ORTX - Earnings call transcript Q1 2019 | Orchard Therapeutics plc

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Operator

Good day, ladies and gentlemen, and welcome to the Orchard Therapeutics First Quarter 2019 Earnings Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] And as a reminder, today’s conference call is being recorded. I’d now like to turn the conference over to Renee Leck, Director of Investor Relations. ahead. go Please

Renee Leck

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

You from access section can slides of the going our investors to website orchardtx.com. today's call by

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

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

Mark Rothera

and Neurometabolic everyone progress we of call, last update a expansion announcements the on Welcome related weekend. morning’s you you this clinical-stage we made with our plan to Renee. over today’s discuss holiday long I of quarter have the On Thanks, Disease exciting a Portfolio to enjoyed many and new the hope program.

into scientific platform. spend Before just about our and a the I’ll dive recent news, you we vision few minutes Orchard’s reminding

vision transforming of pureplay therapy world. focused lives gene and company the a are singular with a We around life-threatening with patients the diseases approach

diseases, deficiencies technology area, because has In WAS, The Recall today now concept majority stem EMA systems. XXXX a anticipated primary programs remarks we proof today diseases, gene in have in with with syndrome, in two filing but immune validated. morning’s in three filings filings physiological and lies regulatory and the immune focus platform also that five hemoglobinopathies. deficiencies cell autologous with will disease preparing in neurometabolic developing on that genetic or news, Wiskott-Aldrich rare this in clinical our different and expertise different ex areas our anticipated have primary HSC clinically in focus of XXXX. ADA-SCID we of of hematopoietic FDA FDA across Our vivo five been franchise we on therapies programs an are or a

we addition, at making in In are last regulators in quarters. good and the like clinical with discuss proof coming progress registration would designing in declared end of and X-CGD concept in year trial that the ASH we to points December

clinical pivotal next of Our to steps program further engage design regulators. as development CMC intend work, achieved proof in concept, recently and and transfusion-dependent, we also a beta-thalassemia trial, conduct process

more, designation or and has clinical therapy. portfolio datasets reminder, in a years gene prime follow-up going treated XXX out we we patients most response field and one have in the to believe this As of the X across showing With over of program of durability robust our Europe. comprehensive

prepare for to next developing that and product In the over to we expand Orchard multiple understanding deep diseases medicines a three potential invaluable treat rare continue team our global the to as and of years. will two addition, commercializing be has footprint launches

talk this where to franchise unique we diseases have by news, our of the items neurometabolic news Turning progress and I’m expansion morning’s to three thrilled about.

First, add to six we our exclusive in secured to an MPS-I for program license gene-therapy clinical-stage, worldwide portfolio.

signs share first regulatory Bobby Sanfilippo positive, of are Type finally, patient encouraging we the Europe. some in treatment to license. where timeline our more with the of was the of the for your news shortly. draw And Second, we attention Syndrome University Specials MLD Manchester will and wanted or a brought MPS-IIIA treated A program to forward under details out Early

MPS-I focus our on deal let’s announcement. Now,

that Italy Telethon clinical-stage asset entered onto and collaboration us with using transaction another This in new includes as expanded a our us clinical ex gives this partners an and We we vivo in forward be exciting Milan a that bring Fondazione us a diseases helped potentially to to academic technology. gene the development with drove HSC patient. portfolio to collaboration look San and their MPS-I patient. with in Orchard therapy through into these systematically patients. will treat program congratulate amongst a neurometabolic for continues common functions have with Milan, That of companies choice current for driven more We therapy an Ospedale data Raffaele is last want serious the and gene this to impacts intense by to Orchard which help and other a team patients centers debilitating care year which our transformative a with standard on condition, say his I of complications, cognitive inadequate. innovation skeletal by and highly focus delivering driven in remarks. completion within cardiovascular the the Bobby partner relationship to about secure market science the to built opportunity the medicines purpose GSK and company make the of to and

be to that and platform and We capabilities. to out our continue seek opportunistic leverage will programs secure exciting

results devastating our filing in in advanced cognitive function a] this and a loss at program very OTL-XXX of has leukodystrophy most MLD, the its is is age. rapidly progressing clinical characterized neurometabolic impressive indication metachromatic condition first Next in by [often approaching motor shown Europe. franchise, disease or young a and regulatory

Agency a early pre-MAA with As in European we conducted positive we this meeting announced May. morning, Medicines

submission half narrowing to We previous bringing our our XXXX are of guidance. forward the now first

leadership in we have of concept in as possible world. Our neurometabolic goal only to Further Europe, cementing proof this in diseases MPS-IIIA arena, will third trial starting get is in this declared that around the as therapy and patients the our the a to but be clinical year. condition markets the U.S. rapidly not

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news several diseases, achievement neurometabolic exciting plan, including milestones significant the thus far we’ve important of XXXX executing in year. made our also the progress on Beyond this

the In registration addition Marrow to the proof-of-concept we I mentioned Blood the previously; Transplant in multiple for data have releases we meeting MLD Most transfusion in recently, lead dependent and data beta-thalassemia programs. presented provided at March. European dataset our

to using line for have all dosed cryopreserved pleased programs registrational for of trials strategy the launch trial cryopreserved in clinical using a with Recall, formulation. a our our for also our WAS We’re fresh products. to OTL-XXX. were formulation first a the of formulation is in three conducted with patient the lead This

rest we will time the following of For use agenda. the our together,

our our quarter Next progress more will of financial our Thomas, up, Bobby Gaspar, milestones. opportunity portfolio franchise commentary detail. cash current about announced to how of today and detail which discuss will we market by Bobby? Chief provide also new some our credit Frank in facility, remaining neurometabolic review more view Financial and our our and runway. our in our XXXX Officer Chief with the return Scientific that our results Business Officer the and Chief Officer first review close addressed I’ll then impacts

Bobby Gaspar

technology entire using] first neurometabolic share you encouraging insights of to have therapy I’d and to Thank give three today. then the you, you time the some some portfolio. our spend across and reminding like into news where some we the [we Mark everyone. approach are And gene programs very hello

step a take back. let’s Third,

HSC with ex self-renew see give use gene means to the self-renewing. This modified is progeny, to This patients to stem is engrafted why or vivo gene approach approach indications. each Our our deliver rise once HSCs durability rare diseases. continues cells the which autologous marrow, powerful is HSCs severe very very rare in bone we across effect to modified because disease and cells of hematopoietic to the are

important talk characteristic has natural HSC in blood cells, and of microglia immune opportunity very multiple system, us process This already related including by to because blood and important conditions therefore we HSC going correcting and through other for that that the demonstrated cell importantly a the to exciting subset of therefore lineages, severe to back deliver macrophages, an are become genes is today, which correct in cell prospect to HSCs This in correct bank enzymes different the to models. diseases. red and microglia The the is diseases. going different is correct platelets, been the correction brain. a brain cell-types CNS that with multiple ability what for become multiple barrier pre-clinical is the extremely can we allow they have across of Most gives neurogenerative

platform HSC where Now, we let the setting. demonstrated in clinical application our me about have the MLD of talk

a with this MLD. this As month, EMA the to we we meeting announced morning, had pre-MAA for earlier positive

a We Enzyme forward for half our submission time limitations effective of specially condition blood-brain XXXX. as to there no deadly an replacement The to exciting are not neurogenerative is cross during line given development and are now therapy the in this. patients. ERT associated MLD brining options of due of significant first submission this available the for targeted acceleration is disease severe allogenic the such with to that inability barrier, treatment option the for the MAA transplantation

the MAA our patients. biological FDA. into globally benefit leveraging committed our with of are commercializing on to therapies We for application the the plan a license We

the undertaking activities our bridge plan U.S. approximately filing. current to development requirements our expectations the file in this Based year PLA regulatory after currently We’re of on to to EU anticipation the from a submission. MAA is

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clinical the in of exciting with ASGCT license Raffaele. as and to which program data part Telethon Fondazione recently The dependent same month’s the showcased collaboration transfusion patients, and expanded given an San for made at stage the emerging WAS we strategic meeting, However, was beta-thalassemia. our decision MLD, partners Ospedale last in fully program

different underlying replacement system. Hurler disease as systems efficacy form, comes ERP. brain limited and which Current which characteristics is There transplantation works multiorgan importantly requires patients a the neurodegenerative levels. products. therapy to severe limitations. one has activity caused the genotype, affect a nervous The multiple a including Allogeneic with is most syndrome, absence This mutations that central treatment an limitations, Now, in and and barrier the of is is talk have let the gene, its young disease. are The also multiple a blood-brain disease cross restored there strategies is skeleton, in down the that IDUA systems, best the could underlying commonly about the in to a of to buildup that breaks and of condition, of effective compounds its MPS-X. affects age is most treated enzyme to and heart, me and when the morbidity including and related a cause joints, bit the most lysosomal This enzyme allogeneic also is and of the significant multi-systemic mortality the genotype correct at when IDUA inability normal other Due storage the reactivity. risks by Transplantation little means transplantation with storage body. neurodegeneration. enzyme significant enzyme of known correction

successfully defects. to cells cells. high who The problems. intravenously the with approach, that corrected severe blood-brain to MPS-I gene the to also level being have and is which in is term approach deliver in then Our correct gene encouraging peripheral has HSCs approach data the become IDUA and been the to This clinical express gene cells produced correction cross over brain modified of enzyme HSCs in gene gene enzyme microglia, leads the secrete autologous patients of neurological that barrier modified preliminary similar autologous to produce long in used treated This allows so IDUA disease. to patients with the those modified MLD

suggest Institute clouding, partners I A of syndrome, our hernia, will just by vector correction patients, the form patients in thoracolumbar tunnel in levels the to assessment few weeks in at these follow-up supranormal we patient overexpression parameters. on with growth, severe and the expression presented umbilical the cerebrospinal periphery forward Engraftment of of observed in disease, motor is to gene sufficient updating four peripheral ASGCT today point. this of congestion, improved we’re early carpal regime achieve the Rapid has conditioning contractures. trial. the concept marrow clouding with was and follow-up this for Telethon as bone total copy with and clinical the longest In assess metabolic more spine, enzyme As months, interactive, and resumed treated been with patients. summarize. all The age and selected number well-tolerated patient airway Clinically, gene such Hurler proof had with CNS the the fluid knee therapy of regression of nine and time three seen to the Raffaele ago, have all the you patients improvement. the was looking of the corneal San These all central confirming a correction upper of with in suggesting look the therapy. under skills, first of IDUA at urine We gag two features at advanced corneal this kyphosis approach.

confined from available of MPS-IIIA data X in severe to storage follow-up to the expect MPS-IIIA like as pre-clinical first last complete thermal space intend patients and in the programs where are MPS-IIIB enzymatic also update enrolment to to The XXXX. known licensed of University We results disorders buildup onset and We XXs. of give you deteriorating are I’d in in the leads wheelchairs often to is MLD like disease caused options. HSC teens compelling will Patients early in and defects. another gene, which disease impairment. gene vivo with This effective motor mortality and in cognitive disease. and no now early MPS-I cognitive our clinical showed later produced which In MPS-IIIA, believe and SGSH MLD to by data benefit this results function has the mutations clinical in One-year Sanfilippo B, up life to neurometabolic compounds leads half XXXX. will be severe MPS-I is have is A Manchester. defect and with MPS-IIIB. approach, significant of thermal ex therapy similar in These life are storage in and and treatment

trial use need of application modified UK there this that of year, MPS-IIIA later Mark when option MPS-IIIA enrolment later in research this the for recently HSCs the of to we used expect SGSH University the such We’re and Specials treat under treatment patient] the and and is suggest wish meantime, exciting on In well same other in technology family especially the year. of patient. to in grants investigators treatment to mentioned, patient the [at Manchester file open track gene pharmaceutical enzyme. unmet government overexpression We situations with same a this available. with Manchester clinical successful trial the team this is of at This of the three-month upon unlicensed for this University trial patient. embark Data the first an were the a licenses high no and engraftment product physician-initiated The treated its license. of development, as utilizing procedures as

an at to licensed our to and clinic on closing, the ASGCT. the regulatory the this program advance the program add European excited technology. also Manchester the data to also University of of from was MPS-IIIA shared have generated MPS-IIIB we’re of the this uses for summarize, of by We same MLD preclinical six University timing the and encouraging presented portfolio of programs. clinical program proof has heels into recently and To data which MPS-I In filing the now move This our year stage Manchester. concept

remarks. Frank me now to over Let hand his to make

Frank Thomas

Thanks, Bobby. for position. addition XXXX capital plan. today year execute cash has new business Financially, also have with to to the exception. very We no busy of strengthen a to already report new Orchard been news and well-capitalized some we was our remain our

We an and accessible we attractive million with regulatory balance into new portfolio, XXX the our next senior ways period credit available. extension and upon XX current with of As we The XX X%. with finance plus months. the closing out capabilities, to up Financial, two to million with million XX manufacturing interest that us facility tranches five-year of ended credit cash sheet funded months on position Today, QX builds to facility LIBOR we find MidCap under capital runway in which and facility this our borrow is capital cash footprint, allows have announced a There to investor already coupon our the into entered a million up and strong plus approximately build available at an base. of XX and continued rate commission XXXX. clinical cost most balance only the the growth provides the to certain is XX in the achievement and milestones of now we

the company we’ve a burn achieve milestones our an capital believe to cost the secured near-term allows to and interest impact and on minimal us additional we to with capital So, this period rate. attractive utilize only

of This our with build of support capital. Freemont, facility You construction some California. facility and in non-equity-based that will recall in equipment commitment might our manufacturing source to credit investment a

business in plan, our approach with our our to manufacturing the relationships programs in three a while rest for As of online This launches. includes as multiple redundancies preparing manufacturing. manage our CMO product our own we chain bring for supply existing are pipeline first for programs transfusion-dependent the the other such strategy beta-thalassemia, to This initial leveraging we our preclinical needs X-CGD, the enables to while us strategic commercial advancing. the site are establishing and taking Fremont and potential manufacturing launches, capacity

complementary for already structure therapies on part as a to because of risk, long-term our the of on MPS-I programs therapy and We future and utilizing reduce business deal And and These strategy. the focus world, drug maximizing our with our strategy. a doing efficiently to small patients milestone undertaking And to are the this find HSC The be remain we and both us we programs the high have possible. represents we grown work program. enhancers, the balance shareholders. payment good one this see to lines, to appropriate by succeed our touched potential Mark opportunities As probability optimal as delivered deliver continuing programs. quickly followed processing. cost gene innovations for with to products space The on capabilities we’ve in we our to were can vector of are production bringing automated gene way. in committed focused strategy, over product value while a therapy MPS where sales of new structure as also example upfront royalties development transduction new efficiency part enables of new and deal and the the efficient in capital program a to will technologies and net payments around stable and cell the success-based are improve cell goods by of

X.X quarter which You here. results financial can will this find briefly in the R&D million full morning's million market for the press the for commercialization first quarter first potential for cost expenses a to of to XXXX, XXXX XX.X highlight were used I late of was compared operations, release, first to cash the primarily as which company expenses acquired Strimvelis in cost GSK included the driven period due in in public prepare quarter three million accrued XXXX, This of in associated of pay for stage as same were first same primarily by XXXX. We cost million programs with The X.X expenses. period the development the down XXXX, and SG&A well to in in personnel XXXX. XX to clinical quarter April stage increase increase support programs. compared the from of XX.X about significant was of million XXXX.

that, related transition Mark. And I'll back call the to over Some on with for turn to services. GSK payments deferred now inventory transaction the and

Mark Rothera

Thanks, spending areas. a and market patient to close for want three market preparations. Frank. discussing by clinical stage opportunity The access efforts, minutes identification our few programs, I

the patient populations and X-CGD represent prevalent factors; not and into MPS-IIIA. the ultimately patients these speak various take two for will identification revenue underway MPS-I, clinical with work factors by MPS-I I’ll exist and of the programs. ADA-SCID, primary for team populations efforts with define that estimates and each market for ongoing in This deficiencies along indications we First, populations commercial year. Based stage these WAS in addressable the potential. of reimburse opportunity for for Prevalent of program, does opportunity our the let’s two and and the on our covers number per only account our to This include programs cover each countries moment. of neurometabolic additional orphan franchises and over is the billion These market immune indication. is diseases only that now now market that addition a access based the also $X infant drugs. estimate six on MLD, these

beta-thalassemia in that patients program approximately incidents clinical Our that and respectively countries achieved orphan X,XXX proof are drugs. there we of transfusion-dependent concept recently reimburse and in estimate XXX,XXX prevalent

Given the program. our size see in significant for the indication population, generation for of this potential patient we revenue

lead we ground MLD in mind regulatory multiple initiatives first the launches. ADA-SCID, as a As in prepare their filings, our for we key programs upcoming approach have few

The first involves identification. patient

We are deploying focused screening. education a and newborn on also two-pronged symptoms effort early around

ADA-SCID, For focus listed RUSP. educating on physician patient panel community our ADA-SCID the in the also symptomatology well on US that is documented is literature. in as and the is around the recommended uniform screening known

been in all XX screening the has implemented is stage US other been has in states over Newborn pilot or adopted countries. in XX and

For the to patient physicians for look of and diagnosis. and well-characterized of development. early assay This shorten as these make known time pilot newborn more much collaboration organizations. to also define work with are and is widely A underway then screening signs could in MLD, through we disease the this studies

to patients’ our few views extremely market Now, access. The with therapies let that transform outcomes notion. the These are reinforces potential a valuable sharing of durability demonstrated administration. minutes lives about date to have spend this me single a

is appropriate now, done scientific therapies to as faster structures pay as possible. and as goal is evolving for ones many than these these in innovation get Our as therapies. place to Right patients fast to the possible

time, we’re our with once payer different arrangements, sustained various collaborating include, with tanks given better reflect evolve by with a will at-risk the working These we we’re focus that the options on created the advocates, partners. therapies. current be policymakers, system options payors, the and more preferences outcomes to At of to are which to limited offer broadly to meet comfortable think system of flexible payment same paradigm near-term range not the creatively the and these needs also seeing. done payment current is but and Our

therapies. health For US and really of the cures MIT NEWDIGS active some these the with instance, paying where initiative, partner the across care to in of approaches implement financing tests we novel for we stakeholders very identify to are in continuum

the like XXXX. Q&A, remind for on achieve Before of to we the open I’d milestones of the you of track by we list line to important remain end

present in for product Firstly, cryopreserved OTL-XXX the formulation studies ADA-SCID. data for OTL-XXX MLB from

OTL-XXX data follow-up registrational WAS. published new These Secondly, questions. on your for for and and engage regulated now filings regulators open products And three-year trial in XXXX for lead I’ll trial with for to registrational X-CGD. in for the from for patients the XXXX. finally, three catalysts call Operator? the patients design OTL-XXX X all potential

Operator

[Operator our first comes Rama Thank from question from you. Instructions] And JPMorgan. Anupam

line Your is now open.

Anupam Rama

for the congrats Hi, the all guys. Thanks and on progress. taking question

leaves MLD, of XXXX? two that to four the For you to program between And ASGCT second I/II of half the so kind through phase OTL-XXX several you patients that up ongoing then be be in need the that MPS-I, think to patients enroll the enroll, that patients that EMA has remind gating FDA moved and in have timelines completed to what EMA for first I of was also in can the question, discussion allowed the it there slide identified. slide a that, been suggested us would suggested the and factors six, know, filings? been And you

Why you given can’t complete the all potentially is first been XXXX? much. of that’s the for progress the sooner enrollment made? So, the so half why guidance Thanks

Mark Rothera

and very you Yes. Good thank much morning, the for Anupam, question.

to regulatory, as said, upcoming address forward really legal, XXXX marketing view that is -- showstoppers going as meetings, to you that view which, the means essentially, aimed facilitate �� first are our first of positive in assessment validation scientific in and So, will We from had the specific, order starting guidance finalizing we’re know, an that this, the authorization and of meeting with XXXX. we MLD, with today, pre-MAA And to product subsequent applicants issues of meetings typically application, you in at you know, going they interaction. and didn't any, half these that kind them assist information see to that’s these providing application. and a we in we a Europe, know, bring

a you know, now You first get about and from of development being you done, filing we’re cryo required. and undertaking what the know, way US we’re anticipating demonstrating on elements XXXX. to, particular, US, then an know, fresh of work. EU need as those formulation half in formulation, to undertaking filing we’re we're and regard that In as we confident CMC performs sort of focusing cells, focusing again in really cryo same know regulatory activities bridge filing that or also cryo-formulation And is what we the to some engraftment with are data the and to on

the we inform the we points use refine multiple to think strategies. learnings have next to and FDA filing the with need going we’re the with what over from FDA program we our months on XX expect touch to to that we ADA-SCID as conversations So, do, already the

think I that So, question. answers first your

today, four they in of regard with this patients their data that patients really inadequacies the quite really to the that have syndrome they at patients, that presentation through, additional the felt coming we on Now of therapy the would current that us all MPS-I ASGCT allude issues face which the on but to what presented question, available. first and treatment focus second you're patient, today did was we right inform address Hurler the start potential particular, of on to to

Operator

comes Cowen. question from Thank you. And next from your Baral Ritu

open. now is line Your

Ritu Baral

to data so the data the a patients four you in enzyme activity guys. on morning, to for question. in VCN, talk data little and the on XX, more. bit on taking this ASGCT want set I Good the far? seen maybe the well variability a into Can dig did the MPS-I as the Thanks Slide little

Mark Rothera

question few a with, hand Yes. Bobby. I would over to maybe words for Ritu. and say Thanks start the to

to and data, not suffering with care And, of syndrome about everybody reason is really providing those of proof advanced of four where alluded that think I function needs we particularly to program in cardiovascular, skeletal know, think MPS-I which issues. patients the with the standard know you encouraging. preliminary sort concent that these off You the we’re is is current important already is well just addressing Hurler it start the the patients, clinical is its thing remind cognitive, excited study to this of we they’re

in to patient follow-up of date Slide that the so could follow-up, particular, data and you're nine speak to think longest you I relating I And then XX. maybe this patient is so, one months ask Bobby to just far on with to alluding

Bobby Gaspar

Yes.

– by think we’re far. so encouraged got I that data we’ve well, the – so

We’ve can So, dosed. two mid-term at we reconstitution. as patients look where know, relatively have been patients four the you got

hematological in the patients, levels now the have months and levels these of CSF, So, also been good in has in seeing periphery, patient those patients, we’re engrafted, both numbers IDUA GAG that there patients, evaluation in and also both the vector supraphysiological seeing has IDUA reduction we’re significant of process, periphery expression of one in also conditioning patients in those therapy seeing to and copy increased the well. reconstitution as supernormal CSF nine the GAGs in after in the is start expression who and reduction in CSF post-gene had now also – in we we’re

so and peripheral longer in that seeing the reduction follow-up, in correct patients in above obviously, the these GAG at we’re will of in – disease, increase aspects long correct should patient, normal CSF and over well. levels, enzymatic levels in both the of as an So, of expression enzyme one peripheral the term the expression and – peripheral IDUA

correction far concerned. as which CNS is encouraging – as So is

Mark Rothera

with And is because what expression well. physiological its levels as MLD we super saw

Bobby Gaspar

Yes.

And over to there’s of underlying so, that is expression yes, demonstrate lot MPS-I necessary what is I that correct to think data disease. the a for

expression, aspects you achieved outcome periphery transplant. after the patients demonstrate an So, the transplantation, best correct When you different – in condition. normal the IDUA show but alginate to course the the that been have of the of look in that levels of MURI this transplantation, over can't is alginate best models who at it required outcomes expression over where disease expression states the – all of

of all talks ultimately this express that So, you the best when enzyme. IDUA you fact to results get the the

clinical, So, again, this in follow-up in we’re but in there’s and months early is activity. nine disease, IDUA that patient encouraging data one this it's seeing

Ritu Baral

lines green the the in lines So, column, spot individual data, spot, XX, third are blood green on those Slide patient the lines? blood the

Bobby Gaspar

at points. multiple one No, different on. that in That’s just patient time

you're… what So,

Ritu Baral

Okay.

Bobby Gaspar

product and at of all one-month, in vector copy the is post-gene drug and light months X looking So, consistent, those the number months all bone it’s they then which look DP very consistent points you're VCN therapy at three time data. and of shaded, accessed what’re and the been seeing which marrow, has and six the at those, in in bone is so between its X.X so different marrow very actually a at

Ritu Baral

patients it. function, abnormalities? you an then, skeletal the Are measured needle neurocognitive it the – what where age these to the the especially those? And as move actually mentioned, Got could in relates the what clinical are the of being Mark endpoints on study

Bobby Gaspar

which to at some neurocognitive So, also have designed which something a at proof-of-concept bright and primarily functions, neurocognitive, what we we There the study, activity at safety an function. it’s spots. which this planning. look secondary at to in – This you isn't study, know will look other looking – so is is designed and to endpoint in is look IDUA look at in to it's look that are blood would at look the are also endpoints, mainly not study primary but a follow-up to registration are early

Ritu Baral

when it. additional MPS-I? from these present patients you follow-up do think And, might patients additional and Got data from you early

Bobby Gaspar

we little in it's that, presentations, through on the same have I or well. that think a we given that abstracts meetings medical you but do guided guide data sharing advance to as program of this will past, the in in at early

Ritu Baral

might for it XXXX, a or this expect an be we thing? Could to end of XXXX the update

Mark Rothera

I something to have think that we'll that's discuss.

that. know and of made see the we'll when to the be we'll announcing license program being we're going more of are course, this once obviously follow-up we these of at patients, Of presentations are future see and inform dosed, conferences, today, then you patients how

Ritu Baral

questions. for enough. all Fair Thanks taking the

Mark Rothera

you, Thank Ritu.

Operator

next Thank from Securities. question comes David from And, Nierengarten you. Wedbush our

is now Your open. line

David Nierengarten

had Hi. taking a couple I questions. Thanks my MPS-I. for also on

the to double-check course, with that company differences then, an a design, there, Just a – the program, differences out Thanks. to there's at between MPS-I on level ERT. program wanted in increases the in these preclinical in keep you stage. Are just program preclinical not important your And are patients of there that. without anymore? your peer had I exposure previous or double-check or as advances? all there ERT taking mind any confidence on programs the comparisons to the to Are on study and with

Mark Rothera

over I'll Bobby. to start off with the then second hand question,

are point I in this it's suppose And, of the most easy a severe programs, this Hurler highly again, with and way so, important the slot But, portfolio and care look fits and track well our is that standard study, well current think engaging and is is is well syndrome. this we've patients on inadequate, the down completed so, of them extremely an we data. and has down of with approached by debilitating think this, a which condition, this particularly into we we preclinical I strong you that at form, encouraging proof-of-concept clinical already phase the franchise, already is area, have first SR-TIGET. WAS, that that fact relationship the We diseases neurometabolic to and beta-thalassemia this of program. in MLD, enrolling know, working this we because with is work most also them, in clinical-stage with expertise advanced on program lot is attracted program, a and are have a the with this you and the we as a work on when

was the it reason current complements felt primary we was portfolio. license this, and So, our that to important it

So, with I'll question. hand to to maybe second that, Bobby over address your

Bobby Gaspar

who is with so, treated with are and who this patients patients it David, both study, open have So, ERT not ERT. been in have treated – there been to

a mixture that is so there far. in study of this So,

are developed who ERT, who obviously and patients ERT some any have there have previously exposed have So, been patients there on therefore have not are don't also some previously, to antibodies. and who antibody production to and been ERT anti-IDUA

far ERT mix therapy regime to procedure. who The patients and answers gene hope the to as The developed that adjusted prior your patients conditioning anti-IDUA a I is induce to all tolerance concerned. are also those it as have question. may So, is stop try antibodies.

David Nierengarten

That's at. exactly I Thank does. what getting you. was That

Mark Rothera

David. you, Thank

Bobby Gaspar

you. Thank

Operator

next Goldman question from Suvannavejh Thank Graig you. And, Sachs. our comes from

Your line is now open.

Graig Suvannavejh

your congrats could. you first could decided Good your comment impacts they've we shared had beta-thalassemia on you. and appreciate you. the the shapes thoughts that market Zolgensma the thank seeing to just remind Novartis just could comments around announced with if just, second they and strategy. all see competitive question was program? progress. morning wondering you on Thank the that what access. where two or you My data Great, – – given then, where have opportunity how on with on and given is, I that week, just approach last how I for questions, landscape their I us, you're the go product my program, added And just exciting how ahead and I the you Clearly, pricing and you're thinking just overall how approved Novartis's if pricing? you

Mark Rothera

highly XX,XXX per with begin you to personalized for Okay, if the is great. Thank this prevalence is globally, you, beta-thal very I'll The Graig. question. remind is XXX,XXX, indication so available. large year. a incidence about that I'd of a the near patients beta-thalassemia medicine you be look made to

the a we the proof-of-concept there to therapy ours, and is data – that conditioning, entrant as itself, of speak in is the seen study, allows well a primary that vector, population, patient ourselves. like hypothesis second to have Bobby, those and and number So, for how you elements not of the to study us elements maybe room our position the speak could to but gene really encouraging study differentiation and just

Bobby Gaspar

we've cell is a think about than treated the well years severe age so, high – who one-off transfusion-independent to year. have of cell correlates severity terms you this is remind blood one which the as underlying per have to the younger pediatric Also, of levels administration. Just so, patients patient genotype, XX transfusions after patients red population, that, patients of the become treated blood of also of things so in population red transfusions, that the been I of far,

some are population that other to in in differences there comparison programs. So, treated the have been

remind you both the to to certain using thing also, beta-globin important the wild-type The of again other an in region a is we're that locus mutation. control there that beta-globin enhancer The within And differences of are vector LCR, say, vector terms differentiated gene the doesn't contain vector. and just within anti-sickling region, also, regions intronic the regulatory itself.

vector, what programs. different other So, conditioning between those the the The has and patient well. been been are in done has factors also all of as protocol different population,

to says, an is forward Mark So, program opportunity patient what I there's population. very this large move differentiated think, as into a

Mark Rothera

good transformative delighted to recent address question, your journey, announcement. another see families, option to world, new suffer Novartis' be we're that be see patients. for it's coming to made could second available and their a the and treatments these SMA terrible with perhaps so, like to So, Patients

we Now, complex, a looking individualized at things treatments. our take and of clearly, technical, really medical when programs, it approach to we'll like comes individually. highly all them are These be to need highly pricing, personalized all and to value

person's and about, talking as looking We’re these curing you stem the or in gene, a know, at and harvesting stopping own potentially single returning a these conditions defective administration. copy a inserting to patients, we're of cells, working

shouldn't and that So, impact to underestimated. would be that by the free-up otherwise potential tremendous over once-and-done nature of therapies has these patients, long-term resources the healthcare be financial consumed

of also, over the said, We in programs as of ourselves system. programs cost one-off to part treatments. possible point and the which I at offsets of long we're medical far about term, call. open conversation can reimbursement and thinking earnings As the how and view curative that value chronic was care the as do going to to adapt or see the to really we these system, payer originally as be of the transformative a we that, set up actual but the of structures looking our each different successfully We're integrate one for that from really to a alluded in

that how model. payers do in possibility tailor engaged payments, to think on think a are we proposition, the or are once stage There we the things have to different open we've need value We're stakeholders needs. but and one-size-fits-all don't We about risk different many payment agreed like needs, it's to many I sharing may the their conversation. this with model of

Operator

And, question-and-answer to to you. our concludes turn like Thank conference Rothera remarks. for over that Mark back for today. the closing I'd session

Mark Rothera

arena the making just I exciting today's neurometabolic call, diseases the for across to the this joining rest really, especially with wanted announcements progress we're the morning's everyone and Well, of in thank that portfolio. also

realizing and the vision closer transforming of closer diseases lives the innovative through around rare I we're of really gene therapies. with to think advancing Orchard patients our world fundamentally

with you. next that, I to very opportunity forward So, to the with look much, speak thank you and

Operator

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

all may good You Everyone, disconnect. a have day.

Orchard Therapeutics (ORTX) 28 May 192019 Q1 Earnings call transcript