Orchard Therapeutics (ORTX)

Renee Leck Investor Relations
Bobby Gaspar Chief Executive Officer
Frank Thomas Chief Operating Officer
Yaron Werber Cowen
Anupam Rama JP Morgan
Peter Kim Barclays
David Nierengarten Wedbush Securities
Anna Vorobyeva-Schiano Goldman Sachs
Call transcript
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Good day, ladies and gentlemen, and thank you for standing by. Welcome to the Orchard Therapeutics Second Quarter 2020 Earnings Conference Call. [Operator instructions] At this time, I would like to turn the conference over to Ms. Renee Leck, Director of Investor Relations. Please begin, ma'am.

Renee Leck

operator. Thanks, and second everyone call. XXXX to welcome morning quarter Orchard's investor Good

website, latest information the found our with slide Investors today's of Our deck be supporting corporate call can section orchardtx.com. on

like implied to we make this by materially and will call from started, events Before forward-looking Actual could remind on statements. get include results that statements everyone those we I'd differ expressed or filed a uncertainties, our statements as make. result the XX-Q including quarterly set forward-looking risk we and of any any factors and in those forth Form that other with SEC various filings

disclaim forward-looking the on views obligation as our Bobby be call revise or In turn statements addition, statements. to views should Gaspar. CEO, upon our relied call as only of any with update today not any and that, forward-looking made I'll specifically any over this any representing represent And our subsequent as to of We date.

Bobby Gaspar

We're over everyone, few happy made we've to welcome. today Orchard's the progress you join new strategic the Hello, and vision. to last executing months discuss

You our vision Orchard investments pillars position placing portfolio feel and prioritizing our that long-term on programs. first, of neurometabolic comprised emphasis growth are: we value for that sustainable and four our creation. is These remember

establishing Second, model. focus a commercial

in two next within Third, disorders of Officer, Today, and our those Frank strategy. fourth, including manufacturing Chief neurometabolic generation investing developments our creating manufacturing pillars, in momentum Operating focus the will Thomas and innovations operational progress and on efficiencies. advancement and of our I

that at hear we Starting work of launch. the potential investor indications, informed quarter. thinking more in in we've fourth an also genetic and of disease event with FTD or in on you'll with neurometabolic subsets potential important OTL-XXX, larger and plan we host has value our Crohn's year, to the Frontotemporal Dementia including about a diseases. Later and our of how close analysis as some approval approach the price Finally, I'll insights around a done thorough assessment

have migrate improve by has this therapy is emphasis patients potential unique the and genes dramatically their often which that portfolio. We curing due is children disease. are of believe seen potentially the into placing of of to lives why are brain the them HSC to and gene to who the these therapeutic we to proteins HSCs diseases in the special properties deliver on our naturally CNS We

developmental Our preclinical a stages that MPS-IIIB Two promising for proof-of-concept would metachromatic MLD. including our and in registrational field, among programs a pipeline order. as in subset or and known in and pipeline includes devastating a a multiple stands these programs of program in FTD updates genetic the this MPS-I indications leukodystrophy first MPS-IIIA and approved condition be that Taking therapy for programs others.

that the OTL-XXX Medicines pleased as Agency we report to now Libmeldy list XXX For be Europe, to questions. in have which of responded known are the from questions initial recently for known MLD will we the European Day

medicine in very year to approval to are with EMA important affected potential this deliver important at to a to this be arrived and We the working the juncture this and have path families. children their incredibly to pleased

to designation RMAT to year. and IND end the drug MPS-I, its of need for an given disease program treatments for one to pediatric FDA OTL-XXX critical One well and recognition new and for orphan life the our limiting bringing this the develop for recently to the OTL-XXX therapy file rare two from is We of our received are committed nature. it this in seek US designations. by plan as severe disease In this

and improved primary with over biomarker motor have measure proof-of-concept Engraftment treated translated outcome scores patients will XX study in months also ago. and that were normal You the presentation activity who in ASGCT met our recall first was cognitive two engraftment. achieving growth patients at May, from eight into first all our hematological skills, from stable the

input plan registrational date, are results generate Given initial design to XXXX. you program commercial initiate study in intend clinical protocol, represents the success. the and to FDA promising this working with phase, our MPS-IIIA preparation to we'll the we with a develop seen endpoints in protocol third for are provide continues for OTL-XXX. for in trial and registrational data. more for to next three patients we've We and the have EMA the enrolled the details to In proof-of-concept internally clinical trial we the a experts the seek OTL-XXX pleased relevant and in to to we early from this and year this on as while clinical including field space we program both finalize position

We developments the recent call and over to in progress Frank second are pillar trial plan expect to the to interim now our this neurometabolic strategic of for organization of continuing discuss present prioritization has enroll on data patients The manufacturing. will year. programs. new And next I turn made considerable the to in our

Frank Thomas

technologies. well a where investment, three is our capacity supporting term recently meet near Bobby. our time we've core I'll to on Thanks, another had months. objectives, over to Aligned news and here. moment manufacturing past recap Manufacturing focus scale take with the both investing which in executed We pillar our to needs objectives new over

is the stable deal fact partner extension for that parts with can obtained the and an this Libmeldy. line in that in the emerging multi-system manufacturing. of for The of access to date. for second on of some presented the of for multi-year which producer marketed they our of work opposed are vector our at due the product. from MolMed, in to process their for of product MolMed a increasing with our last development be enable gives now while was produces advancement future a indications. upcoming this for build The to our with rely must around other space. authorization our supplement the time be our company, be potential been at line, GSK retaining in signed we signed collaboration time. of agreements GSK Strimvelis, clinical the is manufacturer a OTL-XXX supply, Aldrich each current our step for focus a The we production, announcement and into in a option security to scientists the treatment along in which The at to way cell produce and evolution applied us was who cost batch processes beta viral provides believe leveraged that we of the ADA-SCID lentiviral produced. this need therapies a dramatically technology This EU. GSK a The OTL-XXX can those step. MolMed, programs, to a reality. of integrate month as drug license of significant the inefficient WAS approval transfusion-dependent potential Renewing good a restarted a cost our our multiple transition program. a capabilities many manufacturing with technology own vector plans future or cell from Wiskott time is announced potentially thalassemia made. efficiency work can our previously improve chronol plasmid as has manufacturing to has and vector line ultimately and Syndrome basis, vector stable vector. And cell which is A that in making drug including collaboration scale single manufacturing a The of Currently, alleviates comes an the snapshot collaboration and launch and with of cell us involved our titer, lentiviral will at important the stable programs from of both practice produce uses in This vector methods significantly using manufacturing key technology components vector capacity buy making to line ability component that ESGCT we year reducing technology stably of viral the have gene to producer of all largest product us First, marks the the the the commercial regular our past consistency high work generate in and vector enables select from on that

funds fluctuations which This us year, the to three half is several expect investing million of quarter, in important XXXX. burn changes a $XX position any milestones up depending plan. support have implementing operational data was next in under million with of certain of and into saw operating the approach activities that launch as milestones footprint cash the quarters, facility gives on into quarter strategic a spent decline $XXX second or Beyond XX, being our second in our past restructuring, what but activities. cash streamline of activities including $XX the in R&D this fund EU achieve With used high of some the capital. we strong releases. this excluding available months to cash programs, We Cash have down investments XXXX we and in indicative first the additional value runway we credit manufacturing, million many numerous and disciplined were for anticipate of approval operating approximately within and broader the non-dilutive to first other regulatory and our a Libmeldy the able potential and timeframe, heading the the and XXXX we after to June

of quarter point We second our believe Company through close of term XXX% exciting the making committed create need find that back a financing. investments press all a our to an can of in stakeholders. with You to important the to to of pricing. are for pipeline for as balance context Bobby way commitment of release, community the commercial value we've we hand our from in patients, product in for but our Libmeldy and for managed inflection to we I development. value therapies financial moving the benefit our focus without of activities business a I'll the the discussion the phases and and And now an promise it candidates, importantly, near with reality had results shareholder

Bobby Gaspar

which like few in now about to I'd regulatory this Europe a talking Libmeldy Frank. key Thanks, year. approaching milestones spend is minutes

parents terrible You've disease. skills most had most as is a then It's and but child that moderate added progressing we Libmeldy, nearly genetic also and the said starts lost begins each about and And a MLD with child. disorder. she disease many and family. overwhelmed rapidly the need. or to cruel economic common wages really the fatal for level found MLD to heard had the their yet emotional I he to baby previously of a talk aged physicians reported ill neurodegenerative with looking severe, difficult manner. to walk, those fact It are to With severity half parents In in all with impact describe conditions symptoms means normally depression. this among gained. their five, severe and the MLD to what that imagine humbling. undertook, to develop and transform child option. to on terminally potential very only me to of write of Usually from rapidly lose MLD is this whom It the and care the pass a lose ultimately of anxiety approved, reported the have ability form a Affected unquantifiable Caregivers impact interact notable today, even away would unmet usually to with like child's MLD or children But while administration. life of child around at we die. we their them a as around families they with talk in XX/X feeling of to watch [indiscernible] research regards is who often anguish world the households they before we worst of and year, experienced caregivers single of expenses engaged, the dealing if pallets the have for the in carrying

assessment Libmeldy in the of or treated of rare is key was look In NICE. methodologies health In XXX therapies taken estimate On which applying follow-up encompasses factors than less differentiated to Libmeldy if Health Libmeldy a entire other certain ago. not we top full million therapy cost today, value. economic average approved, like widely HSC any one-time of bodies in to Based for potential looking a the have patients. offers in Excellence we we chronic we EU, the replacement price have the a approach outcomes. Our markets used and disease we robust treated on value years correction health we on quality adjusted in standing today. Institute that clinical be million years, data conducted potentially contributing believe for to less With last number and million and cognitive priced. positive exceeds decade market Libmeldy, impact. therapy data than first to expect the market of cumulative highly such €X.X which gained is the the Libmeldy patient anticipated gene have on €X we years €X.X assessment XXX into our by data effects. full base patient significantly set data eight durability robust nearly to on as on therapies this the key ability to the or XX follow-up its patients. us Care on as extraordinary durable first genetic €X HSC Libmeldy Indeed technology and a range the have therapy. program an and And range more Libmeldy upon administration. lifelong come with over is total, many Libmeldy designed potentially National of the the XX impact offer our approval is by potential across our the six curative If price for In for extensive set years economic market to years data human at the million UK's positions offer of NICE analysis appraisals, our account portfolio for the and XX-year do overall spans QALY the longest as than This of or determining to enzyme well we which life or other Moreover,

We to innovation committed stakeholders path for with also all are for the potential the the balancing Libmeldy. sustainable in value need to for forward need share a of

of based to durability payment open clinical and the effect arrangements, Especially are the this of we including gene flexible the options more. approach. such HSC given five As strength or value over therapy payment years with seen data

-- approximately very discussions on severity of another and magnitude in that of population our just most MLD in rarity clinical regular the believe currently in price On is the the to of incidents expectations into estimate and size Europe been put range of line numerous have perspective, with the disease. of impact our the factors, with be We we payers importantly, expected about it based point, to incidence one-fifth Libmeldy condition. SMA global rare and

in diagnostic Libmeldy work MLD early markets therapeutic is national As payers we our be with screening, governments working is treated access urgency, it degenerative, treatment Because we can and on newborn pathways to this within in for Since the identify get rapidly across and and are the regulatory commercial of access. child to the line issues. with crucial, progressing key European local awareness centers related and Europe, finish collaborating team is window.

engaging avenues. stakeholders countries to the a and reimbursement with provide preparations in on number and we early options that for determine potential best are access these track pricing Our are of

have by to my seen families for HSC to me it to potentially Orchard, Let close genetic work. transformative XX been as helplessness, spent this anguish bringing I diseases have pediatrician means charter close gene what academic, disease. us are genetic many the for young to so opening offering our outcome over progressing has therapy be deadly brink their saying, the I life's severe years therapy and and of gene diseases has society. children, certain pediatric the therapy a and on and years, a many, fundamentally with and their for that when gene believe the Operator, I changing deadly and line feel families the with the but possibilities of rare questions. for child many firsthand, we the fast please It taken condition. open diagnosed is community


[Operator Instructions].

or Our comes line from first Cowen. comment from Yaron Werber question of the

Your line open. is

Yaron Werber

Hi, thanks Yes. for and good team the info. morning

some MLD Can questions an and mind. the respond That's Day starting of first that FDA. you also Maybe question. overlap getting a So were the of bit sense, us for you the you that needed where XXX just and give are They the update EMA, on asking? don't a were questions if the questions with kind and EMA FDA. typical to few the of to with were you what little with

Bobby Gaspar

that Okay. Thanks. Maybe I'll Yaron. take one.

-- pleased it's able so very we're in Day from yes, So, the we've say to the been that respond to XXX to questions EMA time.

to line is with we exchange around both a after itself the through. submission, the in active EMA the and really And actually So and questions expectations. submission very the that with collaborative XXX prior dialog Day Day the XXX had questions the came our on

covered topics. address are to able been we've those responses they and And now So their and clinical CMC end. back

be approval again all at if the other well, FDA. no And a FDA or a CMC and year. of the had hopefully topics. and -- the both the questions, goes So full CHMP they your But in nothing specific was EMA hopefully the questions end fall about before clinical looking around EMA the and was there we'll opinion that to question to

on wasn't they same in picking it the So topics both were agencies.

were that through. there So different topics came

Yaron Werber

no that that Okay. our been is from of the and little adding fulfill maybe of, communications I but cadence say would the remote, little probably of the an and Bobby, sense to of who've further how interactions SeaBird we've opening everything bit long-term especially help work that's things is don't happening Great. slowing And have a bit are things more companies interaction be FDA that's over communication? and probably continuing them you, maybe much the little distracted, consultants a is than Do to FDA. sort COVID-XX. IND from more be harder want had one really little and interactions down is a everybody might FDA with a at along essentially those with with to you that Any is slowing It's then any that now kind how hearing down. sense other

Bobby Gaspar

don't this syndrome. we planned. year, with both on I mean that know interactions on as and light have much can those had been on that. I earlier I too And have the FDA Aldrich MLD shed Wiskott

for to IND And later course, any to for But submit we been this be once an any haven't we we -- given any of As RMAT seek that in indication planning we'll should IND. are happens that need year MLD. and we way. submit delayed see what designation

I let's as you through what than known we interactions has can't anymore have been far, so tell So, say, our it and planned.

Yaron Werber

Okay. Any Great. final Europe And are patients how through sense maybe -- question. in many you. already registries? a diagnosed Thank with actually MLD

Bobby Gaspar

maybe, that you? I Frank, to And Okay. can one hand

Frank Thomas

are to that and question benefit Libmeldy. early identify to place going from the they're early the can identified sure certainly to from benefit to [indiscernible] Yes, progressed course. key think launch get going is of And Of the to that is And we've ready in as is get diagnostic eventually of and is these incidence too about patients, as that newborn them There our do Europe, screening Yaron, disease patients far activities whether and progressing of opportunity the be an in opportunity. because make of they for in nature so putting one the very based commercial on the on we place. a rapidly Libmeldy be, we I the trying patient they're is focus think do team for the, identification. undertaking here key got much but

team is could of to initiatives focused out, patients. again disease awareness the in more very also identify and and therapy diagnostic the place these eligible the that putting on for on identifying So patients be


you. Thank

Rama from Anupam JP Morgan. or question comes from next Our line of comment the

Your open. line is

Anupam Rama

outlined? lot question. was should we of and much but XCGD get I MPS-IIIA strategy MPS-I broader thinking the that And context so update if I Thanks recently, an the taking we've a MLD, Thanks on wondering so could talked know you've much. that we about program? about for kind be in of the your how

Bobby Gaspar

Anupam. Thanks

XCGD. So, yes, on

to you, So this remind just program proof-of-concept. showed so

ago time we So for that responses some the patient. and we the in talk XCGD older saw about best

late were young and adolescents these adults. So

we as patients gene pediatric why affect the it's did of then see some case. and does well should unclear of and those also the it be pediatric engraftment not robust to actually cells modified patients as Now that

population move section -- have pivotal of able study, to to in entire the So want order to the a be we one than rather treat to population. just we

are And protocol. more an treating so what pediatric we're patients amended conditioning with doing is we

can successful the as we say patients that in see population. a so I'll engraftment well treats move to And I'd those study, and pivotal that treat, then say entire

work the next So, the that's piece on pediatric front. of

The that for to such this a in a large other place want manufacturing we is the right thing and large have indication. indication is

is the transduction. work using effective there make terms how how for example patient ongoing also -- about enhancers So more in transduced to and we we of and cells efficient vector transduction facilitate

a treat is we'll able there on pivotal be So also Once on patients And in study. amending well. have the the manufacturing. then those as pediatric to piece the to we protocol to place, move

So that's ongoing. that's the work


Thank you.

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

Your line is open.

Peter Kim

history? question on Peter remind give is still able guess, I IND one MLD. clarity might whether you do IND Do to move you. all half pivotal the be second GMFC-MLD, could you to you're the current to to you for -- our on Thanks to just questions. sufficient Thank to integrated population as you relative submit the when natural you and for data well that Gena. post to have for submission, another whether would do needing us study think taking as in secondary secondly Relative active maybe expecting study? that This versus endpoint data be on the And collected. data

Bobby Gaspar

Okay, I'll take that.

end on the dialog the an FDA, the the that you interaction interaction opportunity with we the rightly much when had doing more And very that, to our timeline, data be be will to give And able in had for once file of you that discuss have our that information BLA with an as that's hope we And is about by said, able RMAT designation with them. package intention FDA full you question. and thank to submission that so we'll IND so greater early seek with year. we've we've them, then So to 'XX-'XX. clarity by when have we timeline, will

Our to has FDA the already the and analyze patients that we guided is to. in us the intention way treated analyze have them

our put we And in we've is so that would course is clarity about all -- so that be GMFC our will the package FDA final point that of to forward far it sufficient as will only submission. and have And but concerned, what interactions patients as IND, score. we with BLA are the had the the scores far the history that treated we your analyze patients been able our about that will that natural once whether to with have we've

Peter Kim

you. Thank Great.


Thank you.

comes line from Our comment from Securities. Wedbush next question or Nierengarten of David the

Your open. line is

David Nierengarten

Hi. for taking the Thanks question.

here the approval your in difference between Is anticipated Just anticipated process the there one indication. regulatory and on €ope. any

or States So approval an covered anticipating it all [indiscernible] the similarly €ope, and on United through is you require Thanks. between with split from -- early in IND full juvenile to are that later progressing? the all infantile early on juvenile how in be ages would to and

Bobby Gaspar

Thanks, David.

number submitted topics well, did that. included we as, as clinical through at around Day-XXX responded and of cover the Day-XXX questions, far as all so, a And the from and that discussions questions came the to EMA CMC we've have label of

able to be we think through the I will the greater as clarity on label. CHMP comes give opinion So

that. we will And you we only at around can of give will similarly, subject FDA, lable it's think we And And more that what so discussion at the when, I give again, be clarity the patients and the discussions interacting more time. be we've those the had we as that included with well. can with because that you FDA detail

treated we Just disease. of treated juvenile just in to juvenile the that have have of the kind form and early patients patients we highlight infantile

So those been are have all our development. treated clinical patients that in

David Nierengarten

And a follow-up. quick maybe just

identified mean, initial -- population. incident discussed Frank be more the such of course fair will to put I the on screening that focus assume that as You infantile is to patients. it in your

the rather infantile early juvenile population than population? the So,

Bobby Gaspar

this all really. two there's to it's and One diagnosis. strands early I think is, about

mutation who clearly very screening, identify gene. Newborn in have gene, patients being in able MLD is important the to ARSA the that all

And very hard so if on working I'd that. be

of the a anticipate year actually starting in we pilot in Europe and before starting end US, the before also year. So end the of the also Italy pilot a the in

But and we quickly as we're pilots diagnosis putting so in and is screening. and about starts, lot as as before a have newborn pilots all of trying into wider testing, there so we And of other it well get of diagnosis early implementation diagnosis is genetic to -- a that disease effort screening get you that those a so do so a that diagnostic place, get awareness, disease activities the if better -- suspicion possible. newborn

losing are of also be have right could able that aware do to are MLD efforts made ongoing but who all to So both that prevalent those that the diagnose motor population for skills that will is for population and incident individual have may they'll physicians of test this exist the the the and importance early. -- that be may fact as chart well so

of the activities population. incident will kind So both these and the prevalent help with


the of question Graig Sachs. line [Operator comes from comment Goldman Suvannavejh from Instructions] next Our or

open. is line Your

Anna Vorobyeva-Schiano

can Hi. get is just a questions. programs? you We bit FTD? taking little This the on you. FTD you for a in about And Anna for Thank tell our wanted if Thank the see and see Graig when to more more opportunity little data Suvannavejh. also the us on you we bit next clarity CD might

Bobby Gaspar

for asking thank Thank you and you very that. much

picked has our invest And indications. gives also interest us we tells to gene plan, insights FTD, because part that therapy indications, have Frontotemporal new and you to as biological dementia HSC HSC indications. so us subset And reason mechanism want for again seen that larger opportunity ultra disease as can in the understanding address we've on rare as that gene what Crohn's of some conditions, know, and and the we larger take a of of larger genetic because of strategic Dementia is And well. we therapy and these activity, believe these strongly out address

migration the able delivering about of an HSCs genes and it's to FTD similar benefit. has we've pick have we've CNS the barrier and the the to out seen proteins mechanism and FTD, into where that in it this blood-brain been example therapeutic for So MLD, may natural CNS, in a and of subset effect genetic how through a across

so genetic more subset. about talk will And that we

will the HSC about is and of and We seen timing more chronic once that you involved gene Day therapy colitis an in Investor the is the Similarly,for can seen that about that gene correct talk how action at granulomatous detail in we've the all of give disease. content that later disease, day of the we'll mechanism Crohn's year confirmed.

colitis children that modified terrible can CGD. Crohn's is tissue CGP and it's the So migrate and that with invasion can histologically colitis they like response and HSCs looks in to they HSC gene and respond because get into gene when bacterial the therapy abnormal resolves that disease prevent have inflammatory

in inflammatory by the subset and HSCs of correct the response from tissue again a using prevent there prevent gene macrophages we disease, and occurring. resident abnormal Crohn's and So a those defect is colitis macrophage again, modified can

genes, later and mechanisms, those are the in these larger our patient indications Crohn's population the HSC at we of and the disease, So about broad Day address why gene believe more year. and talk the the we'll specific Investor kind therapy opportunity FTD mechanism, this can

Anna Vorobyeva-Schiano

and covers particular just is of you. more we Thank it I on focused that you assumption genetic make really if follow-up, quick or But genetic color. population Great. when it one for you say one ask than FTD Thank can one particular the just genetic CD defect? that forms can

Bobby Gaspar

subset At one you. it Thank FTD subset specific of and moment genetic specific Disease. of one genetic the Crohn's is


over conference additional I'd like for the this to in closing no showing management turn I'm you. to any questions the queue at remarks. back Thank time.

Bobby Gaspar

for much. this hope much. XXXX. I us very I'd our thank all in again, the prepare that value been you potential of to progress opportunity in to and update for Thank of And for end disorders you the you launch able approval this Libmeldy neurometabolic a tuning like be Thank and and has by year give an to on very a as you we also


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

Stay day. safe. disconnect. now Everyone many wonderful You a have