Earnings call transcript Q3 2022

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Operator

Thank you for standing by. My name is Rihanna and I will be your conference operator today. At this time I would like to welcome everyone to the Orchard Therapeutics Q3 2022 Earnings Call. All lines have been placed on mute to prevent any background noise. After the speakers' remarks there will be a question-and-answer session. Instructions] [Operator

turn to now Bobby call the will CEO, over Gaspar. I

begin your may conference. You

Bobby Gaspar

everyone, Orchard and Thank XXXX welcome you. and Hello, to and Webcast. Gaspar. Financial Corporate Therapeutics I'm Bobby CEO Co-Founder, Results Quarter Third

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

Libmeldy. the Chief Frank will Q&A. on for today's commercial Parker, our Commercial I Operating will review me activities President Officer, have and Medical joining call. our for Thomas, joining Meltzer, for three and Chief financial Officer, leadership team our Chief be members the recent Officer, of Leslie highlight will also Braden quarter results

modified hematopoietic cells to you cure genetic and HSCs of severe stem potentially Orchard's treat many to know, gene leverage diseases. is or As of aim the power

in Our is area focus twofold. this

pipeline commercial is is for of modified HSCs clinical We into the the have where conditions, late-stage delivering migration CNS of a where to gene gene therapies the and neurodegenerative needed. key it

Secondly, increasingly of Libmeldy, sustained developing and susceptibilities, for NODX next IND With indications studies the disease gene commercial such benefit, HSC MLD, success. of as for this with approval, form therapy we regulatory enabling specific can metachromatic how HSC lead disease, research programs leukodystrophy approach genetic one year. our approaching have are therapy or we our Crohn's larger gene seen to clinical

reimbursement our Europe, for therapy. Throughout this first commercial in curing with and made year, patient Libmeldy for have qualified strides MLD treatment we establishing great centers, treating

growing treat eligible awareness believe especially patients. diagnosing to are the refer We for among and making and physicians disease encouraged managing progress we and the by are MLD is continued patients identify,

unlocking early through rapid Libmeldy's universal because the progression adoption This to full will programs, of the of is be potential. commercial diagnosis, key given especially MLD, newborn screening the important

screening This front. from on positive now In newborn screening we first important regions. ARCHIMEDlife study. MLD of this the pilot their critical to potential MLD evidence this fact, morning's necessary release, of in case news announced advance for the universal key identification provides has in confirmed adoption

Newborn of understanding our screening true disease. this will also epidemiology help of refine the

for from they newborn changing will to life patients As potential. prior benefit so may symptom MLD anticipate studies additional Libmeldy’s onset, be expand, we screening identified

U.S., to Turning and we ongoing on leveraging interactions on the feedback in active request our completion the this to an FDA B are Type ongoing in natural RMAT Prior pre-symptomatic update arm. CMC meeting receiving elements discussions, release, and the the clinical of of the package, the classification clinical provided a we recommended morning's to with are interactions elements a our scheduling pre-BLA discuss patients has of CMC history that designation comparator packages. meeting, requested and specific of particular, FDA Orchard and has in

we to with XXXX meeting we move data have our Type to are submission. the clinical and submission that occur intend committed OTL-XXX with update. a prepare provide a subsequent in and which the forward to B BLA and needed for early working requested We've to will anticipate agency We urgently believe information

for program syndrome. MPS-I OTL-XXX is Following Hurler our MLD

only between payers the of a difference This transplant. show developing care, Our trials for stages be some for ultimately commercially. proof-of-concept we With regulators, cell aspects OTL-XXX current will that that registrational the protocol can a showed data study allogeneic for global has final of stem meaningful not hematopoietic of the in are addresses compelling standard very the disease. from but and regulators, important feedback multi-systemic

at enzyme ASH very with from and oral encouraging pleased that levels, Royal we're proof-of-concept the proof-of-concept and that shown our supraphysiological SGSH study December five-patient Hospital. meeting at has announced the in sulphate. has sustained we data a data, of Earlier so Manchester our far of this Children's MPS-IIIA University normalization is partners substrate engraftment heparin and study presentation ongoing including Manchester, This an upcoming been accepted As month, for the in biochemical

be will the outcomes from study time neurocognitive Ash presented. the first will be

We December doctors to XXth host two the study's Rob are with the to be principal data able Simon investigators, IR which also attend. the hope of an review webcast Wynn, planning Jones, on to and you'll we

study data addition who seeing clinical to study, In any treatments. they not to to formal outcomes the patients they'll from be are have able the describe undergone the compared in patients,

therapy in very conditions where to severe, are see difficult these our to need. is programs that excited gene continued there such making we the So progress high unmet HSC treat are a

review progress in the Braden turn over to our I'll Europe. with Libmeldy call to now

Braden Parker

XXXX, of really many We're identify as eligible two made the we've and MLD treated network in first. commercial the progress the in We first including Bobby. patient under year did Between activating pleased infrastructure agreement. Thanks, those possible. currently we we in process, as and treat our half the confident second patients more quarter, than and the treat UK in with the the to are NHS Libmeldy so third patients far patients in will others patients needed we

four from has have the XX days. approximately averaged treatment to patients centers five apheresis qualified out we where infusion in addition, treated In now of turnaround time

and terms recently expand Technology Assessments Finose. HTA In joint are consortia our of to actively European and reimbursement we access completed to in initiatives, markets, with Libmeldy have additional working Beneluxa Health

APX the process where of we renewal the under Program secured Access we year. patients Early the have France, treated In for have two another

Orchard in reimbursement we During agreement. patients treated this for France eligible negotiate will time, any a revenue while receive

existing the support network additional design, our the We Continental of ways investments Europe. are qualifying our this in in across sites are By addition in MPS of to the to programs built and Libmeldy and by neurodegenerative enlarge to also the treatment centers centers framework administer undertaking UK, to space Sweden in in in plans future. five Spain

Europe, the identify eligible mentioned universal marks be initiated treatment step an the study also could to These total, via East, early patients Bobby drive with screening in been have the U.S. In newborns. studies the treated is commercially, studies programs important over place. in a and newborn dozen actively to diagnosis MLD if reimbursement Middle screening newborn six As efforts earlier, adoption ARCHIMEDlife identification in the through positive and of screening.

In the patients have at team. innovative our receive type work an abroad identify of geographic entire Middle eligible capital Libmeldy pioneering is of treatment we are Overall, may treatment in Europe. we proud who East, of through are expanded partners footprint and in partnership commercialization strides to a how centers be This example this continue qualified reimbursed great in to we international the the efficient of Orchard making of manner. the therapy our

Frank the I'll and discuss turn to our of revenues rest over financials. to it

Frank Thomas

Braden. Thanks,

of release incredible Operating and in this some that is have serving technology X-K, Some morning's of on ability a Orchard's mine in you in may impact always an previous I patients. have decision have to I've news to believed we President the noticed a at Officer. our Chief the continue reference have Orchard as and to personal which and made

While expand the ingredients gene to and reimbursements, space encouraged to our a as challenges establish to leadership make team the a faced believe the breakout gene leader Bobby, Orchard over establish ourselves technology forward ability of gain field, focused a and I'm very therapy to has the we've the couple many all in company broadly the patients, organization as by committed the the find past globally, I portfolio. to success. successful to therapy to model. has Orchard years, progress and to extended with move I'm the company this we value potential a as working and business to create made with demonstrate

$XX with we with upfront quarter flow after most revenues. these $X.X recognized the to prior sales of treatment turning Similar to revenue is quarter revenue In bringing Libmeldy, recognized in treatment. paid the revenue the the third commercial first to were XXXX. starting million of total time with Now nine treated Libmeldy, for our the from Libmeldy for from the two financial patients the is quarters, cash who performance million of at product patients from months and

We also revenue the Cost in totaling product quarter. patient sales recognized were another product the Strimvelis million with $XXX,XXX $X.X period. of for treated and additional

and manufacturing, third of up amortization This party quarter, royalties, and gross the both a for primary the As of XX% goods, cost periods. resulted make sales reminder, product from cost items for three non-cash margins approximately of year-to-date in products.

COGS, and in this morning's from of of release. the million. $XXX.X third press We third now in from and full burn ended our financial results full We're financial quarter find quarter rate revenue the the restructuring seeing can Libmeldy Beyond reflected savings the investments March XXXX and you results. with cash our quarterly

operations. $XX tax in of periods. million the the fund from to down refunds burn and cash in QX third excluding of those QX $XX During this rate in This $XX year, million million this is used year, company one-time in about quarter,

growing we're on guiding expect burn downward ongoing management. trend referral XXXX. We number strong in currently see revenue Libmeldy the into and quarter quarterly now cash based continued patients of last in the as And rate from our to the a second of result, runway pipeline, expense a our to

beyond about also as our existing disease state gene of business but for As platform infrastructure, the [ph]. just have with partnership was we funding commercial think than therapy development or The the with new set ideal that XXXX, could indication bring partners farming case we capabilities, existing would opportunities geographic platform. our HSC leverage business also the across capital, see knowledge, footprint or more an

of in active and the our We potential platform. manufacturing business be leverage continue to to development scientific full

become to breakout leader for to gene in as the in gain prepare promise therapies of a filing, our and poised And we community, I believe our and advance as as clinical Europe we're continue the exciting research on Orchard and we pipeline we stakeholders. the U.S. launch Libmeldy the our to candidates. reality is of therapy traction XXX% patients, dedicated our for making of benefit a

call the for I'll over that, the With to operator turn questions.

Operator

you. first with question from Wang Barclays. Gena Thank comes Your Instructions] [Operator

now open. line Your is

Unidentified Analyst

can Hello, hear me? you

Bobby Gaspar

We Gena? Yes, can.

Unidentified Analyst

is Thank much questions Libmeldy. Gena. have this for hi. Shu two our questions regarding Oh, [ph] for very you I taking

expect not patient first have how you so many but patients quarter in fourth many about have of treated, trade this The year? treated you patients the thing far so do and how to identified,

the question is second for Libmeldy. My about U.S. filing

with Type to color as meeting in could you more what or BLA Is to on be So was B what looking FDA XXXX? filing the And you. you more like are give granularity do elements? FDA, Thank expect for? the specific FDA the

Bobby Gaspar

Braden very to one Okay, to first to thank discuss. that, much the those going over you I'm questions. hand for

Braden Parker

for thanks that Yes, question.

number outpacing fact referrals We only with in community we're that to this year, of the of that and different come general. can year due the and Libmeldy also of though MLD launch this or speak to treatment are the in the speak that referrals I to we leads receiving specific guided the well Libmeldy have itself increasing that's of as but to year as the as not awareness last centers haven't number leads the significantly

In number that at the Italy. terms this first And five you recall the date, year. half previously we've in Eastern in said Middle the to year we've the of half OSR, we of we second first treated in of treated was if including of three treat patient patients patients the would more in than the year, treated the half, that

of process for we the two So in the with that in three and half. second half patients in quarter, third more or first patients those half remaining the the quarter, patients these treating year, in we've treated anticipate the in and the than first are

Bobby Gaspar

much very Thanks Braden.

me asked about the the meeting, B put context. clinical so Just this you in let filing, on Type U.S. just

the some had very we had see like package. last and clinical remember, then clinical package they what on we to constructive you FDA would we and outlined an submitted in the they IND, If RMAT year, with we had, meetings designation the

the discuss And Type now so we've B and clinical submissions, of want those to been to that, been some They've that. FDA. they making to very submissions aspects specific hold meeting a reviewing of

saying the So coming active want elements. around and we're they're to the they program, had encouraged the focus and we've fact with that very by this that FDA on specific engagement forward

I'll detail. into on So more our go and me then -- a that, just couple elements let I'll a CMO, highlight to just bit Leslie of ask

will sure can know that you're thing we've treated remember, the FDA phase, to when in Libmeldy because do support think to treating have pre-symptomatic is, and valid they late we're be a juvenile have what would of information phenotype? phenotype the And that's we talk very effectively. the first of And infantile them and Leslie will its we we about number And a how that early can or greatest effect. pre-symptomatically, The to that's know a patients we able why that. question have like is, all so you that if

history. MLD the that for history of second appropriate there's our the that is comparator. evidence to to again, the support And the, understand again, comparing And And whether natural is, it care thing the what population. FDA remember is against and to an no package natural about treated we're standard wants

like are So discuss those two meeting. to the main B that they'd at the issues clinical Type

So questions. we feel be to able to the support have answering and we evidence those

detail I'm just bit ask to more to about going So that. into a Lesley go

Leslie Meltzer

Thanks, been topics information questions. discussed Sure. and information are that providing these that objective Bobby. amendments have I'll we the And And over submissions, agency provided questions. addresses reiterate previously, in to discuss those with this is meeting that the year the to IND those past and response through of the been really that's have

diagnosed. symptoms, patients remember being our led key regard in them, development with classifying pre-symptomatic to older that that the an experienced patients, to pre-symptomatic that thing to the sibling have So of and program appropriately which is XXX% sibling

subtype sibling between this and to phenotype. experience. genotype and MLD due them a There's very between sharing concordance of in they that relationship the terms is will high the of pairs And genotype

been has classification. how validate this That that That additional agency U.S. on an those review for will discussion we FDA, place. part So of background perform and with the patient the that results. to is independent patients The shared the assigned. asked taken how have U.S. around aligning also been experts be and KOLs, asked has and takes some place, with assessment information this on

there history history, trial study. questions the in With largest conducted about done regard natural history conducted to also of studies the history one MLD. the this that's And that natural were representativeness of the the population. been site natural It's natural the

have history studies MLD. conducted in As comprehensive part the we across a of that of review natural globe

and compiled outcomes between We natural consistency supporting FDA will be demonstrating natural those populations sharing elsewhere. the studies history the literature our population and and history history with natural

discussing that and those the And Independent we with that natural those well. also literature agency we validated discuss review with chance generation we conducted activities that we study to as classifications the yet conducted, Review by been activities patient that previously, have in agency. haven't a data Committee the we address can The the have history believe have questions raised in-depth had

Bobby Gaspar

B have you, Type more final to filing. for that update specific after Leslie. then that And meeting intend point, we timeline can then you of some information your we Thank meeting clarity and the give to we once about

Unidentified Analyst

you very Thank much.

Operator

comes from with question Your Cantor Stavropoulos next Pete Fitzgerald.

open. now is line Your

Pete Stavropoulos

taking all progress, Good morning, the Bobby and team, questions. congratulations on for and thanks our

on I First year the XXX publication, have is, ago. New MPS-IH you've several of program in England since updates question given Medicine Journal a just about

want you what of on from transplant, disclosed, the the do patients. clinical being just get three terms patients into of your administered or consideration So in observed data manifestations you've when syndrome, especially allo-HSC is perspective in two where superior? in And I Hurler younger that see undergoing addressing XXX taking benefit to various

Bobby Gaspar

and Yes, thanks thank the a Pete lot question. for you

in disease, therapy. associated XXX. about we're of allogeneic graft back a has reactivity neurodegenerative is to care know limitations to again, are transplantation, there allogeneic There some those neurometabolic, need, safety. excited standard mortality go There's is unmet and a have very there's we high it condition, and XXX disease, with So, versus another limitations leading host enzyme onto patients replacement but around

there's So well. number there's issues that. of major And efficacy around a then as

with area as that such concerned, So problems, is hearing, transplantation, in patients we especially in mobility as and far the who other joint after restrictions there still limitations are have as in vision musculoskeletal there cognition well. know are as in

final of factors superiority a EMA, together, really study talked we've that's allogeneic it's many from lot been stages and of in global feedback a to we So the FDA part the address. and over disease, complex registrational taken want putting And to that KOLs. we've that we're shows transplantation. a the different both a And

allogeneic. think very that I therapy that's our a to needs very, important over to make, point superiority show transplant.

talked we study a about. able and have designing it just as that those to show will, feature I've So aspects that, endpoints been to be

Pete Stavropoulos

registration of in to terms there and in are identifying if so, and screening patients Hurler you Okay, newborn in that able in actually may your effective syndrome, studies? again, enroll they are be place, programs

Bobby Gaspar

mean, in very if screening MPS-I. already very, place. but in are correctly, remember I XX a that's being point. fact, important good MLD, talked MPS-I the no, states screening think And for I newborn about Yes, in newborn in I already we've screening U.S. for is

that that identified for patients being study. mean are will So be and early referred global registrational can our

that's So right place a good start to beginning. from the

Pete Stavropoulos

screen in there? and right, All you also EU, of the did terms

Bobby Gaspar

interest -- condition But of the Yes, it's these there -- the in have. you the in can treat better the think and the so again, think the Leslie because is shows where sorry. there in fewer this can for a you another that lot EU are these just… screening I when EU. more sooner national patchy that for is again, patients, as there's MPS-I programs literature I'm I outcomes MPS-I

Leslie Meltzer

I'll late distinction just a syndrome add there's this, one thing, infantile which is MLD. with that Hurler and

All the the onset in patients with a We believe symptom treated that MLD. pre-symptomatically, be were Hurler already way syndrome we patients would diagnosed don't were taking infantile have late in our in that's be there diagnosed study, the study proof-of-concept of requirement place. that that

we outcomes, identifying care, screening early, in even anticipate on also the of So the improve best but in newborn there's we an current is standard where helpful to opportunity patients believe symptomatic would population.

Pete Stavropoulos

different at neurocognitive younger a it there, I the mean, of you then would and terms -- and sort it's endpoints? my population an have to you pivotal symptoms so you actually have are, subgroups in to a older you that affected guess cut and is, I to of and assumption population, then, older neurocognitive Okay, study, would perhaps look already possibly and

Bobby Gaspar

patients have stage is our we together. to all I the if a to intention patients. subdivide At think moment later that's come something at put to we'd

Pete Stavropoulos

my Thank for you questions. taking Thank you. Okay.

Bobby Gaspar

very you much. thank Pete, you, Thank

Pete Stavropoulos

Bye-bye.

Operator

comes question final with David from Your SMBC. Hoang

is open. line Your now

David Hoang

Hey, Thanks questions. the good my update for and morning. taking

you So the the about some would I to But requests MLD here, things that the ahead any us of just the U.S., B be in just sort you for CMC BLA if there's requested, on of CMC had OTL-XXX, need into couple Type of just outstanding a do to that which or remind pathway talked addressed product FDA work need meeting. filing.

Bobby Gaspar

Thank point. for another and David. very that's much great important you that question Yes,

very again, So like package. package. would they see again, FDA FDA with the discussed just the in what was us it context, a to a had guided XXXX, And our where meeting, meeting in to we in and we late CMC the constructive CMC

And so over to submissions the those we making year, questions. this to been have FDA address

One questions manufacturer. comparability is Remember, has XX the going And study of development final package. years, the drug is the manufacturer been iterations to a program product over around are we there have been this in with product. cryo-preserved and vector in obviously, the that a

So needs there's comparative to a ability package generated. be that

of So have different to feedback. we've that are submissions we've these all FDA, so and to all and making been material. material waiting of on And we're what for they've they that points, and that the indicated is their they reviewing us

in that something we'll we the the Type that have will update feedback, CMC clinical we filing get back B timing had clinical feedback, is then and to can the hope I we both our will the And externally terms hope that so of guidelines. our and again, of, soon. and on on time very meeting have

David Hoang

And question landscape. helpful, on a I broader just really thanks. regulatory had Got That's it. then

some recently pure that could approval FDA guidance a proceed aware I'm the I receive, from accelerated therapy company, they on did gene think, pathway. So which

was so syndrome. for XXX this granted, And MPS

into things approval for looked have franchise -- gag looking So wondering as surrogate you thought at reduction MPS accelerated routes [ph] if your I'm looked for or a endpoint? like example, just potential about

Bobby Gaspar

good for MPS-I specific this to very is as as And pathway very, concerned. very question. this we space about be far again, well. accelerated biomarkers, and carefully, a use watching again, as able opportunity is an for talked example we've been And That's to an MPS-I

is that up discussion. we're into. looking So, again, much something this for It's very

as be the manifestations disorders we of try if encouraging other the for in only and used these I it's where a sometimes field, I period that could happened longer is of indicating for think FDA accelerated these those kind severe programs, tackle biomarkers time. very think that approval, over very

something did anything watching will obviously, So about. we're think this to want is carefully, add you we Leslie, that? very to and we'll

Leslie Meltzer

therapies of endpoints. recent accelerated Yes, And exploring with of being companies where action just also of comments mechanism biomarker supportive predict quite able the are the the to that likelihood say to clinical FDA is leveraging there's approval of a use quite benefit pathway direct. reasonable gene these

So as process. through we're something and by programs We're comments. companies to very would that absolutely news these the very programs MPS encouraged -- we other are look shared as it through have progressing those going regulatory discuss is encouraged by are their

David Hoang

right, for question. the All thanks taking

Bobby Gaspar

Thank Thanks. you, David.

Operator

you. questions Mr. the over call further are There I no at Gasper, this time. turn to back

Bobby Gaspar

thank everyone you we for as Thank Orchard's advance and very Thank attention you. your continue you mission. much. your to And to support time,

Operator

concludes This today's conference call.

disconnect. now may You

Orchard Therapeutics (ORTX) 14 Nov 222022 Q3 Earnings call transcript