Gamida Cell (GMDAQ) 13 Nov 192019 Q3 Earnings call transcript

Ladies and gentlemen, thank you for standing by. And welcome to the Gamida Cell Third Quarter 2019 Financial Results Conference Call. My name is Sherin and I’ll be your operator for today’s call. At this time, all participants are in listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. advised request. call be at being Please Cell’s Gamida Instructions] that is this recorded [Operator

would to Relations I Madden, Please Now like ahead. Vice Ms. for go President Jaren today, of Investor and Communications. introduce Corporate the host

Welcome the Thank during today’s XXXX. third update the quarter which afternoon our review an call, company provide of to and you, will financial good Sherin on and everyone. we results for

Executive and Officer, Lodie, Here Lankry, Ronit Chief today, Adams, we Tracey Officer have Medical Officer. Chief Financial Chief Scientific Shai Julian Chief Officer, Simantov,

our we Q&A. Following remarks, the will call open for up

afternoon We the also issued summarizing on a our is which results press our across release progress www.gamida-cell.com. this and available website financial company,

the operational and cash During and make future statements this expenses and our plans projected we our operating about forward-looking may product candidates, potential and therapeutic of development strategies our plans, call, objectives, runway. expectations including clinical

over actual Our number and what other project with XX-F And XXXX caution filed the a today on not Gamida differ I'd only whether to February to in statements our that described Form risk time due factors, forward-looking considerations XX, from results the and the Julian. submit now filings turn result of SEC represent to may you SEC with the a of call will section factors important update events, as may we we otherwise. of our like of future materially that from we them including or These in today future, views time. as the information, the new to

cures patients of through hematologic this afternoon. rare and the taking everyone with committed us blood Jaren therapies. next diseases At to join and cancers we’re you, Thank to cell development to for thanks Gamida generation the for Cell, finding serious time

in natural or malignancies is patients continued transplant. development development option and solid NK programs. important our is expanded make candidate and a We’ve X Our could need both across life the a treatment killer potential in GDA-XXX, omidubicel marrow tumors. Behind hematologic Phase and omidubicel both progress for saving immunotherapy with offer product of candidate most advanced bone to cell in

potential omidubicel. has do the first curative the not bone orphan in is in transplant eligible This received breakthrough estimated than find transplant drug marrow from and the for FDA therapy receive match is donor. Europe. to of first of a patients to marrow a designation also more reasons, of U.S. Turning it receive designation bone transplant, the number product the XX% inability a including Despite that the and to

infections, the treatment including Additionally, donor. marrow related transplant to as and has bone and outcomes who limitations disease. match risk not patients the for to have such improve graft these a do potential omidubicel believe We that has risks of host address other versus complications,

Our evaluate international patients to omidubicel hematologic in efficacy and blood safety cord to of malignancies. the multicenter with is risk randomized compared umbilical omidubicel designed standard high

for omidubicel XXXX half We the BLA U.S. complete expect the FDA line Upon we this in enabling enrollment in approval top by with the of data XXXX. read-out positive year, in anticipate submitting second data, XXXX. end of potential first and of the a launch half to the in

omidubicel required for regulatory application filing, to enables a to document our use the the streamline a in common EU. for MAA which will or us We technical authorization work marketing file

we Work bring both to will to We’re sufficient omidubicel the approval. manufacturing patients reliable our own ongoing following Lonza help for at commercial ensure potential is FDA facility, supply. that also have to build advancing key activities and required infrastructure and

to We’re to patients. also comprehensive services to designed working assistance develop patient hospital omidubicel programs and bring

or of initial in an blood investigator reported at and first data Aplastic disorder. Earlier year, the X/X life Phase meeting second graft. of into are stem bone Therapy and consisting patients omidubicel cohort as sponsored severe plus patients three study a marrow this The Cellular we designed is cell underwent threatening rare haploidentical we standalone a all cohort, meeting. which Transplantation evaluate patients Additionally, data transplant, enrolled with that now Anemia, three from in omidubicel the being successfully to showed omidubicel graft. the TCT the Patients evaluating a are

formulation which encouraged number a early proprietary a Emboldened earlier non-Hodgkins study we're in multi-dose with GDA-XXX, approaches. by This our lymphoma, prior to responses designed Phase multicentered reported advance patients Turning functionality multiple a X/X Phase year. were an data with functionality by multiple the technology sponsored this our we’re generated represents in developing to investigator the myeloma. key limitation Allogeneic non-Hodgkins patients complete study next platform, to the cryopreserved enable is clinical X year, enhance therapeutic been lymphoma cells. important of omidubicel because NAM and Both created has to using in preserving and GDA-XXX donor and

report further instrumental transplant in she's I'll and to Chief of several Beth Officer at now Her completed Israel biology more important strategy. Ronit Hematology fellowship action data Simantov, Genzyme obtained GDA-XXX study our the review present has Department development an the and in American to technology regulatory and been postdoctoral detail. Immunology reinforce summer, Last Medical in we will ASH platform. results research BlueRock, Medical we approval transplant for PhD our we platform area and on the our our Annual updated appointed enhances an Tracey underlying Deaconess through has turn a or previously worked December and new at our Hematology over preclinical Syros Scientific and the impact Ronit? session of already Center the and in making on provides who data She the Oncology. Veronika as on immuno-oncology Dr. call the That NAM from a Pathology Tracey will abstracts that on also experience Officer. month, team ASH. Lodie understanding the day, Sanofi mechanism Chief of and X. Next Bachanova, Society our Tracey oral Meeting of GDA-XXX same advancing research programs in and during autoimmunity. NAM at of Monday, in

summarize Thank what you, the provide see Julian abstracts and pleased expect month. next you GDA-XXX meeting everyone the can I'm on the to for today. to update and good at to afternoon joining an ASH call

binding First, properties, are step killer trigger NK of NK is which NK enhanced to natural functional generate inability a cells market transformative anti-tumor of increasingly cellular recognized in -- to that greatly immunotherapy. dependent antibodies back, potent and utility a NK numbers tumor the The killing antibody sufficient a an cytotoxicity as of in potentially cells destruction recognize by the ability the ADCC. being tumor activity however taking a for have or highly therapeutic or cells antibody limitation to been cells, The approach past to cells, cell activated of contract. key cells has by

X XX patients non-Hodgkins by dose. to Minnesota. with ASH patients Dr. Bachanova using University doses multiple of multiple is from potentially overcome in our recommended study all of technology GDA-XXX This patients Veronika to lymphoma three are to myeloma, non-Hodgkins well accepted NAM this includes the seven lymphoma first the of the at The limitation. data safety which and with is for presentation patients, conducted and XX being increasing The We as Phase in-human to study assess with was myeloma. designed abstract determine as submitted

months dose which a was to patients with clinical observe duration also limiting XX in evaluated, with of population pleased toxicities responses. partial patients encouraging disease. with responders important GDA-XXX We that of five was responses particularly to median all it's heavily observed. to response advanced three First, generally response. pretreated a seven partial the complete there note was doses were were activity at well with Among no see and months complete for patients The lymphoma, non-Hodgkins two for and the tolerated

XX showed tumor clinically history shrinkage very suggests patients post four one Richter's patient had and an because with myeloma, a patients response adaptive multiple transformation that immune the GDA-XXX had of continued interesting stable was therapy. One CLL Among complete of response. illicit disease. and it with a This the

already the from the abstract time for the longer-term are as in as to of who enrolled patients include and submission. well follow-up month Next we presentation, reported, since evaluable oral patients expect additional data

underway Julian turn Tracey in non-Hodgkins call review mentioned, the compel abstract. now our to move next clinical for data preclinical and these submissions plan As with enable to IND activities our to I'll lymphoma. study to forward our over are us development an

Thank everyone. and afternoon, good you, Ronit

a understanding of During our NAM technology at poster mechanism the reinforce session ASH, report new action we that data preclinical of our underlying will platform.

findings. that want preserve expansion gene pathway found leading gene engraftment Our the by research included we next compared down the stem to expression the after preservation omidubicel transcription cells NAM in to of and of of analysis that of generation factor Broadly, absence involved proliferation expression I transcriptome grown pathways CDXX them. regulates in three elucidate and highlight up and ex-vivo to key regulates differentiation pathways NAM. to sequencing the cells pathways

nitrogen First, feel of we and suppress in genes involved minimizes expression species oxygen to saw reduced which during differentiation. reactive production expansion stress we that cell the NAM of

Second, environment micro bone of feel of expression the in metalloproteinase NAM the the We cause genes for stimulates responsible decrease this a Matrix marrow. secretion.

Telomerase, observed remain and to more cells we Third, of increase enabling a quiet semi-state. expression in

bone Taken together, preserve omidubicel. previously Phase and we the believe provide observed clinical of cells. reported rationale These the patient mimics marrow outcomes the long-term study for ability of in favorable function environment of and engraftment data engraftment to hypoxic stem NAM scientific of that the the presence a X/X

technology our spend which We building believe to to platform, used focus. could scientific of we understanding any are really be pleased our

action We I'll characterize Shai omidubicel mechanism to produce of the we action expansion during are GDA-XXX. results. With turn conduct our studies and call financial mechanism that, the are to enhance over reveal continuing with beginning to to and to for of similar process

everyone. and Tracey afternoon good Thanks

milestones we our set are will for a have results quarter we We advancements for in financial the the I foundation year, far XXXX. pleased review third so This of multiple made this XXXX. key with afternoon,

our SEC results can You filing. in our find months nine

of assets XXXX, September of and $XX.X XX, for as December of to million cash cash, $XX.X total compared available XXXX. million equivalents sale As we XX, had

proceeds due and same As the million. development to GDA-XXX expenses million programs. quarter period to was from $X.X to for resulting associated $X.X and $X.X following the expenses compared XXXX. increase $X.X $XX.X to of million net revaluation advancements a being a $X.X XXXX, of million for of compared to expenses, income aggregates by reflects liability. well increase publicly XXXX primarily in a increase was services, company. in of in the Israel related Innovation as for XXXX. in XXXX of period in increase loss in same net Finance third as increase compared This the the and million the third third earlier of finance the Research to XXXX. revaluation $X.X reminder, which offering $X.X expenses General for year million, period due $XX.X third Florence million $X.X million offset royalty-bearing non-cash for from income a commercial expenses gross net XXXX the was million, loss the of this quarter other the net the million million quarter with mainly for the professional expenses Authority completed the to quarter administrative including omidubicel traded increase compared of The of $X period activities for same and readiness XXXX. was The in grant the non-cash for we was were Net of same

call one in cash from advance tell to be of than reflecting to for XXXX range that cash the to the million, based expect range. cash into this We runway activities This used operating plan activities fourth end ongoing total ongoing company's in We expect our is and support to With to on will go any continue business activities. we $XX in position the quarter over anticipated additional can turn With I quarter operating Julian. upper the we you will clinical our or funding year, expenditure current development operational million current less the of programs. that, anticipated guidance to XXXX. $XX our to excludes back

bone for we patients. important the for omidubicel, have approved opportunity transplantation Cell. cell FDA executional Shai. year Gamida has Thanks to With been This bring marrow for the to first therapy an

the completing and filing key at BLA bring next reliably enrollment We’re advancing omidubicel cells working readiness launch forefront generation the the our to X on we’re patient NK in of patients. study to immunotherapy. Phase With activities as advancing required GDA-XXX,

a in of several activities enable non-Hodgkins working study on lymphoma multicentered year. We’re GDA-XXX next to

over in place updates a and these progressing our forward look team progress to have strong months. programs committed providing We coming to on the

Now we the open operator? questions, will call for

question first comes Your Instructions] JMP Butler Securities. Jason from [Operator from

Your line is open.

questions. for thanks Hi, the taking

and versus Do that to lymphoma First Thanks. a just may what then that preclinical your ongoing you trigger have you patients the any could should studies? preclinical just adaptive ASH elaborate little an a question GDA-XXX. GDA-XXX additional saw couple in immune or comments that In response. data on of abstract the we any you wondering presentation, speaks expect see if

is I'll take the clinical Ronit. piece. This

So of ASH in the lymphoma duration see of data to abstracts data patients a couple as that's well ASH, more both as you at patients and were And July. for expect presentation the of we those. the in on know longer a submitted as worth

So between incrementally and who now be the additional July information approximately patients valuable presentation. for at will patient available are

In the terms that in the for response, patient GDA-XXX quite there abstract. this response response and after modest that was that that to that was of over patient and period initially the interesting deepen followed we had a month saying adaptive receiving us a and reported time to the that because be patient continued cells, reason think NK one their their an might the

us probably from GDA-XXX mediated now response. by there's adaptive longer continued a GDA-XXX that response Julian? by but So that mediated told T-cell by triggered and no

the for a NK And reason the week. about for is that only cells belief persist that

continued lot looking of scientific tumor occurred. has immunity So continues a and from this that we're to that have some This shrinkage adaptive with be samples of must hypothesis patient we rigor. see mean to and to demonstrate the

just one your to then Can Israel give Thanks. to well on Lonza as expect manufacturing. you supply? be are just this you launch And an as facility do And as quick update Great. at where the facility? omidubicel online follow-up you with us

in Lonza currently facility believe And will is site be a our we Lonza, in under by Israel, pre-existing online thereafter which will followed construction. has later launch facility shortly XXXX. Netherlands. in the the So facility that the come

questions. Great, thanks for the taking

question Renza Your from Gregory Markets. next from RBC Capital comes

Your line is open.

progress thanks Hey, on for Julian taking and congratulations my and team, the question.

you. Thank

omidubicel or potentially the just with making wanted could progress enrollment, a With start point respect on if wrapping to that considerations and there. put year-end, the the you. first the sort to we as certainly or context finer of for mind, we by just timeframe release around that that understanding day the you two the within perhaps the in Julian, Thank you're to just can I’m endpoints as how expectations wondering and fits venue, half the could in that expect data keeping well develop.

over to very question, I'll clear clinical a turn Since that's it Ronit.

thanks and Greg. Julian Thanks

complete enrollment, the that and confident track by year are to end quite about of that, to we So on more complete hope the happens. that will think that we enrollment when

we that In terms of you venue for I and exact timing for the once primary we to endpoint, point you I information think and don't certainly complete, is finer posted. put will be it enrollment able keep have will a today, on

data abstract. have meetings, certain to certain that reportable abstracts appreciate certain to eligible with at clean and we and do to have presented times Please that we be in be an time submit

color commercial just your side the versus how latest on Absolutely and payments, for latest understood, you your subsequent you there outcomes or thank be think thinking you. that towards maybe perhaps and team great? Thank will but you. could the payments And more framework moving baseline either omidubicel, that about provide then just Julian pricing

that pricing. can't case a will to is comment Of of to market can mechanism payers what lot believe transplant to course, a the say arrive had work to is rate But on do reimbursed we that from that to at talking carve-out we centers. I be I price. We offered

Appreciate All you it. very right, guys. thank much,

Thank you.

comes question next BMO. Your from from Luchini Matthew

line Your open. is

is Hi for Matthew. this on Nasha

one quick So me. from

preclinical does of this to you where you? does it, what the inform omidubicel of apply thinking how can strategically it action next. very information help illustrating the think it How in I is for terms mechanism data interesting, about

answer question. that you Tracey, I will let

that about for really really cells. you Tracey. excited our Thank to the of because This comes sure. first the adding data question. elucidating data that is current and does nicotinamide to Group what is RD We're from understanding this Israeli on Sure, the

in and that distinguish So cells NAM our really really saw of is clinical Phase Phase directly we that bone stemness I able environment, CDXX to to and improved X really two-fold. NAM the we it where improved the marrow preserve how think helps is is outcome understand engraftment mimicking are the the X platform We now to believe the study. related us and

working academic of work that hopefully expression this we’re profiling. in to metabolomic the extension So an now profiling look addition, investigators with at gene of is

of that will in and help sets work other will cells, analyzing also can there data So expansion we expanded currently to think said this without for for our we NK from other And there, NK terms out expanded NK us it which to broadly. in phenotype distinguish NAM. and technologies of manufacturing understanding the cells preserve and will of which those the sure, the we're for omidubicel in out cells platform natural I NK addition GDA-XXX with think apply expansion process are NAM compared to this as we addition, improvements expanding cells

days can of maintaining the culture process cells, So and improve of protocol NK expansion and the that manufacturing activity key. we the the and is which the improve while improve the

I So and can where understand we of well. the in understanding is think an and leading the pathways doing overall is what go hitting future just us NAM as what will we existing to help products improve

very Thank you. Okay, thankful.

Thank you.

comes from Your Mark Breidenbach next question Oppenheimer. from

open. is line Your

the our for Hey, NK Thanks on taking for is program. a also this Mark. had questions. Matt couple guys, I on

obviously at other trial and that, but Julian, being of tested plan product first, product and So That's first will Minnesota. guys resemble you protocol any anticipate cryopreserved tweaks I mean exploring ongoing engineered the that's that the enhanced you I my mean potency. a approaches, guys to other kind an could mentioned know University in thinking generation I us multicenter give for next making about the think treatment it's idea to the in the XXX, a any tweaks and second or otherwise of? types persistence variance Editas, you you of Can Thanks. start what you possibly cell of modifications with you of are as collaboration path is have question one

first first so your Yes, question. to

are are and We there to continuing yield donors. for the consistency. Again, optimize process GMP

the can possibly multicenter And that we next so are set initiate study our And then variability, patient so we we specifications to year. examining product. cryopreservation and so patient can a necessary, the is for multi-dose NK

In there that next generation contemplating. NK, terms genetic activities we're modifications for the are of certain

scientific present announced haven't But so when We data what and have a publicly are. do those we'll at data we venue.

And then require require are first of of any like to approaches six doing NK HLAs? the that? question. out going adoptive maybe follow-up seen degree NK to matching of one From donors, you healthy my I've like some matching Will also you be four

for we requirement do any The Currently, using trial believe not is in haploidentical Minnesota donors. matching. there's

half don't haven't And NK the any there's requirement. they're nature X that GvHD any observed So matching the on primitive setting. based believe matched, cells we we but of the in Phase

a is So, think healthy we donor adult any donor. legitimate that allo

Great, congrats progress. the on

Thank you.

[Operator question comes Piper Instructions] from Your Ted next Jaffray. from Tenthoff

open. line Your is

tackle very the questions. about thank much. great much. U.S. a feasibility lots based was or study, file bit mentioned potential Great, Europe just you commercial manufacturing more on thinking yourselves, to next you call, the wanted have could X positive you commercial you from not a that scale but territory Phase the from also year products is I A very I little to you Europe Thank data good get and of think in yourselves? the sense

question. great a it’s Ted

exploring. are definitely We

very to try sites, it's globally. in omidubicel and investigators many European patients we interested bring to as and our to as have possible obligation omidubicel We European feel

But filings ourselves will this So open we some launch up ex-U.S. for it in or partnering enablement through doing for all whether strategic potential an our necessary do point. and the question partner. Europe we at either end or

looking really next to I'm pivotal forward and so then year. study Well, hematology excellent. much. the data Thanks Great,

Ted. Thanks,

& from Your next Messer Co. Needham question comes from Chad

is Your open. line

had Great, you patients. thanks. for and the questions. my taking Good In XX of at evening months thanks durability CR ASH in the abstract, least

significant As do update durability obviously important do you What what this is of think, more with with for amount we question technology? an look is to an durability get you interesting patients think here.

this Ronit. is Ted, Thanks,

They the therapy had within So refractory or to all patients, pretreated, period patients these prior of great of are responses short relapsed their after a all none prior heavily time. therapies. relapsed very

certainly the for itself And disease is will quite So you'll of impactful is the the to this for enough ask seen presentation I in updates XX study what forward think question far months is but CR in with and think patients. and get ASH this us continue we've at want go so I means next indication. of to in no which get, to

one free that, months. the is idelalisib Let we or for kinases, approximately and rates which me survival to PIX duvelisib XX benchmark the is XX monitor add that progression

potentially response of a a administration and think kinase it year makes the a And of we the makes this single certainly PIX that So tolerated, it well setting in treatments. fact significant. ahead treatment it's positioned

sense look and I to Okay, great. action. Thanks that forward the makes updated

no question would time. to at the turn Julian. over to I There's back call further like now this

that the a Thank today. you, on NAM GDA-XXX call providing look and at We hope to everyone We for at ASH forward technology joining to us you of Operator? month. further next update see many time. our on

today’s concludes call. That

disconnect. may You now