BLCM - Earnings call transcript Q4 2017 | Bellicum Pharmaceuticals, Inc

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Operator

Good afternoon, ladies and gentlemen. Thank you for standing by. Welcome to the Bellicum Pharmaceuticals Fourth Quarter and Year End 2017 Earnings Conference Call. At this time, all participants are in a listen-only mode. [Operator Instructions] As a reminder, this conference will be recorded and available for replay. to host like Chief Alan now Officer. Financial would introduce today, your Musso, I ahead. go Please

Alan Musso

and Tim. you, joining today Bellicum's Officer. Bill With for everyone Chief new the me Officer, Medical President Grossman, Fair, our Rick Thank is afternoon call. Good and Executive and Chief thank you

quarter We for afternoon, investors year-ended be section and and on will of on the Bellicum live our bellicum.com. can using which slides this released media results financial webcast fourth be Earlier XXXX. XX, today's viewed in December the

you not would site. Web do page to list, If have or Relations received if company's can like the this you you so release Investor the added on the to be of distribution

our I'll to on filed Rick. XX-K Form report for any turn of future Private those forward-looking our light a over Securities statements and these of ended And annual approval Securities to statements new statements to forward-looking of review information to Reform numerous XXXX, differ risks of due statements reminder, including involve and regulatory with the discussed plans or of no undertake research publicly of XXXX forward-looking Factors Litigation of December conference As include only and revise a year forward-looking as the indicated those results of the today's Act financial regarding events. plans, section will filings, opinions regarding the obligation XX, in or uncertainties call release clinical development may revision results outlook. these the our Actual in the and call. We trials, statements Exchange under Risk the Commission. our from by including number call factors, and now this date These made reflect

Rick Fair

us joining Alan. for afternoon, Thanks everyone. Thanks, today. Good

a he Dr. February Grossman, He his to Bill Genentech also group trained in transplantation. Alan, on development career immunotherapy. where Bellicum spent pleased the global cell their stem Chief in joined am role on from William in and Medical leadership our addition clinical pediatrics, call today played be focused I to and joined by Officer. academic cancer In

terrific will and background some at hit reviewing during doing are ongoing results has he available running today the and from a for Q&A what has be ground Bellicum. the be he So we session. He studies

progress the made Over three We past moved cell programs vision delivering CAR-T TCR year controllable and we cures our substantial through of towards clinical new trials. into therapy.

XXXX. initiating BPX-XXX validate our CAR-T platform on for filings to switch We and control, including MAA of the dual switch cells for We continued in molecular enhance the European plans track progressed in development XXXX. clinical trials first our for of are first and

each plays manage cell T all safety used BPX-XXX important extend an strengthened we will leader that product potential number to is expand new into report how Lastly, on our first its on will BPX-XXX. collaboration, be in But switch clinical impact therapies, to organization team. report hires I the interim Each how treatments right the progress would serve strategy fits data projects our cell highlight BPX-XXX and they the in treatment. of to on to of toxicities on therapies. we these our can own of our each to validating leadership make be programs a our CAR-T we our therapeutic and expanding CaspaCIDe role the but in in platform BPX-XXX, first our made cancer window like controllable a clinical of evaluate of CDXX and key programs for the also promising and our some our with and summarize Today, can an case

Our MyDXX/CDXX our activation call what switch, inducible iMC. of clinical evaluation we BPX-XXX provide will program the first

driver the T CAR-T performance both gas and the have brake projects enhance both to allow us technologies pedal construct. will efficacy. persistence our to study generation hopefully same enhance these and ongoing designed Our cell use optimize we switch have In and To of of incorporated CAR-T metaphor, the use a expansion iMC we cells. automotive to into we next how an

haploidentical without match completed to European have cell top enrollment with children transplant BPX-XXX MUD will improve excited have shortly background, in in partial little that marketing year. expect line look unrelated And year, treatment deeper to the arm from BPX-XXX. filing generating patients about to CAR-T would to in are you data potential a and for into of trial results most applications continue recently study each expect their starting expect to receiving for of progress our I first in approvals outcomes We donor Comparative forward non-malignant summarize EU these support the rimiducid validating BPX-XXX We preview technologies in of the programs, coming stem hematopoietic matched to children cells we Clinical next at progress the and XXXX. a undergoing XXXX. switch with clinical BP-XXX experience and advanced thereafter. for transplant. C-XXX of our our date in clinical take diseases, the malignant a BPX-XXX like candidate, of the end With treatment to of and what we authorization made dual and follow as the observational or results strong provide

We events, neurologic cover update response our patients management agency proposed known This I risks cell stem for full response for in the the week. to are U.S., hold. clinical the to changes on quick allogeneic monitoring a protocols undergoing guidelines in transplant. plans included adverse which last and our submitted of Before FDA a with BPX-XXX comprehensive just

later to encouraging impressive look the patients that BP-XXX primary confirm for forward and the PIDs. with these the clinical the data into conditions that removal are and We changes announced belief to an survival strong satisfy today for we patients Bill our weeks. interim call. in of advance data significant commercial optimistic with and important study in or AML these on hold the opportunity. The BPX-XXX from a from the from hearing that data. BP-XXX agency Based insight the is pediatric Earlier in will will be immunodeficiencies share and continue represents and takeaway more results detail coming

We protocols studies. the to transplant regulators marrow the gained insight Lake for potential review are with coming adult FDA in and bone at from finalized for working protocols recently the meetings develop Salt pediatric in registration valuable as boards with opinion our months. convened we We City, and investigators and advisory leaders Tandem

PIDs the basis in global AML evaluating AML the we reminder, a for serve are support As to filings randomized pediatric to are U.S. as planning registration study in in either adults or and with a studies

be at provide trial designs mid-year will and more to We able timelines. specific estimated guidance on

our CAR-T to program. Turning

afternoon's that we of use press first this of You may have the reported our iMC activation seen case release the humans. switch in in

to robust research, of cell saw the rimiducid As observed preclinical with in expansion iMC. CAR-T our administration activate we

While treat of intended set this early in talk additional solid to proof data, encouraging case highly the a vivo more continue it's that on Bill very as about is our tumor sign patients. the will working solidifying is obviously and patient. shortly. concept as sights enroll are we in here novel technology From

meeting cancers treatments. is for adults medical or We later one expect recall findings nonresectable cell other in in with expressing as you more T therapies report PSCA initial cancer, of may The this challenging to study, pancreatic year. the

cancers this expand PSCA to the other expect trial We later year. expressing to

sites our clinical BPX-XXX, affinity to receptor we working for to incorporating As AML MDS, rate to cell enrollment. is recruit T study targeting team X Phase CaspaCIDe, more add with the of with high our and continue PRAME patients our and increase

at medical findings expect We Phase meeting a to BPX-XXX year. report preliminary this later for X

While managing of we we and just the switch acute potential toxicities positive our CAR-T previously TCR BPX-XXX beginning from therapies. in on are evaluate have to CaspaCIDe impact its reported data safety studies,

have being future leading a enabled patients first CaspaCIDe treated hospital. better The profile a European pediatric X programs. which the characterize for and help in center CAR-T a trial We Phase results conducted look research of to CAR-T at been the us CD-XX forward CaspaCIDe may of OPDG,

XXX more the turn to to data. Now the you I over will give on XXX call color and Bill

William Grossman

Rick. Thanks,

by of to and am and be been oncology. how would has start excited just I like at here saying transplantation whole First career immunotherapy focused areas off the Bellicum. My to I in

to team. the am that share be briefly here my the some in decade to controllable with TCR on cellular mentioned the I would in deficiencies generation to had cusp and I cancers. hopefully past Bellicum over of curative bringing stem his the With therapies academics next of review. thrilled threatening very the cell like the transplantation focused Rick transformative in therapies biopharmaceutical and believe emerging industry, data at in on and innovative both space, I and During is patients immune time with and CAR-T, the of now that, you driving life I

pediatric demonstrate morning, data First, two of graphs or their two-thirds relapse analyses here clinical cutoff for survival may included patients analyses post-chemotherapy The study. failure. shown cohort release the which transplant and cohort our was CRX from [indiscernible] our our of you AML this of in XX The in line press seen both in of this free a of estimate these year. overall second nearly top of patients, in AML remission interim BP-XXX XX have January of recent outcomes

in relative one the estimate estimate appear up the see, year. to survival median of undergoing As of with survival XX.X% alternative you and can the for literature year results a pediatric is rates patients donor XX.X% one XX%-XX%. overall approximately free favorable reported transplants relapse is follow AML These

[indiscernible] with cohort rare which PIDs including immunodeficiencies. with immunodeficiencies wide We disease, many range line syndrome, severe and primary other our outcomes also [indiscernible], combined chronic [indiscernible] top of immunodeficiency a present had

survival am that strong and As cells matched PID newborn of free transplants median up my pediatric shown XX much the SCIDs in follow in of infusion followed T investigation. the also see I demonstrated one here, We overall developing program further don’t estimate statewide a XX.X% to excited year. part have of latter believe patients Having XX.X% with this screening donor. haploidentical career of these for estimate by Wisconsin, an survival spend personally first academic cohort very patients these disease results results a undergoing and in warrant are identical with PID BPX-XXX of

Over and as critical help which the years, for first as Puerto step very patients well is last to a states screening adopted the program ten obviously SCIDs, these XX of have earlier. infants identify XX a new Rico

a which or makes realty to match will outcomes these matched could of imperative approaches donor toxicities. related XX% related with have transplant BPX-XXX unrelated provide only minimizing alternative as us haplo-transplantation This donor. to those such while with infants develop similar associated transplants, However, cells,

our to initiated an presents Slide study overview patients We BPX-XXX Phase Turning cells. dosing with trial design. BP-XXX nonresectable rimiducid in our therapies. X study of is of This BPX-XXX. a pancreatic BP-XXX is in standard recently GoCAR-T failed clinical

the in the rimiducid we were cohort by in dose limiting reminder, the lead to dose no a patients without themselves. safety As cohort required with BPX-XXX evaluate this completed toxicity. of to phase first cells or sequentially observed X We

the The escalating We rimiducid [ph] peripheral the safety combining cell I evaluation next BPX-XXX doses vector on of are phase of [MTU] BPX-XXX cell single the analyses shows potentially or dose be with presence copy escalating, post of X a day Slide now infusion. for cells. number

first and BPX-XXX patients of level demonstrated peak are row, As in blue our XXX by occurred low of alone the [indiscernible] arrow. three infusions post-infusion. the day in cells around Cell XXXX zero one levels cells. vector top week copies The noted to had detectable

after peripheral patient detection to again rimiducid with the first can at of Slide, the see level cells you Moving bottom of initial the BPX-XXX our low infusion. dosed

of following we week trial a These to observe plans This proof followed later copy orange this the early on for patient by results nearly expand in safety continued demonstrate of to be seven clinical activity. the other However, expressing day iMC vector to rimiducid XX-fold noted arrow, numbers cancer one increase a switch and year. concept the activation after dosing. dose as single approximately PSCA rimiducid potential an with for types underway

solid preliminary, hand could to is while an So that, to achieve and a be back in CAR-T rimiducid And BPX-XXX results cell very very you, these efficacy to dose Rick? high of pharmacodynamic like would step cell expansion important I such single tumors. seeing of are with encouraging just levels for

Rick Fair

Thanks, Bill.

We potential We active to current the several in can novel Turning the company are have cells I promise able technologies therapy we by acute to into then of development that in optimize cell preclinical they discussion leading scientific dual best and believe today, T that switch product the finally last areas and to confident a can vivo. preclinical continue for platform see therapeutic first switch and reported we to see dual our we and clinic I we ability XXXX. our this control year therapeutics. combination that activate expanded conferences occur. a the cells, be we believe to than platform just am organization. enhance the as platform data over when two of an at it. industry first apply create half in more I been our to that exciting the I the with of efficacy powerful right exactly on them control cellular needed to on enormous do be To candidates technology and two also has toxicities now and in XXXX. switch dual the to where move ever administer areas path. when our manage intend in class many the We for have have

strengthened a broad on capitalize organization them. of have our have and to We opportunities set

we we talent and Ed our to led vector seasoned who completion the manufacturing promoted has of teams appointed months Harrigan to of quality key clinical to last facility directors. out added our addition the of of in made Operations our In and leadership industry Technical to Bill and Alan President Executive and recent in cell a Grossman, impact Smith, recently board Houston. have Vice viral big has build year executive

our has opportunity and as CAR-T who positioned been has we Business to of renewing turn past I to strategy Chief that, our productive last highly am our months With business Alan Greg forge a with platform progress the the place to of year at well and capabilities, lot of potential our the we strategies financial the I The integrated capitalize Finally, XX our and for contributed team I efforts. of we joined work execute broad product confident proud to Naeve, collaborations Bellicum's believe am sharpening hard has over space on are development ahead. technology. a discussion. it I in will made year and have the the have for over Officer

Alan Musso

$X.X Europe, million facility quarter for to period quarter compared $XX.X XXXX $XX.X expenses $X.X respectively, start-up Bellicum reported were costs, periods charges quarter including million net $X.X during $XX.X during of in $XX.X million $XX.X million to XXXX $XX.X December comparable expenses. Rick. XXXX, loss related BPX-XXX, million of XXXX. for year Thanks, XXXX. periods December the and in due million expenses The included non-cash and administrative year million were ended December $XX.X XX, and XX, development million XX, results costs, in December million ended were primarily in-house other due a for the particularly primarily the for G&A in million and to increased our million million ended the XXXX $XX.X and were increased for XXXX, comparable million in million and $XX.X fourth to of XXXX. General overall and manufacturers of million year in expenses increase costs, and for loss $XX.X and the clinical personnel trial to and respectively, and for share-based consulting Research the comparable net and our XXXX fourth XXXX. administrative facility costs XX, contract compensation and in an XXXX personnel costs. for a $XX.X and million compared the respectively in to growth, fourth Higher fourth quarter related The and the and manufacturing comparable period $XX.X our ended increased and compared expenses the and year $X.X

to will over through payments Based million of quarter Hercules of interest-only the the our restricted Bellicum requirements back we a loan first February loan will current with XXXX. fourth quarter XXXX, cash lower rate. totaled that investments to call off until interest $XX facility you, operating And a provided XX, new $XXX.X I be plans, debt December X, balance This resources and cash paid In and sheet, liquidity, $X.X XXXX, of Rick? sufficient additional million. the as Finance. million from XXXX turn Capital on current cash, On Oxford operating in approximately expects the meet

Rick Fair

Thanks, Alan.

and we heard In PSCA you a expect four have to the progress we As and making expand pipeline strong readouts data additional opportunity, programs, plan to expressing XXXX, we strong two operations have plan XXX cancers. today, and place. to team initiate are across across in explore the key new expanding studies, our XXX trial our

back product come we intend you next to to early clinic these milestones, targets switch move indications this dual In summer addition we year. the and into with for candidates will to the the

about forward for you our where progress. excited questions. look open will and keeping the are on now are With we We updated that, headed I to call

Operator

comes Citi. question. with your first line [Operator Instructions] Please Karnauskas of Robyn Our from question proceed the of

Greg Harrison

This Harrison is on Greg for Robyn.

there just partnerships as you potential any the what expectation sort that expansion expecting they sufficient And from on results forward into, that have activation enter T order or be activation have you of cell for persistence would do as could partners as far in what and are to of interest of they As is your do potential partner. from well? you see technology being cells switch expecting discussions to with looking

Rick Fair

partners. of would to expansion Sure. the and are us say that Thanks, Rick. It's persistence interest to Greg. both I both potential and

those think important demonstrated and efficacy to longer able studies features. T cells persist T both cells CAR-T T being I expansion of more causing create have cells so is that with for correlates and

a expansion tumor see so patient. are excited of to this And we early solid data in

Operator

from Jefferies. with of the your question. Amin proceed Please Our of comes question Biren next line

Biren Amin

X, after little cohort bit the there experienced or patient, and events just on tell that adverse any how I expansion if that start doing after is about Can expansion. you were are XX-fold seen one a patient the cell rimiducid. Maybe could just us XXX the a

Rick Fair

it's Biren, Rick. Hi,

early We was I T expansion it's show interest in comment cell share are clinical not our today experience patient. status Obviously, proof terms the think of since going of to have to very to vivo we concept the it's our seen with on in today of and biologic what first that.

We results at in patients and of this will cohort a year. remaining later this clinical patient meeting the the present

Biren Amin

it. Got

And so cohort? the enroll or to you is on that second gone to cohort have continuing

Rick Fair

The cohort continues enroll. to

Biren Amin

Got it.

we what Okay. in just year? Can enrollment and you on at you terms us data PRAME. just where And patient expect then tell later of type are this could

Rick Fair

classic de-escalation trial. about is X single but Sure. comment relapsed that won't and numbers X and little a I It's exact center. say we talking we dose are enrollment will BPX-XXX been AML ongoing. enrollment than patients escalation I a in by slower refractory MDS mind a hoped, keeping on and patient has that at

also about to talking We HLA-A two needs there population and both are be where positive match. PRAME a patient

be So is, engaging the late we look in new number the patients meeting screening are at this a record the total experience, that process patient to sites sites accelerating and of will enrollment and we expand the year. in that hopes will of whatever of on

Operator

the with proceed Guggenheim of comes your question. [ph] Securities. question Please next Our line Tavasoto] of [Daniel from

Unidentified Analyst

of interest I Do CAR-T an you would already And have question increasing cell. an enroll the technology have there or into you an you moving that PSCA to for is idea if an positive? your Thanks. wondering GoCAR which developing be, in an other allogeneic cancer since asset in some interest CARs, are was plan allogeneic allogeneic types BPX-XXX second

Rick Fair

investigators, tumor that’s GU the field. Sure. there Answer as types that interest that believe to finalize we more. expression will on GI I based PSCA. and we and And question, But understanding evaluating the express primarily communicate to our from are we playing your first

area CARs, about today. As have programs that no to to do question allogeneic interest your but specific an we disclose second in

Operator

the of your comes Fargo. question from Please of next Our Jim Wells with question. proceed Birchenough line

Unidentified Analyst

in. data diseases the -- in data [ALL] afternoon. is the and all [Nick] from Jim in representative you the that AML Other presented diseases? are you interested afternoon. just of off PID. those the I data. hemoglobinopathies Congrats today [ph] on It's data understand that this Maybe on XXX starting the Good [ph] and you interim other for are progress those and on presented similar

Rick Fair

Nick. Thanks,

You know types by outcomes vary. patient

know mean you So representative. by I what don’t

I the there think case data previous set would what overall maybe that reports consistent data and our is with would say in are particularly of say AML relapse-free data survival survival the but compelling. I the is overall I

Unidentified Analyst

have correct? XX% of think around Because you Is in reported a Okay. that cancer rate I previously think patient. I relapse

Rick Fair

That’s right.

Unidentified Analyst

you Okay. interest have with changed you clinical your have FDA Has data And in not PID. perhaps for transplant as is sickle the the or of cell know for as strong as discussed thalassemia investigators. then with hold, where this the level beta imperative the they

Rick Fair

have events No, adverse I specific about FDA from concerns don’t hold heard think neurologic clinical we the investigators. or

transplantations. we what I adverse is in these are from are have them that stem commonly think that heard seen events allogeneic cell

Unidentified Analyst

on is XXX, a the from And rimiducid? of would you then activity single duration infusion Okay. what expect of

Rick Fair

to Bill, you want tackle that?

William Grossman

obviously on are collecting in still early we so very data collecting No, are that we data. the

We infused patients haven't soon more are to yet rimiducid. scheduled with be enrolled any that

now we comment don’t So on. other to right just any data have

Unidentified Analyst

you comment Okay. CAR-T patient that terms of And that activity? changes. of biomarker Can the indicative then maybe biomarker occurred changes in on cell in

Rick Fair

Yes.

I look T think are at year. biomarker in expansion. to vivo. later clinical this that stick is going a was iMC we and We first additional Again, forward data to reporting cell showed we today our trigger the that that data to chart what, working meeting

Unidentified Analyst

particular samples Okay. don’t am talking they or multiple on Thanks. the the is are than just or bars if patient from one chart. that a is patient? expansion. noise from then and XX-fold one, this I bars bars, I And reflect more this arrow last about single

William Grossman

the multiple a the on bars represent same seen now That’s analyses single sample. Yes. and patient arrow

So that’s [indiscernible]. the bar

Operator

your next question. of of Thalmann. Please Our from comes with line question Wangzhi Li Ladenberg the proceed

Wangzhi Li

start also I with would BPX-XXX.

encouraging day detection I expansion or you of I check one that. So -- showed you XX, figure like how XX wonder day [ph] [data] mean cells to how the around the I say in wonder only the also let's the something that, you expansion. see very a after

Rick Fair

at that time samples Wangzhi. We question, Yes. point. did weekly test for Good

high evaluated administration in one samples to the hours So We after you weekly then remarkable thereafter. be the XX looks of and first really what figure. rimiducid see

points from later So this see necessarily those what That surprising. we the in not did that can that is particularly on time you infer is cells graph. disappear

We to want our what future patients, and there. monitor theories couple be will comment certainly you that bill, of on in might maybe

Alan Musso

understand seen you So pharmacodynamic that here. have results to we are still the collecting the data

of And potentially environment. the seen we [expedited] so [marginated] [indiscernible] micro potentials that its one is are have here the cells or even though being [ph] tumor

of And so re-dose the without data have continue the answer these that we we can and activation expansion hopefully have biopsies exhaustion. of be cells. a it multiple to fact lot the could obtain parent done Alternatively, of times questions. re-dose will see tumor to have And that we T rimiducid to rimiducid cells these we need in pre-clinical and where ability shown this to

all information go will be collecting So we that as we along.

Wangzhi Li

to [indiscernible] cells just Okay. there is like is showing day XX, I or are [ph] you just there any gone. helpful. [masked] to the results because said you figure the not the or XX the But so on day measured in figure, said, clarify, wanted are all in but That’s clarify they there. day, something, you the in I

Rick Fair

cells from they that tumor could Bill, The marginated so one peripheral the no detectable heard you or longer the theory micro-environment. as is blood be penetrate are in

repeatedly need use Another to we to are gone case rimiducid may generate theory persistence. is which prolonged they in

Wangzhi Li

just And the half is remind it. life maybe of me what Got rimiducid?

Rick Fair

Yes. XX less It's than hours.

Wangzhi Li

Got.

release said XX% a PBX-XXX survival press is In the free alternative What I compared donor for XX.X% you one PBX-XXX, want press to for exactly to to a this and XX.X% Okay. relapse the the morning, overall release And the then donor and helpful. survival early alternate right. for transplant, transplant? XX% clarify is Great. point. That’s

Rick Fair

[ph] to of basket we blood core matched [search] that transplants. unrelated glean haploidentical the refer a donor or papers through we It's

Wangzhi Li

a it's of different types. mixture So

Okay.

Rick Fair

Correct. Correct. literature We at really matched transplant. expansively siblings looked the beyond

Wangzhi Li

beta Got in any is overall survival one alpha, there data pediatric regarding the Was and year relapse-free any transplant data there historical it. AML. that? survival and for TCR lung And like

Rick Fair

haplo beta been alone. alpha T I on that could we don’t None data AML any believe find. depleted with presented has

Wangzhi Li

the trial you Okay. pediatric U.S. trial, When also the in kind would that’s AML Got pivotal, it. mentioned hope the Last then to or pivotal? already be in question the this FDA year U.S. or PID. of initiate AML and that you you adult is is, pivotal front of say

Rick Fair

EMA that FDA in be coming is the FDA the pivotal we be and the plan case in of will In case of the the pediatric adult in the the discussion trial, months. and the on there Our will both engaging, study, to on the chance that to be adequate to ensure That’s registration. those to will a we view them may health on design we trials would year specifically have had why that for more after authorities. they both report designs as talk

Operator

Our next proceed of Please with of SunTrust. the question comes line Pete from question. Lawson your

Peter Lawson

midyear we on or XXX, that a data? Rick, just of year-end expect Kind when could event event?

Rick Fair

say I of would later in the half second the year events.

Peter Lawson

any there. I trial could any long see but approved there we when kind or your know of will is going this kind do sense of Got it. interference around an from it's have out you if CDXX, that you. And then update not how Thank of and CDXX

Rick Fair

hasn’t to recruitment The already Yes. patients. and any we have barrier Italy being trial heard what have multiple so they in enrolled there is been from conducted

I just So enrollment started earliest would the know at say this that. might expect the we since late we year, beginning results XXXX. would of be

Peter Lawson

Perfect. Thank color the you to I details around response And just just and sent appreciated. be hold any hold back you the clinical FDA around, further then could so you that much. would to guess, any

Rick Fair

Sure.

adverse terms the really or in of have I our us investigators FDA clinical occur update these of clarify the the neurologic basically that communicated, we events patients. think hold, to as protocols requested neurotoxicities clinical to the monitoring hold, for previously and management

the in updated has updated types it have active and risk has events. our protocols BPX-XXX these all And the patient team discloses three of inform FDA consents adverse of so brochure -- which these updated the feedback. team submitted investigator protocols. And of to their teams has has we The potential U.S. our The for that

we are in So everything them have We hear the need soon. FDA. hold optimistic we believe lifted FDA like that and from the back that fully wait we responded will feel hearing we order but be the forward to to asked short to look to from so

Peter Lawson

Got any you. of PID. the for you Thank to you. for want regards from got Have sense the And AML, this endpoints PID. what then just as data just FDA finally, AML, is morning going to the

Rick Fair

previous From appropriate. the be outcomes to in survival be transplant have would AML or related And think we Yes. PIDs, type relapse discussions, would I more a of the in in of endpoint model regulatory an like, Europe case think similar that more I acceptable.

the on versus if of So the the endpoint disease, cure outcome shorter each you [ph] the of transplant focused [month] will.

Peter Lawson

do there for enrolling you change the a think would be any And in pivotal? you be would patients

Rick Fair

I see look on Bill that? any would thoughts if he Maybe to has to

William Grossman

other as populations are point be that once will pursuing we this we for designed any considerations that believe likely pivotal At in trial we well the and don’t adults have that patient pediatrics.

those So the discussions with clarify population. patient need authorities have to we to will

Operator

Reni of Instructions] proceed question. [Operator from Raymond next with James. Benjamin of line Our question comes the Please your

Reni Benjamin

been starting on relative XXX. how Congratulations have on Maybe non-malignant progress. of the and remind a XXX the you many versus can you study. off patients give breakdown with recruited Can us patients like the just us malignant

Rick Fair

closed transplant out are terms where And that believe those malignant, we our BPX-XXX have all point a point all also point reported I last we XXX along that patients is total and cells. the We out we to this was cohort recruited are. approximately we At we have their that need of I as patients. filing. that of of trial support those XX% to breakdown maintained screening non-malignant survived patients presentation the longer enrollment, and that’s of to Europe would no I not but But unfortunately to XX% receive enrollment malignant, in the overall lines. the of So believe will enrolling we have in total non-malignant

Reni Benjamin

can And patients how a you as are us evaluable? sense give to many sort of

Rick Fair

No.

Reni Benjamin

Okay. And then just...

Rick Fair

hit think will the communicated evaluable that MUD cohort we and our I need on based number. we a powering XXX patients previously confident that comparison feel minimum the we we to of of

Reni Benjamin

split but can you you how is the then MUD that us data going be. And one us might the [ph] Got what's you it. Can the out and as to certainly regarding after That’s would know sense, and the a prospective give sense data, retrospective give a enrollment I groups [probably] between okay. just come mentioned cohort. the

Rick Fair

Enrollment okay. is going

our are opened have patients. actively sites we recruiting and So

we that a of that And month year, midyear of trial have maybe us around the end course endpoint we a is accrued the hope give with our completely So update. results will would provide six when XXXX. early the

'XX. mix As prospective timeframe far expect accruing pre-specified given the majority and late be will from that of are patients them retrospective we middle but the that don’t a of the as have the of XXXX through we mix, retrospective

Reni Benjamin

to encephalopathy at from just time, then confirm I call. And regulators more just both the reported the the the was submitted U.S., I cases to the want were correctly of there no regulators, same remember to previous information and to switching Okay. regards gears EU if in

it’s [draft] You been guys European feel pretty confident be are as a response -- and that has been reviewed will Europe it [ph] largely by just concerned. regulators

Rick Fair

our can't are share speculate we they possibly has so engage them the that regulators the the have of as FDA we reported additional the to had U.S. they that where lion's far in European Certainly Europe. is view been But to the that focus on responding I received of this. I have of BPX-XXX. of received encephalopathy we request. regulators cases the relevant And and in the can discussions been related all questions. will reported to What have adverse events haven't say any

Reni Benjamin

somewhere Like previous question for incorrectly example base be to is maybe the very That smaller, regarding thinking hematological penetrating would a larger trying Am we the back understand, in a the in was I so final [indiscernible]. originally have of Got of in it. about nice I great. you that of theories know cells? and see of be on one me we for kind there guess that it you tumor sounds Is good give I from to line CAR-T still periphery. could expansion would microenvironment. type be, where cells that so, and the but one this sort cells injected of cells. then going would so would that am see think And sense, the the some I to can us XXX. it just expansion those some I

Rick Fair

to in response on over that that scientifically offer to I will Bill to one let it So turn me you the then Ren raised. points and of speculate comment maybe some

is that CAR-T cell to date. I tumor we it with that accomplished my can robust been would say that trial a what T combination cause cells in excited because data we expansion are solid knowledge CAR-T these think PSCA. I to really hasn’t with shows a about rimiducid And in recognizing

a proof and that’s like and more that robust able the future to important drive trials. expansion in efficacy be we to to concept the So apply of of hopefully we the feel really in ought technology persistence

I comparison be hematologic will might across but speculative. As malignancies, imagine that over it for the turn trial to Bill, I to

William Grossman

Yes. Exactly right.

we as the it we as level get the very potentially doing idea of cells relates more I able the we as will solid. tumor think are expect and versus results now more patients that like data collect be we hemo collecting beginning of we I the of potential more understand PSCA this data the we will here what at enrolled. versus give eventually to think co-relation the is that and being insight think just data expression expansion to to and look from One right expansion other into on

Rick Fair

we repeat study. to to in we this in ought we technology apply takeaway again, hope apply that able vivo data, first The ought cells from etcetera. patient technology T to if induce through that believe have and that dosing you we we the expansion the we have have the etcetera, be it achieve today very is, application of to able a now be platform of to be we on solid to takeaway our will even goal tumor requires that able will you limited But will hope topic,

So a it's really good start.

Reni Benjamin

Yes. I agree. it follow rimiducid? seven did of rimiducid doses, have just daily doses up. one dose one seven of definitely you or And quick maybe Was

William Grossman

This cells cohort for initial single much zero too No. of day the dose a safety again perspective. X. This required was, of rimiducid on is only, so a from

are see sorry. so rimiducid administered you have trial here, we right this a increasing And you can is single our if it's back that dose day of go evaluating on that cohort that dose slides, rimiducid -- we that the still and earlier self will we doses to see two be of increasing there slides patients, design we The more of X? but showed single

Operator

Cantor Our next proceed question. of question line Kim comes Justin the of with your Fitzgerald. Please from

Elemer Piros

math patients. Elemer what if I XX of data you for much is that did the go a this submission. the How into combined, Justin European ask correctly, analysis Piros This Kim. I data reported for to like is would from my would the And

Rick Fair

You good question. are raising a

Europe of for split today. tell are Our the analysis U.S. split duration which are top you population for current the know but my European we I timing of of majority the European plan a off geographic the I includes trial the of safety. think to is the total the I of up the follow to the include them them patients patients enrolled the don’t U.S. European based that off in efficacy on mix head the versus have and don’t recall and in patients And of which these

Elemer Piros

would that good it's a in you include like chunk or cohort. evaluable XXX to of So the the so fairly

survival question the at you is, looked months? So at six rates have event-free

Rick Fair

were present focused we meeting more where free this free disease these the as carefully. get looking relapse In survival. we that on or more this We and will we data, not. we Certainly at overall at to medical analysis look have survival analysis on closer

Operator

the no further over management over audio are like conference for questions to remarks. back would now of to conference. the turn I closing the portion There

Rick Fair

everyone. today's call. thanks, your Well, Appreciate participation on

who to great every and a and their always, I inspire have for participate would effort our effort. most Bellicum investigators patients like in our day. Thanks, evening. families who And team importantly, As collaborators our our thank entire the and clinical trials

Operator

you today's for concludes Thank your participation. conference. This

disconnect at your You a time have and lines day. wonderful of your may this rest

Bellicum Pharmaceuticals (BLCM) 13 Mar 182017 Q4 Earnings call transcript