Earnings call transcript Q4 2022

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Bobby Gaspar

Hello, everyone. I'm Bobby Gaspar, CEO and Co-Founder of Orchard. I'm especially delighted to host our fourth quarter 2022 conference call, where we have a number of important corporate and regulatory updates, along with providing our full financial results.

Before we get started, I want to remind everyone that this presentation contains forward-looking statements. Please refer to this slide and our latest SEC filings for more information.

and we this morning moment call evolution our news will that in in therapy of during gene discuss constitute company believe announced and this we the platform. I HSC our a breakthrough I me today's have call. joining on three the team of members leadership

Commercial more morning, results Following my Chief along Officer, our Parker, summary our financial provide with the Meltzer, President the quarter; financing our Operating Officer, and joining a and remarks, on Chief we for Chief Braden also Frank will will for Thomas, Leslie view Officer; announced this of Q&A. Medical be

Our call. remarks focused plan on this our during topics is to keep X prepared primary

provide to in an commercialization Libmeldy Europe. First, update you on

FDA. cover Second, the recent announced morning the that off review significant this we we with financing for table with capital will foreseeable for will additional the future. need third, take And the the that the to in U.S. developments our interactions

be start making with MLD. for Libmeldy a We've HSC severe always Europe, treating launch believed Let's for patients therapy reality Libmeldy. future disorders. are with we and the the in certain of approval future that neurometabolic And with will gene

we and disease physicians eligible identify, treatment to Our MLD managing teams patients. signs and awareness diagnosing seeing refer support patients and medical commercial is continue growing the and are that of among

In fact, figures we expect will XXXX. be reflected revenue in our for annual

in million only its during XXXX under first launch, in place. just few a with was sales reimbursement and of generated that $XX agreements Libmeldy year in

grow sales for XXXX to screening to in we while in see a implementation newborn the and continue studies, in prepare As the and launch also Europe, we that of optimistic expand other U.S. potential markets XXXX reimbursement Europe in we access are additional will

identified importance the the to newborn after MLD about screening been have date, X frequently And XX,XXX of confirmed globally. cases of newborns spoken screening. We've of

referred the identified other are pending clinically with X has assessment. these for assessed clinical patients and of One recently more cases treatment been Libmeldy, while

talked have MLD about XXX,XXX we X in previously births. that recall live being of the may You incidence

the data be previously incidence pilots are are these that may U.S. the East does additional published. year. consequently starting the suggest actively X screening than this Europe, and While In the total, with and studies commercial still opportunity early, in newborns Middle it higher

U.S. Turning the to

the held As early the BLA including this dialogue have presymptomatic B been and the constructive ongoing clinical FDA, elements pleased Type planned patients. requested in MLD. for are has outcome in confirm, very now the meeting, with a XXXX, which quarter on This for scheduled predictability the FDA the of package is we history of clinical XXXX. course meeting on of meeting by second a clarity OTL-XXX our the to we comparator FDA with to and provided clinical granted Based of data meeting, for submission natural potential the on been key pointed our that we pre-BLA

are also of addressing questions We package. comparability CMC remaining on our in process the

following mid-XXXX Assuming OTL-XXX. a we for the multidisciplinary anticipate meeting, outcome pre-BLA a BLA potential now successful submission

important continued bring partnership patients to very the year, first half with as to of and U.S. the look And to XXXX. with have work in expect a pleased middle believe this review. to launch granted we submission and in our are a We potential are therapy approval the be point we the FDA priority an this of come likely can We this to we forward in to

Frank news, key strategic cusp which of minute. couple about believe now that some is on financing months, during of we inflection and will have we OTL-XXX. a So the other in of This the progress the part to for points way long led come talk a the are past today's

of as progress our of rest to genetic press initiate NODX releases turn preparing MPS Before calls. We'll MPS I and and also to to over Crohn's conference syndrome a talk future our registrational a Sanfilippo Frank, the mention the IIIA want make we study, that programs where to be disease. these Hurler in syndrome, including to are it in about well, portfolio form in programs I I continues position

that, turn Frank. So the to call okay, over with let me

Frank Thomas

the significant fully therapies same drive with organizational infrastructure believe as Thanks We're our of distribution strive in leveraged many clinical for we're capabilities curative setting commercialization and a growth. therapy Libmeldy matures, programs. can platform. as HSC manufacturing, company an XXXX a for lot, near-term Orchard utilize will Bobby. our that developing potential will now that be up commercial I and to stage we our be preclinical is pivotal Libmeldy gene pipeline year And other revenue the the and with potential of to a today. unlock

X focused employees are year months delivering quite first be to and proven our The on the for for company, I have positive the rest the know of also. results

near-term for All group health cleared at financing could for capital Conference Bobby a progress additional attracted our company the future need to patient accelerate strategy. the for IND the negate but investors, and focus through of allow provides an growth and progress and company. screening. WORLD with plan and on that it and that were of data FDA presented the clinical OTL-XXX, financing closings We for that structure to care significant Not very able for interest our ramping only we up the newborn OTL-XXX, business to does the regulatory also described execute have leading financing identification the the had a commercialization, provide

and Libmeldy RA and used a Capital as led company launch potential portfolio us million financing preparing for The total U.S. and vision larger of to a with long-term the premium up and proceeds price. These simultaneously extend be a shares, for approval the elements: $XXX based of million will MPS $XX is expected at alone be indications. and structure X programs XXXX. fund comprised quarter a approval is to could well financing of pre-BLA a additional that while closing our advance second build both research and A second of closely in successful support as next-in-line investment shareholder our to are of transaction meeting There of runway a upfront worked The in occur to is which Friday's $XX anticipated authorized will proceeds sustainable into closing the XXXX. to business. our million XX% on in priced Europe, to to

OTL-XXX structure upon a that potential FDA The shareholders We This progressively priced the existing provide U.S. good partnering for to and our also for $X.XX supporting this per gene warrant final structure element to company warrants advances a and unique at prices. incremental share closing of deal structure valuations all attractive field the to towards It's build investors with exercised potential in We committed for XXXX. approval up approval regulatory innovative MLD thank share. each $XXX are participating want OTL-XXX capital therapy. believe and to with is each as million with in U.S. in would at assumes special of is their of the higher investors participating the the us in

to Total $X.X for in in manufacturing sales goods plus year-to-date $XX.X sales revenue starting sold million This amortization. and royalties million, same XX% our for which the in Cost million quarter $X.X XX% margins from the of of primarily comprises periods. costs, XXXX. was product noncash approximately gross third-parties was period for full revenue fourth Strimvelis Now to and Libmeldy year. resulted from and was in million to collaboration includes $X.X performance, which financial which fourth and the quarter, the million revenue Libmeldy, compared of with quarter turning the to $X

investments the from pulled XXXX worth Beyond we you financial been quarter morning's calling Libmeldy this release. a the can quarterly cash burn in fourth March We year in the revenue reduction rate and against with the results overall the press XX% results costs, quarter first comparing that with restructuring fourth quarter of approximately that our ended full million. in out of has the find It's completed $XXX through the and XXXX. when

excluding quarter, to fund in items. fourth from $XX.X million onetime cash the During used company offsets the operations, about

our for lead expenses, XXXX Libmeldy Our in focus to our operating to and annual trend continued should continuing which a financial rate. goals sales over and downward are grow burn managing XXXX

milestones, therapy launch leader Europe gain I sustainable breakout Libmeldy forward in business believe is Orchard the positioned gene the become upcoming model we on a traction demonstrate to to therapies. Looking and continue in as curative to for well potentially onetime

we commercial for clinical and will also U.S. a launch and various prepare addition development. objectives, and our pipeline a to potential stages in of approval research In of advance candidates

your for with So the to call questions. operator over turn I'll the that,

Operator

from [Operator from Instructions] comes Dae Stifel. your Ha Gon first the line question And of

Dae Gon Ha

Maybe on me. from on two meeting/pre-BLA. the Congrats B the Type all progress. One

the the meeting. and I sticking heading that, CMC there you the the fully be resolved, any before on discuss ironed to So Bobby, more color in know into discussions kind Has to aspect, around been pre-BLA? outlook to pre-BLA kind that that that's leading Type guess, of history maybe been B more you out Or wanted the have is this natural additional you provide aspect? still meeting? the predictability latching what a cohort had need to can point the to of and be aspects more comparability optimistic with I

to get And With first just XX you Frank. you for tranches already XXXX. XX enough the out, regards into question then laid to OTL-XXX, XXX then was XX, the second and

that the And eligible OTL-XXX does cash ask, the to that me already wanted include that monetization? Just the PRV? XXXX, sorry, does remind on runway can include already PRV you program, for is

Bobby Gaspar

Thank you, Gon. Dae

ago, think path as bit, just address meeting. also remember, questions which So months able I ready FDA, this meeting. a our may overall be X because, And they and comment said Type second to review context kind you to some clinical is for FDA about the CMC outlined as just B comparability for in pre-BLA pre-BLA that well. wanted clinical we a I'll they they maybe to take about meeting. back we all the the the weren't that and package. And I'll Leslie of of to step question, discuss to would first the said But talked

we we of aspects, patients. Type points is could pre-BLA regards talked think and resolved FDA to the was about, them, said that so had these validity we we robustness which the that I course presymptomatic clinical you to those the meeting. And clinical a and constructive that arm that natural specific reason And a and and in issues, B with predictability part history the meeting advance the with clinical comparator now where of addressed again, also the the the that of

clinical a BLA the talk the on side, as We're the number as had submission far as the something the that some with about also On CMC with FDA the it's is interaction we've in side can for pre-BLA as questions side. as narrowing of we're the comparability specific CMC where concerned, at also did we dialogues on down FDA meeting we and concerned, them as that well. is during far the to

So that is ongoing.

ready a I thing we been we that saying the now in ready for granted think meeting, meeting request is, main having diary the pre-BLA obviously, that, meeting. has is the the But FDA The and are weren't QX. for said

Frank Thomas

to I PRV financing other with just and the generally look years, a see bring several burn U.S. the eventually about expect So respect we'll the as to market the especially then period, growth globally. and over on burn would mean, think markets we I I to maybe in multiyear Libmeldy next when question. that continue over

from that have will the contribution offset that coming burn. you'll a So revenue

I the I R&D submission on think operating flat come to a we'll OpEx of down start think slightly as to complete would see down expenses expect bit. expenses I the BLA, we the line,

tranche I into overall, the 'XX. like of feel So us gets the financing first

to us, being warrants The the then second tranche third And from 'XX. think the into gets of the beyond. we say, are and would the of capital I if certainly the opportunity runway to extend proceeds profitable through our exercised, all with financing that I then come substantially piece, in well have Libmeldy

think I the financing for proceeds this a the So you important company existing is very plus when cash. combine the

PRV. With respect to the PRV, to the rights not unfortunately, have we do

would they sales we in PRV XXXX licensed upfront, in get from program. little royalties the you MLD and back very downstream money GSK consideration the was recall, but get for would that on they that from this If

So get the essentially, get when will if be PRV, it transferred we to or we GSK.

PRV from monetization assumptions. So included proceeds I've runway any my in not

Operator

Co. line your question from comes & next Hartaj Singh Oppenheimer And the from of

Hartaj Singh

bit questions, Just patients. One is a on couple little quick the a of three just disparate.

in find the to able that higher seems been been You've to the I academic one than quite know XX,XXX work done, be to that that's patients. Bobby. of sort the seems over XXX,XXX be

the you difference presented going any quite the study. on a academic FDA, and much the at analysis there the some what insertional from what there in-depth you out of and just any? of there, were type discussions that on integrated around how integrated on safety see as issues Just both thoughts side, we've were the B meeting seen discussions if with or safety and Just the you analysis that data sort looking in the secondly, do especially And -- forward? specific It's trend then mutagenesis were that?

Bobby Gaspar

Hartaj. you, Thank

screening let So of piece talk first me the about newborn all.

we So I of numerous from, an It's bias X average in XXX,XXX. patients Remember, there's instance think, have in to those XXX,XXX. figures had ranged was analysis. -- analysis, around incidence talking in different come XX,XXX MLD. the missed. retrospective always to a the X up will X And been quoted ascertainment previously those retrospective of that the importantly, analyses an anything And from about I think, retrospective in

incidence in every And child. especially higher so published previously start now is the to capture usually, than where because you diseases, rare these

And newborn do that with through screening. analysis only could prospective so you

And we so started that's to do. what

it's that in first was also X,XXX conducted babies of deidentified there screened a And and add X State. MLD so screening, And that Washington identified. nice study I prospect baby was screened were MLD. genetically the XX,XXX cases there and babies will of was year confirmed been X

that's we've So a screens. prospective seen. study again, And are separate to what these

we think how higher, to does think published exactly picture. to I is may what on to true at But epidemiology. predicted. just it just I point. the baby the But let's difficult screens not my remarks, previously than get that, can full fits incidence say slightly higher was might a that suggest of in prepared point screen I enough said will babies And XXX%. I more to this a time It's does it's and all higher So much as say to incidence it basis that be need something be say give than true probably

even So coming XXX,XXX we'll the planned. and doing pilots keep more this that, than are actually anticipating all that we doing with screens are year

So what true we'll is. that get exactly nearer understanding to figure incidence

And point that never insertional the respect I really overall mutagenesis of integrated about comment a second Leslie the will of analysis case to kind facts. insertional vectors. about the we mutagenesis. I've the had clonal previously, even expansion in for insertional signal there On with that, and we've picture I'll wasn't never that a used reason about or But of the talked molecular the that We anything let have within had lentiviral this is and mutagenesis on program. our comment promoters because

are that we have promoter viral, events vectors believe of using to safety seen is the not have enhanced reason of activate and as remember, that the we far And were promoters that So used insertional activate And genes. a the human can as concerned. neighboring in have strong capacity why do regulatory is that such mutagenesis our we to elements sequences genes. that viral [neighboring] use lentiviral have all programs, the internal the related which profile

Leslie Let other talk aspects... to me hand about it over to

Leslie Meltzer

safety of to points very all been robust regard been it WORLD, deaths. to add -- XXX was treated. anti or we've sort clinical at of not with also very we whole well robust some these engraftment not we with clonal exposure. no There's cumulative outcomes. see but very see evidence on that with And treatment-related about presented tolerated, SAEs the of oncogenesis expansion, associated la OTL-XXX that over to years Yes. safety impact patients the that It's see antibodies, insertional are as consistent all got profile. instances of a very patient, no fact We've in has any to which lot We evidence presented of is no Bobby malignancy, in date, everything or addition made a

all our significantly clinical these meeting outcomes that efficacy is which survival, the well of present results part The see includes some disease. among manifestations the on pre-BLA XX improved movement were the from the patients consistently the meeting. development in treated a on B with other WORLD data on as we Type of at impairment, program next And use discussed of all update presentations clinical that as supported patients clinical the of treated our as stage results and cognitive motor an And to FDA programs. we process, impairment that previous compassionate were, is on again, in

Hartaj Singh

just B Is a of want the Type pre-BLA Bobby, preparing the that around And question. actually efficacy, is would exercise and a discussions that pieces box in-depth use that. safety pre-BLA I think imagine, the mass and that to integrated of and follow-up the BLA meeting. I core to in that I pre-BLA and way was the And the that where data about actual meeting, quick and the the of just to question the of Orchard the into it? sort sense filing other -- term had ask going. the on you the going for lightly that's and B don't sort check the sort was has to going meeting the to of a just Type all getting Is

Bobby Gaspar

think I so.

I think questions to did clinical address. the those generally, especially pre-BLA addressed. gone to that And have they we've a the is clinical previously, questions feel that I that they as I clinical on said think been wanted fact have meeting, the think I front, that And case. we

now And be a so -- lot will of this procedural. it is

may of on is is that be is just pre-BLA for still at they're we that where -- FDA the We are to discussion the comparability the process package. and responding, a that ongoing are with waiting et making that us, And again, that, package. there CMC comments about the meeting. feedback cetera, comparability And a are we

Operator

from Werber question next of from Cowen. comes line the your Yaron And

Brendan Smith

a us. Congrats from one progress. Maybe on on great just This for Yaron. a quick is Brendan

for the the to You filing things a you next filed IIIA, to bit assuming that of IND And which And mentioned you pivotal proceed an able for Crohn's the I sometime MPS Can in would still is if to what relative we a timing remind you if us Or initiate expect after is IIIA year? MPS study still that IND you expected, be IND? a kind expect then program? start MPS pending for not, study. pivotal in flux? that maybe little should as maybe the

Bobby Gaspar

Yes.

those take on IIIA. ask forward just So MPS the let comment let me But for and on I'll first by one, to path all. me I, one comment MPS Leslie of

with And the both beginning having that some the the study so to remember, for going transplant, safety has by been a plan study with composite patients our we for the announced cleared that a to end the registrational allogeneic study year I at cleared, details It's year. the filed registrational of And that around of endpoint and design the study includes the features. and we MPS efficacy FDA randomized for be IND by a that. been XX is approximately against gave pivotal open and

are we of and program, IND the end to the the anticipating are year, of proof-of-concept we aim data, Crohn's announce preclinical XXXX. of on half an On submitting going the in with IND-enabling studies we're first moving forward the by

ask these so on Leslie milestones those IIIA. And are -- But that comment X to for MPS specifically the I'll programs.

Leslie Meltzer

Sure.

that still out X normal patients history natural follow-up X are cognitive in outcomes WORLD, in are So recently study [encore] patients, with And where X the study, we are in see follow-up. patients, presented or development of X-patient out in consistent results the presented the for have year improved IIIA, year early end to X at X we've MPS patient proof-of-concept least shown we is patients. at what relative all that last that in an either we and of at from then of

skills outcomes. see gain of So these we in patients, we clinical encouraging signs some see and constative on

this quite early disease the in However, is still course.

and with of quickly decline around at a If will X. of then A X ages the discussions, of age until point which you recall and go X, patients around normal they'll plateau from some previous period Sanfilippo development the Type to through

these this proof-of-concept if patients for we looking from follow-up things natural of matures matures. we as reach would history. study for age additional the a from expect And regulators additional start deviation this are discussion study data some data designs presentations potential we're program, pivotal as clinical an in as And and a this So with potential where to as working, will study. our see on inform this data

Frank Thomas

to would Brendan, Brendan work. a Frank. to I pivotal we supports we would the -- sorry, if a how period up just develop data anticipate follow question about mean time study on a that could have that that into of need the soon this I it's year CMC your of study. moving some pivotal

we and year. a to don't working position we'd think to can be study next as registrational start get working I we'd a in So in CMC. the quickly be certainly, parallel on But more that as design

Operator

line your comes question And next the of David Hoang from from SMBC.

David Hoang

financing. --on the pulling together on Congrats this

So had question potential a in XXXX. I launch Libmeldy or around of two approval a just and

that in to to just look the have activities could potential is -- cadence need U.S. compared how possible, launch then ongoing may would expectations U.S. market? currently And develop bit how to you you what's that, do the as talk the the U.S. to of guess, a you Assuming prelaunch about in EU? any, what little may pursue I a commercial

Bobby Gaspar

to that over Braden. straight Okay. I'll hand

Braden Parker

to disease state European in prepared to as overall access clinical Libmeldy advance there's somewhat impact of MLD, we payer Thanks patients want well. the educate to in preparation as as sense from centers an with In quite be you process Also disease, education might the for prelaunch would of as for for them on activities treatment imagine, the to side Sure. the have that has well the rare take that the of would payers U.S., the an on of question, the working qualify approval David. a access place mirror identification as of number in activities overall. to a

place will be U.S. that and taking well be identification approval and potential efforts place leading mentioned others, And some then screening the certainly that as other to There taking launch. a there's would activities in are Leslie up newborn patient as

buckets at So building prelaunch a those at we're some in are to of perspective from of activities addition some the the are team to region. cover U.S. the be the looking major the the looking in out that we'll time, Those at right

the XXX,XXX terms of U.S., in estimates In taking roughly at launch, EU, opportunity cadence look the in the in have U.S., the it's to to live kind size X identify. of of births, current anywhere similar that patients if of births at would the XX potential and the looking we new you the again, year you're each of XX between try to find and

So of in but real is difference of it's a access U.S. opportunity see in the similar reimbursement the type terms the sizing the you and where side

a in quickly U.S., lengthy So quite reimbursement followed technology reimbursement in expect by than health we've what Europe. in I Europe, the happen seen would to we see negotiations assessments in more terms much of process

and So a recognize we to than once sooner U.S., to Europe. we lot get the get would approval, we identify able revenue start have be if we in approval in

Operator

from comes of And from Finke question line your next Robert the Guggenheim.

Robert Finke

financing, This is the outcome speak next Debjit. defined you the Robert mega and is meeting the on leg? confidence the outcome pre-BLA of Congrats on how the next in to the a From positive financing. for perspective can for

Frank Thomas

on the to level. can speak the Leslie Yes. Maybe Bobby is of that. comment how and then of And confidence financing I'll to sort sort tied

the yes, in saw deal, the press you structured release, So success-based. as is

more more and the program, business. to for comes So and in derisk fund as the we growth the strategy capital make and progress

a pre-BLA to meeting that milestone and defining is essentially successful tied second what means. the So exactly

as to in not a then minutes long there's precludes It's as to that forward from an financing right. us think come in. obligation, the I that of submission, second a is nothing tranche obligated moving

to then to as that is money the we meeting successful the before as and that scheduled So come expect in, we're second we long meeting, happen, have sometime a midyear. because would expecting in quarter

technical of let that's the but on further itself. the comment Bobby aspects it, I'll meeting sort So of

Bobby Gaspar

Yes.

we're able We've a And what far side with We as had confidence time of years we number measures is agreed far what we X feel past think the we've the in concerned. side. been say in think and and off FDA, the GMFC the our we that, to very FDA on this I designation. I what good talked use, of things we've over and as on pre-BLA as place. the a different RMAT is discussions through to that during as essentially a happens we been -- function. both how like meeting motor on an period, the to such IND, in the think I lot the I'd clinical of look questions do as with filed CMC score that file at

presymptomatic independent the items of an constructive that of FDA, else the We've natural detailed composite on the had these to on the had patients comparator is We've meeting all clinical severe review a is We history natural type were agreed primary the the meaningful. endpoint clinical data, committee the addressed of clinical motor cohort, where be both of clinically review and robustness treated patients, history the needed use and of clear. conversations B developed the course cohort a survival. free called score side, with to what predictability the clinical

So to very given meeting. pre-BLA been and has that forward positive move has us confidence all this of to

we're under answering of we've started say, could to CMC there view of meeting. through pre-BLA review, as again, that again The a proceed was they package CMC come side, written a we as a questions -- is I And needed response comparator a addressed multidisciplinary number On through. started we the questions addressed. be with documentation. only those lot to a We that

think things up through to forward that move with those we're to where meeting. we to us are. And I get having that's a you place that, all so why led where And have But we Obviously, as that stand. meeting and I'm we with we very have transparent good that. of being of that feel kind are in after to the

Operator

the from Pete line comes Fitzgerald. of question next Stavropoulos Cantor the from And

Pete Stavropoulos

is WORLD, the recently used? of had Can it get to you difficult So to just implement? to of hit presentations on a sensitive but MLD genetically How is from patients. is how to screen, newborn identify you they which be patients? How it assay number easy other screening, going specific or for the is it? words, couldn't confirm? In sort important discuss MLD And

Bobby Gaspar

start, be able and to Leslie on Maybe I'll Yes. may this. add detail

So and the is for is an sulfatides levels. looking tandem screen basically -- that mass in is uses a increase used spec

enzyme. sulfatides of accumulate a substrate deficiency are patient ARSA result the And of as so cells in that the

So dried little a that the you in you the on look a that. for and that the -- is sulfatide. do is the eluate, run spec punch blood mass process the And can what can take from you first tandem rise is from measurable. from And can shot spot, you thing the that you

in the with Leslie One, or look analysis. X subsequent one in you using sulfatides could But rise go MLD normal you accumulation natural are to children. to patients, And get kind maybe the threshold is me, itself of define a diagnosis. or And events. you high is tests genetic correct different will a to use this associated have at Now is But of spread. of compared always natural level see And threshold course, can or sulfatide of you which But you to of the time. consider is in both I gold to that above tests. individuals need have pilots that, MLD. After just genetic you standard And to screen. a over that those just can confirm think screening you history, be positive test. actually second-tier a either that but those can fact, whether activity set to

it So to I'll Leslie. over hand

Leslie Meltzer

narrow universal research right. spot that precise these as and are an in point, at or blood there's different in And to that first Pete, Bobby sulfatides make levels. X-tier an the that's I collaboration intended mentioned, that pilots you either methodology will that results down patients with these screening to are share ARSA all do important part Yes, elevated the as worldwide assessment is takes subjects sulfatides, could have shared goals think you of scenario, improvements to would methodological the pilots. the have of ideal is really screening these And either of same share and the testing research how methodology in groups a But the the becomes pilots genetic go place. or a state starts testing in and between country be dry level. of inform or to who And in a or confirmation regular X-tier could directly be conducting that that

start because with directly rate they the don't actually what people a of in have general there's high of population You ARSA call ARSA. pseudo deficiency who

confirmatory you screen to X the you first where roughly incidence of dramatically positives to samples XX,XXX Washington which and the to state in XX,XXX potential you X for lot point being testing in If ARSA, referred de-identified genetics, the a by the first ARSA, of get in for false sulfatides, idea then for can But to you showed. false just is positives. what pilot is for were reduce get

are specific worked online. the details over speak, pilots that course being those the anticipate are methodology pilots of alignment coming of out would improvement this So as the in in more and I some as we year significant

Pete Stavropoulos

this earlier, have the with site agency interaction experience answered don't it? any the the I from other for inspected if does know And or site CMC the you Has but color also, any the companies? And visited XXX? agency on

Bobby Gaspar

Yes, just so the on interaction.

on CMC questions I've from the process questions the think previous answer of I the in outlined we're comparability FDA package. that in

approved On inspection EU. -- the they're that in Milan, the they we you'll are that commercial the manufacture which know is for so manufacturer front, use, AGC obviously site

asked monitoring for an putting are done We have pre-inspection and processes inspectors with and are FDA place in there them we those using a inspection. and that procedures questions ex-FDA were

will we only readiness as get understand pre-inspection instill nearer we actually when occur. think I actually to will So once the

comment later stage. we So say that on a at I'd can

Pete Stavropoulos

And can if just one in. more squeeze I

give For composite bit about MPS detail survival? more complication-free endpoint a just I, little the program, you of can us XXX the

Bobby Gaspar

Sure.

have Let hand who'll detail to over that me Leslie, more on that.

Leslie Meltzer

Sure.

care, all So composite was I endpoint current an by and allogeneic at standard the this MPS are clinically in suboptimally particularly outcomes looking for served almost designed meaningful which that specifically is are patients of HSCT.

failure cognitive It growth that of so death, graft, need aren't well too and includes follow-up incidence with a as replacement outcomes associated of of associated also requirement HSCT, clinical for impairment. served of the and forth. such in morbidity components to by this So includes with therapy as incidence decline outcomes mortality with enzyme includes that reinitiate the and as transplant, as transplants MPS-I well

the that directly superiority outcomes care. to against be we'd is again, OTL-XXX outcomes so relative and designed And OTL-XXX show comparing to of with standard study of care with standard of

Operator

call end hand reached will now the I have the We session. now Q&A of the Bobby. back to

Bobby Gaspar

Thank mission diseases. severe we on what advance patients as bringing you. to to continue you you our Thank a HSC Orchard everyone. gene support attention for Thank for therapy very is very important with think in Thank you, much. important this your your time, your I devastating day and

Orchard Therapeutics (ORTX) 6 Mar 232022 Q4 Earnings call transcript