BPMC - Earnings call transcript Q4 2021 | Blueprint Medicines Corporation

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Operator

Good morning. My name is Lydia and I will be your conference operator today. At this time, I'd like to welcome everyone to the Blueprint Medicines’ Fourth Quarter and Full Year 2021 Financial Results Conference Call. [Operator Instructions] Thank you. Kristin Hodous you may begin your conference.

Kristin Hodous

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

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

Becker Chief will Chief Q&A. be Hewes; and Our and available call Percy Officer, Scientific Carter, also our our Medical for joining are Officer,

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

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

Jeff our Albers. is CEO, here Now

Jeff Albers

our in key today. fourth and through for and therapies particularly commercial, to notable Thanks, precision This lung cytosis development joining we areas. everyone of cancer for four expanding toward the you continued Kristin. the with patients efforts. masto In impact was And systemic the call make progress our quarter, thank research

First, advanced a U.S. with launch of path realize SM, launch with we in mid-year, driven PIONEER the by base a non-advanced continue from opportunity. on broadening prescribers. plan AYVAKIT be data cytosis, Combined masto the continue in expected growth we to trial will future which systemic of to supported potential by blockbuster advance our revenue the

all of XX setting will will the as Second, up EGFR in three our programs, begin the BLU-XXX as clinical AACR at well our over months. clinical in CDKX of from of of wave data string this This clinic trial updates data XX meeting our with SYMPHONY sets to quarter, a next program the be early April.

compliments end and our making we're month, the Chairman over as patients to of committed our the our and recently I note, protein company. partnerships. remain focused platform. them. thoughtful have vision, years. of of thereafter. the decision technology in And the April. in create Medicines team exciting to a countries through advanced beyond. supported opportunities of and role that what achieved exceptional bringing role I by this world, applications in their including degradation marketing activities in for and Chairman and expand for current to or the And consideration we drivers with execute progress CHMP we GAVRETO and I'm these to make place capabilities have kinase year deeply also success received systemic room Altogether, more strategic we've driving than the Third, to in Executive to positive where a we our including opinion therapies the And team seize for a to review authorizations excited as AYVAKIT in right under cytosis. Europe, Non-Executive or now planning AYVAKIT Board, our place masto around XX and precision into beautifully Blueprint personal putting our my as research new for we proud in fourth, after growth, on executive on succession we're on XXXX long-term we finally eight of in last medicine targeted All growth transition then And leadership Kate tremendously Executive I'm to I came plan last Chairman Haviland announced near-term

the jump Now, Kate to updates. into remarks we let me brief over before call Kate? our some to business make turn

Kate Haviland

become everyone. for Medicines’ Jeff, employees I'm confidence. to CEO. morning, next their you, I'm Board, and Jeff. Thank incredibly the our to grateful our And Blueprint And team good honored executive

Operating team to Chief build to as I are company and medicines Jeff of years our to first Officer, worked Business six portfolio grow closely fully last industry-leading with of the love therapies an Over that global had this bring we the on and to had Officer experiences and integrated their have we the and best-in-class Medicines Chief our today. have of Over experience precision been for the and patients the at time, Blueprint that lives me. them. I've tremendously have touched many people opportunities families impact we the rewarding These the patients, lives

better expand step more hope around many in I us world CEO people reach future. the role, As the into – who to are for our I'm driven to hope of look to a

excellence driving continue this execute as culture has building continuously do a portfolio and medical to to strength to our a we patients. and that us which and continue so served well delivering our will from is innovation committed with passionate of on team We strategy, of position

the let beyond. to Christy to impact and dramatically me medicine With commercial We the foundation precision turn place on patients have our performance. on Christy? to review year field over of in increase the that, call our this and

Christy Rossi

morning, everyone. Good Kate. Thanks,

This QX product advanced And overall. revenue with the year was $XX me of build. now approximately and quarter, from about XX growth revenue quarter, the disease our adoption was to million patient real-world is From line by the QX fourth roughly broad base for or AYVAKIT growth. year-over-year In the with approximately volume approximately to new supported prescribers full revenue performance. advanced fourth grew SM driver continue million of AYVAKIT Let begin from Total SM subtypes, net $XX advance XX%. XXX% primary across in accounts for AYVAKIT with is the community pleased that the of prescribers time. first the AYVAKIT prescriber coming half for we and account all utilized see population. to experience We're that representing

a drug has point pathway disease our as to AYVAKIT first mix larger In expectation indication. see Through trend growth over inflection for become our PV continues see our for XX%, approved which continue line utilization free for an expectations Program strong With and our to in XXXX. AYVAKIT therapy Similar to to Patient Assistance akin launches, approximately this rare solid This access other its non-advanced Jakafi, product. addition, brings we first-in-class a patient to steady to with growth across the fill the of was XXXX. of blockbuster time such was its Patient SM a in indication trajectory. approval growth prescriptions. payor of in for to we that course for guidance lifecycle, through with the consistent foundation, Jakafi's our accelerated Jakafi myelofibrosis me rapid

the XXXX our in to more AYVAKIT revenue to this we this As over double press Key expect revenue net in AYVAKIT than noted million to morning, year. product $XXX achieve we $XXX million release of in increasing drivers duration, treatment year. of half revenue, will starts be we in acceleration approach as which performance will drive we particularly second our growth patient and the anticipate new

to addressable each base as to the XXX patient see continue expect U.S. the prescriber as as already our XXX who growth new broaden to year. advanced newly into starts penetration estimated we well in We increase our patients, and patients are SM diagnosed diagnosed

they with the the disease As launches, they right we real providers at field other in providers most time a their are to innovative when targeting adapt patient care. educate to setting. approach are This leveraging both in and ways and to academic data community potential our our an may to proving receptive, evolve the in is enabling reaching time, rare which be in effective is have

COVID We impacted hematology efforts and patient anticipate pandemic in patient U.S. our oncology to the that with aid the has landscape will the ways. improving also and many We care know AYVAKIT. increase in in identification treatment that

For trends. in line roughly observed ebbs half visited SM that seen about patients only number flows example, advanced claims in COVID noted XXXX. SM in the a hematologist we've also data advanced of and We’ve of with diagnoses in

further science We in are of the combination the from Omicron are starts seeing expect In environ patient year. well new we drive our efforts, and past growth an peak. to field totality, own that throughout targeting as some education in improving external improving as our the geographies access of

two improve well. over will then at real-world anticipate the clinical addition, studies, revenue this years. and in treated longer a of to advanced In treatment durations durations often a In low as with average setting, duration the expect driver be that trend disease of approximately SM launch our their AYVAKIT are we therapy growth. and with In of course case treatment patients AYVAKIT as we time be earlier key

States growth in patients end the the encouraged XX% We're only in past or our in SM the quarters We SM diagnosis opportunity gained two first to infrastructure insights patient that drive and several over with and we newly later By the SM that an are believe diagnosed the improved healthcare of in first the reinforced starts. Over will a diagnosed with expect claims we of over the by treated comprise potential provider XXXX, of of now we have Overall, newly for to their estimated half in unique patients visible is of course have patients. two AYVAKIT. could diagnose United blockbuster XX,XXX in diagnosed launch, majority growth of more seeing disease view testing my years the new patients quarters education been XXXX. launch, data. our us continue patient the

well I'm disease will and non-advanced focus in approach pioneer we mid-year opportunity the about increasing us. we in of execution of diagnosis readout continue As as excited disease. SM, And on as and front to the advanced launch awareness

Let me to now turn GAVRETO.

and execution the grow more SM Overall, for to launch share. Fouad education R&D revenue ahead than and for ongoing that XXXX, Genentech's to On over turn We note, to to QX as U.S. revenue RET and and In our the overall flat transition the global in in in driven expect by led to on QX. market model launches drive XXXX, leveraging quarter-of-quarter RET terms the will were in excited opportunity we our a market look GAVRETO double Pharmaceutical. provider and patient call dynamics through the Blueprint our Roche the of to infrastructure and I'm new of road in U.S. overall starts by the review Genentech anticipated about identification launch progress. advanced expansion to continue of the AYVAKIT prepare by impacted SM. outside patient to focus growth multiple I'll U.S. we see CStone non-advanced distribution

Fouad Namouni

and good Christy provide Today, Thanks, I'll three focused areas. on updates morning, everyone.

Our ongoing continued data plans to platform. Meeting, and AACR research efforts clinical to progress, our new present at the expand

important me updates. begin clinical Let a few with

enrollment top-line First, data pending earlier And patients the an to PIONEER AYVAKIT announced of more trial this We quarter to we we the track are report end completed of in XXXX. sNDA in on FDA than our as non-advanced mastocytosis. plan to submit by systemic of mid-year results. XXX

lung For first patient in program the our EGFR of January. BLU-XXX study our we dose in HARMONY to were pleased cancer

for we Exon addition, the from clearance Lengo the XX Therapeutics. FDA from inhibitor BLU-XXX, In acquired our study to EGFR received

tumors. study CDKX aberrant well Last patients report Pfizer and BLU-XXX, for the best-in reinforcing for encourag CDKX begin class-potential inhibitor patients, ovarian We to CDKX into to inhibition clinic. breast we excitement tumor evidence cancer in as has our with of were FDA providing reductions as clearance cancers, and a bring inhibitor received two which week, showing BLU-XXX, the Cyclin other our to in their breast E see also data

let potent abstracts new multiple that against our we selective data include with plans wild-type for and combined am the in well lung supports that agents cancer. patients presentations. EGFR, ability a for accepted Now, data pleased in as I Triple BLU-XXX the me Annual feature to BLU-XXX, is AACR These turn Mutant for as to at preclinical BLU-XXX first tumors. April. over EGFR data Meeting highly other share present its to clinical and BLU-XXX with uniquely is lung have

other will us mid-XXXX address or EGFR Our prevent from strategy cover XXX the second and the setting. majority resistance the This in agents of leverages about the and and XXX, cancer. acquired to BLU-XXX treatment in EGFR the synergy through it first-line to between allow in complement mutations lung study following third-line initiated we emerging dose are We osimertinib escalation. SYMPHONY today in halfway EGFR of and

is pre-clinical performing see data. we are BLU-XXX that encouraged consistent So far with very our to

gaining the inform combinations. monitoring which our tremendous development will tumor are we insight into circulating profiles through In disease addition, mutational of tumor heterogeneity DNA, and of

cohorts data will presentation dose AACR translational well clinical SYMPHONY data and activity. from will of escalation safety, early the Our as highlight and the of first signals pharmacokinetic Phase at study, include X initial as

shift our highlight let's Now research to gears efforts.

the the scientific drug our approved was that Blueprint One drove This XX our which quality of XX and are a accomplishment for In most and have candidates, platform incredible important my world. team. now of of we Medicines an factors productivity two is to forward around size join of age. decision just brought the and company years

to when of steps our strength further our expanding the on taken discovery transformative we've to build Over the the patients. goal and to last with diversify deliver ability therapies year, strategy,

protein targeted degradation harnesses a oncology this part degraders the body enable adding effort, machinery and that to approach to precision degrade protein Targeted unwanted cellular in of design of therapeutic we proteins. are is As hematology. capabilities the

this new additional for targeted approach inhibition capitalize degradation target part challenging our on and The succeed kinases, capabilities. ability compliment our to provide optimize approach kinases, traditional target open to will kinase library and of of classes. strategy as to annotated with implementation will our protein pharmacological incorporation discovery fully chemical Our of research

We will updates. vision our selective roughly growth, we sharing later on I'll able XXXX, the day innovation. internal will the of sourcing of forward capabilities, in are historic Based this the and targeted our over the output our to believe overall we by plan and we more of to area which now expansion our look Mike to research building at including our supported year. external financial engine protein turn exciting R&D degradation an discovery double of be in second half be planned on With represents partnerships This that, call and review to XXXX. by

Mike Landsittel

detailed touch and results year the Fouad. Thanks call and in today's we few reported for release morning, Earlier financial a quarter. press this the our from on I'll highlights

total including $XXX.X AYVAKIT XXXX million in $XXX guidance year of revenues full collaboration in the which to in million $XXX.X product net For XXXX revised is of line million. a $XX.X GAVRETO, revenue sales $XXX with million, million revenues from the in were and revenues,

collaboration $XX AYVAKIT revenues the million Off were revenues product approximately product quarter. XXXX. million recorded In $XX noted, and addition, in as AYVAKIT Christy in million to of in these fourth grew net XXX% year-over-year revenues revenues $XX

major which saw driver SM, For revenue. solid AYVAKIT, advanced we growth in of product is now our

the our addition, CStone we by including collaborations Europe, AYVAKIT through of In important as GAVRETO GAVRETO growing approval China. achieved in the Pharmaceuticals well of progress as sales royalty revenues in and on

quarter. million were In Zai develop and BLU-XXX payment to also recognized strategic a revenue addition, we China, the $XX in Lab which to BLU-XXX we Greater and commercialize as sign a upfront in included that pleased fourth collaboration with

was This integrated year in research of completed Our company's one-time Lengo respectively, acquisition and and total charge we operating million rapidly the a and expenses full $XXX.X fourth BLU-XXX since acquisition that of assets have our associated into the were for time portfolio. Therapeutics. our quarter other and with driven $XXX million by $XXX.X million primarily December

As $X are in which we data in exceptionally strong our we XXXX, us business our portfolio for over position with billion guidance. to our XX revenue as over we as to towards potential in invest the shareholders. cash, an drive will This we are patients a enable range year, to catalysts detailed mature, of enter XX continues months create value more transformative as as providing well next with to just

the positions of over anticipate our source, our million questions. We over will performance which product turn advancing highlights AYVAKIT and This and call which we revenue in uniquely will prolific now us continued XXXX research $XXX drive $XXX For product $XXX continue for for as our that to operator to well With both to million net anticipate years portfolio that strength, with a AYVAKIT Operator? our thrive revenues, course to achieve back significant of includes the the financial as $XXX ahead. revenue diversity I'll combined the to that, engine we expectations guidance growth. XXXX sales. million million approximately total

Operator

line first question Instructions] you. of comes from Your Marc Thank Frahm [Operator Cowen. the of

line is open Your

Marc Frahm

my Thanks for taking questions.

cyst truly might robust When early at kind are triple-mutant the the You to a presentation preliminary AACR, of terms going be to kind to much see some extent we completely you can in with be and understands efficacy signal, populations start enough patient escalation. likely of data to maybe for single how work-in-progress But XXX be context to updated that's very in very so of are agent really a there? the could determine AACR. going give efficacy dose of many that patients it's to

Jeff Albers

Thank you, Marc.

So I mind. what at think XXX the it want Number we for way features we confirmation wild-type X in specific was look that agent EGFR designed is, from the some do see our with of XXX, so to selectivity.

that So in X, tumors that Number think activity are for like see would Number designed for. I chemical combinability that and mutational all of to in profile index we we with profile was designed these was allows to see the safety other a coverage mutation X, like harboring XXX therapeutic agent. that large a would mutations.

escalation earlier, midway consistent the the in with as the profile I is So preclinical is, mentioned we're the today see happy that clinical study profile dose is pre-clinical the through to profile. and

patient at agents importantly disease of and looking We combinations of are that our within the really for ctDNA very strategy is of the even heterogeneity learning disease. sometimes tumors single a EGFR profile in in the mutational tumor these informing lot from terms the a of

data present some ctDNA data, BJ of early and we'll from don't number and dose namely, the [ph] clinical dose yet the translational and data, of from signs cohorts data, data so more show tumor activity. patients and cohorts. we'll specific but have this escalation So I data present third point, safety escalation we'll at first first

not Phase mutated of have all patients based but we'll profile, that all escalation patients show EGFR mutational and we data by able are study purpose time. but selected that providing of X, included the in patients, we and We the have their to have, be this we subgroups tumors on dose within for we the will

Marc Frahm

Okay. we the of about building assuming use maybe And AYVAKIT then the are ASM half update? that in thus second some of driven as building and Christy Is up, by helpful. accelerating the market your a year. sales also kind in, kind maybe comment coming of get will the you into Thanks, and off-label is advance positive of entirely level acceleration trial ASM, pioneer assuming be or there very label

Christy Rossi

Sure.

patients So to provide use terms the think I SM, do I terms penetrate off-label durations headroom increasing we assuming the factor. begin part in surround then starts and that another am cetera. factors of It's we see not we will a we grow pioneer of But as be advanced driver. lot of patients to diagnosed continue to positivity are into I are, sound pioneer nice the study mentioned primary just a – in true earlier in of expects factoring positive. et more, sort as that year additional newly treatment AYVAKIT to to be will new

opportunities year. SM and some, next of study think then anticipating course definitely primary driver in into being launch that we're the year looking at advance we positive. I But the being there's So really this non-advance larger get and sales AYVAKIT of from a benefit

Marc Frahm

you. Thank Okay.

Operator

Thank you.

comes Dane Our next from ahead. go of Leone Please Raymond today question James.

open. Your line is

Dane Leone

the congratulations questions of data conversations been into heading all the the line what had Kind of from first lot the with disclosed has the the taking and investors is guess progress. trying X.X that. context for we've you teams I of a questioning, understand a ask, thank similar I'll to given Hi,

the Phase dose not to You're established by a recommended X have being expecting presentation.

or actual a maybe mentioned broader of index And do biology, co-mutation being interpretation. lower potentially about a up with then obviously we early you clinical do, patient you're I things you to the is think wide a with the from at in when it to mean several you. that more patient how AAGR there's And the frame disease therapeutic in not would drug open goal a activity, Now activity responses? of you which think rate TXXXM whether the activity? trying of drug to count control benefit range CXXXS, about presentation, if to any resist there's a does of Fouad, you able Thank clinical which population as mutation or bound framework clinical think that establish the

Fouad Namouni

escalation Dane. data said The dose is be I will we presenting in the first escalation. are that Thanks cohort, we from as through very midway the

We time specific the at able be your that expect time, to running. osimertinib that and and mid-year that activity monotherapy generate an point be to up then the expansion rates sometime to by combination around RPXD reach typical for and time response at and would with would also,

like of in that the the and shrinkage seeing, what and response the don't that are one, talk molecule. and the of safety molecule we is sure too it' because some amount make rates tumor in we to to activity? about XXX Number what and others. be and the terms think really agents designed design seeing cohort And into that consistent we I from of the CS. seeing and with mutation In and with designed patients? data, osimertinib stable terms that drivers of what therapeutic was is it's get will activity, the in rates, about we data presenting, so mentioned; we'll TM early. disease the specific covers is really instances rates signal two was of for, that be When of first with I As other which to is of able response some terms the and be means This it combined need we index molecule are mostly

are we specifically from in the lastly, extension we'll would are. the one and the later it we And escalation, when and dose define I thing to an rates at have we Phase – to when learning important enough the the RPXD I starting be the this mind very that quickly And think but dose say we very not opportunity do that also an really second, have in add with combination in the and I because what have monotherapy X yes, mind phase, osimertinib feel a as comfortable probably example? will first at at combination third-line only escalation expansion we'll would looking also important and be line in too. we that's with response looking

Dane Leone

you required Could the for mutation, see you. just to Thank that you the question regardless clarify expect of in last line of drug given preclinical you the is both one, drug you the that CXXXS active with be this Do hook expect being compound? when to modeling, those activity TXXXM say whether co-mutations in activity? that part existing the it important patient really is population the or of

Fouad Namouni

from really you this development is the learning helping What and heterogenic is is and the X, are multiple our Thank mutational question. tumor Phase there for we combination profile strategy.

your On other whether triple we hand point will CS mute to monotherapy, double mutant. look have for we the in in activity

activity we escalation, this it's like covered dose to mutation of think terms see by XXX. are in range the point, by too early at would across that design I of

Becker Hewes

Yes. Hi. This is Becker.

be the of of them and bit expect disease to establishment winded there a wild-type, protein where with to at heterogeneity, but responses for for activity is then until a broader point, want we to the of as would than the beyond looking that really detail for I there front these and Just that's potential the means respect just this situations rather they've hold. is And the end that of might more stable biological once And you lack combination of and are strategy mutations monotherapy the established that of very and to tumor that. a a once. That it development optimizes see with heterogeneity but mutation large and in the wouldn't TXXXM mutations try sometimes is optimal a the they're the as on all the molecule in waiting for frontline be has cover molecule. hook a it, classify prevent get gotten little activity that where we TXXXM.

Dane Leone

much. very you Thank Excellent.

Operator

Thank you.

Our question comes Stifel. Canino next Brad from today of

open. Your is line

Brad Canino

you tumor XXX, and a a Hey, great. for have, to through Thank question. on conversation that even you'll the, into safety? And based treatment I something providing early combinations all the clinical Will you these movement the would PD changes as you. measure well on Thank triple to assess to with guess the on, and said support be the in you that better within ctDNA data on mutant that it activity heterogeneity BLU-XXX possible extend over like as be scan patients. the the in

Jeff Albers

good see activity in clinical surrogate strong you, and have We on been happen data capturing that patient with for marker fact would in the of with our ctDNA is be of that for Brad. it what I the responses mutation. majority the pharmacodynamic variation When and patient. the surrogate time question, from showing a you, for activity level, your could to point shrinkers at like monotherapy we'll the the tumor tumor evolution side to the a for the obviously Thank very specific to treated agree marker and Becker's But over patients compound. point XXX, reporting of for impact burden and EGFR we pharmacodynamic is ctDNA, ctDNA are

Brad Canino

you. Thank Great.

Operator

Thank you.

Salveen Goldman Our comes question of from next Richter today Sachs.

Your line open. is

Unidentified Analyst

call. Thanks the This is Matt on good Hey, [ph] taking morning. for for Salveen.

about quick questions. change the you potential out Just heterogeneity? you the a in already And patient what context a would ISM? Do you're two data, thinking be trends then considered this advanced in upcoming of in respect you're little how Thank secondly, about the but again. talked launch role with way, SYMPHONY bit, to positive and SM the for seeing uptake there you've

Kate Haviland

So I'll with start question. first the

a – go this through as lot think do we go I learning So, we're launch. through

in don't think are how around to being of so advance are primarily insights move view able much rare place the reinforces we've building, market onto patients kind that blocks effective a hone We've identify where are critical just awareness, disease and rare the seen, right known think facilitating clearly advance that's a it I those diagnosis, true I the non-advanced. disease opportunity. in lot broader to that in SM treatment. is my, a my trajectory the as we can This the patients is and anything is, of at of targeting making SM is key right treatment. building at of help market. disease sort offered if Like be sure as

we've the this patients with diagnosed in been And encouraging as having SM to non-advanced see in U.S. encouraging data of that's And healthcare that that really we in a them. help critical trend. it's the It's something into hard for we drive to over think overall. treatment that continue encouraging in providers, indication. support. see I growth think the available are I interacted in to in effective And something to the expect we're population market this be, overall develop to about and hear will think opportunity also the an the and interested just working very AYVAKIT sort what time to terms gotten really certainly of I

Fouad Namouni

tumor DNA, tumor is and for And DNA what the secluding heterogeneity and is and what not. positive

but the patients. and in of what given at to also focusing only are DNA, number tumor or we have Crohn's just step think see in primary when on a in what in circulating only or difficult do to the are type of level, not access the bit biopsy that we that tumor little one metastasis. That mutation, we now are composing is tumor the to a about of back tumor I think able type

in what's and good at and And on us picture in over the or terms how treat that mutation And this tumor that creating the are lines is when we the patient. lines earlier is what us tumor given over to when really what burden we and are mutational what happening patient a profile. means tumor DNA change – really like pharmacodynamic molecule has just what of targeting with on the its multiple surrogate a ctDNA are multiple mark-up we a really clone very of in what of given even type we for the changing of type telling level, So focusing evolved time. give see would it

revolutionize deal time even the very early the also are doing, to you field know the but in knowing way expect an which really overall variety or and to for going to activity, do clinically in are and development doses what terms can is Phase before can with it over tumor. that is of So reaching what at of at terms what combinations drug to impacted. in you of tackle terms extremely I use you the this process, And you are really this predict it believe of in knowing will what which Xs of helpful, medicine

Unidentified Analyst

Great. much. Thank you very

Operator

Securities. next Reni from question of ahead. comes go Our Benjamin JMP Please

open. line Your is

Reni Benjamin

the be kind since of endpoints has for approvable still secondary fatigue world And Thanks. as the not is And obviously taking potential or way impact of and any to into today? the like know I guess be secondary account might account the need endpoint. an more or there shots changing might feeling I the and in COVID un-vaccinated? be might How taking do going vaccinated to for primary a quarter. of Thanks we're you guys. fill things around results be this total at and congregations not happening morning you are PIONEER the Can and endpoints where middle able and patients big may questions component, ultimately score? question the time to who to us mainly Just year. how assessment's remind great symptom I that good that it's on the whether by you how guess patients is booster kind whether subjective use I'm things this and scores? just is My with is the of Hey, the symptom segregate a expecting kind

Jeff Albers

don't Sure, that? why you excellent. Becker, take

Becker Hewes

remind trial. Sure. about just is randomized I'll this a question It's blinded you, thinking really a and COVID good

can remind capture the is and there goes that able as effects, it on Total we even at patient we if well the outcome, benefit to do the see arms. data. Score. score, measures but The We some I'll of collect beyond also both have on so amalgamated are COVID objective will that patients we reported looking you, and specific we'll the primary be endpoint, data vaccine status, so of are domains, So, whether deconvolute Symptom had disease

have been to is season, flu need So, at things through would we that. that are a of There would so the other capture like – continue gone through look we we'll kind this to anyway. thing we've

got all trial. we've that So, built into the

Reni Benjamin

Great. Thank you.

Operator

you. Thank

Morgan comes Stanley. Our Ulz from of Mike next question today

is open. line Your

Mike Ulz

the taking for in thanks one guys, just Europe. Maybe question. AYVAKIT on Hey

Now advanced that about in the strategy opinion the the in market launch talk how maybe for U.S.? CHMP positive you've you got different the can SM, Europe and might Thanks. be than

Kate Haviland

Sure.

to in beginning looking year half available to are go growth forward. also get forward the pleased revenue we the of in and to Europe as we a and to contribute kit making this positive that launch yes, very So topline we're opinion

than Our strategy the Clearly, that a we're opportunity. have that's dynamic, coming countries pricing focused U.S. is to in taking approach for you strategy similar of from in different cadence Europe that little advanced is sized the the right Europe bit an reimbursement which to U.S., organization the in the system build a and really online a is

system Germany so, early the see and along the some et bit that markets about is interesting ways pricing is we in markets the think, some be perhaps those quite And I continue And One secure more expect other we will cetera. versus of which terms key U.S. particular the as Europe an of SM market access come and we'll of some ET&M, timelines, then called something in is the what of that's thing, registries, driver mature, on. that patient the to Germany, like in online. faster kind is in innovation markets driving organization a treatment, been particularly active advanced there's is

with the And cetera. so, And in been et of that we're investigators key there Many members engaged highly a practice community is have of place ours, community. in Europe. of

for of the forward that AYVAKIT. So, enthusiasm there's lot year. upcoming We're availability this I looking of think to a

Mike Ulz

you. Thank Great.

Operator

Thank you.

today Our Eun of comes Jefferies. next from Yang question

open. is line Your

Eun Yang

Thank you. I have a question on EGFR inhibitors.

compared think combining of kind with be on osimertinib. So, you BLU-XXX, you of BLU-XXX advantage data, you and what you. the think you when I the best got be have know the the data you either with and with seen, preclinical around do would but on combinability comment of what can the strategy? the Thank and of BLU-XXX, trial could a And to based see of potential kind osimertinib what also,

Jeff Albers

Thank you. Very good question.

are learning we lines, in either patients believe with heterogeneity, order or as I the progression even of of that tumor osimertinib, or are majority As reach earlier to about we EGFR is showing our way can combination the together after for in mentioned, mutant line EGFR EGFR important. very and really we selected have so component design really of as types key for over a have was have we combinations. it type And five it very, to multiple intention was wild the agents deliberate

combination BLU-XXX least the who osimertinib look their data, the at it we at after in progressing with strongly So, addition them preclinical preclinical by patients activity in help is if supported of these and with and responses see osimertinib patients And will by will or supported data. induce disease. and are hopefully

only BLU-XXX including TM. in it really discrete, both start osimertinib combination good BLU-XXX coverage cover resistance We these a we by expectation combine the the will a that strong level to the Phase be multiple agents, and Obviously, not to majority when combination subclones can reach will patients most some way with of cover X, from so for does is subclones be occurring. line way and to brought will where will covering tumor. a the the mutated mutational we with very BLU-XXX. able cover constitute be and really My from this even the BLU-XXX, to to addition that will prevent the BLU-XXX can first TM are and very you osimertinib that BLU-XXX, to important resistant be majority combination combination. that some studies strong we all will at just have when similar combined the a bring in two started in see of in BLU-XXX

Eun Yang

you. Thank

Operator

Thank you.

of Joel question comes next today Beatty Our Baird. from

open. line Your is

Joel Beatty

the Hi, progress. the And questions. taking thanks congrats on for

is question important question endpoints you might them? physicians primary to expenses there then the of discussions expected just SM, the expect what's describe And your from secondary these could in them, over is First trajectory year? see PIONEER the important results you are the from just, in results the in what for study on non-advanced endpoint, to second with or course

Becker Hewes

is This take Becker, I'll that.

score. about TSS think the Let's bit little a

systemic Symptom designed Total a have manifests. in disease with experts to and that we the mastocytosis capture ways different So, this regulators is collaboration with Score many in that

very of are So, a rash patients some others to predominance a and a that symptoms lot have of different there diarrhea, of score. and that then high have who up some add

sub domains just by total when score aren't understand captured And so, that ways we better the are at benefiting to look will at patients total the and score. also in that

reduced. benefits. have best patient with best So that'll XX% lower look or looking way that mean also from And large a a one because, time with going from their who the of with reduction we're keeps life, at that we'll a patients then family to change reductions quality for responds their at work symptom is them example, stops in them or meaningfully spending

are deeply also measures there looking the components are be score those the into. of So more objective that Then disease. we'll of TSS

burden with their in the at and population mutant tremendous story that benefit the all treatment AYVAKIT patient reduction reductions look with in impact allele life. really of cells, quality in we of of varied the this tell aspects describe in So, a reduction of tryptase, mast on these various will

going XX call in new of be or what the not really and scores life, in be to ability at their looking of response. XX their to and be we'll and to normal, return not years measures concomitant in which the might for thinking some they patients a just we're with the normalcy. work, will use living cases they've a what don't things is return other of These at objective looking medications out, going The normal been we'll to normalcy have

Jeff Albers

second the you take do Mike want And to half?

Mike Landsittel

Yes.

$XXX expense respect with saw so first, of in the kind QX, of million expecting acquisition. as the to related to see, you a operating So, charge trends, one-time we to we're large had Lengo

way it the about think, quarter-over-quarter right. by best growth is So, adjusting that, R&D, driven continued to think for OpEx I modest

in think in be BLU-XXX into the putting about clinic. clinic, we As the we’ll the BLU-XXX BLU-XXX clinic,

the past moderate up over we've seen you to could so. expect what quarters in at or ramp year see we year. course similar throughout to the As those the expenses expect be of a historic But development pace, to increase it

Joel Beatty

Thank you.

Jeff Albers

but of position. we've enthusiasm in biotech. an financial add anchored becomes Jeff, interesting we're think, moment. the what programs that noticeable the opportunities seeing, an Blueprint more that. that's, And a clinic in I the around is now into in this collective then SM tough pre-clinically, about It's of in in market position, pipeline financial revenue to is being this expanding blend strong strong noninterim maybe talk all a right our got And position and the We now, I'll the that of ASM expanding seeing we're and drives what set growth for moving underlying driving and all

total sometimes never today. minutia which I strength, stronger. in questions it's market, clone has that never lost is our stronger think, vis-à-vis specific of been about Blueprint a Medicines the it has gets the perspective, the that And differential of been So, than

Operator

you. Thank

Michael Schmidt Guggenheim. Our comes of from next question today

Your line is open.

Michael Schmidt

look systemic I mastocytosis. of could you. guys, thanks product And thinking launch opportunity, of into ahead a an had for launch example I well, just as possible that question. type as market? the sort taking my commercial of Thank non-advanced in in are a guess of serve what Hey proxies this one as so potential that perhaps we

Kate Haviland

Sure.

if are I one. some think, think, because some do interesting, of the look of is evolution, there curve. at, right, some looking we market Jakafi I leading broader no I is analogs, for of look brands, a that sort building into When myelofibrosis HAE Alnylam’s is at too. PV I that, indication more think and mentioned in terms the the there perfect I of lot and particularly we as in of between looking at shape market transcript, SM. has Certainly, analog trajectory a sort is one interplay than think, I interesting, of example, similarities at for defined it similarities So, of a a is which of

disease help patterns, referral historically then but those challenge, a kind et patient analogs, having So, diagnosis diagnosis of and when I that's defined population, perhaps rare building been effective catalyze a cetera. identification, think really treatment therapy of can you patient having in that and a growth it's

catalyze to we're in think patients said, as is drive AYVAKIT for forward. So, of go excited going about therapy is potentially we of really impactful we're growth to the continue driver some of so terms being transformational the then look diagnosed that addresses continued which disease, to is patients. we'll education as we truly, really And to the trends seeing I as be in first that important this about something

Michael Schmidt

before, All patients treated but been right. then addressable, time? the had diagnosed some in just today disclosed And, might Mike, non-advanced market being having how i.e., estimates many you us over remind to I are immediately and believe think, be as building you opposed

Mike Landsittel

Sure, yes.

has evolved has, at I last our the with how market over So, the actually the market. has this think, evolve own engagement couple deepened understanding the of years, as look I and of even SM continued deepen community our as to and

Historically, and sort another patients still way U.S. a third patients about diagnosed being their making of of major thinking of and missed XX,XXX we sort talked in about XX,XXX a see in towards diagnosis. kind had third third about a completely, diagnosed, some we the markets. of being So,

about XX,XXX, nearly, of see half approaching we Now, patients so diagnosed. being

to more really our trial with moderate that patient are in patients practice. will something kind severe. threshold. Our first think, I clinical In a candidates be that be population of, AYVAKIT not used will we're that for defining that's thought to TSS be That's

therapy. on controlled actually on seek will and therapy. So, not patients I with lower, their often that scores than well and are well are struggle symptoms are we TSS struggling in do care who that controlled think, in find reality, of themselves cutoff, with burden the patients know fact, not And

think therapy expect in some and data on is from benefit profile, risk initial really positive AYVAKIT, I which of patients itself want with treated. like see So, to we the a based drive what PIONEER be who to will a

Michael Schmidt

Okay, helpful. Thanks much. so

Operator

Peter Thank from comes And question our Barclays. final you. today Lawson of

open. is line Your

Peter Lawson

my taking questions. Thanks, Great.

tracking into around in go of cadence, of or leads, Just anything XQ? about week-to-week and of on you we XQ? or around or COVID kind takes revenues, inventory talk And kind AYVAKIT or scripts as – us around how the month-over-month those into just are in can interested from how put about should thinking XQ kind impacts? Anything can we you tell be

Kate Haviland

Yes. the Thanks question. for

we in acceleration in to course of increasing what added starts fourth the driven prescribers. continued two factors of expect talked quarter. patients in We We've terms the see to the really I duration. over So, and them that and year, treatment add about is patient by revenue new

We across first biopharma, quarter insurance which case same first be sort a right, the in compliance be in is to cetera, taking will I changes. see copays, impacted will et for net to we quarter dynamics typically do impacts that first lot for you be hit expect tend a to see quarter. to gross of thing, by that the of do think

that expect is of part all play through to dynamics the so, ramp first in And a those quarter, in our expect which year. see we of I grow be kind would to why

beyond. think optimistic those do see starting are that benefit also the COVID our We of ways quarter so, that improving and particularly landscape to will this most coming and We out get best that we're QX come – many in us of We're as and the territories we already are our that. can out. signs into performing see open

Omicron trend to As peak us we this will move past that continue. start see

Peter Lawson

And we're growth XQ? Got suspect going see do you. you in quarter-over-quarter

Kate Haviland

comes a see we're I of fully specifically in to mean, terms leading I indicators, growth quarter, of We lot have to we'll the see hoping picture want but guide to for those together. to see don't certainly how sure. growth

inventory we're of in better cetera, of coming March. that et some around growth sense you mentioned [indiscernible], And have the as out we'll impacts a and

Peter Lawson

kind Great. we potentially about heterogeneity? patient the Thanks single with about way to as tumor on think this just gonads is And to SYMPHONY so patients responses tumor AACR of shrinkage think best for less much. then data, as around

Fouad Namouni

This of This cohort, safety development, is escalation stage think, first already and the about be is Peter. we from I important dose going data Fouad. to PK. are Thanks because talking

then earlier, circulating around activity And be when talking in surrogacy you the about within expand, patients, terms some an midyear, of see remodifying from are activity will clinical perspective. shrinkers mentioned response some you diversity the I perspective, from tumor time burden so they that's data, in to As DNA and and like rates, how RPXD from would is and patients including PD on. tumors. the of we expected in tumor

Peter Lawson

Great. Thank you the much. Thanks for data.

Operator

Thank so the this queue Mr. are call over the you. no you. at Albers, questions to further in time, I'll turn There back

Jeff Albers

a was, a growth as on of operator. the see on have we going upcoming of lot as a conferences, perspective appropriate well a facets research as you, perspective. I as call, patients opportunity and through to just in timing question, commercial of to it development a Obviously, have I us we on all think, feel our medical touching this Thank opportunity perspective, fortunate commercial end business both to and AACR. impact the in and such from And Excellent. from on from front ahead came incredibly

the to months. seeing And of so, conferences, medical going range coming period a at to you more lot look all of over be forward busy a conferences And It's investor to come. time. a

next forward the to Thank update. look Bye-bye. So, you.

Operator

This conference concludes today's call.

You may now disconnect.

Blueprint Medicines (BPMC) 16 Feb 222021 Q4 Earnings call transcript