- Earnings call transcript Q3 2022

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Operator

Good afternoon, and welcome to the Sarepta Therapeutics Third Quarter 2022 Earnings Call.

As a reminder, today’s program is being recorded. [Operator Instructions] At this time, I’ll turn the call over to Mary Jenkins, Senior Manager, Investor Relations. Please go ahead.

Mary Jenkins

Thank you, Kathy, and thank you for joining today’s call. our for released XXXX. third we today, financial the results quarter Earlier Exchange afternoon. press our filed XX-Q and on is with earlier at Securities The available our website release sarepta.com, and the Commission this was

statements. Estepan, number Joining us today the These take formal the slide Sarepta’s and call, of webcast, call the business, Please which that on materially common forward-looking control. call our differ will contains for statements of of beyond for Louise After and making we’ll results are like the forward-looking risks we Dallan to during to Ingram, our Rodino-Klapac. note moment Actual many can and forward-looking are Dr. any uncertainties, remarks, review these adversely could trading Murray Doug risks on the forward-looking Ian a prices materially statements. open stock. Q&A. be a from I’d this and and affect Sarepta’s operations results involve which statements, our such

a President we the the The encourage our overview statements, CEO, risks to quarterly circumstances. an on the our you and Doug? other filed SEC subsequent not call as Doug filings. provided And the provide publicly does events Ingram, detailed progress. will of obligation now any update projections and Company’s undertake uncertainties, as forward-looking financial report including to over or its with who SEC any review today based recent most recent well Company’s I’ll applicable Company to description For Form on XX-Q of turn

Doug Ingram

Commencing Therapeutics seen results and approved therapies joining you everyone, afternoon, financial for three VYONDYS release, and with the XX, our Mary. call. had with in we strong our performance, XX XXXX quarter have another quarter as community Sarepta Thank EXONDYS you, XX. will conference AMONDYS third you serving Good thank

second same in of to quarter. million net million second for representing we quarter million the in product performance, total total that, $XXX our you, and was And year for was strong remind quarter, million. the revenue and on between light third approximately growth year. comfortable that revenue quarter $XXX.X $X even about quarter the XX% mentioned with and $XXX the and the $XXX.X full revenue our guidance continuing between we booked our raised $XXX product quarter $XXX revenue came over to million, last our And guidance million and we with remain in To in As nearly guidance. call, net third million second anticipated a was

therapy a move for SRP-XXXX, platform one let’s now community to that XXXX So, our gene and momentous has dystrophy. therapy muscular for and been the Duchenne SRP-XXXX to our we serve. for specifically Duchenne gene

the with FDA possibility an Over basis. we half submitting of BLA application of biologics approval XXXX, first course discussed the license the of SRP-XXXX or accelerated on the a for the of

patients. And a for As and the third intended treat of indeed submit ambulatory feedback a did our in the we discussions second we announced the call approval SRP-XXXX we for Duchenne submit to that received, SRP-XXXX. our written on that we result BLA quarter to of BLA quarter, those

is accelerated anticipate Now, launch BLA the of approval trying so you are but here and me XXXX of To why the by date accept those our who a appropriate by moment urgently, about at merely the asked why for end just is and in have efforts. science to seeking XXXX. let not if to a filing of year. of compelled FDA we talk of move good if May goes the all for this a middle plan, November on We will BLA PDUFA successful,

living with and time And patients It justified is approval good. time, brings an noted to and that approval extended modern appropriate. patients in including is innovative to Duchenne. with AIDS, pathway saved FDA accelerated has do same accelerated evidence. The And in ideal in HIV approval tool patients opportunity gene and living therapies by with disease from for for the intervene appropriate We’re patients countless therapy cancers and perspective, the therapies at living has it of the actually SRP-XXXX itself results repeatedly accelerated innovation. an And review. where candidate explosion our an lives,

should this of robs the exists patients ultimately Time well-established killing to to living patients irreversibly enemy consider, that now. daily family someday need to them. every Let’s now, and suffer eventually. Preventing irreversibly their further is and with is when and therapy the need an bring Duchenne. have Duchenne hourly their muscle greatest basis, of function children not a first, those pathway or not of disease progression therapy them on regulatory It

shortened we and clinical Duchenne and by on benefits to supported monogenic the scientific a the accelerated endpoint of surrogate results confirm approval. for to a is wealth founded the disease, disease as precedent underway for are Second, is biomarker FDA addressing functional by one evidence well date shortened robustly proximate preclinical, the produced and on a dystrophin therapies a of cause based functional accelerated well-characterized upstream using approved endpoint. an surrogate well-established one dystrophin has as SRP-XXXX related functional of And one approval, that finally,

and approval is to approval EMBARK. get in children this accelerated can Our risk dosed, invariable. the rapidly the fully accelerated granting expeditiously harm of not proposed risk be EMBARK patients is we The if rolled accelerated fleetingly small, is and and of while confirmed so will Duchenne is seek confirmatory trial is did treatment severe fully certain, through approval

planning ramping manufacturing. BLA, the up are of So we for our success

services bolstered commercial, site on are launch patient access readiness. our teams, focused and and medical have and affairs, We we

as SRP-XXXX we as for patients soon commenced theme SRP-XXXX safely currently expand of of for to possible. look a the Duchenne are finalizing and study ENVISION, the the we population. expanding early we to have already percentage thus, the as exclusions on availability non-ambulatory to mutation a Additionally, EMBARK And protocols continuing patients the exon if our of expand larger of label to the commencement addressable population. Likewise, non-ambulatory for to the successful, study narrow in and

Next year, our in the commentary LGMD in goal effectively IgG with we to our those use and dosing our and positive Hansa plans SRP-XXXX. cleave with Dr. on well. partner, intend of of start safely explore with Duchenne Biopharma will XXXX the gene further permitting a rhXX imlifidase patients plans will include study therapy as to provide for Rodino-Klapac to

franchise. RNA our to moving Now

you As aware, approved AMONDYS. EXONDYS, today, and three PMOs have VYONDYS we are

XX, years The first EXONDYS six came of in those fall ago. XXXX, nearly approvals, of the

approvals, Scotia our the time. results in benefits our therapies EXONDYS of PMO in post-marketing had assent to continuing effect respect of opportunity we October, to addition versus to natural EXONDYS main prosecute At mission, two the with Conference we world on Muscle So, have Society evidence history in to evaluate the study and for presented the commitments those of the Halifax, over controls. Nova World

For those basis associated the the findings Dr. with interested, momentarily. you the EXONDYS. dystrophin. functional our in survival using discuss poster her will on Rodino-Klapac of Investor remarks these benefit internally on page will EXONDYS find an accelerated shortened approved website, including was

over denied perhaps additional a that would this have would with that those of degenerative AMONDYS value themselves And and to the lives with data as degenerative full encouraging disease, able well, without been VYONDYS therapy was living As to support is only it, as data that an benefits the generate never of and time. its approval disease. and and accelerated years this patients deadly long-term Without been least mature the a to of early at by have reveal approval we approval. patients

So, moving to peptide our PMO SRP-XXXX. next-generation RNA PPMO or therapy, the conjugated

on track year, We on this are dosing and are in to we MOMENTUM complete and XXXX. data have that a Part we’ll B, trial enrollment readout

commenting Finally, management on a transition.

had position Manufacture, be Bill fair of give that Technical a when to retiring. I able mission year. to I some Operations After an Bill our we Head retirement and launch to next impressive largely I’d would in SRP-XXXX thank Bill to goals. But persuasion I and and not announcing from been coax without like are big will to would our our X believe We of in was career, him science, tonight amount out approached be Bill and given whom you give of XXXX. Ciambrone, him retired important like the me,

to the SRP-XXXX launch and need capacity fully for analytical meet SRP-XXXX process to development to and the ensure and we had to First, of complete the community.

relatively has his to achieved that one a sustainable function with say a the short the Second, of intended large proud and to of in I period every focus as Bill a time. those make successor Sarepta’s technical goals. prepare to scale talent had to to operations build robust, that And impact third, am Bill ambitions.

strategic in with could involved more who the experience, Arif he and over has an the aligned Bill our best-in-class leadership and missing years XXX have intimately make of has the continue from Shire, absolutely forward. year. the transition imminently us the a process, of requirements have where to organization, Bill And date someone to has we drive ideal capacity will presumed activities including with we and over for operations an the of confident it beginning now will X and all at adviser manufacturing to and not our successor And Sarepta then has have the was I’m me XX over a with prepared end Bill. the we SRP-XXXX. success. our both PDUFA in with for Bilal for he will driving a through will He Bilal technical our that of launch SRP-XXXX the bill, Bilal, it personnel, We operations beat, organization as years successor. including aspects continue and a been Bill built success. stay XXXX closely without technical to after progress to of familiar operations us SRP-XXXX organization and XXXX. and priorities technical June well to people to at manufacturing this technical worked

executive partner at an enormous SRP-XXXX. me my well. will the for and remain a am colleagues by he’s that of I know thought for Now sentiment Bill And is involved that has pleased shared of the Sarepta, been all our he and very great approval us on advise and through impact I as committee. Board made

call for you’ve to the R&D you us Head Louise? over will So, in thank me Sarepta and you, and and turn Rodino-Klapac. Bill, that, putting to with of for great Louise Officer, for for Chief all let our And Dr. that done do be successful. this what Scientific position

Dr. Louise Rodino-Klapac

us for for bring our achieved beginning. and milestones potential their closer of SRP-XXXX to team the prouder community these forefront. both a FDA commitment, Duchenne at R&D is the of dosing mission ahead for schedule. could for Duchenne BLA study enrollment far and completion the of treatment program. therapy What submission I and third my and In The execution meaningful the we are the with of On regulatory quarter, and to note, critical [Technical our a Difficulty] EMBARK two gene perseverance accomplishments as keeping not be the Duchenne. and just the personally SRP-XXXX realizing we’ve thus while of professionally for personal accomplished of

genetic learnings are candidates I the have parallel, time therapies finding of therapy the SRP-XXXX genetic to to research. market, career them therapies. is my the medicine and paradigm My the forth from entirety strategy never been dedicated best our platform one with deep our development been We In Nearly and just singularly targeted array bringing aligned leveraging shift are of instead to focused producing widest and to a the applying the multiple our patients while treat professional genetic pipeline. that has create drug way to to on in bring shortens diseases. a team and

breakthrough compared therapies to efficiency deliver significantly early has is Broad lower greater with capsid. of adeno-associated new delivery of expression lower viruses at are platform the with doses of modified at greater potential the a another to and capsid MyoAAV advancement our doses. outer AAV in MyoAAV to genetic We MIT the natural Harvard. genetic protein that of shell gene group The a to uses Institute platform research medicine announce showing with pleased and offer or serotype

impressive Duchenne amenable for World a October. Sarepta’s In several when of compared at new patient-level Muscle had XXX PMO data focus X,XXX resulted The place data controlled presentations, eteplirsen Now years longer eteplirsen analysis a natural in patients with I an skipping. treated a with patients XX end to on Duchenne for XX from patients treatment our to significant containing natural benefit genetic highlight total, Society evidence compared presence to an our new exon that for Conference comparator key history survival study, took late-breaking in This moving portfolio. showed survived treated with that the in untreated end analysis real-world of group want across found medicine we to had history Duchenne patients. to on that posters moment and reproduced X.X therapies. of eteplirsen XXXX which presentation statistically I’d like to patients.

for lower XX most X.XXXX. models. to X.X-year to eteplirsen increased years benefit less two analysis The survival Patients greatest the than baseline natural and eteplirsen-treated equal Duchenne eteplirsen, be we of exposure hazard less which X.XX conducted longer on of control eteplirsen to between evidence X.X-year benefit years therapy p-value years. add likely of with patients ratio survival sensitivity had consistent rates whom includes XX natural and showed was were Additionally, controls. natural ages where XX X-plus to with in survival to increased with delay than compared including The for with accomplishment for of is aged This years natural benefit. higher to to baseline equal have to history subgroup also all a new and on data totality on compared natural significantly a XX benefit compared the patients patients history of finally, patients the survival a hazard Duchenne And treated These those Other of for a is a arm slower lung ambulation, significant associated appear of cohorts, p has survival a of predicted p free eteplirsen was with with X.XX with patients p of from in X.X the in mortality where function, from milestone observed difference Also generally X.XX. of ratio across controls, and have decline history X.XXX. for prolonged were consistent to Patients robust and findings had loss history observed X.XXXX. a analytical to with X.XX to a of survival X a ventilation. in p-value findings of to the controls, was than patients finding And a are a of years longer time X Duchenne for p-value most XX% for the the included X.XXX. experienced is therapy of the where with years patients hazard with less Duchenne rate the to analysis X.XXXX. history leading conservative survival ratio for

Investor As Doug posters page our of available noted, the are full of on website. the details

Now, to and beginning gene turning therapy SRP-XXXX. with

critical mentioned outset thrilled time submitted study to to I we for commitment team. have a acknowledge dosing so effort enrollment much are deep activities moment like the team’s I’d we As undertaken Many period by so accomplish X can our quickly the to move of in the tremendous parallel, were able EMBARK so done work execution. a but Phase at BLA are FDA. of to of in short because completed to and the my take to remarks, we in the our

from SRP-XXXX data a preclinical supported to years and SRP-XXXX profile. time points, and was other Difficulty] across an of accelerated on score to dystrophin control. these for results benefit. a reasonably propensity trials, matched likely as XXX clinical SRP-XXXX positive approval biomarker or studies functional includes efficacy functional X after surrogate version well positive XXX, clinical analysis BLA endpoint Difficulty] results. Studies of safety including X safety functional as Our by addition The external to three ENDEAVOR demonstrated treatment consistent the In comparing X, Among based integrated in XXX, expression as results [Technical and multiple it at things, BLA is [Technical clinical predict

trials therapy, western the time these the manufacturing and treatment functional SRP-XXXX such data [Technical In X our [Technical for Difficulty] SRP-XXXX across the clinical motor independent commercial further studies secondary confidence consistency the the by functional endpoints process. and improvements material clinical and X and commercial clinical XX-meter success of data from of show process EMBARK. probability demonstrate Quantification supported the both rise is protein particular, our mean The expression from our expression key as NSAA ENDEAVOR to our improvement Importantly, confirms increasing and results our from Phase effect intended across immunofluorescence. of in by consistency function Difficulty] reinforced walk/run. blot of measured across

level conviction Additionally, to conducted being these EMBARK ENDEAVOR are the serve both EMBARK studies material. and increase same of for our because using

complement Importantly, across profile studies XXX, with activation. consistent safety of evidence and XXX, XXX, an clinically integrated analysis, relevant SRP-XXXX the of remains manageable no

the over our As are I enter because milestones agency, wanted remains leading to data consistent month I’ve encouraged a and at anticipate [Technical mentioned, dosing period note prepare our completed do sharing Difficulty] readout. EMBARK experience. with publicly review study up to completion. data we in and additional regulatory with safety that EMBARK cuts as XXXX previous we to The one not the profile

reaching We which mentioned, early we a Doug EMBARK. with are mutations possible, cohort currently that living narrow designed many the in the committed individuals as as of exon have to ENDEAVOUR to new Duchenne as study are part as commenced is excluded why,

working also in a ENVISION, evaluating are protocol the We patients. SRP-XXXX placebo-controlled for to study non-ambulatory finalize

we’re of the follow study enabling of for starting years. to in minimum study, an patients us gene process Additionally, extension therapy X out a for

limb-girdle dystrophy for priority wavered to Sarepta. a our Our has continues be commitment programs key and not to muscular

key natural component continues study are of that development pathway. enroll represents to history our We pleased our journey and a LGMD

for LGMDXB, is a absence the process We the [Technical SRP-XXXX, also third to a treat our will for in using ambulatory to commence the we is finally, mediated interest to It commercial dystrophin. science The vector with commencing in soon the goal exemplifies addition, non-ambulatory our plan SRP-XXXX which study LGMDXE for And Difficulty] the pilot of advancing with to soon and study study to quarter start in protein individuals best therapy XXXX. clinical by living be in systemic for commitment disease. rare an of material our dual SRP-XXXX, patients. progress additional rhXX gene characterized material

[Technical to grateful guided clinical are to us to We update dedicated them, turn who teams, bring call to enormously investigators trials along have support where to activities. who Dallan? are who and the clinical to mission thank you Difficulty] today. and in with the tirelessly our scientists participate professionals I’ll a forward we the and [Technical their over who Dallan patients our for again extraordinary families stand Again, experts Difficulty].

Dallan Murray

you, Thank Louise.

The third totaled XX, franchise. another in AMONDYS $XXX.X third revenue EXONDYS $XXX XX million Sarepta’s execution of for product million quarter with of for million XXXX the $XX represented for Net PMO RNA-based quarter quarter, $XX roughly and for XX. VYONDYS million

As quarter, and timing $X disproportionate a you’ll the for a We AMONDYS including quarter had in the results very to third guided July pull-forward the quarter. played anomaly we second of upfront, impact The quarter-over-quarter to Overall, million much recall growth from holiday due on XX patterns Doug on modestly for quarter is as had net QX. pull in fourth forward expected AMONDYS the ordering XX and to are as U.S. see the from we revenue population the patterns the to rate exactly the the AMONDYS fluctuations out vast revenue expect and guided. The the U.S. mentioned and in resulting important introduces rebound pull-forward gain by continue the revenue at quarter. third Xth third the an exon some XX than are as access XX. contributor the identify in we is revenue the to revenues U.S. EXONDYS XX been and grow XXXX. ordering quarter-to-quarter growth to the see an becoming XX what of EXONDYS our Ex has from different currently expectations, positively ex to majority as net and ex our and time, This growth impacted that sales these also coming continues note with important amenable product the skipping. net It’s same year-over-year, in we U.S. for rapidly Duchenne track

revenue revenue ex U.S. third grew relates the XX in while to it modestly, revenue, the quarter lumpiness the As ordering for overall the EXONDYS was by U.S. impacted patterns. quarter

quarter of whole, net $XXX.X nearly to to achieve XXXX. of revenues year million XX% We growth remain the revenue million the product represents track guidance for in over third as $XXX full third on million. our quarter a between Taken net the product $XXX total

now of on the to performance our quarter. of each PMO three the Moving during therapies RNA-based

in third the last third the XX For revenue EXONDYS we X% year. QX. from net XX, million EXONDYS $XXX revenue of note, quarter nearly in quarter represents To growth delivered net for

$XX [Technical XX% light $XX For of nearly than AMONDYS million. over revenue, pleased revenue particularly pull of compared million the XX are XXXX, XXXX. nearly we forward, the to quarter in of net XX, effect grew the VYONDYS third very the of with more quarter third of Difficulty] net in the with representing

and on leadership to is maintain have new therapy position exon amenable We our and population, our market efforts continuing in XX continued the team existing patients patients. their get

mentioned we we call, the on changes substantial coming VYONDYS any the As in for quarter expect quarters. don’t second

execution generation, we’ve In addition to revenue another accelerated of mid-XXXX. strong launch-ready for and quarter be to by SRP-XXXX our efforts

sites are what that believe lost The and will and will be be for office not the in the launch of the head launches. feel opportunity experienced a stride. ready SRP-XXXX’s we so our were as on each schedule, of of the one patients submission successful out lead magnitude have for ramp-up we of Further, quarter, BLA will organization were approval, R&D the our Duchenne, urgency customer our X colleagues as we day support this we at just leaders ready of building Currently, that executing started third is launch engaging on PMO ensure to our patient gene hit are on SRP-XXXX In and full teams us. they As team with all approval. ahead field community date. key to the most of therapy deeply will we we who

are the poised R&D therapy therapies. To absolutely data with our that to first with Duchenne launch gene incredible. market RNA leader be X Duchenne have is of the the approved colleagues the We generated X

want therapy note, On with colleagues share to to ready years the X build that take important personal the torch, R&D this a of to community. recently the Sarepta. celebrated I are our deliver efforts and I on Duchenne We

privilege a unwavering During serving has place take extensive The are this mission us witness execution we’ve over that, Ian the time, well, of since I experience ago. And has our to no an on it team’s we prepared and Estepan and proud our strong our launching PMO been six a I’m gained the dedication patients. to first to performance years commitment than that better to of on I’ll in and call financials. for is launch feel to the one SRP-XXXX. update with our Ian? turn

Ian Estepan

Thanks, press Dallan, well a This reconciliation financial third and of details all. on on available non-GAAP financial XXXX for basis results. good afternoon, non-GAAP afternoon’s of to GAAP press results quarter GAAP refer website the release our as for Please provided full to a our as release basis.

third the of recorded Company the $XX.X consists $XXX.X compared same of of XXXX compared X exon for was revenue to ended period net Net revenues PMO same XX, of revenues the our product total for to revenues which the million from period and months September collaboration $XXX.X skipping product XXXX, revenues increase XXXX. million, million million million. of of XXXX, quarter For $XXX.X for $XXX.X an franchise of the

collaboration For a On $XX.X manufacturing. primarily XXXX. of $XXX.X and is the the demand XXXX, GAAP September million a In of XXXX, U.S. third a $XX the certain to for GAAP approximately of AMONDYS million recorded and a of share quarter for in months expenses million XX XX. quarter of around EXONDYS, cost individual XXth due The basic product third of of $XX.X were in and $XXX.X reimbursable in XXXX write-offs XXXX, of due in VYONDYS $X.XX period $X.XX XXXX million to product and of We income sales XXXX of with per net third or The share million a product the batches months primarily period recognized million. year-over-year increasing the for $XX.X the three increase to of of reflects XXXX, to specifications $XX.X we terms. XXXX, respectively, in due the and an XXXX $XX.X basis, third the quarter which continuing diluted the diluted under The to sales or in quarter $XXX.X third million XX, $XX the or cost R&D of expenses share for increases and recorded world. basis, million In for primarily of approximately changes to in million three SRP-XXXX to loss the to third basic revenue, revenue $X.XX, the same XXXX and we a million per the for our net The third or million and to costs reported co-development the relates Roche. $X.XX compared million million agreement $X.X million XXXX respectively. $XXX.X of in million for net our for collaboration of quarters ended for $XX.X Roche XXXX. increase each XXXX, and totaled per the products payments in increase sales manufacturing our increasing for quarter in period to $XXX.X $XXX.X is increase third the compared the three loss the due XXXX, basis, of for increase $XX.X arrangement increase the products same million. million and of On and of same $XX of XXXX, reported XX, net R&D the we compared were diluted quarter non-GAAP quarter expenses our we non-GAAP as XXXX. September in of On basic offset net months our ramp-up ended for royalty a BioMarin not non-GAAP million compared $XX.X an of quarter royalty compared of September a meeting in during quality ended XX, partially by decrease demand September ended

of and for months On to compensation three additional million period ended expenses expenses GAAP the $XX.X CEO an due for recognized due XXXX of approximately $XX.X third $XX.X was SG&A turning to recorded third in of and is increase the basis, an the XX, over XXXX, we of Now for million of by respectively, in service expense, grant June to SG&A. to an recognized The XXXX, million. basis, stock-based compared On The XXXX the as the $XX.X were a primarily XXXX. expense non-GAAP million increase repayment of a the net in expense we a which XXXX, primarily our same $XXX.X period to of increase modification to quarter income months same basis, executed driven increase interest the $XX.X the period. the an quarter of for the million agreement XXXX incurred result the from senior had an other our reduction convertible GAAP increase third notes well due decrease In recorded of a quarter, XXXX. XXXX. expenses investment interest in we investment portfolio expenses net $XX.X the million $X.X mix million loan compared billion million. due quarter in other third ended of in December due as proceeds issuing On is three the September for of to $X.X for a term XX, of XXXX of as primarily

a prepayment the the of debt loan. XXXX the portion repurchased and convertible of XXXX outstanding million. resulted loss $XXX.X outstanding full prepaid extinguishment our notes amount of of term our and we a debt Additionally, in The to existing in repurchase on related

to XXXX, our the current We XX, as approximately cash approved. to Assuming Doug fund of XXXX, billion well this if turn I’ll call therefore, to along launch operations that sufficient $X.X we Q&A. offering investments with Doug? over are support of to and in start our the of projected that had SRP-XXXX, approval be and an profitability. September we now, And anticipate our to cash, capitalized and the will equivalents back revenue, cash

Doug Ingram

the questions and let’s for Thank you, open for that. Ian, call Kathy, answers.

Operator

Our Barclays. the Gena of Instructions] comes from question Wang line [Operator first of

Gena Wang

likely see you question SRP-XXXX. Any that And that it’s be have I that Study you the expect review possible review? FDA think the would mature mentioned May the concern X-year Doug, regarding if XXXX. in XXXX? to data will one like XXX you PDUFA date standard that in do could January

Doug Ingram

with in submission BLA, review. -- priority a we have requested our we course, So of our have connection of

our current expectation. for planning at we the We date, that, for that’s May PDUFA are and the mentioned that’s deadline have least why

that commentary That’s haven’t believe any the now. us that information right for planning is from we’re assume. anything received what agency different the So any to or lead what we And for. we would division

Operator

Our Tazeen comes line the of question BofA. of next Ahmad from

Tazeen Ahmad

are Thanks. get upon Doug, would you on you your complete need wanted would manufacturing do to to launch And commitments. for commitments some from What whatever show detail I FDA? you if before review, do have the just enough agreed allow time you you where to you this priority proceed? get with a

Doug Ingram

Thank you much, very Tazeen.

great review have So shape. this the obviously, division we’re of with but dialogue during we’ll in all the itself,

placebo-controlled bulk are work that CMC the the last well. will our of in basically required that we of done then and some and -- You dosed, EMBARK connection assay study, trial were our our with as to recall got the now we commencement before completed fully

CMC. good that So, shape I very think we’re in BLA and an is submission made the enormous respond very to of right the to of agency’s we amount any now questions, has detailed

right shape good we’re in now. very, very think I So

Operator

Blum next Our Company. of the comes of question Gil from Needham & line

Gil Blum

is helpful patient? this for maybe put difference a DMD in us. Doug, for context Can six-month a you So just

Doug Ingram

have between end so versus first working longer for that, submit on that just much day all assumption didn’t delta traditional sort accelerating every we It than QC not an the start were months. question would underlying that -- that is would just a -- a By all, clear would the because be I in is probably submission to and approval done we’re approval about of remember, let’s accelerated formally with of we the because compile time it that if -- of sometime wait be think We’d since seeking a your BLA. X non-accelerated be information Six versus ready were the to BLA existed might pathway exist XXXX. that. approval, of has we we as ‘XXs, that all EMBARK, was

getting priority the we end of get in back the XXXX. approval probably review, we’d half Assuming XXXX, very not be of

do. every day, not damaged, would and will that exaggeration, would Duchenne. a XXX damaged the been get currently probably that with so six But therapies of therapy basis therapy ways this delta before, months, they died. is Duchenne daily the the damaged even of on It I’ve some means this sun this the at over So, that then significant What is in Remember, or United a and other does those a monumental really sun therapy of and neither in patient when end it’s a not be comes arrest down the the have means is also has an reversed. children and not day our goes And X-month Difficulty] up -- States, that which very patient. what this rectify. time, or That’s in in patients life any clear, like of envisioned Duchenne is said lives that decline. a would [Technical

be And hundreds some wheelchair for number their the to of would rest a other lives. of confined significant

group will other wouldn’t lose And have. they additional of that milestones some them

of be provides sort my and on we that in for isn’t an that a is this should valuable good that. to founded And is really because XX seeking just And is to between will certainly maybe or be it bring upwards And on again, to XX,XXX that a , ventilator, accelerated XX,XXX and an there it’s here therapy inevitable, signs an script, we fighting compound and it to patients, that going X And should inevitable. with doing XXX a as opportunity months that months, hundred approval is delta every And of to XX% time sometimes X-month approval and be for when therapy them. will served an any single that period. valuable. damage ventilator life-ending It accelerated the or long XXX are to and every available realize unfortunately, accelerated is that that damaged There I would it’s of day purpose-built, is wait. those devastating have takes not this isn’t respect approval one science Duchenne. now. right the valuable to only is additional argue, population augmentation I be a not of But enormously real It’s on children science, about is Duchenne in few well And for mentioned I a because be neurodegenerative XX, some when XXX pathway we difference disease. like here. then that a a it’s something a approval the

then relates We this to the correlated children they’re absorber the and have downstream disease, XXXX proximate in fully EMBARK, these They’re as we why And Duchenne, I these why it’s a It’s perspective discovered. missing be now to eventually results here so final the since but monogenic say trial, we shock gene, placebo-controlled in is can relatively today, as directly rapidly. approval our protein thing our that when the and will are surrogate is is know protects it muscles. dosed. were its muscles know, dying accelerated cause endpoint some dystrophin their that their the on least, dying. you fully at from compelling of that because sit seeing confirm endpoint, actually enrolled. We’ve we known

so And basically about will be be isn’t search. will on trial confirmatory argument when a day there some It the complete. complete

they now saves in muscular innovation patients when clear, a associated we chronic I a in innumerable so diagnosis, would who with the the and Duchenne sentence be a availability history, lives. argue Cancers let’s with it a one had pathway, And can disease. live something with time dystrophy. that accelerated HIV had and pathway, AIDS like at death of that

that for wealth had dystrophin truncated we that support of associated our And see an that to have We to data the of perspective, certainly than as the AMONDYS. results EXONDYS, matured, SRP-XXXX really we now, emerged functional accelerated perhaps that. is our of time, has from data greater brings very VYONDYS we the an can So have, PMOs, the is that benefit and justified we the with order still Well, even that seek the it of numbers amount benefit and compelling and experience use patients. over approval and evidence magnitude functional enormous what the given patients of it. we have

unethical I hadn’t we So, mean it the had But -- sought thank as for it really question. evidence-rich in you my detailed very discussions with said would border script, think much it, on very be approval, FDA. I certainly and we accelerated after that would I --

Operator

of Our Bristow next Colin question from of UBS. line comes the

Colin Bristow

side. my from couple a Just

case C this I the here, I update the had, think regions believe on on contains you that region. change any lesion with recent of was protocol the course? XXX, region in further terminals First the then screen second, the Cohort of spans to just the exons it the out And any around in Pfizer’s myocarditis, patients insights XXXX X near [indiscernible],

issues concern immunogenicity proteins lacking that index of patients side region? potential in any on of Sort your for sort or around making

Doug Ingram

can to turn No of going provide question, to over. questions the Rodino-Klapac Dr. but those latter color. I’m additional both

Dr. Louise Rodino-Klapac

for our follow Thank Obviously, that question. you continuing to patients. we are

as continues occur as patients cardiac baseline. the other sustained As myocarditis recall, do had continues Duchenne, normal that. no, well in well, follow. updates with you increases these to There cardiac know, other you so in to increase. was and function, patient X But history function than can a trend in Cohort natural troponin Also, the

EMBARK, in of to to terms but excluded exclusions, mentioned, conducting X XX that down. narrow we’ve as X In we’ve study genetic the we’re Cohort our

program We the our for terminals with have had concerns any not C

Operator

come line from from Sachs. Goldman Richter next of will question the Our Salveen

Unidentified Analyst

EMBARK wondering potentially but sig MSAA you Tommy the discussed we’re XXXX, or on an on -- variability on approval get the progress. the what is scenario you accelerated would on due get you Congratulations for like could you’ve is factors? And stat This if AdCom? comment where Salveen. for And of other be that to likelihood can Thanks. not

Doug Ingram

Sure.

with So clear, I let hypothetical, first, on first start me be EMBARK. before confident one comment very let very we’re the that me on

powered. well very We’re

into its design study very informed. the XXs. is entire in We’re It informed. well is That well very well powered

confident is if question totality evidence the is underlying you the I issue And And the someone based certainly And we’re to the standard therapy remind entire the that from very justify question? what that availability confident. removing? Now not that, the of that at just notwithstanding it’s of I can actual removing that of the apologize. we on And didn’t we’re the with course, not then the the commit to That for the the if would totality therapy, have therapy. The the power to commit think And look That’s all the dealing is, standard standard. hit. second and data that essentially you but the the of of of on directly. you all on study, would would the is we Perhaps already EMBARK actual me of yes. the evidence of fact accelerated to continued answer do market approval.

Ian Estepan

AdCom.

Doug Ingram

The AdCom. short an agency from whether we that going the yet get haven’t answer we’re or is to heard not

late know and class, November. probably occur an certainly BLA, and the of after planning in AdCom spring that get XXXX. given for we’re the our will But we We that that which that its Apologies that. occur in would assuming should first shortly for the accepts would agency is in this

Operator

from with comes Matthew question Morgan Next Stanley.

Matthew Harrison

some just I just wanted primary real-world the that you of thoughts endpoint to for EMBARK. the ask in around your of and about some point the time data presented

just longer time think primary one one point if about the of lot you’ve looking that wonder is that study? concerned from in your right are year is in of study I confidence Thanks. potential the investors at a endpoint and a time I patients. things variability considered the for that

Doug Ingram

for for those and this and, us, very exciting it over the really kids opportunity to years for real-world see Yes. is first Thank all evidence a EXONDYS, follow to number of The had of EMBARK, we’ve you. emerge. --

different, all much it very of across truncated course, right? -- PMOs and reliably are therapies but across very amount a of dystrophin that ones remember, dystrophin, consistently our Now they making XXXX makes. all quantifiable of make and studies, it [indiscernible] of smaller the amount and of the PMOs make The does our it’s functional

Becker dystrophin, different to or life well over because again, as functional of clear, I the misunderstandings. been We’ve then, I we’re study, the XXXX, thing. the we time about informed dystrophins have So the very of of different And Duchenne kind well Becker all there as the does so makes design truncated the full-length and and is as that it. the XXXX approximations they’re of look impacts taken and And of version as say, on rigorous population a PMOs. a been frame. type child think we’ve just reasonable the study. And the again, a account of variability would heterogeneity into patient of the same

of to we own history addition our data a from experience, think the organizational have. an don’t least knows, everyone and we of experience perspective, has at wealth As have, in I natural kind anyone and

XXX data, Part XXX data, X data, the data, the X have multiple XXX, and Part cohorts. We

around So that And of study, the we’re assumptions think that study. relatively that make including powering informed in of that, we design conservative the on and well the of I in based all we assuming, XX%, assuming well variability said, we’re is amount standard very even as over we’re that and study, feel that of I’ve the of deviation confident the which, conservatively the in assumed. powering

feel good where about So, we and about time good feel are, XX-week we very frame. we very

of that’s I a XX Now weeks if delta can that say in that at you see on look XXXX the versus so a a trajectory time, keep period And XX it also see trial what of in life reading will already exciting actually but when that’s Duchenne you history. that the makes is out, patient disease expanding. significant weeks you short the natural you XXXX been for of fairly

X in expanding it seen You’ve years.

X. points the deals X-year you of XX-point where the a the the the with truly that at over would see get kids you XXX trajectory out cause with as change expect something literally root from disease to on you’re a when You transformative X and in mark way, the disease-modifying of disease scale

that now. with we’re the of largest it’s kind and a that, I we’re One, X and years, X we it. powering years, -- results. this what years, studies about the [Technical right its are It those on of and Difficulty] think hopefully, far we very means years, exists X but X excited X beyond year, XX approved. if get years And confident around of things very is lot two of the one conviction mark, So, have very comfortable the

Operator

next question Schwartz of line SVB Our is Securities. of Joe from the

Unidentified Analyst

for This taking question is on today. Beth you Thank for our Joe.

sufficient in X check, your just what consider have BLA I SRP-XXXX therapy than doing FDA current Pfizer use doing patients later. support there, if similar with X-year-old were we package regardless gene of a Thank if broadly initiate just you in their to is was X- the kind DMD maybe wondering patients to from filing than younger in desire ambulatory you also age, if data is to study support sense a to of to use ambulatory for For curious patients, our you. sooner doc study?

Doug Ingram

that we patient about of regulatory the for the approach population. a and parts applicable So, have as that standard taking remind to greater be of for the extrapolating understood. to standard feel well presumed broader well mechanism good a the we’re the population do would is confident seeking you approval as action and ambulatory us population When legal

then in the that You population expand were have a to to narrow ago, it the moment it population larger see the a to study broader deal populations. ability from to heterogeneity we effect, with comes discussing a as

we’ve why chosen to that’s population possible a ambulatory placebo-controlled trial we want on population. So, as for non-ambulatory soon get started the as the

the old non-ambulatory rapidly. in for we kids therapy And then are dosing ongoing go it actually kids about the want patient are population but because those than significantly our in lower talk phases this We that today, to X-year I’ll Louise -- EMBARK? I you think as as possible soon population non-ambulatory your XXX expand the to can turn protocol, a in but we point, planning we study to lower, need Rodino-Klapac, going are even to to

Dr. Louise Rodino-Klapac

go study Difficulty] younger [Technical as even that, to to years than Difficulty] old and well. that plan for [Technical Thanks we X the say

are we certainly, in this therapy So population. addresses that [Technical Difficulty] broadly

Operator

line RBC. of Abrahams Our Brian comes next of the question from

Brian Abrahams

to assays but be, -- in misheard then I I guess, going studies it, Maybe with to shifting then and there’s gears What’s commercial perhaps with And for subsequently, like scale CMC process material. clinical on X material there? it the the plan materials and latest it, development limb-girdle. a sounds now

combine how path? Just conduct might support lines these time and of to potential two pivotal any a sense maybe studies

Doug Ingram

essentially turn Yes. still color. of ready have for process the done assays broadest commercial additional to that of that is remind we’re standard working study a and agency an for of use, start I’ll commercial in on But the those assays isn’t it approval, I’ll To provide who XXXX. basis use can clinical strokes, could for the the say to everyone to sufficient. Louise, release the assays form the current

in But working we’re have study and is that. It and of So continuing that to goal on our to all does that. we’re XXXX. done commence that take time,

commercial X -- really we they in concurrently. of additional Why to interesting not for of but population really when assays clinical different then have both should and studies, is to out, studied And are have doing are not for we done the which significantly we very than trial you insight is X of pointed not material -- purposes They’re population material our you think our some the parts As in be year serially just patients available study occur other benefit process very unrelated us that and that pivotal answer your sense, complete? the give will XXXX. done that your would in what why have usable the is, next process. with intention question studies. are a be And us them studies? the is These real would the can commercial do with

time, study patients. our from and so, while from that a we at use And our same makes least perspective, that the perspective, and gain additional material insight only do benefiting sense it Louise? at

Dr. Louise Rodino-Klapac

[Technical Difficulty] non-ambulatory patients.

it significant mentioned, population. so, the And as Difficulty]. into insight us And entire [Technical Doug will give

population whole we’re And the Difficulty]. [Technical so capturing that with we’re sure making

Operator

question Skorney of the next at line comes Baird. B. from Our

Brian Skorney

have patient prepped think presumably and approvals. be like on this payers I payers? that I know launch, pushed guess, of I about guess, recently as potential demand sort back been we with pretty clinical H for are your sites far would conversations Subpart high. obviously, have what somewhat so

of Just the curve about trying think to the launch.

there’s in you accomplishment, H just of But Zolgensma. have you put approval? initial a to payers flexible that until think up some do out, be or EMBARK obviously of idea You’re ton of think get payers utilization at that relatively do the you on will with hurdles going negotiations data Subpart be

Doug Ingram

know. I’d guess, things. And me if couple say of miss I let I a Dallan, anything So,

able think open the make is with what is compliance we everybody first we’re go on to process news Dallan years, overcome to your and kids wouldn’t That approved which what say prior them we’ve battle-hardened been is ability been up get with has XX% therapy. We’ve been lots what want. the reauthorization get definitely therapy. well good to tremendous. hurdles to probably from if and the process go ability sure a for over be XX% humble overcome. very at say, with that is think patient CAGR therapy But even really to with from or demand of the been you the now keep between know think chronic that working to point, what will X enormous. remind number doing it it therapies be for I’ll point. will we’ve Because to and The -- We XX%, the our we’re would significantly X let’s we I perspective, obstacles understand work and can gene there relevant to payers, therapy, of makes on news in we been good be constrain too EXONDYS, rates things I good perspective years to that about adherence -- kids a these their demand, and just they and growing for that nearly doing. are therapy, is our respect we’ve on as First, the payers,

from at are not concerned patients desperate We’re get to this like therapy perspective. our all a

in physicians same be think place. will equally I the

as is the thing relates to prepared are third to capacity-related The it at that. well they issues sites, infuse

to served patients is are about XX infuse. sites available have fact, by expert goal to Our sites. XX of And XX% in about ultimately

very vast should the we these So, shape. experience good And have already gene significant majority of in be with therapy. site

feel fourth is the is And let’s the payers but waiting very I months I payers, or for a it. innovative I our of with -- very We’re And and proactively simply get know thinking working approaches. X have think be of need They clear. thoughtfully payers, this, get some very luxury I’ve launch they And about don’t to befitting therapy one strident is little I goal now. very X therapy, with and waiting. it Zolgensma. about the then of very, and earlier, strongly from kids said inappropriate. well These we’re need

for you think, working payers be issue, the be but are we’re a with at in think a our value going access, payers and this manner. want there therapy, when I And supportive. suggesting look exact therapy, the want work be payers won’t we our be to we And on timely which to -- certainly, lot we’ve value don’t So, of just is not very that I are value done is to going of challenges, going compelling. of access proposition -- on objective well-informed

Now from get through let an some uptake very time takes the it to process. be me perspective, clear,

take get think to couple we the assuming kids a uptake approved I envision, well, I to probably and Dallan, on do on? have start our and But commented and will have the get to is team the very process of think do time So line used I ability quarters forgotten robust through we’ve getting going job then we it very well, the the and like. should’ve be this done that none. if Dallan to I something second

Dallan Murray

it, No, covered I Doug. think you’ve

a of that the think there dialogue level to therapy, receptivity couple of had I and payers there’s the payers, high is things we’ve one. -- constructive a with real gene number

with We’ve EXONDYS constructive Duchenne, the in R&D well And that The place well I of very we’ll was publications I a work launching here last we’re well think a prepared that the Doug KOLs, -- have has engage talking Secondly, data we’re of and and be important colleagues. XX. a support then a XXXX is thing that would that in community lot than of to about. we in learned Duchenne. to Duchenne the and you a -- very going folks last the there’s we’re our dialogue and neuromuscular particularly and think to in in the framework learned value outcomes lot years, the now X And in KOLs, about this here. I to will thank Louise as say different be community value our the launch thanks different payer the to our the were as health --

Operator

William line Tim Blair. of Lugo the Our next from comes question of

Tim Lugo

AA the progress. year. on EMBARK Congratulations I and know we’re laser the focused next even a on data

heard EMBARK just on Europe your while. -- going haven’t us that’s a about what one I in making Can be AA? Roche occurs in no and we the to you dependant much Europe However, an for give with update relationship beyond. with and frame the assume time impact matter

Doug Ingram

So first, great relationship Roche. a with working we have

think I I you’ll public their it think you can revealed see -- in statements.

publicly, about certainly with reflective at colleagues therapy this we working that I relationship of be our seems positive think as about are bullish Roche. have to we as is very and Roche the

timing approvals to have the that world. development think aspects will And you’ve the pathway noted, across program, Roche’s Europe that inform other my regulatory like. other readout is have and got we then colleagues of world that what external the on to it and will HTA think they’ll I and a on upon United around of the statements leave Europe all approach the discussions commercial their EMBARK the then parts things to they Europe and Roche for very I of Tim, know than in to that been public planning, world at which the good rely the I States, as beyond the collaborative outside the

their from statement, that hear we upon. different that’s should and Roche, that’s what something think -- unless I public So, rely think we I

Operator

One for moment JP our Anupam Morgan. next of comes which Rama question, from

Anupam Rama

about wanted of on I when think earlier. that constraints or a you’re next are it And planning highlighted scenarios to supply considering are should up supply you’ve launch comes little approval? this be we XXXX any to year, access side, Tazeen’s looking to this follow the But the hurdles sort pending for as of And there think what question on we curve? manufacturing I kind bit.

Doug Ingram

Yes. Thank you.

the did the good which was We’ve different So that one, the capacity? ton launch? is point, a a the to fact, capacity And answering how I supported? question a a very so your which that was, we yes, shape. to assays slightly in good And work -- CMC about a your from in -- before of good And done shape I shape. a EMBARK, we we the good think with is perspective? was And ton answer are then, which be answer good going in and to have Tazeen work. we’re question, and yes. Are of fully is from data done we you think in I a perspective very to is in Do shape were is very

current assumption, planning community we patient a assuming a which that working to aggressive even this and very I alone. That serve ambulatory launch without is be this delay. even to goal that and speaking, fully the fair is, population. think would at our our assuming, broadly We’re is for fairly uptake launching assumption And point,

Operator

from Our Oppenheimer. Singh of comes Hartaj question next

Hartaj Singh

your into best-in-class have Louise, over when DMD, a you The you rhXX? there. LGMD? vector Great. Thank you. you it the it And kind to do is seems What have had really know, lastly, update. in clinic been thanks colleagues for I you quick a Thanks program. see attracted Nationwide, were it for getting AAV question. question. the rhXX better you guess, the a you all decade, for of I than how And and MyoAAV just MyoAAV? significantly And on I at working and

Doug Ingram

I’ll Louise this to turn Sure. comment. to

Dr. Louise Rodino-Klapac

you Thank that Yes. question. for

bullishness [Technical research it. seen we -- validate to clinical continue love well. we’re of we once as And our path certainly, So what about Difficulty] terms for It’s the very on we’ve rhXX is [[Technical in is Difficulty] that MyoAAV exciting. a in phase.

Doug Ingram

I apologize.

breaking, sorry, -- Hartaj I’m Louise. You’re

artfully less love I first we Louise just what think rhXX. cutting look, repeat I’ll was, bit. was firmly, a said, out I which saying, repeat Louise little think I’ll is what Louise

serotype current is on perspective That tropism our neuromuscular coupled a at excited. are focused no no neuro And from cardiomyopathies and We better really therapy a and well. now from there is is -- there as for platform with the least, frankly, perspective, perspective, gene right rhXX. than safety our

this isn’t on any about rhXX. So negativity

breadth can and with around increase more therapies dose, make thrilled enormous cetera, world, future one to to would the Obviously, and it. patients find if like. an is and probably patients safety affordable are of et the ways amount to decrease of We the there which value

go. have long We to a way

meaningful. approach a dosed some magnitude treat takes kinds healthy to serotypes, or the tragic. have with us current rare even other disease could gene is that animal and many MyoAAV. of potential At with haven’t therapies be patients would cost which any thinking we very, be -- diseases, for excite to than a MyoAAV. We but some tropic things model to kinds be MyoAAV, literally any that more It that’s human do least that at about currently acceptable. And very become at very difficult. the volunteers seen be other the today, order probably even really would next-generation preclinical the an of If and goods perspective, we’re not would diseases of therapies, seeing everything, places currently we the we’re really benefits of challenging us breadth thing about capsid, It ability meaningful would to really XXXX. therapy. probably and That’s really be cost but Frankly, MyoAAV, gene it. to other seen best places of going kind are to of to to a where today. full we’ve that’s It we’ve of these it if Africa around find certain -- ultra-rare be world transformative states there MyoAAV different the And India have our like goods of and the in And ought ways maybe including extraordinarily not have of

the of potential rhXX, road. down about So, are we we are with the excited MyoAAV but in love

Operator

from moment next One of questioner, Cowen. Gupta which line our the for of comes Anvita

Anvita Gupta

Ritu for to I Anvita the is wanted program. on This questioning continue from MyoAAV Cowen.

that a transduction So durability in explore? patients, that cells of are to get potentially hopefully muscle hope any you seeing sense you of or satellite improve to something guys is DMD

Doug Ingram

like. have know we mic Louise’s I certainly, we will -- see don’t the is working. if satellite Well, if and cells explore yet

Anvita Gupta

already…? seeing you Are that

Dr. Louise Rodino-Klapac

We will explore it.

satellite. Just to also Difficulty] [Technical our have with mention, there transduction explored we

as it well. So with MyoAAV not something but it’s already we certainly we’ll evaluate see,

Operator

One caller, line moment comes of the of which BTIG. our next from for Yun Zhong

Yun Zhong

more that the launch been about of that before, It’s with question therapy gene asked program talking an now probably related. old strong actually but had you’re so confidence,

please? you you. launch into skipping we going on do impact our expected to of your Thank kind to what have build And So are revenues? strategy, that exon launch

Doug Ingram

Yes.

things So, are is one we organization patient about that of organization. patient-driven, as often talk the we a an mission-driven

cannibalizes as indeed, as believe let’s platform is are has we So exon from premise. a therapy that If the greatly we of skipping it, we and start the current it XXXX with is children first a the or result are have impact and as benefiting thrilled. beneficial that an

will be clear about long technology our PPMO best though, therapy just lean be and significant do therapy for first all, kids. PMO our We or to come. perspective. a that next-generation for very into of exon want very believe, So place from I a time gene the that skipping both or there We for to

First, cannibalization of I see for term number any reasons. and a event won’t -- would we near significant the everybody in in remind

our population -- are about therapies of first is XX% thing -- on exon that our over non-ambulatory. skipping to of are is current the So, XX% our patient we note current

while they So, they’re get therapy, us they XXXX need this need waiting approved. to for

-- XX% there there related that it, about of neutralizing are therapy. kids, availability antibody gene Second all, something so, maybe do until we have won’t or for

that. do something to about trying We’re

and the data our very very until the and continue encouraged not. benefit a a we’re the and in for going lots well preclinical we’re with therapy of where around U.S. that’s we’ve mentioned that. addressed, both to in partner, study to I seen, world, be that PMO where remarks, may the world will able therapy the the opening availability As or those start gene a address PPMO be by course, then, kids. places, But to is around Hansa, of may that there we there’ll be And

go the which time. do PMO least we gene and the would and a If to is the the with beginning. doesn’t is the that you’ll is so would of we’ll basic of if suggest some as have of some benefiting a enormously long-term and I research some believe So preclinical that of you add are even to therapy doing cannibalization therapy. But I together. the XXXX be consider we’re to coexist on chronic therapy be results course, it a at even and in both at over it, run, long And answer. answer that too. or exon-skipping PPMO models kids some And case, that’s thrilled back then in we then literature think and for value for and therapy then, an to issue eventually additive significant that it co-administration our thrilled, And this the made they explore onetime the with of will statement will continue kids

Let’s be honest.

population reducing excluded. early As with that can now. an even also XXXX amount that you’ll we’re started study investor, of dosing enormous current And to reduce restrictions. is It’s because the the already be enormous be actually right thrilled this would have a addressed the -- be with exons we’ve then is have to

get we’re on on us. patients, for that into we’re all label as and as kids study and frame if positive it get another in of the like and kids a kids, going getting to non-ambulatory antibody non-ambulatory fast bring to of works, the then, that to expand the possible. work is neutralizing focusing that We’re would start to And course, of going the done XX% biggest that

Operator

Kluska line of of Fitzgerald. Our next Kristen the Cantor comes from question

Unidentified Analyst

This taking on for Kristen. for you is question. Thank Rick our

of importance EXONDYS about high in data the real-world Society, how unmet presented the the need these non-ambulant talk you’re at you Muscle the population? XX about For data World specifically thinking could

Doug Ingram

-- respect look, Well, interesting dystrophin, is it guess The I what an the question. does so beauty impact with to have on of question that’s ambulatory.

of is a itself applicable is journey and by of kid it’s with data the they move of and and really approval, we that beginning very because functional over They really imagine clear, I And is so think with value VYONDYS, on the what will finally, all, in which so the all across result issue. different we of muscles every of this time in example, induced the real-world is and value over the -- we may of it’s a sort with saw them. same every period long course, dystrophin that place is I of a see the part be And when have over the at we time be to the about post-marketing makes they time. a of amount you of number EXONDYS And the evidence to it AMONDYS. of suffering their for think proved equally are it’s population First me -- reveals time VYONDYS accelerated is missing or what important want which XXXX. very respect important metaphorically damaging but And that understand let there as of AMONDYS, exactly all are important, journey, EXONDYS to they’re for with dystrophin EXONDYS important that’s significant back just loop regard. And commitments. then the XXXX it’s point I of something for from important

We I as have to everybody excited hundreds submission. with really mind obligation of our do in to And of notwithstanding absolute and our continue top to are timely about them millions to literally it’s respect as we commitment very those already progressing that two and what possible. of studies, both be clear real-world of very, dollars and of complete studies ensure invested very evidence, we’re those assent want

work very evidence our We well. is post-market to real-world as the need encouraging. So, on commitments it’s -- to continue

Operator

of question, next which One moment Berenberg. our for of from comes Shu Zhiqiang line the

Zhiqiang Shu

on opinion got I there external in negative a accelerated I’d control is have was Committee. wonder, -- in submitted ask integrated you alignment the the based the like the recently, product that on any we’re control gene Obviously, from analysis you with Supervisory approval. control to about natural aware history. FDA external an your that package therapy for a from included oncology

there. it your got wondering to have alignment if any data to is support you have how approval? And Just accelerated important that

Doug Ingram

first important. is the so of think of I extraordinarily totality Well, evidence all,

the XXX, broad the all how review it FDA going the valuable. more rigorously ranges, broad way on all control preclinical age and not related extent very to -- clinical exists analysis, them. the Obviously, And exists, the which including work, why their that the very we XXX. Those we’ve information itself the then FDA good. analysis way seen have, X feel it the work. the But X to Part biomarker that pulling all we’ve the it of a data are all reveal So, itself. done is shared years would broad integrated of in X year, X all at from is the the that We’ve we together are a kids design, And the I’m integrated of X with it. all data say views this approximation a this additionally got all on have, weights Becker dystrophin, does like. Part it’s those out. in one X X, years We’ve XXX of of we kids now, has And out. I process certainly, of X years XXX, is kids safety very got external than perspective. look sure a an year, and reasonable What

people, that external masked group We a ensure means to But X is I and control, that And distribution if I’m very but misremembering. not well-masked rise this -- X external this of I covariants, -- used regression different not we then had walk/run. across a we representative, then age, time, not simple covariate, not we we the and baseline, matched. did propensity which external on Just got We to it’s matching, between possible. the closely XX-meter synthetically NSA a there. remind was our is control tight stop simply closely correlation even think identical believe, X. crucial didn’t which a as distribution, makes analysis is simply actual essentially the the

developed. within from on with placebo actually one very the a is that at wonder have that trials, came to actually because to time And of might were so information a associated that. similar the as was should the trial a note rigorous about people placebo-controlled extent approach it, on their patients this as They the then placebo-controlled came in you’d that they external gets. many that from of the top control, limitations I external about So control

it’s the any So, of we’ll agency. I review think with have full data. this don’t one piece

it’s functional into question dystrophin. go because to shortened the respect that’s the think to is, in discussion with case, talking surrogate I this remember, this about -- of going does all accelerated fundamental the data we’re endpoint approval,

you very likely on so predict this imagine compelling. is, So, is based the reasonably all to it data, clinical We’re of likely benefit? perspective the obviously what can sponsors, it’s believe but reasonably we our

Operator

from One line moment ISI. for our Evercore last of the question, which Gavin Clark-Gartner comes of

Gavin Clark-Gartner

arm For thinking for wondering include those of placebo to LGMD, if a on registrational next either and current you’re trials endpoints I’m planning your what just is?

Doug Ingram

do want this? Louise, you to touch

Dr. Louise Rodino-Klapac

the who arm a [Technical older severe as is [Technical going placebo be X Difficulty] more Yes. Difficulty] are [Technical then non-ambulatory. And think agreement. that and Phase study include Difficulty] about to patients we ambulatory

Doug Ingram

to Just to respect with everybody remind XXXX.

we’re done in we’ve So, we’ve cohorts seen our -- X with -- material. clinical

the safety rhXX, see same would additional You’ll seen not very it’s we’ve across to ought both promoter. things the study profile gotten that when surprising similar And a X design XXXX, you it’s of and we about look good what expression of I of cohorts. think And we’ve like. remind very

reduction that demise characterized remember the kids. of their We properly protein, enormous with like. in the that regard. and side first is amount generation beta- unlike Duchenne and to of XE, the lack lack is beta-sarcoglycan, causing know addition this of associated a associated well CK dystrophin-associated what’s in native of localized to of dystrophy, the protein functional these properly on the localized the The sarcolemma the and It all with being exactly not complex, an And XXXX It’s XXXX the of muscular muscle. beta-sarcoglycanopathy. upregulation it’s signals induces -- is treats in functional which sarcoglycan protein

ultra-rare or that trial the undue So with fact this mind, way. unnecessary all take lean without disease, to get thoughtful enough registrational of view and to that that delay therapy in for and be it be this and needs account this into kids is the and get rapidly trial that the to given is an our done

Operator

now Thank it you. Ingram I’d Doug like to remarks. turn With closing no to back for further questions,

Doug Ingram

the for history. evening, Obviously, with most long spending us next all and more All that, gene fairly patients, And history. that in the the we Duchenne remembering very XXXX, insightful least back I if right. thanks, very among among probably months Well, questions. perhaps a correctly. dystrophin this consequential thank have are I’m the seven of are your our consequential And for the most much than in believe, at time for you discovery everyone, since

that we of pass And looking through to we evening. of important would you so as have looking have updating with with I we’re to ahead forward the lovely these to everyone work all and us, that ask And we’re said, milestones. a forward that,

Operator

does so Yes. conclude the today’s now participation This disconnect. for Thank your conference. you you in program, may

Sarepta Therapeutics (SRPT) 3 Nov 222022 Q3 Earnings call transcript