bluebird bio (BLUE) 7 Nov 232023 Q3 Earnings call transcript

Good day, ladies and gentlemen, and thank you for standing by. Welcome to the bluebird bio Third Quarter 2023 Results Conference Call. [Operator Instructions] After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions]At this time, I would like to turn the conference over to your host, Ms. Courtney O’Leary. begin. please Ma'am,

or me current opening will momentum Officer and the and provide the will Commercial Krawtschuk, lovo-cel. intentions some and call for to Operating results, turn results everyone, position Andrew. A well Director on in addition on the these from company's lovo-cel for financial for our and call, Klima, the Chief materially plans, it of plans, risk as preparations of up I number that expectations some of Financial business potential subject begin, risks CEO description Andrew differ joining Relations are Investor will Today's expectations assumptions and involve Obenshain, regulatory Investor including Tom our our Harbor Such discuss the Good future which contains color financial in results financial the XXXX, Then contained is website, SEC, to on Securities discussion business of of Officer SKYSONA commercial our today. thank overall section Litigation positive uncertainties projected are factors our ZYNTEGLO My launch as quarter and Q&A.With www.bluebirdbio.com.On statements regarding to let opening we Chris provide to bio.Before call before going over in at approval. risks our and today's with results commercialization forward-looking filings are launches And Chief O'Leary, that, our expectations progress, results. statements is our statements our name could and that third that remarks Safe Relations statement. actual a to under morning, are bluebird operations. you review Private based is regarding and of on Act Courtney cause Reform finally, available

update of you morning. call has lovo-cel. FDA field disease and results This on potential thank our to been going platform excited for approach profitability, bluebird's and we the for we joining of morning my preparations Tom years for the extensive I'm December.In and transformative today everyone, you, approval importantly our cell for for focus QX a Courtney, details will potentially as bluebird this as financials. Chris gene for this validated milestone. Thanks, FDA But I upcoming reminder, are unique year and gene and to therapy regulatory our ZYNTEGLO comments key minute, growth, gene come. And occupies strategic our a an and for most sickle bio. with an commercial a potential of and patients and the but cell a expertise to more for want model SKYSONA the only are on as standalone position therapy lovo-cel We And therapy on this companies in one commercial of of lovo-cel launches. therapy commercial gene exciting with to incredibly our for enable provide launches approval that in moment we company, the approach expansion bluebird as

gene sickle to is a cell therapy sickle breadth our vaso-occlusive to the wasn't requested our will approval and testament FDA for a of and a any older package with disease the team remains be weeks main away date for the driver more disease profitability depth events moment BLA program and and fact our lovo-cel PDUFA We individuals for of December lovo-cel potential track a of company, cell the This the for be it will of for community, approved, Advisory cell remains sickle of bluebird are our the X if or from The that disease. believe the VOEs.We history milestone for robustness, than confident and significant XX. for review on Committee the lovo-cel data maturity years XX significantly other major come. in

lentiviral gene familiar third and with. the has is thus, are they the Additionally, very from therapy that FDA a this reviewed technology bluebird vector

lentiviral to I technology measure years therapies unparalleled traceable, up that for how highlight and following week, anticipated will over the commercial the XXXX, of cell Last giving we uniquely years our long-term launches progressing. early our Meeting modify cell a Annual the be follow-up in ASH unrivaled program.We in can progress how our to of ZYNTEGLO beta-thalassemia XXth it from at over our in us ability We follow-up understanding we with to monitor in our and turn an lovo-cel is disease to launch and rigorous also Tom of monitoring. approval.With effects it an to will presenting early vector programs we understand its follow-up that, time. X FDA data X Our to continue announced December, conduct sickle transfusion-dependent plans potential are lovo-cel

and good Andrew, everyone. Thanks, morning,

have to our to advancement discuss tremendous here this We progress made and commercial it's greater quarter, exciting morning this be detail. in

or starts launch. ZYNTEGLO, collection For to since strong continue growth unique with linear we XX cell patient see

leading lovo-cel XX to XX company.Our this with the track QTCs nearly between doubled since QTC quarter to qualified we and and result key commitment XX on and remains patient XX year pediatric watch a activation We the patient goes and the a QX, they once patient. early scale as that As ZYNTEGLO will of our end the states. of the treatment at moment value-creating activated experience our a the the now is metric accelerated starts centers on network this from for collection, remain commercial reminder, company for and represents stages adult the for by to as has center will this revenue launch the in continue treatment launch. in has the ultimately across prepare through it's journey, cell It

outcomes-based new footprint continue demand. patients.Shifting for reach to experience demand with steadily likely ZYNTEGLO to Looking I'm for our into ZYNTEGLO reimbursement, We to our year, patient carry patient to we our morning grow Massachusetts and will XXXX, will we agencies. multiple Michigan XXXX and momentum the announce 'XX treating anticipate ahead state extend QTC have are into through and QTCs of activated and this meet to end continuing as expand to more thrilled the Medicaid agreements signed gain that

agreement an enable and may significant large finalizing types reach with innovative This portion bring to demonstrates for beta-thalassemia. patients. arrangements are we third of of state, a government a therapy these which us development payer patients to with eagerness Additionally, support gene to

weeks. drug commercial see ZYNTEGLO We approximately ultimate have across and access continue to remain at strong Prior denials authorization government for consistent approvals product received X payers. X and for

beta-thalassemia year.Moving All out note, We of high having burden delivered look strong forward this that a XXXX regular require ZYNTEGLO the recognize patients launch of anticipate year every underscore the on milestone, payers metrics on SKYSONA. on and to disease for early value continues progress to who transfusions. with closing the key next guidance to giving and of commercial

an to nearest competitor. SKYSONA therapy. XXXX, in to We manufacturing have start seeing received head remain our on and capacity.Shifting across increased and are and to for are demand in our track signed capitalize in ZYNTEGLO X completed next XX underway therapy Preparations of recognition year. guidance of gene we patient We anticipate growing starts, this operationally we with denials X enable early patient transformative with Lonza X to ready ultimate lovo-cel. government from starts are activated date. start and to And early against agreement this our patient X Patients guidance or XXXX launch year patient well on waiting we launches, payers XX-month QTCs, commercial giving recently are for

the Demand was more We patient of clearly of sickle ZYNTEGLO by partner and community an QTC for QTC scaling. XX feedback.As therapy strong an bluebird over a there a in indirect response to a consistently is recommended have of models patients reach that cell a competitive leader gene and cell investment community And which XXX% our they mentioned, if lovo-cel and recently no would an our be been miles is part trusted excitement to lovo-cel of The our market which XX% sickle gene in a at is decade, and expanded of as and doctor. payment intent weeks, patients reimbursement. their of has miles seen about is equitable long to bluebird to XX% focus network, access ensuring launch. is timely patient therapy, A research been team and synergistic sickle continues within and exception. planned education in than say XXX patient grow. and to value-based between advantage. patient estimated XX% we particular consider planned been network on a key QTC.Moving for include lovo-cel within within converted can to rapidly cell access has has lovo-cel

our potential in positive cell gives are FDA on in as national of with number pending contracting offering value early covered lovo-cel a we on disease significant approval of manufacturing. negotiations innovative us We currently sickle The represent underscores are advance approach. with confidence our XXXX.And living in seeing of therapy a response These in payers' the percentage of sickle individuals gene late-stage we to contracts confidence lives. make finally, prepare and further cell available our recognized for payers and

from hospital. delivery to many applying the therapy are cell ZYNTEGLO commercial optimize of learnings the to We from through manufacturing collection back

details with over that, compete. We We share to month, that are the it next to plans well-positioned time.And more plan excited through about financials. a I'll approval on Chris and we are potential lovo-cel talk launch to to at turn

morning, Tom, good Thanks, and everyone.

$XX cash to SKYSONA. by and SKYSONA, for continue This XX our $XXX gross in our on strong driven remain deploy time translates As our capital ZYNTEGLO to bring revenue. full-year in there to XXXX QX, reported million prudently $XXX therapies in guidance million from have a combined ZYNTEGLO revenue Tom over we been mentioned, $XX.X product of to primarily We growth. patients. to revenue, potential that total date, million range track starts the linear and and In million again, to underscoring with burn patient

to week, sell lovo-cel important sickle review This XX, financial marketable not in source of that, further for and additional our cell advanced granted, QX opportunities million. cash runway.And of have the launch our runway the cash, next announced entered would proceeds and provide or an cash an from early we've turn September extend our potential including $XXX for cash of the of into be has anticipated for had to potential capital to year. strengthen agreement release cash we equivalents, in to plan last of sale that PRV voucher, over lovo-cel.As with Andrew. of to million updated securities, a to million guidance I'll into position and it a $XXX explore We restricted an runway cash, back if restricted XXXX the non-dilutive we $XX by ahead cash, we disease Additionally, financing priority the continue

to potential through for from X and stand a PDUFA Tom, therapy we thanks, and living largest as sickle bluebird opportunity for disease yet, It's those Chris, the date in with weeks time cell walking us lovo-cel exciting the bring updates. Thanks, out an our gene individuals to our U.S.

therapies Looking promise further delivering to the and the like to potentially committed gene Operator? and patients we'd it near-term horizon, of questions. up their bringing with remain our families.And open for to curative we to that, onto path profitability on

comment the [Operator Dane from or line RJF. Instructions] comes from Our of first question Leone

the of Congratulations Maybe progress few on me. towards and the for lovo-cel. approval moving questions all a

of give patients would some you the burn year. go a probably for before that little actually expect required long-term burn and the lovo-cel? just down the that after just And the expectation just will recognize the some That's So secondly, year-end? is and R&D recognized or balance bucket obligations infuse biggest were remainder you as bit that of is the revenue provide given guidance the more there could the Firstly, bit still color collections general basis. what quarter of clinical follow-up in cell from in R&D the infused the expectations studies? on of patients a could relates that on then remaining color it as revenues quarterly the us Is and to terms had maybe to the approval, you us burn a around of still

versus infused rest how So year of guidance change. for it I and think and How R&D the the patients and the in collected in X burn there. questions will

those? address to want you do Chris, So

Yes.

So start the patients infused. with let me

to -- ZYNTEGLO cumulative guidance launch. we're both continue we'll and not the in SKYSONA, to stages of because we're where commercial just are on early we So in going provide individually our

what balance we and our R&D, will Again, on gross and it's patient has million -- starts it as infused question X? your about deferred, we've time. products, question both over this then relates million revenue, X in is to revenue $XX in see, been I believe? can deferred sheet. sitting then you you $X.X that's translates on the been company again patient for about patient in XX and to starts.And that And your repeat KPI in that's have come As our

Yes.

incremental $X.X recognition -- would just to million what follow-up of that still $XX still head of XXXX Q, you I then I there post go the think of correct just or on year-end. secondly, recognition commenting revenue there's we the expected around R&D be that drug the occur million, with into a is clinical the And is somewhere there's quarterly studies? for associated there beyond of interpretation deferred million substantial still $XX before just And said amount? on deferred R&D, that would revenue lovo-cel. commitments subject, So on as approval Is basis lower are long-term that the there remaining from burn that was in

Yes.

take So that. let me

sits the in unapproved. and SKYSONA. for that with So quarter contributes lovo-cel as got associated of the to relates R&D There's date. ZYNTEGLO because our predominance programs to is also And attributable product any and lovo-cel spend follow-up spend R&D long-term to and spend R&D, ongoing then to it the in that's work line clinical R&D both that we've that the the

to either in in As will expense R&D go certainly as down, or costs inventory longer and approved, through when lovo-cel it see gets you'll COGS XXXX if those and the coming beyond, and and no R&D. relates

However, there costs patient all X for long-term follow-up and will likely optional programs. be registry

the or Gerberry of line comes next from Securities. of Our from comment Bank question America Jason

pent roughly that understand recognition So wanted in just long you're revenue be appreciate you with then just the about sort just [ you How launching patients in comment place bolus when but little dynamics I of Medicaid expect to to to there the cell sickle dynamic. you about that when could you states? reimbursement more we can early expect you just would absence patients policies phase that the more speak take ] of curious deferred? I and of of And an dynamics? that bit So how the key if at Would And up? in. be lovo-cel, are to do expect those the launch, speak think Medicaid to interested, a if early XX% to

to that going to I'm Tom hand to respond. Yes.

the lovo-cel therapies. the me would expect gene of would last like Let We question that first. launch look start with other

through patient But Specifically, ZYNTEGLO it expect And and it believe then the the through upfront. QTCs, we've there patient gene patients And excitement seen, linear that, on that centers get treatment strong top the we to getting time bolus growth. like would look going demand. We of takes process launch. that be a are qualified of therapy. to process as working of just the is reimbursement

strong although there's don't growth qualified launch. that larger there's that with a much But seen much place. a bolus scale given in and -- it for higher traditional we on already are patients So be therapy, and gene patient we have it's a population oncology a believe of expect centers would treatment like we a eager a expect will that excitement bolus linear we've ZYNTEGLO.It to we be like bolus of

who As estimating Medicaid by sickle XX%, patients the dynamic, are cell of have Medicaid. far covered disease as we're about

equitable on fair through we've exception payers.So or back the And payers Medicaid. patients, medical with early no expect outcomes-based reflect you We either get Clearly, coverage seen now take for with an payers and commercial are have with working the as have go and the might as possible. place, more than denials commercial if a But coverage. hopefully, both, patients Medicaid process. close agreement bit we agreement payers commercial will payers Medicaid with both the launch time in and patient and and place a ZYNTEGLO put right would soon outcomes-based little Medicaid possible to patients commercial as launch, to who as in with

in a Were could exceptions place? an just squeeze Medicaid I on in coverage getting was with policy through If medical ZYNTEGLO. patients follow-up before official

Michigan. with now, Early that's Massachusetts Yes, now agreements it's right coverage we I outcomes-based correct. and in believe, Medicaid -- policies. policies, launch have have X XX have, states the coverage there We

have close. And were to we the early and approved getting through the medical lovo-cel. through We exception on, that's But a for third true patients be process. would expect going really same

the or Eric from of comment from line question comes Joseph JPMorgan. Our next

I guess lovo-cel capacity terms treatment? Can year. expectations at at or about patients thinking just undergo of bit next expected to that us looking about talk for centers bed capacity, of And beds least looking treatment qualified a -- helping in launch perspective, curve from the for for outcomes-based in sort a reimbursement of and the reserved ultimately these then reflected sort pricing their What revenue sort outcome of outcomes or just a protracted just frame capacity of are -- pattern? in get of negative a success? a in kind -- particular that's you be how or a form on are color for whether more then sort sort where little at going ultimately, top-line, I the repayments agreements. looking to of And might the guess in tracked on of you recognition

#X, X one agreements, measure how reflected in financials. capacity what And So bed of to on lovo-cel? going kind that's #X, with you're there's if the metrics the gets for in #X, then if the questions that outcomes-based case, hospitals. there.

let me the Chris. first to and to those hand So Tom, X third then

events capabilities. at or big convenience luxury the and the less and schedule not an out around don't times these our not schedule issue, the have and hospitals because to many centers have So Also it able being qualified going life are times that issue treatment being therapy busy wings acute, at an and huge Part diseases capacity built of forward. and and cell busier have of has we of hospitals hospital. patients is been anticipate gene around

price you hand #X, in So, Obviously, asked be then a seen we not little have issue. hand. a far, at agreements. go about bit outcomes-based so QTC and an those capacity

disease a obviously, population.And Medicaid closer with the we've give we to agreements, received but reasons, endpoints, for to talking into consideration, I I'm different Outcomes-based then sickle what going will primary higher a more now about not very disease, specific did different competitive payers. will We price feedback and launch. be endpoint is from right get that take positive state we cell say as

the In fact, received agreement, we're outcomes-based the -- it's that a close we few right reflects payers signing now momentum how feel national around and which being we to with have. large positive

constrain effectively are and of particularly we'll coming if that And rebates capping XX.X%, Medicaid, recognize that, constrain the statutory and off offered, particular associated Remember, of the by that number through Perfect, that that's revenue is offer particular the class with on we then revenue we'll of then any product. in patients that a commensurate trade are Tom. for the the that like revenue just rebate constrain SKYSONA XX.X%. with that being way the you'd it is rerate estimate

require So we'd to as here no coming of accounting through then estimate us outcomes-based standards that. revenue a amount relates take revenue. the conservative to of of estimated it ensure agreement, approach the And we significant reversal there's because

through outcomes-based estimate of So terms. number constrain in markers outcomes-based market we'll with we'll and the analysis, on the accordingly based conjunction and the associated then the revenue and agreement with at look periodically agreement, patients Tom's that accordingly the contractual that revenue coming the release

comes Jack question next Allen comment from Baird. of from or Our line the

commercial of on and dynamics progress. to so. discussion the A about Congratulations sickle team around the rightfully cell lot the was

and any to One long-term ability you to shaping how have how see had you up? that commercialize follow-up Do of that label questions to are into the thoughts on the I look you looking as get leverage you was medication. your

Go Tom. Yes. ahead,

talk Yes. back last have most of amount data many Obviously, the when thought therapy you in or QTCs both we prescribing any in a you over physician, years gene recently, and to a physician either long-term a market that community the in been there look out that's sickle disease of cell to difference leader, research is the referring robust talk a a as transplanter. package the the we big

to and leverage we an long-term will follow-up advantage us. continue the that for we So to of be continue obviously will amount that have,

point-in-time say, just all would long-term I a this be follow-up. just you In efficacy. times, And this will we case, lot Yes. a of particular see publishing

will the product the of picture robust in product be more overall the much a of the important which it's differentiating I profile in market. think So

comes from Our Barclays. line comment next Gena question or Wang the from of

Yes. questions. I have I think X quick

Could and regarding a to revenue, manufacturing is Lonza The the this question then the first little ZYNTEGLO And down regarding capacity. product regarding give SKYSONA the million. amendment quarter, bit the break $XX.X you revenue? increased my more is color question second

regarding -- how numbers? have is the expanded ZYNTEGLO? that what -- Medicaid is clarification. to for Just wanted a the increasement And patient much what terms capacity And lastly, the So of in is? outcome-based outcome-based then

into or of say, recognized like your revenue a possible a proactively released, you the recognition? track also will discount account outcome for taking failure, For revenue XX%

the going you and And going bit I'm to -- bit about breakdown. to Lonza hand is that, of will then first Chris because to which the are actually ask do, take regarding I'll the I second through if once then your to what you X, I'm I those But repeat forecasts. we sure not amendment, Gena, providing can not I'm question manufacturing the get Lonza last got we for it.

to been And pleased completed and ZYNTEGLO. has process scale agreement QX, see always demand. for in up we this initiated this commensurate demand encouraging we have as plan we're with to Our

plan.And So quarterly our Chris, just the about it's talk part really do you to want revenue? of

are the balance to revenue. numbers, XX And process, gross starts, approximately we're which and cash that but effectively reporting patient while through we you reinforcing completing patients in look, can see our have moving effectively $XX a million collections And is had as and represents translating sheet. not reminder, infusion then which

the that I'm you do and you question on as agreement? then favor So me not sure understood can it. we've I'm see will a linear repeat outcomes-based continue.And and growth that strong it's sorry. described, I can your

Yes.

maybe XX.X% discount you like outcome-based. prebook, you layer, assume or proactively book success. but XX.X%, is you you very like think when Do XX% out say, I there clearly, So another lay recognize, Medicaid, say,

say, failure. So you XX.X% the revenue? Will be of, XX% recognize that putting on top XX% discount and XX.X% proactively you will when

Yes.

of And and will complement course, here. start this. I'll Tom So I

question. mutual I think a it's

the they patients will which look a And based flow make we on at channel, that. how for. particular then So what qualify is rebate estimates we do we through

out Tom of outcomes-based think I'll his, I threw in what estimates number qualify rebate earlier rebate. call through for the that on research will expertise, patients market outcomes-based on or estimate how patients would an the many an would come professional

So that coming outcomes-based evaluate each a that the information regard. that the you constrain agreement, particular quarter, that that patient will marker, kind duration health, we we down on our then using in and whittle et informed revenue through agreement, rebate.In then patient of case, the at look of patient's patient's And it's we not and that associated only then trial that the we'll the outcomes-based in we particular patient in agreement cetera. with population is what patient but enters and through the hits rebate as that do then outcomes-based time follow actually, clinical

a it's make estimates So on and effort at that agreement. collective very that look outcomes-based we

be We recognize will in never a situation revenue we where ahead.

is And We the we'll what do particular will we'll met. constrain for that when revenue. endpoint will purposes, release conservative revenue the -- then we is

Tom. is to And that. to this just maybe -- add Maybe

For through ZYNTEGLO, keep and data through remainder in to of are covered XX% about historically, mind the that on Medicaid. commercial claims insurance, are patients covered XX% based

in payers. we would still we have announced an where majority even of morning patients through the covered to that states commercial So the outcomes-based agreement, this we expect the be

of line comment or comes Mani Leerink. from the next Foroohar from Our question

you hoping that quantitative leverage. clarity can bit around of a help little I'm if provide operating

that about couple should until that's it terms be improvement you and that? launching, relatively Is the operating and margin has flat of improved? in got if think that that scale? how presuming Should products threshold so potential amount Is revenue to leverage absolute about level expected about proportion prescribed of should you've linear Is we well, a see some So in which gross think think number true, expecting we of different some think the we through starts And compounding market? of growth that there's patients? absolute as margin we growing, absolute of presuming How we're linearly. scale? that as we How it profile? should there approval gross comes kick to expect in margin the

Yes.

and to going to qualitatively it Chris. I'm Mani, then So answer that hand

you or are is But from But we're So #X ways. sickle we beta-thalassemia robust a give is SKYSONA do that the the many patient patients different to go -- to maintain margin. positive to profitable. and going need numbers not like cell higher. how anything in gross X or

to a from at more be away you're QTCs, going grow higher base a start it's just there. starting So to going it's and at higher right base,

So linear #X, different vectors. takes off growth from we a #X. start point, switch

So we system and a or efficient ZYNTEGLO of vector much improving a margin that lovo-cel has production in mean rare didn't favor that are relatively which which gross more SKYSONA, will therefore, have things for better require significantly markets. than vector X indications there's working lovo-cel, for change.So lovo-cel

be to really driving so that Chris, you And going to to do what's it. is want add anything that?

level business, our our are of to ability our capacity with we fixed continue see business. commensurate Certainly, our our with a The the in and pace fixed costs reserve we other thing really attainable, commensurate are leverage cost effectively in I'll will margins add and manufacturing there capacity that which company operating sales. believe have that the to driven are to today X from production And the then those by minimum a the of improvement in our XX%. drive the we see is really margins yields. things you And the those volume margin are that and

follow-up. As a quick

And understand guys that Now of about I gross are talking terms the why you in we're margin. qualitatively. answering scaling

expect fairly statement stable grows scale to expansion we we on the Do lovo-cel as potential income of a line little bit. about time? move Let's How over SG&A think down sales we that? should U.S.? the should be expand a opportunities step sales how function? force the Should the a modeling by number Should in expect of force in we QTCs? to expand linearly reasonably a Like

SG&A, of Tom that us, and is forms on at And that to look pretty discussions pace will is our and relatively the up answer at to and part infrastructure, think commercial Tom other opportunities And we grow front that time time our really what spot that's invested achieve well on, head has revenue, and set leveraging in of that. commitment. we've low I the cost, to historically question, principally We focused continue linearly your our described setting described QTCs as kind we of what was that XX-month of start. Andrew in believe in. up infrastructure have and

in with So make will SG&A regard, revenue. see they you sales, and continue with to pace pace but should that investments we'll

I increase not but increases percentage. say it same necessarily revenue would the just will as

That's correct.

lot a transplant leverage. centers. market. disease Because There's rare going a after we We're do very have of good targeted

built of supplement it So adding build most versus been and we actually wholesale it the be to that -- it to has already infrastructure need to we'll adding cost.

or question the from comes of Sachs. comment from line next Salveen Richter Goldman Our

treating have on is just This manufacturing you question Salveen. has to on And to to proportion the collection for on the on Anoumid QTCs just a date seen of to the being have sickle with issues cell a the follow-up manufacturing. What activated end hurdle then that by one been what address? are cell the And date? this able progress. Congratulations and patients of these are year. guess, QTCs I patients ZYNTEGLO? I

Yes.

about with you address? can So why the QTC don't how they much start question Tom,

on or -- XX have the a we designed the within goal community. And be end planned our XX disease of who year getting of to goal network QTC. the board to So plan with XX% cell end to being we XXX XX% this that miles cell our to QTCs have within QTC the was of with patients sickle by reach sickle

community. for have sickle that we cell we feel So coverage the excellent

a expand being want travel unwilling be to we into to to continue for year wouldn't likely will treatment. QTCs patient barrier next a because We

critical just we trials, The regarding This therapy, gene of therapy, show And that same really gene remains Because part is did difference specifically where With we commercial. with T-cells. cells or our can with -- manufacturing. not to in sorry. stem only cells, had cells, a In expand is T-cells, recollections true sickle them stem the manufacturing. on collecting re-expand you them collect you're your clinical is them. option

one have it. do agent We mobilization to

or we thalassemia, case So sickle do case the cell. of the of X in in -- see X

highest standards for various and recollections reasons, see for do we So the uphold we quality manufacturing.

of all to a therapies. the repeated, that need gene this dynamic but stem normal times, that we at be to be in process have is steps I very cell the So across going think means

Cantor or Schmidt next line Our of from question Eric Fitzgerald. from comment the comes

It's manufacturing. about lovo-cel

manufacturing, different drug approved Maybe facilities could the supporting that's just have I the other commercial whether whether whether they've been think and or vector confirm already one have support you these case to that comment for by products to existing one any facilities are and for X FDA. you the been inspected as product.

in So and Fisher, vector we we the Thermo Minaris. to the the X by FDA the just do can't company companies interactions. blow-by-blow inspected Jersey facilities. whether -- go We don't the point, have because quite last you're with forth a and or now. product in for time I with make called Belgium been with a we But we not New long correct, drug don't with with these FDA make back they're do comment working We've

have really established So we relationship.

company. They've we Now worked with those and they ones inspections, have FDA inspection. X through not the through gone all gone ABECMA gone have in inspections SKYSONA their Europe. one gone ZYNTEGLO our where but has, inspection through the through They've team

is time their process. around. So we in confident So feel our very this sixth

Zhu Wells or line next comes from Yanan comment of Fargo. from Our question the

have questions. may, If I I X

on could talk FDA? lovo-cel, whether First, you about also stage Second you're BLA question or discussion whether the labeling with the for lovo-cel. the in

You've talked higher at going to for lovo-cel. a It's starting about be at a this scale launch point.

that a could far? And affect willingness give perhaps that as question patients, than a actually factors or XX,XXX see are TDT cell that's be scale or on Is patients TDT versus pediatric could well this other adult XXx. there sickle XXx color so there launch. you Could lastly, think some X,XXX like get I how you little in severe treated that patients some scale? more to versus launch on

Yes.

comment the ongoing with the interactions don't we on agency. So on BLA,

to submitted cell BLA the the review I'm Tom. for are sickle patients lovo-cel last is treatment the robustness history events, vaso-occlusive and the confident of and we maturity in older So of going package X, have of we've Then disease and with and to pass XX a process. next the -- our who

in it question the third about was TDT that ahead, then Should Tom. play the pediatric. is Go expecting is to market by we adult and the a dynamic be I sickle XXx think versus thal. out see

there have about are larger XXX,XXX are opportunity. have more lovo-cel, patients United the everyone, in remind disease. cell who much And to sickle XX,XXX that a And believe So the States we about obviously, qualified who of just the beta-thalassemia, TDT, there we're XXX and are disease X,XXX therapy. cell about that patients eligible estimating would therapy. could there With be those in for severe gene X,XXX United gene sickle about for potentially States, that be potentially

the greater size. actually it's So XXx market than

dynamics a obviously but for coming end the with range. population.And -- for much beta-thalassemia on because comment seen a such it's larger seen with to a And it's So huge then patient -- we've in patients that's yet, treatment individual just we population hard addressable haven't with ZYNTEGLO. patient huge

lot the we've We and come and have seen online seen meet different We've centers forward both lot pediatric that a a criteria, of of now. stories. adult patients obviously

So from demand the hard a a just there's patients. is is range that of there. there we're But and that wide to say the theme it's demand there common excited

line from question next RBC Issi of Our comment or the comes from Luca Capital.

first there date your after if believe PDUFA of for price the means on X disease? there is Is a AML opportunity par assume discount Just them? adoption where days comment an should is versus two quick picture, drug one pricing implications And here. that competitor maybe pricing. trial secondary faster price to XX your in we or on you'll that's that on to you bigger not? make a their while they're final malignancies And you then Maybe have update much sickle get label competitor the I on could call. and scenario them includes scenario, how before see today you'll for we commercial Any call the think you with the launch? the safety. your the a about you'll does cell how a risk at if Any should a that develop appreciated. Wondering on whether patients there, scenario where which maybe doing gain

to first bit Tom, there? do you the take want

on with into But same run disease. that price it cell Obviously, by impact recognized a in our obviously lovo-cel ICER. chronic dynamics going condition. Sure. are life the do clinical we're we'll our a seeing and potentially other improvements, to like of payers process Many one-time just therapies, in cost-savings to benefit, SKYSONA. by for system, consideration take the different quality therapy the Yes, we sickle we ZYNTEGLO ran curative as recently with values recognized society,

So we have We price these value clearly, the we way that models continue obviously therapies, for are We we and are to competitors' pricing. leaders comment any setting price the establishing our But privy to believe that price. out will on and won't there. in we don't that.

And safety then, Luca, issues. just the on

As a reminder, insertional there with have of been lovo-cel. no oncogenesis cases

However, X been X have were lovo-cel. leukemia procedure And the trials there those where procedure away. X phase in of clinical did we procedures early our pass our A cases to involving unfortunately, those did develop in transplant related since earlier of Generation -- of AML, patients patients cancer the and and the Group

there I something certainly, of in the label X known will while.Again, it although are there. that -- really efficacy mention important or In are cases our the of no what a where insertional the they quite for have safety of point think in determined, is So they label. we've oncogenesis. part is we yet patients events that is That's not in will not in that those be data are but set, to be a likely the

cases And related so to these the procedure. that were are

of Our next question Blair. William or comment comes from from the Corwin Sami line

SKYSONA? of X you produce the be opening about it we closer manufacturing in increase about or closer time all there that decrease to it's QTCs? commercial to possible duration do time across time incremental to then and manufacturing to I manufacturing that products, the ZYNTEGLO Will will think And efficiencies new be long term? then to an thinking be was should it likely your that? lovo-cel, is curious, for thinking cost how And

take If I the first I'll take Tom. second too, one, the

Yes, sure.

a done only our really qualified is we've And the the So time QTC center up headcount of just running. treatment and analysis the cost on the and ROI lot get network. to

So patient for profitable just years, course then ROI they it's treat in the analysis, us. X over one endeavor our if a of very

be be XX incremental commercial how time And to the will going at for timelines, the tests have robust release cost for is continue would think So to those to SKYSONA what closer in right?And tests we timeline. long you to do drives expand that's likely down overall, because we similar year, center terms release, patients, our timelines process qualified the all. times XX your of collect will beyond impact us biggest that network the we will than cells just many it of that question do we nicely and that just about and just to the then to the will that's We lovo-cel be ZYNTEGLO products, bringing timeline time. say driver take. are consideration biggest of this the a from then have relatively any I on will treatment allows hardly anticipate release over those And how to do second we be not said that.

Some are tests incubate. to of just lead release time. have those They long

a those below going never get lead but being to -- time certain out of taken down you're So that threshold, times. imagine you can some

line Stanley. comes or Jeffrey from of question next Hung Morgan the comment Our from

on Michael for Riad is Jeff Hung. This

uncertainties outcomes-based ultimate agreement Medicaid feel becoming I X denial generally they agencies a state for slowly specifically guess, can that understand agencies, there denials, given agreement? have that wanted On is on X the have Massachusetts and date. this or Michigan state given just the has been from do that to the ZYNTEGLO formality outcomes benefit to rationale and the I there with

ahead, Tom. Go

to system. I seeing beta-thalassemia disease Medicaid a the So we're like think state obviously of very costly value that's is in the what agencies therapies state that recognize one-time

transfusion gives if they think that rebated additional paying the for independence independence, least also percentage outcomes-based then of That of doesn't the that where I saying not transfusion patient therapy. them be and achieve something a agreement just they work. should appreciate However, at doesn't a they're the maintain we're our cost security

with So this it's obviously feel that willingness we it's case, the a testament an ZYNTEGLO in and to to us. added value And their work of assurance.

for I'm questions time. additional showing conference at like Andrew no closing the I Mr. to the would back this in Obenshain turn over any comments. queue to

thoughtful time your questions. the and for for call QX bluebird morning our thanks, aim And a to patients this It's and we serve. very for joining that everyone, Great. exciting

like have please out reach to follow-up additional call, Courtney. you. would any set you up If to Thank questions or a

program. concludes the conference. for gentlemen, you Ladies thank and in participating today's This

Everyone, You day. may disconnect. have a wonderful now