BLUE - Earnings call transcript Q3 2018

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Operator

Good day, ladies and gentlemen, and welcome to the bluebird bio Third Quarter Update Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call may be recorded. I would now like to introduce your host for today’s conference, Ms. Pingpank. Elizabeth begin. may you Ma’am,

Elizabeth Pingpank

abstracts you, section yesterday operator, be sickle Today, will be the issued the morning, disease some on yesterday quarterly found highlights. our these and our development topics, website. good We ASH on discussing touching in press addition everyone. were plans announced in and of releases we two to Thank Relations that covering for cell can Investor LentiGlobin which

or statement. XXXX. to let Today’s forward-looking from risk current on our begin, Before materially factors are are to candidate successfully limited that results. the me risks more statements, uncertainties could that www.bluebirdbio.com. in are these of that section Securities Safe Harbor subject our our or actual Investor approved based with particular, website, assumptions contains Additional projected are review are risk and discussion that be SEC, product cause available we of and filings include under not Litigation differ our but which risk factors statements Reform Such on Private In commercialized a the that involve and will information of results contained to Act one Relations concerning expectations of any statements number the developed,

do law. we even by if forward-looking to we change, statements to as update any may required obligation so elect specifically our While in the disclaim future, except expectations

that any Nick. turn these to, on subsequent it rely not as With our as should statements I’ll date You representing expectations to today. forward-looking of over

Nick Leschly

this us for and Thank thank you, Liz, everyone joining you morning.

around flow the made do cell abstract, do development across sense plan, our depth quarterly of As this calls, but not news we thought XX-Q. normally you and ASH disease sickle recent it given time know,

to many team multiple myeloma do and for reality. us up that but get filing submitted patients. our believe to for human inspiring CALD to very everything must of need. also is children cancer powers to is ways when the us success that is critical is reground we visit bring as just make bluebird. of stakeholders. Before thalassemia. remember to our in what old it It I always broader and and never to patients the in intense we promise with shared from must to why, dive decisions The in, and I force for had the quite for face hospitals the across we centering and important helping inspiring. The not aspirations we magnitude the could are are becoming in do need we overwhelming. do. why, significant, The want Asia complicated It in recently honestly working let a and treat gets firmly blue we bring industry to desperate we long-term and and/or on pressures rapidly the that we of EU, to importance is is task and patients of to that What and far believe our particularly the in hope the challenge I is in the shared true that that approval TDT to succeed globally and in of why we’ll deter announced behind. We if what family tirelessly chance several is returned excitement do hope not we our be many blue is

in disease with regulators with on are accelerate sickle to product important with desperate path define excited to families and to call disease candidate need. our On ability that made bring you we collaboration the progress the share our in the to hopefully will front, sickle our we cell development some patients cell have

and of the on update validation on the emerging promise. inside Shifting and of bluebird to This sickle refer today. delivering what broad establishment for differentiators of is the of more the examples. as that are only engine news cell blue for has the therapy capability day-out any the but appreciated as not two through often future. be good least the a tied we as shown The end-to-end I most breadth ASH to well being company can gene to important abstracts day-in, one our execution depth and this gears one product Lastly, building the soul. and and built of perhaps products

of some accomplishments. Here, you’ll key the see a list of recent

the target of as many company the ago, completion the of is It set filing huge months first for course a amazing. team and our foremost and date ever on we First delivered a milestone TDT. is

The thal reasonable important validate but have other we built further certainly The only and these theme disease. are the CALD also sickle the is our regulatory and in not also relationship but have two-way They details constructive engaged been regulatory now accomplishments guidances. requests with progress authorities. and terrifically cell on their

line our earlier aiming Celgene More partner It active establishing and Gritstone. this We XXXX powerful franchise come potential and BCMA is to have with to is busy Regeneron and with building into say, oncology future high getting product on on for also both studies a as get we pipeline safe XXXX. relationships also been our for build very into to the future. the

longer no to seven update β-thal, was sickle and into have to study and saw genotype. served Northstar first this abstracts separate study, two follow-up year were one Phase genotypes. I’m ever since Again, yesterday the presentation also productive data those of year, get let’s biggest study, busy on from two EMA filing. will time cell be sickle little oral from HGB-XXX have the cell what This part days which ongoing with follow-up the endogenous We’ll beta C. who we we for the gears It’s this the a quarter, early updates including our saw that ASH EHA and to In presentations. an at we’ll from at and have for impact have from at cohorts as presentation patients, Coming broadest additional of where ASH β-thal second longer our original in our a off We’ll and basis and Group first in β-thal process B, for have hemoglobin. showing. disease, also is we patients with on development completed In a first share from which studies of updated our manufacturing the our of the then. refined in show β-thal. which patients non-βX/βX follow-up The for of look longer important we produce on and our will we you of Northstar-X plan Northstar-X patients LentiGlobin X data βX/βX placeholders sickle, any patient but patients disease. XX be treated ASH share largely very presentations we excited these in third we A Before and of shift briefly

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turn to over it I’ll Philip. Philip? Now,

Philip Gregory

Nick. Thanks,

bbXXXXX result process published CAR cells. the bdXXXXX abstract persistence cells the cells, manufacturing at survival. million generation to dose which increased BCMA product, next a model. cells and yesterday the cell studies, anti-tumor positive a patients T CAR preclinical for T lasting durable with called As eight stringent T longer and tumor of CAR these evaluable drug on in that of but demonstrated more our provide responses employs T you eliminate T comparable known in appears disease with to enrichment of bbXXXXX long improved with the and is activity the ability targeted tolerability cell bbXXXX, results subset product for re-challenge of CAR The toxicities may Safety of In bpXXXXX of so therapies. persistence is the reports same seven first therefore know, level and XXX improved response. treated construct including T progression-free as memory expected the functional ultimately

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from expression. this novel cell gears of restore I’m is both in the to form, in the patients has and Dr. that have changing the report cell form of HbS, to goal shown cell knockdown clinical for collaboration using β-globin sickle program others of to responsible and sickle by reducing is BCLXXA BCLXXA, the to with to approach properties. of we David the can BCLXXA of and a Now, data disease The at pleased the HbF, program “flip Vector-Mediated switch” HbF Lentiviral disease. with HbF our the in anti-sickling first Children’s disease. patients with which fetal Boston we use levels the sickle unmutated from Hospital is repression Williams sickle

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settings. megaTAL toward been editing of we solid Specifically, goal of to the T improve T-cell liquid cells have tumor working the with technology and the genome across our both our function

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Dave Davidson

month with Philip. industry therapeutic held Rockville mortality. have cell academia, methodically of patients recent expedite cell options the to endpoints sickle from prioritizing and FDA-ASH design high The disease clinical development suffer gather and therapies from to Thanks, morbidity by Despite trials. to the few stakeholders progress, effective groups agencies, Endpoints last early symposium sickle disease assessing in across address for patient to and need and of government new facilitate

an the endpoints development novel Hematologic this for science. clearly and on parameters of from therapeutics approaches of to staff biomarkers potential as possible with more speed to promising targets sponsors designs compelling using a development treatments discussions During innovative expressed engage were cell events. definitive strong need acknowledged as by disease demonstrate demonstrate would efficiently and of reflecting supported could meeting, it sickle a the accelerate openness compelling in such the the traditional underlying trial FDA vaso-occlusive that of senior pathology impact novel recognized to and to generally clinical benefits efficacy be and endpoint

to next innovative for Our and on disease proportion development based at and and amounts composite use we primary cell globin, approach, hemoglobin HGB-XXX represent LentiGlobin novel from LentiGlobin a These plan and presented the mid-year on that endpoint, sickle of this reflects TXXQ the data the level. EHA. we on slide plan study anti-sickling the total figures

benefit total to potential hemoglobin LentiGlobin clinical of rapid relatively hemoglobin released amounts saw podium Dr. will the by recently and at the John C of at data sickle on from robust updating cell We Tisdale from the relevance Monday, disease. presentation NIH, ASH Group clinical EHA, with a December presented endpoints demonstrating Xrd, production as of levels, you be TXXQ demonstrate abstract. the of we large The highlight results and

of cohort blocking Importantly, associated red hemoglobin levels TXXQ parameters expanded hemoglobin endpoint on alter to levels globin in our way, with sample forming In of and emerging the TXXQ the physiology improvements cell size patients and our key profile in sickle a on profile eliminated individuals primary validate level events. the to events. a clinical endpoint of and objective, be meaningful of and as of will S, of resembling introduced sickle more trial sickle will in profile between were attenuated hemoglobin total The disease, allow TXXQ and plan endpoint example, we in XX. this classically in obtain study VOEs patients and include to underlying VOEs, amended comprehensive had markers high as of profile added The composite hemoglobin robust, a reductions LentiGlobin for the vaso-occlusive reduction secondary ages us hemoglobin reciprocal data improvements size the C S point advantages the have hemoglobin XX the hemolysis in a between hemoglobin. substantially the and of data hope environment, occurring with We’ve the hemoglobin laboratory TXXQ being introduce and that increased pathology. rapidly. have the hemoglobin we of polymerization and based by of innovative the on blood the impact us correlation of the severe have hemoglobin evolving a reduction sample by C. of HGB-XXX surrogate fundamentally Based by reflected to we primary associate vaso-occlusive events for similar the This with of trait. ability addressing the that to the the or S novel Measurements cohort cell the percent including gives will

study, In XXX This disease a a primary addition intend share events. open-label have same introduced of consider for secondary two with into international registration in history Phase in development arm, and cell we These single to Phase form expanded patients the of who will trial potentially to trial. to sickle the conduct us through path warrant. X that basis to multi-centered the trial plans HGB-XXX supportive LentiGlobin the X accelerated clinical start enables HGB-XXX amendment is XXXX. the outcomes endpoint as recent planned a studies vaso-occlusive the and

with engage back will to and And over that, We Nick. in will turn share regulatory continue the details more to I call authorities with the future.

Nick Leschly

Dave. you, Thank

in across where our has basis. reality year a we’ll From by of programs adequately expanding and clinical In folks team course ASH collaboration, summarize just to and to close on launch, you saying, highlight this to I’ll truly I in stage all to-date, December to by remind commercial to capabilities, provide and our to With visible but closing, seen on to just transformative X maintaining it while engaging planned not updates. Xrd, at captured advancing our call alone, event briefly fired team and the next up are only and growing I’ll Here Q&A. forward culture, EU Company this team investor want and fundamental not XXXX that, in at close areas alone our across I’ll perhaps early of families year including many potential of our need. XX% programs, and not patients greatly accomplishments Monday our year, regular another clinical bluebird and programs hope our our also our letting research, manufacturing yet look in of as our and -- our for been thus here is many, purpose-driven blue one make many truly about Operator? in manufacturing our scaling for building platform to p.m.

Operator

Thank Instructions] Richter Sachs. Goldman Salveen Our comes you. [Operator from question first with

open. Your line now is

Salveen Richter

taking for Thanks my morning. question. Good

you the So, help Maybe interim you I timelines regard associated And And with us in endpoints this with? they One an program. in study be let’s you to you. two. for time would be study. understand the and you file in How then, monitor on the to quickly then Thank validate then, basis say do or and you do the X XXXX, years? post two do period have parallel? enroll expect to XXX have start planning will is, could or endpoints with study pivotal Phase are the to the what associated

Dave Davidson

this Salveen, is Hi, Dave.

that two questions. was think I more than

straightforward. possible. the initiate which is one, Let to as We the as the soon me trial plan X tackle XXX Phase most first

So, on milestone a going be it particular is based within to XXX. not

need. parallel. be we’ll one And the accelerate will to trials cumulation in the those in running fact, way So, of data key that’s

to later timelines In those about addressed the and have the terms detailed more the at trial, be a of date. will of questions XXX design

Operator

with next JP comes Our Cory question from Morgan. Kasimov

is now open. line Your

Unidentified Analyst

to you translate a doesn’t but have the a for to trial? secondary Curious during This is for key Matt [ph] a this FDA, with forward, on VOE the there is of the Cory. on path take duration profound endpoint. on understand benefit little continue your more as Young effect to a Thanks bit my the questions. Based inclusion if of about discussions VOE HB,

Dave Davidson

Yes. This is Dave.

I agency hemoglobin with I that at sharing more time, So, can’t as mentioned current on discussions and the we’re during powerful our share presentation, is sense the endpoints of the detail beyond course meaningful. are what our now. very the But,

We as transplant, essential also population, are context this benefit to endpoints in agree there demonstrate a in that particularly clinical meaningful well. the of

evidence the outcomes, of time. really evolves. will discuss be something those that’s later based at can a that’s terms totality In the how going weigh how the agency on explicitly to we And of

Nick Leschly

is be ultimate timing to be also key collect for This up of well always us this as is will registration and on kind point, and a was then, dependent, the endpoint. which focused forth as right, Nick. That’s this and so data hematological thalassemia. sets And and it is data

with So, approach consistent it’s dialogue. our with pretty ongoing consistent that and

Unidentified Analyst

Okay. VOC? and agree question. guys the Did One on a severe definition FDA additional you of

Dave Davidson

Severe severe? I’m sorry, --

Unidentified Analyst

VOE. Severe

Dave Davidson

are emerges, Well, definition is. detail able trial Yes. we and the using of stringent a see of more relatively as exactly that what And to you’ll VOEs. conventional be

Operator

from Chris comes with Cowen. Our next question Shibutani

Your is open. now line

Chris Shibutani

VOE of is might some As typically and kind commercial different Could bit, endpoint evolving, go think we the commercial regulatory threshold references the the there’s clinical clinical open of of are patients, about would into literature, there’s mosaic a have threshold of pharmacoeconomic perspective regulatory clear Can standard. about providing a a benefits outcomes. markers that there’s FDA of It’s kind how context then to considering versus or to more and be threshold that talk the various helpful. would these the little of to you you relevance inevitably kind often benefits be? when clinical

Dave Davidson

And with of that a transplant. and commercial The I therapy outcome. benefit we transformative aspect and think platform to myeloablative therefore, the for commercial the gene high, way answer regulatory have from we expectations quite is the a cell a context best expectations. reconciling said to which of our are in what really reiterate truly beginning that that stem addresses

will be to expect unequivocally both clinical would successful, the commercial treaters be to products and setting as our patients, clinically magnitude benefit the well? in meaningful, one of For

Nick Leschly

Nick. is this Chris, And

of the cetera. side for impact think what But I magnitude will, also, on commercial over to of sort on we’re quite et understand a well important. certainly is certainly if to is bit of the an effect, the as analysis the this the obviousness, patients the you doing time doing system

to that mechanisms subsequently. that’s package and is life of and that. which it something rest And clear patient. consistent trying cetera that clearly, of That’s for also of what be, novel sort also to the it the Because hopefully will one, data under understand thing working what system, is then is payment with handle registration assumption on but we the the that will, we’re et digest of outlined, over value, explore if as We’re of something continuing a is time. deliver is which that we’ll as duration day first, this of about are certainly tracking Dave and clinical lot if hope think will the data this and the not first only we sort clinicians, regulators the sort will delivers and value you to

Chris Shibutani

Thank you. to from you Can your your regard relate may you quick cetera? Great. of cell certainly competition, of with that that, takeaways it to us to activity, you follow-up. then, with as business a returned regard And in own been thinking particularly with share which has property mentioned particularly et development, hotbed therapy. intellectual from Nick, some Asia,

Nick Leschly

question. Good

just in for is a our the our the hope mission really a if make the is, at developed that of make to bluebird things hope one U.S. of reality, end that live Europe patients that’s for reality and example. think for day, is I important not

make this on economic our to from of all and all to able the an will had locations. the there acceleration, we’ve sort we the be global come products and do over of way what is get understand time sure patients a it, basis best to and classes to that and to around we make was geographical sort that -- So, world sure physicians platform that all hope we of

hard. is that Now,

Exactly own the either working that to that that And something conclusions do be So, Southeast East, impact or we the able is collaborate through, might But Africa, various do these understand. to how not treatments have. Asia, trying certainly you is it’s we’re is need accordingly on it’s of it’s how of as like the think and what basis Middle about China, it it’s can sort regions. we’re just it it’s said, that to diseases the there. important to in as appropriately our this are and

the and that’s I it. of over nature So, read exploration. wouldn’t trip the

Chris Shibutani

Great. Thank you very you ASH. seeing at much. Look to forward

Nick Leschly

Thank you.

Operator

you. Thank

Our with Harrison Morgan next from question comes Matthew Stanley.

is open. line Your now

Matthew Harrison

question. workshop hosted, or other Good guess, from why sickle things VOE were obviously ask question that Thanks that like I have sickle I and moving was guys been endpoints you us TCD have sure and opposed transfusions for think And or just watching the discussed to endpoint. taking And an chose there the went FDA alternative there as Great. other of the endpoints. was talk about you the that. aware with could I’m about away cell companies the another Obviously, obviously morning. to like of wanted help the cell well. as maybe you dialogue why or as OE some to

and just of settled you I’m So, on the why curious your with you increase? secondary best why think that’s sort hemoglobin endpoint associate that to

Dave Davidson

Yes. That’s Dave. Matthew, great a question. this is

clinically disease, among we given if you of improves endpoint benefit As phenotypes as we’re endpoints, it certainly objective, are them. magnitude disease, will, hematology the cell simply of meaningful I syndromes, our from have But, of itself. and away based the seeing, classic of prominent innovative primary when it’s being the indeed that you mentioned, reasons choice an cannot sickle VOE event-based think And sickle moved ignore in you we rapidly, the the for it’s the different of cell primary. and can disease see associated classic with from

suffer. any it and marker hard morbidity us would a success up for without what addressing so, pressed to if ended of actually is And hematology be to feel in for as problematic causes altering this achieve space patients had we patients really we’d if

see that’s that hematology it’s And a we’re classic correlated a why the events. so, to endpoints the over rates. secondary with endpoints be read for in And innovative taking the are to can improvement that to out demonstrate have those then, And And events as is from essential. in expect do us. we approach improvement initial collect our There are substantial and endpoint the key associated certainly time we other hematology event data. you’ve rather corroborated quickly, that as seen

certainly evaluate patients who Velocities. You meaningful we subpopulation a risk patients would Doppler and recognized, very endpoint. clinical stroke as of Transcranial promising in mentioned TCD, would elevated was that the be that at clinically future. And the And for instance, have another major for instance,

Operator

next with comes Matthew question BMO Capital Our from Markets. Luchini

line now is open. Your

Matthew Luchini

Good just for taking Thanks the Two for Hi. morning. me. questions.

the patients think enrolled on ongoing as rate if us that VOCs and XXX, think secondarily, the where to And endpoints? was that for things takes effect criteria Thanks. a might the as perhaps higher stand there? modify in mentioned, to give them? help as you in how recognize you the about be we are last the EMA over design I two a years, you are something Said just amplify expect then XXX, curious what First, perhaps with endpoints already like the sense we the targeted little could color discussions the should provide that another about I puts of could the a types and study? and do think of age, the way, I that going we of and you about think which new to are inclusion beyond

Dave Davidson

are you right. Sure. Excellent questions; and precisely

be are following part the As enable in VOEs reduce effect to cohort amendments requiring we demonstrate the expanded patients, are that who to of have a VOEs, XXX to treatment LentiGlobin now the us the history treatment. a C trial, of enrolled going to in

with our we I a dialogue that of future that terms the all on share provide this with EMA, of and In at we’ll date. additional the dialogue point EMA, clarity outcomes the ongoing in can at is an are

Operator

Guggenheim. Schmidt Our question comes next Michael from with

now is Your open. line

Michael Schmidt

then. be with out just the leveraged then, one that had data of Hey, associates Is for VOE a guys. Thanks more I taking some levels HbS the questions. inform topic. any there the to existing my could And frequency the of I had follow-up there on features? already study that VOEs

Dave Davidson

Yes.

of engineered So, physiology clinical the a powerful outcomes anti-sickling hemoglobin many between directly patients. in stroke hemoglobin disease disease. terms know certainly hope, correlation introducing path risk context attenuate makes a that risk interfere we in fundamental the little is cell LentiGlobin in is globin an sickle levels correlating where extrapolating most with we are patients existing with to, we that challenging of of of indeed What can stroke going data events with think and the transfusions I it which is in the levels markedly

where path might contract radically So, hemoglobin quite instance the altering is S taking simply from distinct it’s are and which in the elevating of for be that of of hemoglobin hemoglobin total actually physiology approaches S we elevating up a made levels of disease. strategy

events. So, hemoglobin reason this show the think that’s I essentially to the building and TXXQ-driven primary or we correlation correlation changes really why trial showing are this between

Michael Schmidt

now under there? into to that in there maybe thoughts insights ways and discussions review, latest gears, give your more Europe, is Nick, with payers switching treatment potential your wondering on some question us pricing could you interact maybe and I then, model the obviously approved And is European β-thalassemia is if to filing just the what was Okay. on

Nick Leschly

Yes. unsatisfied my Michael, and question, to going you’re fair wholly with probably answer.

imagine, as and understanding as and what clinical well that’s is, But I engagement an that’s that’s sort So, I as can ongoing you dialogue. team apologize. of last say European on ongoing for focused the can marketing across of access, years. built the first foremost we’ve been Because, couple Europe

access engaged actively very sort approach implications and the first to sure the make we and to Europe on are many each exact in are of foremost can understand countries of we topic that with best of the that what the this the So, system opportunity love the value we’d this perspective to and provide that can at rewarded to from digest to have innovation is the make in a appropriately sure how regard. understand same time patients, for

for determined. and could our love appropriately How can to tracked. sickle and ALB to Those as of [ph] in that’s to cetera. is in be time things go consistent these far we’d as very broad then thal over over extent high sort of get to products that et are of time list. all it sense a fans a performance mechanism. know, pay that’s a cell we’re hopefully payment But, risk and value-based relates the productive that direction think way and think you incredibly as participate also also enables can that sort that We there of sort a share and So, we we get are time for can over powerful a we in because be stakeholders range, are you

I’d to access so, market relates -- equation. those the as open it access we’re the what And say of to side pretty advances

Operator

you. Thank

Our Fargo with Jim next Birchenough Wells question comes from Securities.

Your now is line open.

Yanan Zhu

taking Thanks Jim. in This questions. for is for our Yanan

is And about believe how if like curious individual the composition relates VOE the hemoglobin. mentioning trait, what obviously to So, individual VOE very endpoint? -- Because just I of the has the potentially also trait incidence. it any, few, of

think cell of that cytometry be workshop of or very share an is kinds for flow individuals you half might the sickling not at data any treated cell not flow is expressing you. Thank Could timeline probably a case To related from cells end, is LentiGlobin with trait alternate goal half assay patients. and I also the And us LentiGlobin? assay when obviously FDA that recording your that is the which So, red single question single give cytometry interesting. sickling treatment blood for for a to homogeneous you hemoglobin, those mentioning cell have data? every

Dave Davidson

And questions. Yes. excellent Those are you’re right. absolutely both

largely and being of individuals be high the clinical have, it what achieve ultimate comparable in anti-sickling TXXQ proportion treated driving that globin. of asymptomatic. is outcome TXXQ Our an goal down which to a hopefully with achieving S added a And hemoglobin have of we to course, benefit patients, is sickle with levels trait to is globin anti-cycling

expect I trait would profound So, peripheral suggest the relative it than the correction. context. to was that threw of should advantage you of heterocellular seen perhaps of a see donors And with that -- certainly you such that data would Mohammed And outcome. survival pancellular versus at in Endpoints bluebird, our senior or have Asmal, a SS Certainly, context that β pancellular the be to normal in kind see some be striving amplified And chain we pancellular as to would cells more the we that FDA-ASH that potent red the corrections alluded for unlikely perhaps on type to wild that. hope Dr. or impact effect comment what Can’t blood on that work particularly recent we from here red literature, done existing did I having at agree there’s in sure hope blood cells there, in is to a cells the following on S for physicians that we extent pancellular distribution, we to red our the in data exciting on develop we’ve one near be do share we comment an treatment. TXXQ would fact therapeutic present able to interrogate symposium are help assays because allogeneic transplant, you And not I’m that normal from are and the we to exactly today. to what either future. that LentiGlobin

Operator

with Our Barclays. from next Wang question comes Gena

line open. Your now is

Gena Wang

C for events the as patients. enrolled, in know at do update, will ask I these EHA expansion you vaso-occlusive you also, vaso-occlusive sickle just I patients cohort, you. XX events number mind? cell. [technical last the a information have prior Group difficulty] Thank for question for had also And on of

Dave Davidson

Gena. Hi, Yes.

into And patients asking yes. if vaso-occlusive heard we the C the answer would at frequency I ASH. is events in on think that the the I question share of first insights cohort

of on the terms the inclusion that explicit exclusion the share in In criteria, will details future. we

Gena Wang

Okay.

the more [technical difficulty] So, at color ASH on OE events? see the we with will

Dave Davidson

sorry, Gena. One second. I’m

second Here, question. maybe And can on then, you Mohammed just question. follow-up last wanted to the restate your

Dr. Mohammed Asmal

Yes.

you in data episode A effect at our even have there initial patients last of this we on communication. from yes, But, previously mentioned an on patients even the expression were that and TXXQ. showed the dropped of in the VOE in just data presented that modest treatment and words relatively So, these a indeed couple EHA Group out with some are LentiGlobin ASH

they data, that the studies as look of after patients well your getting I what on now, question? think history into our both will certainly had be presenting LentiGlobin. have treated answer episodes like we that Does with vaso-occlusive coming

Gena Wang

TXXQ very order much specific question That’s if accelerated Yes. is, meet have But, FDA wondering threshold you And cannot for and expressed in to I helpful. approval? specifics. for just comment the related total you too any know hemoglobin

Dave Davidson

share time. Yes. information That’s not can we this at

Gena Wang

Okay.

Just lastly, very quick question for Philip.

initial data isn’t you have to XXX bbXXXX. difficulty] dose the But, cells the the safety bbXXXXX, [technical For high, million issues. compared some think I

explain there and us Just you differences? mechanism wondering you if can understand can help any if the

Philip Gregory

Yes.

itself if I’d one will little CRS encephalopathy that of be At that we sure but patients, as from And the subjects. obviously have dose, Grade out say that’s a the is and patients X Again, XXXX number said we burden of can you on XXXX XXX at detectable and that as months out I small first, patient, T we’ve the the I squint say X Grade what that well. at you to active CAR think bit an relatively not numbers more at can that with improvement squint of guess, -- with dose, we small call, patients cells. if the I to numbers, XXX than months that’s could over inappropriate. of and stage absolutely to more So, something X seen, I’m a be with high we the XXXXX see want X X you given this tumor have X extrapolate,

the these of increases the right So, see I components, adding way by is stage, think seven do this thing. right the phrase certainly we’re sort we and I XXXX on to at I’d broken durability whether track the mean it haven’t

Operator

from comes Nash Our Robinson next Humphrey. question with Edward SunTrust

now Your is line open.

Unidentified Analyst

on [ph] up morning. to This Good Edward Thank Nash. on Hey. questions. for bbXXXXX. for follow I just my wanted Frank is you taking

vector how detectable amongst CAR, than You level? think Can talk just CAR [ph] it’s just vector copy mentioned patients dose have I you that from patients will better the many bbXXXX. at copy like had us two to that a senior detectable this for bbXXXX data the

Philip Gregory

of cells XX% I detectable the treated so to-date So, T think is of patients with about out six XXXX data CAR have months, presented about the with half. that’s -- been

Unidentified Analyst

one just BCLXXA. question And

Group it have think remind about so, increase? can it’s positive? us over also that sales you’re you sale are and trending Do HGB-XXX of And comparing C TXXQ going of the is And what showed regarding you positive. HbF the percentage you XX% time? And to what thinking to

Philip Gregory

advantage increase, on We guess the don’t the a expression the BCLXXA that of to study. program do could a time F you something the but the for. happen. of and that doing over we’re I there’ll study question what’s selective going that’s is clinical hopeful Obviously, be So, reason we’re know

what relating we’re Group drug product so or we’ve positive. the studies, XXX to the the XX% transfused from into of to XX% range cells with XX% least range think and getting peripheral of of then in in in perspective, the and C, that’s TXXQ cells described I at the similar TXXQ of

So, that range. it’s roughly in

Dave Davidson

typically the add year that the to reach and but we steady months could they engrafted the of based I a therapy all beyond LentiGlobin in to that of cells. out is seeing patients before modified see stabilization to six out of thing engraftments. gene cells, But only increases therapeutic, continue our data, different we increase to The on an engraftments is context many steady and when start of state of in a that way could state sort of entirely

of midst Philip’s the conducting experiment. So, in point, we’re to optimistic we’re actually but that

And to share in able so, be we the future. data additional will

Operator

Thank you.

from comes Huang question America Merrill Ying Lynch. of with next Our Bank

now Your is open. line

Unidentified Analyst

much Hi. if is, behalf I may. taking of one surrogate so the Chen my Thank endpoints. [ph] on the Ying. potential And about have couple you questions. is This for my first thinking a

it be So, to right a to the prior a six as think the think BCLXXA And timeframe hemoglobin about on if question Do program. you’re my second that potential patient, cell surrogate nine to started is sickle actually stabilize you between like looking endpoints? could at looks TXXQ months.

this also also the But protein. up increased So, the actually and is BCLXXA started sickle trending of that this for down. I see knockdown all S proposition encouraging S and overall of of going kind the think ratio and -- the kind was of protein protein to and percentage you

you help how is that wondering target? thinking you’re or can approach the Yes, us this about just Just because Thanks. that? with

Dave Davidson

Dave. tackle that. part the I’ll of is first This

leverage to And accelerate like the development accelerated so, endpoints improvements to right. VOEs to in you’re of see We sort to hope relative be events classic absolutely the would we hemoglobin able increases serve to And and reasonable we really to months dependent. X should window. it’s meaningful consistent X at be might as program. out data that see clinically going very a But

evolves, to the the Philip we -- maybe progresses. second study I’m data see need how the not So, question. caught

Philip Gregory

Yes.

the So, last to still is XX speculate all after the red or data blood, the on program. since days early cell bit There is little adult blood still had I transfused answer with think This that’s to is respect just so the days in it’s abstracts in we’ve -- onboard. BCLXXA the XX transfusion. about

is And S certainly by seeing percentage which so, high explainable not certainly the reasonable we’re transponder I a think the it’s levels, the thing is still moving I seeing we’re of cells, important that alone. think around. But,

is So mechanistically time. nets is encouraging. require sort going -- of I to out think it’s When very this

guess come ASH. I So,

Unidentified Analyst

Okay. possible question to of is, my data second And But, on consider Can Thank follow when think even reasonable are you. we that X quick a So it is have you up, that X in get the estimate. bbXXXX? expresser BCMA I before to durability low threshold? you can please? months a meet that going the the certain update a the the of when you is

Dave Davidson

Yes.

point we we certainly we And of If to everything and will that want don��t about be that. data-driven you development willingness signal about with sickle alluded a clinically meaningful make correct and that’s sort cell the disease be and authorities earlier supports we innovative the the programs, to of flexible. to is appropriate, we feel heard regulators regulatory really I will see very this you So, data are clear decision. But, that that consistent speculate and a efficacy emerging. at

pivotal In terms XXXX, bear at see in of of when in mind, data it’s point. course on this we trail might

data we’re stage or of so, trickling sort where meaningful. updates beyond the appropriate are And

present, and a will that have when more public. we Celgene, substantial we to dataset make new so, And

Nick Leschly

of -- to explore sure about we the how emphasis I’m early know Yes. do we’ve treatment. I right XXXX as think now lines And in you know is

cetera. So, line, second to with now stating et line, already in third

to say that’s that shares I’d on And I’m confident based we’ve perspective. So, focus date. seen Celgene of that more that that the data the

So, on we’re going here. a little short time

jump question. next So, have we think I to the to may

Operator

Oppenheimer. Our next Mark question from Breidenbach with comes

is Your now line open.

Mark Breidenbach

And defining I the to use Maybe CRB-XXX for a Phil you’re us more this of letting dosing? stratified first. little sneak little low just the and Good a necessary in trial versus you morning. curious on give color thanks more tumor couple how bit in for of one trial, high -- wasn’t burden am burden? why CRB-XXX can questions. tumor me the In a

Philip Gregory

Yes.

maybe on stuff, So, can the that. tumor maybe burden Dave take

Dave Davidson

significant ensures burden. responsibility assessed certainly, I in low high Yes. is safety, conducting our based tumor the we patient saw in ensure have upfront, a previously maximizes, signal mean, patient And primary trials we’re where to safety that and on safety. we this that case always a patients way

so, data was from the we’ve seen. program, And that And make. as continuing are, heard with the Philip, on at It a to proceed prudent was DLT. point modification to we this a based you single

do context. ensures that able we a are that in again way doing And We continue so it appropriately. to in we’re

Mark Breidenbach

from registration today. the far given one you necessary of CALD Can give results for I heard high all Starbeam? seen another before program sense you’ve for Lenti-D, the don’t -quality think is new about a us a maybe Dave. so why So, for the at we’ve trial

Dave Davidson

which you you’re conditioning favorite approaches. different in options that’s different conditioning, may the different of which own centers, their trial think trial is community, for transplanters, the XXX a I explore alluding as regions know, to transplant to additional going

provide trying use to will Lenti-D. we’re and of of around inform ability the to practices maximize treatment so that to And sort ensure world clinicians that the data we the

Philip Gregory

question for XXX statements leverage. historical clarity, on trials the answer just And times to registration. going for we think for and that was gate our we’re using based it’s were anticipated is that The a no our I you’re whether about

Mark Breidenbach

quiet we And going at trial an now? for that just from or enrollment Understood. finally, that regarding update they’re get progress anticipating will are you is Celgene something KarMMa to keep ASH

Nick Leschly

do. not Celgene really may or comment may what can’t on We

Operator

you. Thank

Chen question Vincent Bernstein. Our with next from comes

is open. now line Your

Vincent Chen

presented initial how a yesterday this. I of CAR partner see On T including a they you on Celgene’s data and Can to JCARHXXX there? program. to from data have the out T I We ask chance then, CAR abstracts follow-up? And had moment. compares it range released I thoughts for a your saw hadn’t your for programs a other the

Nick Leschly

I Nick. little -- can more. Philip, is don’t a mean, This I think bit you comment

number of I et XXXX we something certainly bit seen, that’s the quite What is superior data think, now right clearly in all anything, there than that of a cetera, a say is the would there’s range one we’ve them. and that what programs of certainly out have sort XXXX. especially really seen use on that of that make earlier data to since I’d

we’ve emerge emphasis ago as our to mentioned just few remains, earlier the Celgene is minutes with that profile along XXXX efficacy So, drive on actually I the based a into and are safety that other seen, But, out if Philip, still starting not options good any which the to than additional… we there. certainly think as is any have if better of you

Philip Gregory

I Nothing. with totally And that. agree

Vincent Chen

might guess, And how switching trend the wanted S to you BCLXXA of goes I the as patient hemoglobin further. to back Sounds revisit good. sort I gears of thinking are program, sort

what term. I sort sort what up quite I gap through getting β-like impressive. that’s S there days you walk can hemoglobin in discrepancy recall of you this though, expect hemoglobin ultimately correctly but or nearly are patients the you gets Should much level, be longer just I us guess, what could between guess meaningful misremembering sort forth? should achieve it XX% levels get something so, expect and to So, you so alluded after to, of fraction preclinically of [indiscernible] and in of and these And go be we due patients as some should range. in should If the like there? early And preclinically, to the actually I sort Help what higher are if get magnitude and XX% of likely XX%, factors likely gaps it’s we further. globin, you

Philip Gregory

Yes.

the your I TXXQ, go. Dave as cells. of of a And expect of lifespan said, a sort a is that it’s exciting. is advantage express question speculate mentioned too very sufficient the There and for really data same things you basis really. to on answered should early. per extend to be to we early on I That those where will So, HbF. to own mean, And cell cells HbF amount think selective the

going that for have cells a the should in case And How time. lasting that’s stabilize approach cell take LentiGlobin. long that so, obviously of longer percentage BCLXXA to drive to is a S new over to versus to higher

wait to and see. So, have we just

Operator

Thank you.

comes next William Our Blair. with from Raju question Prasad

is line now open. Your

Raju Prasad

for the Thanks taking question.

Just a couple questions. of clarifying

Regarding [indiscernible] the of to you regarding total with endpoint there think you and agency the may hemoglobin when that upward other there And regulatory or be one is that discuss will important Is submitting hemoglobin? cap more authorities? the XXX.

Dave Davidson

Right.

composite endpoint. So, it’s a

have of works who think one But hemoglobin, sickle endogenous in way of achieve discussed as production, region. no, do have data determined total regulatory have sickle Because we we benefit to So, virtue for and cell are the control course trait. try disease. to we’ve the not course know interesting past, the And to sickle be the limit of versus as will to terms an components very underlying you emerge? careful to mean to each as They by hemoglobin analogous essential them might over do sequelae patients cell that TXXQ of an generally disease, pathology, other and the to issue, upward meaningful. itself not normal levels the In again red in need the with individuals vector time the case of would predict not β-globin expect associated we of locus point. overtransfuse to contains element hyperviscosity. How with reverse that that’s be I the individually cell suffer be the of

to be feedback produced. So, expect much is of we how TXXQ regulation do there

hemoglobin. expect question? overshoot don’t your sort see dramatic total Does a So, to we that of answer really in

Raju Prasad

great. on And XXX. a It’s clarifying Yes. one then, just

a is of that is the just maybe on that used using, launch that for global catering dealing programs of extrapolated when or Regarding that for regimens tolerability patients, different and you’re based issue conditioning of example? that could other more additional with be younger or more you’re product a potential markets

Dave Davidson

Yes.

a preferences. for for may provide in give alternatives and transplant which gives conditioning, sense that mean in it it be in think improvements -- I it’s transplant specific both, more institutional going and guidelines policies consistent to physicians for settings I with safety patients,

for improving the patient improving optionality and treaters. about So, outcomes it’s all

Operator

questions. would call the Thank over further I not this At to for you. any time, now closing to Nick am remarks. I like showing any back turn Leschly

Nick Leschly

No.

us step time just seeing everybody the think, to we I I’ll appreciate let I forward, this everyone look taking to forward go. at hear and ASH.

Operator

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

bluebird bio (BLUE) 2 Nov 182018 Q3 Earnings call transcript