term we second across NDA advance stream fourth-line to the research

plan advanced data we for GIST addition, both in Avapritinib updated to In and disclose mastocytosis. systemic

share plan PIONEER second cancer. data cancer of year, enrollment thyroid BLU-XXX lung and line medullary and plan non-small to registration In Additionally, data in X trial to cell half line the second initial second the mastocytosis. Phase from of complete indolent avapritinib we we systemic report in our updated cohort for

mastocytosis of systemic advanced initiate registration registration BLU-XXX third-line for in and enrollment non-small cancer. first-line in avapritinib GIST In lung GIST we cell to trials addition, trials complete and second-line Avapritinib in new expect and

mind, and to first day And on details and to expand in poised second precision Chief regulatory this to authorities our R&D this patients. the community plan to bring impact in and Kate? new is GIST half discuss our ask to Officer hold our now pipeline. research Avapritinib medicine business. Operating activity, we on in share we’re Finally, this approach With year with Kate GIST. patients’ our lives Through all to rapidly our first the of I the step path globally work clinical our of

Kate Haviland

populations and in our highly us Avapritinib’s confidence the was scientist at all to to by underlying patient clinical in-house have genomic the of EUX, of GIST ensure our patients one. every Japan. and are and SM urgency like by designed to GIST meet and a the compounds we available feel drivers make We consistency targeting, Blueprint that in globally we strong at have is systemic XX,XXX gives are goal, important areas Avapritinib treatment. range of multiple we medicine. who both mastocytosis significant The focus. there we PDGFR-alpha from Jeff a Thanks of we this good KIT wide made is U.S., sure a selective and patients their opportunities in which the sense of inhibitor, believe To could disease. activity to benefits least Across Avapritinib provide three treating benefit morning

these Our Currently, urgency for fourth-line first and poor. as treatments area submission as our disease to no prognosis GIST new is mutant [indiscernible] effective of there Underscoring initial quickly treatments NDA possible. Exon and focus is the PDGFRA for forward GIST. patient bring XX are

our navigator saw data the to the these emerging to the we data in approach in trial, Phase over were time continued approval increased. had and support had believe compelling results As from favorably we has accelerated approvals pursing potential in accelerates I The confidence and mature the populations.

care. sequentially, comparing current which population supplementary Our third-line the study marketing acknowledges and bring GIST of paradigm, of second obtain which area of so based global submit strategy, right third-line to and current the our of in has in approved study in strategy execution confirmatory is and plan is for move We on in a of to rapid resulted in the standard the the potential GIST, the to indeed GIST Avapritinib quickly, treatment GIST. up approach approval us a voyager focus the development first [Stivarga] product a to the application purse in advancing study that GIST execute that opportunity this gives patients, the treatment This a this larger approach fourth-line of heels the to Avapritinib of we XXXX, could use the approval on full GIST.

focus of treatment advance is area to GIST over precision medicine the approach in third Our the longer-term.

to disease multiple Avapritinib results as is with through are the a GIST Many GIST showed At ability data knowledge by knowledge by is rapidly right the active two our to about clinical design treatment of to to work specific most the providers optimize likely with that of treatment drivers treatment. will optimize approach, are benefits are that GIST mostly of upcoming in mutational Avapritinib and to genomic the involving from experts type Avapritinib molecular pair demonstrating outcomes. solidified patients profile. These challenges This the understand We our a of we’re leveraging meeting and broad our understanding with basis treatment We across CTOS who with this our in of in with study. begin changing that growing the shipped the positioned view the goal new presented we and medicine develop the precision is patients to clinical to view last experience; in the receive in molecularly us impact against and resistance XX% right Our partner KIT we not develop any best These to of data outcomes. group COMPASS-XL paradigm. in future the is GIST way treatment believe to have to reinforced therapies. the transformative therapies fall, team. drive important uniquely targeted healthcare further clinical expert the mutations advisors medicine our do GIST who patient we for treatment combining precision there patient understanding the

participating around in This perceptible physicians, to by their and responsibility testing can value our truly systems if facilitate the investigational meaningful impact it benefits kind the status therapy on physicians deliver mutation developing our even of to of studies. provide This possible a to responsibility it will the and and benefit shaking their patients highly believe Avapritinib, globally and we that to patients means starts a education clearer best treating patients, However, quo. provide up world. the novel [transformative] outcome help the of ways and

leading patients. it effort to is rethink excited right the are the fundamentally approach this We edge for and of on be

Importantly, we with are Avapritinib. stopping not

scientific forward target of efficiently approved to any or are by advance We information more mutations sharing potently at at Our about meetings selectively day medical that later research resistance year. other for or GIST. allowed compounds to our to this not and R&D investigational effectively platform this the up-coming look therapies addressed has us

the a continue we to and medicine with precision which for treatment We we foundation are near-term build excited strong Avapritinib opportunities a the GIST. GIST, approach of pursue by as that upon comprise will

outcomes now call investment in demonstrate Avapritinib earlier multiple programs. continued patient leadership I’d GIST commitment to improving like review all Our efforts on substantially Andy to GIST broad turn across focused patients. with development over along clinical GIST populations with our the our to to scientific and for initiatives our

Andy Boral

were closed begin the trial, as GIST, data quarter. submission Kate. first for top-line and GIST our and results which we NAVIGATOR duration and NDA duration PDGFRA reported in response was will of rate the months. overall GIST, fourth-line fourth-line I PDGFRA was consistent the was Thanks the response previously to response overall In In the want with planned XX% highlighting in in the was early the XX.X January by reached. rate in Safety second medium use response and medium not XX% data.

for Given FDA robustness lack of of period. to treatments GIST our the in the working approval confident the data these we’re of forms approach of with and and review look forward the during approved

first present the of also new ramping the is In up of year. to in with detailed addition, sites around systemic trials half indolent data we plan the of pioneer the world. activation mastocytosis the that to registration share I’m Avapritinib please the

tryptase, As for mastocytosis population. measures cells these with Meeting compelling mast at Phase of compelling bone have of in seven Annual on improvements this we activity several evidence serum All systemic a reminder, cell mast our we in clinical trial burden, marrow reported of provide enrolled data and including in objective the think December. X profound patients ASH which indolent EXPLORER patients

in One risk of population. the optimizing profile indolent goal is the the trials for benefit to evaluate designed Avapritinib three doses of with the therapy of pioneer chronic low

part generate of trial, as second in outcome part data, patient ongoing, end-point expect this initial and plan the in dose, of the validate registration to a pick which and is During the will efficacy safety the which the half which we we survey, recommended present now reported to serve X year.

data this that requires FDA therapies. designation BLU-XXX, that the for for from patients-initiated year, for of continued treatment first significant RET Moving to mutation-positive alternative treatment to now thyroid half therapy positive progress excited plan the report for at This today breakthrough systemic granted acceptable portion data representing therapy is forward the the including BLU-XXX all no treatment present to the recommended breakthrough and updated the first has we’re Phase there are cancer In for which medullary BLU-XXX, our designation program. to ARROW X we trial, the from expansion dose. the first of

which cancer by to in In first patient support will addition, and of the cohorts XXXX. reminding everyone of be second-line a about half cell enrollment like we medullary I’d plan NDA activities. the thyroid of quarter complete to lung clinical used second-line close breadth planned XXXX, in second cancer non-small for of our the submission to

first seven As we’re NDA XXXX, Avapritinib we enter for planning preparing and BLU-XXX. our we’re registration and trials

realized working I’ll to our expect submissions in these for programs now employees We make tirelessly Europe across all setting financial the regulatory and call and disclosures progress see up this to I’m to Mike for of Reflecting who vision and of our in planned to in the patients. are thrilled four turn XXXX. I’m registration on quarter. Medicines, to results my multiple deliver the of over Blueprint review the year data fourth years U.S. proud

Mike Landsittel

Andy. Thanks

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

from highlights today’s and call, just touch the year. quarter For I’ll a few financial on

position we to run will in with to be fund to million us that enable expect First, the financial XXXX. we $XXX second half into continue our a XXXX cash strong and our operation sufficient enter of cash in rate

increased million primarily revenues the Pharmaceuticals. with full-year collaboration CStone to XXXX, due $XX.X for collaboration the from million payment Second, upfront our $XX to

under and also of milestone with payments received a We basis. the recognize to Roche our the on continue quarterly collaboration portion upfront

clinical well in registration increase of XXXX, lung as registration trials the BLU-XXX initiate the compared million XXXX, GIST operating trial and and XXXX, as of of and year-over-year in million to Avapritinib an of $XX In was the quarter reflect increase cell we with we out second-line line of RET-altered for initiation in expect expansion our BLU-XXX new operating quarter, in Phase expansion mastocytosis, compared in fourth the for continued and ongoing full-year of first increases cancers. Avapritinib the quarter-over-quarter $XXX portfolio fourth ARROW as for clinical the there for expenses non-small total Third, X stepped enabling commercial GIST build These systemic our cancer. XXXX. enabling and trials capabilities and the to in expenses

call continue expect [our demonstrates position for which and Operator? execute the program for will our advance to believe with that, the to to our operator also clinical Pharmaceuticals, build and ability now effectively responsible turn strong We development to] manage positions management deliver in plan medicine us patients meaningfully portfolio. research we our clinical potentially our collaboration through and year ongoing our well pipeline approved I continued efficiently our to BLU-XXX and to first to company With the over ahead. questions. Overall, financial CStone


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

open. now is line Your

Jason Jakoby

guys. on is for Jason Hi This Terence.

destination just you drug For do think how out BLU-XXX, both MTC request stands or up setting, just competitor lung breakthrough you. then against you Thank in-line the did And guys MTC? drug the there? for was your for

Jeff Albers

This Jason. is take Hi, questions. I’ll those Jeff,

clinic, and or namely two for March and as in we cell rapidly in protocol year. are guided half thyroid pending being we fully our a around the was the trial, So, either then always the we we it’s remarks, then, move we’ve BLU-XXX work – planned been our enroll on approach we I any and the we accelerated establishing on medullary cancer now first non-small trial on registration we’ve concept, prepared think new is amended which one, from the a in to or second identify about path first for potential regulators proof perspective, of as same the clear programs talked that focus potential lung cohorts, to enrolled BTD, as clinical reminder cancer. as commented line the always of which has last regulatory to second we and comment just with our don’t filing continue line And quarter second in the second

to competition, to RET-altered – clinical way cancers we this second trying BLU-XXX set that a update robust right will need of comparing to is the I’m very a molecule and a continue ASCO the question this at On potentially two forward that have we active to patients believe guiding, broad addresses without look and the year. of of providing say think


our and Frahm Thank question from Cowen with Marc next you. Company. And come will

open. now is line Your

Marc Frahm

taking questions. for thanks my Hi,

had have in managed government filing you were it? schedule to planned the or, have some I GIST, there formal with on always delays meeting pre-NDA First probably the know you or your at shutdown

Jeff Albers

that our Thanks that NDA. take so why you to Marc. – don’t the one, Andy, path

Andy Boral


So, regulatory comment Jeff on as ongoing planned interactions. don’t specifically said, we

We year, don’t anticipate are and for on-track plan. to GIST for of that half both Exon broader this our group delays filing any PDGFRA Avapritinib XX and with in first the in group the the we fourth-line

Marc Frahm

Okay. then, And this and the you about of for this market of And can How team know thinking size third think to in about we label, the assemble kind think do scope build? this research, then, start for salesforce, to that a are initial of to kind in can fourth-line? initial lines you many people maybe need treatment little out you Kate. fall GIST? going building label PDGFR you the once be the into the timing a you’ve When as is talk bit encompassed actually apart start of gotten you of in get you and line

Kate Haviland

Thanks, Marc question. for the

have, So, will and subject together. on come those question that be Chief you build infrastructure the news, year and your be and of looking those able the probably a conversations comment our of that saw plans around last the the Commercial end we're first Christy future Officer as commercial that at forward will higher to to

patients a patients we the will amount to subset the no medical line have In to that opportunities the believe currently treatment know line GIST of about need see really at the terms so in X,XXX our we GIST you’ll prognosis improve options their late of and that that X,XXX they as patients experience, is and the progress patients population is where look that opportunity continue from continue – significant third to we – very to perspective, the fourth-line. to progress a poor has filings and outcomes particularly

important consider avapritinib we an for those outcomes patients. this opportunity for so, And to optimize

Marc Frahm

Okay. Thank you.


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

now Your line is open.

Arlinda Lee

these to When of I of on have then be secondly, were might to And for my that from anything FDA? you questions. any we’ve can heard that curious on kind Could you Thanks couple competitors breakthrough RET what guys. questions. if program, maybe see you mutations any taking show on enroll designation, in maybe of the your Hi you emerging did terms comment resistance a comments for just ask package able dataset? kind trial and of resistance of your or mutations, reflected you have patients’ Thanks. commence

Jeff Albers

Thanks, Arlinda. This that is Jeff. one. I’ll take

So, mean it’s factor is together the also pull in there regard. you or filing duration response an and because those I anything you regulatory emerges, clinical all response terms of that’s worth it I in big in when data commenting are as are areas approval of distinct programs. so our always focus of think is this The packages, of any accelerated rate of don't pathway across true and thinking – unique

resistant of we of subject or future. conference terms mutations, but presentations to seen programs our be date it we for, track that give will is that in any and, we the be always know to closely, with medical any and we’ve In would you watch not something that

Arlinda Lee

Thank you.


question Michael Schmidt you. comes Thank Our Instructions] Securities. with Guggenheim [Operator from next

Your now line open. is

Charles Zhu

progress. couple a guys. question questions. and Michael is Schmidt. Zhu Charles quarterly in for the the I've Hi, Thanks on of This congratulations got taking calling for

TXXXI exclude since First trials on towards mutations, what in the you say in those and and would third potential VXXXA the are breakthroughs that line study the will the fourth all-comers? COMPASS utilization avapritinib, given

Kate Haviland

that take I’ll Kate. is question. this Hi, Sure.

particularly allowed of and the And time. us very and to an you our and paradigm which really we executing treatment accommodated therapy really of latter Jeff sequential in is, ability just in line bit strategy, move treatment, quickly which a of of So, in discussed, have lines to those kind GIST, mentioned NDA our record approach, a current development file studies that’s is the described treatment. enable as as little

their on may what by when which that two we that be is about be it focused of have receive What cycle GIST think XX% opportunity is second-line, Sutent standard active the particularly mentioned, is some at some care, know we I served do of not approach those mutations. and view that patients so do to that data Sutent, we believe who very to treatment in you is will to would with think our an the and mutations will which treatment is point progress have patients with all indicate is well avapritinib. patients patients that those the current those in

Charles Zhu

your and you. NAVIGATOR patients along is frequencies KIT these around and the expect VXXXA what those trials? particularly data additional can cuts as lines, investors on these And progress Okay. and view TXXXI Thank mutation mutations for VOYAGER through

Jeff Albers

is I that take This question. Jeff. will

going answer. first I laid that all therapies, in remarks going where patients, step, creating think treatment out or to those Kate the the both this both evolving a for appropriate the it’s those going. But community be are to this and disease in or be of first of for the understanding really available step to independent as mind frankly it’s as think keep drivers just in is And important just quite development a in development patient. prepared where to avapritinib, planned a articulate we I pair there’s but almost the to right avapritinib, to that providing is they’re GIST greatest well is of benefits are credibly rationale, already going think future as to with it’s the opportunity in really is underlying be and that that rapidly treatment of

should expect share be updates of conference on So, medical our by for put forward. going data, of distinctions as those where to point on will that you we ways finer activity, mutation? part seeing you’re to activity look what So, we a type to continue

Charles Zhu

and between you indolent versus disease? provide mastocytosis, presented what will any some use reported just and in outcomes highlight Okay, potentially systemic then And additional on can SM and great. you’ve color the differences incremental what similarities ASH the one regarding tool at of advanced patient more previously you key

Andy Boral

Charles. I��ll take question is It that Andy,

the of mastocytosis mast we been thrilled ASH DXXXV with mastocytosis the a we saw improvement profound allele correlated burden. in specific with cell end systemic decreases the and measures marrow avapritinib with at and across we’ve data mast presented that our PRO nicely as year with So, burden, last advanced that tryptase, cells significant in bone multiple really statistically

that are mast shows say differences, cells different would The reducing results mastocytosis. the that both that symptoms that around critical that indolent in similar PRO enough indolent At and the PRO. concepts saw indolent pioneer indolent you similar think have in do disease. with disease study. really very reducing expect is many be the improvement for I the of mastocytosis they think we’ve in around is true very systemic symptoms, We there advanced advanced I’d is now concepts, to the PRO. the we used in SM, PRO see created from symptom predict PRO in in that we we rash, that the little GI patients advanced similar, being the that in seven indolent but fatigue, advanced would are that but basic study, are subtle close on And

Charles Zhu

question very again the quarter. much Great. congrats Thank you taking for my on and


would to remarks. for Thank you. Jeff questions like over closing now this time. at any not the call turn to further I’m showing back I Albers any

Jeff Albers

a updating further approval. you we to and our time potential XXXX support for Blueprint as thanks throughout therapeutic us forward And Thank to Thanks programs towards Medicines, you, lot. for continued of operator. this your lead morning we and taking everyone advance look join


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