Earnings call transcript Q2 2023

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Operator

Good day and thank you for standing by. Welcome to the Orchard Therapeutic Second Quarter 2023 Earnings Conference Call. At this time, all participants are in a listen-only-mode. [Operator instructions]. Again, please be advised that today's conference is being recorded.

to conference like speaker to now today. would hand over the your I and Gaspar, Co-founder. Bobby CEO

Bobby Gaspar

I'm Gaspar, conference everyone. important provide to delighted Hello, our Bobby share regulatory our Therapeutics. of to results. as second Welcome and Orchard quarterly business quarter well you financial XXXX with call. updates, especially CEO We're some and as Co-founder

latest Please Before SEC materials will be we throughout this we get call, information. presentation more started, for supplemental I forward-looking filings want refer statements. to and the our remind everyone that to making

As execute the therapy we'll reinforces morning as news our I field. team commercial, on to highlight The leadership today's call, members me on gene joining this [ph]R&D, across business, Orchard's have continues two Orchard today's leadership regulatory, well successfully management. announced call. of as position operational all in of aspects we HSC including the

remarks, Following results joining our a momentum Officer, be of for our Frank and will overview will Chief Officer, Q&A. our President as an provide my Braden Chief also Parker, summary the as quarter. of Europe, Thomas, in Commercial Operating well for financial

We will remarks focused on prepared keep primary topics. three our

a we First, advance towards to continued potential you'll U.S progress are OTL-XXX approval. hear the about making

Europe. we'll commercial the we we'll updates our share and Second, regarding quarterly provide business outline are And building update momentum general in third, a financial results.

world systems. or to These to onset Leukodystrophy, interact OTL-XXX infancies U.S.,which is MLD, late Europe, it five lose children disease for talk of and the Metachromatic in seeing since medical intended peers gene enormous rapidly throughout pass The recognized of develop In the with start are them. the of MLD away creating has years healthcare severe normally, treatment XXXX. the value update need in where their the may patients, XX an hours approved of the status Let's an the ability with families significant, and babies the start form early around is regulatory late being on we majority for and by require and of care, burden walk, most MLD. on our onset, been within therapy

this to OTL-XXX, U.S. morning's received the our put previously track I from completed have clinical and tremendous months collaborators we've first the with pediatric half both the FDA, on of well as the for designations meeting in if of the made of FDA XXXX. want has PLA which, potential milestone. collectively approval and U.S. the this rare team, to achievement in might our press would take progress to who contributed agency. release, announced on which granted, a to role the priority RMAT clinical Reflecting requested we As we to Orchard be the ,for a OTL-XXX seven our review, the since in moment submission disease Orchard partners as gratitude external sincere

work to have and While be currently still options step MLD, with supportive beyond are families important today, done their we significant in U.S. to who treatment to children bringing Living no there's the this care. therapy one closer

hear working third to of year. this FDA expect filing quarter in the the agency with forward looking review the and the process from BLA are We to throughout acceptance of and regarding the

comprising because agreed and medical meeting. more We're they upcoming data data an an also patient discussed OTL-XXX endpoint present with clinical years as the clinically FDA important meaningful. using to full outcomes planning demonstrate BLA of as consistent the set XXX experience These being than are

proteins transmitted moment some a Before gene updates, migrate financial let's and deliver potential and which disease bone, locally and our CNS highlight of multiple data demonstrate systems, we HSC continues our modified correction. into recently our to therapeutic therapy including the and to enzymes presented HSCs notable to we portfolio, across approach to and GI-tract, organ the discuss affect ability from take gene commercial of underscores

Hurler demonstrating in featured included with new in Angeles. and ASGCT Proof-of-Concept the at correction skeletal data This leadership system skeletal from metabolic patients OTL-XXX function Los in MPS-X, the Several of disorders autumn's study normal growth motor progressive in resulting patients, were in CNS improvement diagnosed which demonstrated of remodeling, of form neurometabolic mission and for presentations extensive rates, joint skills. of the ongoing

among compared care the cell stem The allergenic to not growth registrational for of address and patient the current hematopoietic outcomes. standard by is plans skeletal care of global XX Orchard to standard adequately randomized which end. year trial of initiate does disease, of OTL-XXX MPS-X other transplant, clinical manifestations a hers controlled

to with CROs sites, working currently of first this are the prepare our its clinical labs We for study. and decentralized kind

approved biochemical allergenic proof the updated the A followed enrolled no A. that years, three during of study years. there enzyme therapy Similar with the are disease highlighting to of and the or investigators trial in concept to ongoing for MPS-X therapies to two has median given severity compared shown minimum additional medical HSCT presentation continue proof for to and been study the MPS-X effective not treatment replacement ongoing natural Patients of patients of clinical a history, be fact half which favorable report will and outcomes. be OTL-XXX data additional time MPS-X will neurocognitive with in significant from disease concept represents a follow-up a need outcomes, A showed A MLD,

inform continuing to forward to program. the later development look follow of patients in for this We the stages study to

CNS, our and of programs for we preclinical proof also research pro-granulum the for data dementia, disease, disease. address featured preclinical modulate the the treatment express also presented from of platform preclinical first to gene This ability form which frontotemporal indications. data predictive highlighted the form of severe house therapeutic the the included in our larger broad neuroinflammation concept a of showing OTL-XXX potential demonstrate Crohn's data ASGCT, NODX two in of for of to refractory normalize At which and potential OTL-XXX of therapy HSC biomarkers. deficient We pro-granulum the

inflection as they internal time as treatment of. data business in through from to engineered data clinical, the success. for for Additionally, HSCs and potential Together, opportunities are one based disorders. autoimmune provide to continues development providing rich demonstrated development. pipeline vivo near HSC achieve commercial genetically our Moreover, a CAR-Treg cells the same term and both therapy help regulatory on infrastructure and Libmeldy, common investment us points, and a scientific, platform roadmap gene all multiple

financial discuss with let in over our that, me With well we are turn performance. as Libmeldy, the to to Frank building call commercial the quarterly Europe momentum as

Frank Thomas

the We've HSC therapy you, always Bobby. believed other severe Thank and potential gene curative for genetic diseases. of in

MLD. Libmeldy making are in With we the promise for a of and that reality Europe, approval patients launch with

work all members continue MLD, the to and eligible commercial identify, of support refer Patient of community KL and teams Our with patients. and to medical treatment

countries. to We to make reimbursed in additional Libmeldy access also continue European progress obtaining

we secured four exceeding European in our have additional far this countries reimbursement year. annual goal in of So in half agreements just first XXXX, the

Decision with center Approaches to to be it's with qualified. agreed authorize in MLD. Sweden in the fully for onset ongoing Most country early for recently, are Reimbursement New the will eligible Norway all countries. European the several MLD patients treatment once referred Forum Patients in in discussions

through mechanisms during are border our in pathways have through in Europe, treatment in relatively In drivers which and commercial early abroad access important East, early successful geographically been Middle France, cross programs the in of growth reach launch. this expanding through addition, Eastern we stage

Europe. at from qualified on commercial in four treated of now have different we date, countries our five centers six basis patients To

a We drug take rate successfully continue and this it U.S. in to achieve confident commercial are and XXX% to we production success the product model can apply

early the aid of and diagnosis Following frequently MLD spoken newborn of screening a to about potential FDA importance approval patients. for OTL-XXX.We've treatment

in key countries as many To screening of studies these have confirmed are commercially date, newborns identified and cases and following and XXXX been with newborn of regions, MLD globally for newborns patients states. more June XXX,XXX XX, identified Four in nine treated be of through expected screening prospective to beyond. actively are of MLD than MLD screening the Libmeldy

more of Furthermore, studies XXX,XXX identify the end through referrals generated and the newborns for are world. adding screened, to universal screening. MLD around been early to By expected symptomatic provide the will the family U.S. to have creating being in the evidence to opportunity applications data these than from year, and patients critical of diagnosis an screening support and additional the

working implementing implementation year. screening programs for screening together to culmination a represents other this step of U.S., very Act, Illinois and for actively for for start by the Legislature the in newborn laying On all screening Department initiative known big Health that of the front., multistakeholder screening a are week, was states. to support we Illinois groundwork State where in Public Metabolic of MLD last recently, as signed requires will state screen is also which it passed bill SBXX, that Screening Newborn governor the The newborns the the statewide and the forward the MLD. for of the expected process MLD This MLD

the study, the following positive announced in prospective previously newborn with necessary being apply are identification a taken to a that steps MLD In screening for Germany nationwide country. of from addition, in

identified patients to been appropriate continuing have The determine with or be who other through quarters, been scheduled are to these in for treatment Germany Libmeldy, either studies have treated treatment are in future evaluated options.

the these believe represents treatment Using studies price $XXX realized and the efforts literature, of opportunity which estimated market also patients than European advance since cited alone of opportunity. for identifying an we universal potential elucidate in U.S. patient global one births we screening, help applying existing annually. the the per a incidence annual eligible in true to research and and rate million incidence launch, Beyond supporting MLD, more market live be significant project to XXX,XXX Europe average net Libmeldy today's per

will the prior believe screening Libmeldy realized reach considering enabling in the to $XXX market, assumptions we up will like Libmeldy therapies to opportunity incidence potential of This to countries addressable the universal reimbursed. be symptoms. worldwide for million newborn place, peak global be same those the identified Applying to be is if annually, opportunity could population onset only where

are take the community seeing a it these can this newborn to definitive MLD draw entire The time, make conclusions. early coming from studies we're can reality. data the with screening together momentum still While

incidence globally. be the no consequently, the other market candidates clinical the than and Libmeldy in a per the could even commercial in fact births, commercial and long findings by longevity following create be The higher opportunity that year of term exceeding barriers opportunity durable suggest approved potential discontinuation preliminary the high live literature. However, that Also estimated to recent are This of of one could to entry or there other in the previously therapies closer further programs. and clinical development may currently $X news the XX,XXX billion bolstered is impact. its market

and With financing a and revenue $XX a expenses. continued Now it reduction another execution performance, ensures operational turning initiatives in capitalized our to in to strategic our quarter growing continue The disorders. aimed to accelerating strong preparations, of at has been U.S.Launch that we investing Libmeldy closing in neurometabolic in next of in programs and sheet, financial operating our excellence. are advance second proceeds our the solid well million progress growth balance MPS from inline Europe,

which comprising share the securities to premium $X the at price the of on Notably, closing per represents issuance. a XX% were date second sold ADS, the

expect an sustain in for to could future, proceeds additional $XXX would and further our the to from Company OTL-XXX if grow closing share XXXX. financing Approval participating U.S. are our This mid second in to per We sold business. The exercised expect the million all runway extend XXXX, our in needs $XX especially funding to we as Company's revenues could investors. the first level by bring warrants at following cash foreseeable a that potential the of to up offset

revenue. revenue revenue date This the million, of of to which product quarter to $X.X product cost margins manufacturing the Total sales of The the sales. representing and $X.X costs, second million and $X.X amortization. million, was Libmeldy XX% comprising million gross and non-cash in sales for quarter royalties collaboration highest beyond of quarter for in was Libmeldy approximately resulted consists in primarily cost parties $X.X third

release. the find financial quarter from results press morning's this You can in full the second

from quarter second same investment to and realized period are be Total good the reductions across business our to were Importantly, corresponding stewards period both capital managing declined expenses. our XX% expenses in we operating SG&A compared last year. closely The more declining the and expenses XX% the with XXXX. in of our than continue R&D

and of so cash the the a cash and approximately into two We growing of approximately XXX,XXX,XXX ended product with sales. of generating and quarter revenue middle with XXXX, years investments runway

on will Our burn continuing remainder financial sales goals our rate. the focused our for And year year. are annual Libmeldy managing XXXX continued of operating lead which a downward expenses, grow we to to in trend over expect

over it hand to for final now comments. I'll some Bobby

Bobby Gaspar

Thank you, Frank.

for year progress of momentum beyond. period throughout incredible an the to carrying and been Orchard and it's we outlined, we've As rest forward this the look of

gene Orchard leader is next in twelve to breakout become the the HSC well positioned months, Over therapy.

financing, continues coupled on track Our us get sustaining a to stronger the our growth, top-line strategic business with become puts recent enterprise. as self to

many has to platform also We've including early progress the made strategically has commercial, applied areas gene and therapy current learned disrupt to of development, lot clinical been regulatory, care. probability Great from it advance research. of development and of across greatest insights commercialization business, the in and manufacturing HSC those Libmeldy our indications where our a standards have

the because and from focused, approach difficult to we've for face this diseases. of period our driven well funded companies while ability devastating It's environment sector, headwinds been a other number shown emerged gene continue contraction a as and as cure has broader of ever, more leaner, to mission setbacks, ultimately the potentially therapy and industry

we'll the open questions. call that, With for

Operator

instructions]. [Operator you. Thank

Our Guggenheim. Chatapadgy first question comes with from Debjit

You proceed. may

Robert Finke

team. filing. This on Debjit. for BLA is the morning, Congrats good Robert on Hey,

on you for So, the anticipate an do Adcom filing, OTL-XXX?

Bobby Gaspar

Bobby. take It's, question. you. And I'll that Thank

me a need, Let back RMAT with that step unmet strong designation, high that second a will review. get of do there's just or likelihood be we request We we priority will and have requesting review. have we priority review, say the priority to

an as first far for therapy MLD. is concerned, submission As a Adcom this is the for

and we making lack this show quite the we condition. Adcom. And the to, think of of need, disease, the in So that alternatives devastating difference we level unmet the other again, it high well for would significant an OTL-XXX get welcome FDA this are may that with be I that

soon of So if kind very those we do have a then relatively these focused whether obviously, we're in position. But outcome are questions, I know we'll clinical on the not. think an strong at or time

Robert Finke

That's Illinois can for could any follow one you catalyst great. apply and Thank states? provide on to any you that color the screening you. that in Illinois And up, learnings other statewide maybe

Bobby Gaspar

Yeah.

states So by statewide are screen even will basis, There procedures recommended state state the screening implementing Rust different and in different allows legislature on screening the a screening. to where states the U.S. for is for before have panel. some is condition by them

are and there with those some been states. of so working we've And states, those

it passed to is birth legislature high screening has Illinois so And and a rate mandate for MLD state.

year. this pleased we're So they end progress be early or there may with extremely the year the and of well implementing next by

U.S. screening think, significant very, that So MLD is, for a very I in moment in the

there states condition can Now is start which the also placed are screening panel. the before Rust on other

And so well. we're working with those states as

pilot mandatory Now some of there pilot are also other kind states that than studies screening. do programs, statewide can rather

that And other so working are with. are those states we

So there's on may Illinois apart an Rust start in well panel. awful states before lot that of disappears from activity other the screening ongoing

states. with working be we'll so those And

Operator

Fitzgerald. Our Cantor next question comes from with Pete Stavropoulos

You proceed. may

Pete Stavropoulos

and Hi other Bobby, made the filing guess. Congratulations team. the progress Frank completing and this Rolling on BLA past all

any the have if You facility by there's agents approved Just us know inspection being first color us can on in know approved product. of many you any the one help facility the just of for I agents are FDA the the commercial you questions manufactured manufacturing can facility how and being the remind there? this and experience manufactured European authorities gauge at providing that provide

Bobby Gaspar

Yeah.

with So that the Biologics, Milan. based the They we're in CDMO commercial have is manufacture for AGC EU. is which been in approved working

so I And their is other as So commercially inspection. obviously, will have FDA commercializing this EMA Libmeldy, been they first an inspection. also in They in there. previously They products manufactured FDA the through, think well. are we manufacturing manufactured have EU; And through inspections. strumbellis are be haven't which been they the

very We have worked Biologics. AGC closely with

mock FDA FDA performed have inspectors, inspections past using or We

the may FDA issues an may be an to that to by conducted for EMA comparison way the in in inspection well addressed identifying be mock that need FDA, through going inspection. inspection it so

very this of to so And place a by the for activity with lot in has us, been inspection. subject successful working and a of are put them we everything closely FDA

Pete Stavropoulos

broader more with in do a patients about remind you a for U.S. when you for approved, also think sort Thank the approved you label, you restrictive you be us, of just a And if anticipate or that. can similar know, label? it'll the label, melodies

Bobby Gaspar

and let any ability in that, Well, disease, and But hand with independently I the either me symptomatic EU information. to symptomatic the extra Libmeldy late children for for normal to the the or infantile essentially the patients IQ. and early patients defined early the Brain can still in is symptomatic, approved of also have juvenile early who with form of for then it over it form EU, pre start the and is by have individuals, who early walk symptoms as juvenile

similar with applying be FDA. very the a label will We for

I add what said don't there. Braden, to want to whether just so, know And I've you anything

Braden Parker

I characterized think it you've we'll, Bobby.

Bobby Gaspar

you. question. I hope Thanks, thank that Pete. Okay, answers your

Pete Stavropoulos

other question, risk And for move filing don't the those INDs focused disease indeed. relative give shift that does from last you for indications? and larger indications risk indications indications profile benefit neurodevelopmental that you for as findings your on, key of so one benefit you're the of like MLD remain larger if confidence in It what are just sort Crohn's platform forward some you mind, to these the currently favorable the may will and

Bobby Gaspar

we've we've important you. one an very can carefully Yeah. target. as thought Thank which about diseases This that is we that question, selecting considered

with Crohn's disease biolemic is form severe these specific that have suggests outreach disease, Kol and form these a and genetic of disease. our in the literature it individuals NODX. Crohn's mutations gene of called most for are So Crohn's the a individuals And

biologics individuals form surgery, and disease. these the stricturing the high have they and a are of to fistulizing rate or of who are refractory have So immunosuppressants they

high still currently despite what's need. So available, is there a unmet

severe the taking disease, disease, the of form autologous of form this with individuals in trials many of part that know are transplantation. also the We

reintroduced. unmodified to need and conditioning where Chemotherapy So autoliger cells have. where they are

this for with intervention form individuals that is of of something the warranted is So that those disease. severe still kind

Now, so gene will providing individuals cells believe going obviously, is selecting nod and providing with one we be it two step and modified, justified. we further gene modified mutations

In addition more to intensity conditioning intervention to on regimes, make form reduced population. a that, applicable we which again, are this also of may working wider

Operator

Our comes you. from Cowan. with [Operator next question instructions] Thank Yarn Werber TD.

You may proceed.

Brendan Smith

Yarn. Hi, the the this is Brendan on for Thanks filing. congrats on questions and taking for

just Can really or through We're this of have year U.S. would of just I quick the next what really potential to for from Sales one cadence European at XXXX? then expectations understand launch a synergies us. walk point the that And U.S. expedite maybe up. a follow potential later you learnings kind and help trying from Just Launch? into us your a

Bobby Gaspar

thanks well, and one I'll that for Commercial Chief that over hand Okay, Officer. Braden, to our

Braden Parker

Yeah, for that you question. thank

and of terms they In in Launch, we the number the terms of from to treatment how of would the size terms are U.S. personnel, think approaches a have. similar there the of in Europe apply centers of learnings very I would that qualified market,

of market standpoint. an size that You applying and rate, the look they're the at pretty from similar incidence

versus differences the in I see time is advance of be of any think negotiations. main that where there through you of period in you're lengthy pricing will assessments technology health that the not type going Europe U.S.

The reimbursement will come than much Europe. sooner in has in the it U.S.

you MLD launch, and kind think that will ads the launch. early Europe you the as patients identifying But of almost but much be very if of trajectory, of sales this opportunity, sales so nature will similar, as seen So you the than within in happen launch will in of know, Europe, likely soon given number we post of similar window in and we've uneven years of the patients the nature be that patient well. in those ramp will as see anticipate, launch did sooner and the they and about challenges the likely do treatment

will predictability have newborn little a you that until gets of more which place, bit universal screening patient It of provide in within treatment window identification much lumpy opportunity.

Brendan Smith

then great All and up. quick just follow right, one

and I for if of kind just you what's pivotal missed getting in FDA, remind a up initiating this or know sites selecting the a the study? this. Sorry I a is bit, on it us MPS? just before of touched with Can you matter study really left finalizing it's pivotal but running One, maybe design and actually

Bobby Gaspar

take we the FDA, is regulatory The final any far do path as concerned kind to order that. design in have to study I'll the study. with open further Thanks. of is so don't anything as

kind ready sites multicenter, So logistics now is trial. practicalities of it the a just will getting for controlled and randomized, of really what be

of that and study it So into you that imagine we getting anticipate end is the of goes in all the year. opening can the work place just that and by the

Operator

call for I'd you. like to the remarks. over now turn Thank any Gaspar Bobby closing to back

Bobby Gaspar

very you thank much. Okay,

so devastation Orchard's work much I Say. your which support your very approach curative genetic to. therapy. time, platform the of for that vision HSE to has and gene a Thank through for this we by want your questions end these your and just your to realize And you we think as attention potential severe diseases caused

So thank very you much.

Operator

call. Thank you. Thank conference for This concludes participating. today's

disconnect. now may You

Orchard Therapeutics (ORTX) 3 Aug 232023 Q2 Earnings call transcript