SRPT - Earnings call 3 2023 | Sarepta Therapeutics, Inc.

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Operator

Good afternoon, and welcome to the Sarepta Therapeutics Third Quarter 2023 Earnings Call. [Operator Instructions] Please be advised that today's conference is being recorded. At this time, I'll turn the call over to Francesca Nolan, Executive Director, Investor Relations and Corporate Communications. Please go ahead.

Francesca Nolan

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

forward-looking Joining a us Doug Estepan, open Please our our Murray this statements. Dallan note the After we today number call Dr. be Rodino-Klapac. I'd remarks, call, forward-looking Ingram, that webcast, moment like Louise to statements. Ian a and formal to the on on slide call of which are we'll making for our will contains take Q&A. during review the

These forward-looking for risks Sarepta's and operations adversely could are results materially prices which statements differ the and affect from trading the can of materially business, statements involve results such and many these of common Sarepta's any Actual uncertainties, beyond control. forward-looking stock. risks and

For report not with forward-looking today a subsequent to company's any applicable obligation company's events you including of as does well filings. the most other publicly or update and to provided circumstances. Form any statements encourage financial we the SEC on based The risks uncertainties, detailed projections SEC as description company recent undertake on XX-Q quarterly review its the filed

now Doug? who and call will And I'll our over Ingram, progress. the President CEO, provide of Doug turn to our recent an overview

Douglas Ingram

summarizing the you but joining results It not Therapeutics' will placebo-controlled Sarepta the result on for thank EMBARK. double-blind, Third Financial and we afternoon, perspective. by X only I I was Call. trial Good days Results our here, will begin XXXX Fran. Conference you, ago linger Sarepta's Thank of Therefore, announced Quarter that

does slows arrest not shared results and whole, confirm of a with that by a decline, ELEVIDYS as profile are stabilizes, the EMBARK programs other Duchenne. safety so does taken for ages laudable the entirely across First,

its confirmed but benefits mechanism have across age satisfied our shown have with EMBARK environments only consistent patients the the Second, ELEVIDYS that results for of not action. June approval,

and And Hence, ELEVIDYS be we with label are restrictions. they and so rapidly. broaden already an ambulation they that to committed have supplement a broaden have confirmed discussions remove submitting soon productive will the BLA committed to reviewing finally, application and to encouraging the and FDA in we to age label doing to are engaged

Now comment let quarterly on me performance.

The Sarepta. was first third the launched with fourth gene Duchenne in quarter moment muscular therapy for a defining for We dystrophy. therapy our

We on us continue performance more profitability importantly, basis, great territory. placing a biotech to we our an drive X with achieved and PMOs ever non-GAAP rarified

at an have came net release, team's execute revenue launch $XXX approved seen stands million of mean by will merely last The AMONDYS. serve net patients, continuing million, million to and $XXX.XX of revenue ELEVIDYS product at over performance in ELEVIDYS our same VYONDYS Duchenne in revenue exceptional ability reflecting led triple the PMOs, external quarter total growing and product the you quarter in year, XX% X As EXONDYS, came and at and total $XX.XX our consensus.

and non-GAAP major the Likewise, and quarter, near product in $XX Sarepta stood quarter profitable over the year, as to earnings transition XX% same net PMOs in growing our term, a the flow million prior at for positive $XXX.XX we in million organization. cash a revenue achieved milestone

walk and record Murray, a of has of Officer, of done our execution proven shows it has team quarter, these And short a move to will nothing near to will looking been remarkable with in in The expand Customer we the rapidly of track forward ELEVIDYS. and label you future, the ELEVIDYS. and take launch our tremendous the what Chief through working families, Dallan physicians job payers results.

employ to majority the of States. the Duchenne patients ELEVIDYS that can of make United So in to execution this team level available

provide an will progress. Dr. Dallan's Following Rodino-Klapac on pipeline discussion, update our

we billion not approved expect, year, to to update. launch, therapies, would exceed this are me are turn we another And but $X our across updated Dallan? going you X substantially commercial in for call this providing Dallan be the guidance a also, as let that, early important obviously, the Now to with sure. milestone

Dallan Murray

The park Thank ELEVIDYS team ready Duchenne quarter you, and Doug, in they say proud it It and and of fourth by good launch the knocked third launch. date. Sarepta's And most I'm complex have X, day on thus afternoon. to was far. was out of the the was to far the challenging

support mentioned, just our expectations dose third exceeded in sites over generated readiness success going the the us was best-in-class own product longer-term organically risk with to launch this aging today has for revenues commercial out upon us patients sets forward. $XX site and nearly we of what million XX This in up lofty . Notably, for already Doug team ready As at teams. building quarter also helps approached Duchenne the net by medical We and ELEVIDYS. today.

rather have meet therapy building they separate than and responsibility gene up important put everyone board the magnificently stepped We out teams, moment. on additional to this across

early and Our KOLs patients was is our X serve. they gene the stakeholders. neuromuscular execution eligible flawless That external sites first, therapy with in achieved success the ways: of care

payer flexible the the the infusion. of time, patient's team just the the to SiPs proactive site expedite are engagement to on access time model at to get for well Secondly, right who customized label; by distribution and patients establishing each funding care third, in those a Sarepta eligible kit supported for

in HCPs, recognize for I patients came was and for and Duchenne patients eligible moment, were the see together now doctors, would it expedite for community to our to important to together moment to stakeholders how ELEVIDYS, support to their like access humbling and whole led community journey. by are this they come eligible who ready a the take other all treatment nurses

past gratified about the urgency have launch years were expediting our allowed demonstrated We This educating that the access for patients. eligible payers the and the progress the payers several also teams and made Duchenne. by in of experts policies

securing a we still across work of goal payers that, to of more access Saying in much are aging all of played the plan. importantly, regardless key who out. risk and patients to of do and all treatment payers the the patients for supporting of achieve eligible country have with some role Additionally our at

team quarter for have this our which very data we that been patients would Sarepta's robustness established been payers label as today confident fast have well Had The the over start support function when their past a educating the team's to of And building working access years, EMBARK available third a in as sets QX our this gain the the speak, label. data. with on therapies. the We're the in broader of efforts was stage in our is diligently launch, as the the model align newly and we nicely much all slower. importantly, The itself. to Duchenne eligible for as just the the trajectory efforts different X quarter of we

and of knowledge X PMO launches, ELEVIDYS. we glad able the expertise and these gained say We've through were deep apply I'm to to to launch learnings

roughly quarter grew ELEVIDYS, driven the the and to the prior in stakeholders over a our team great serve, the and our support We overall by ELEVIDYS because flawlessly patients large first it So launch revenue we quarter. by for summarize XX% the to quarter. in prepared they part product was key revenue robust net executed third were

Net product XXXX was of revenue $XXX.X in QX million.

quarter cannibalization We represents ] established ELEVIDYS the our in our for population most fact continued business our with Importantly, ELEVIDYS, net we [ that far we U.S. business the less not will to serving success and material X time. XXXX also also over PMO U.S., globally X opportunities a expect this the franchise. impact PMO in product the of PMO revenue. have ever had cannibalization to Since than our XX% of from as the successful with Doug on in said, patients our in see spite addition

to patients. are supporting here of I'd to thank a take those all like PMO moment our relentlessly who

effort, PMO in So the the roughly QX product million, XX% XXXX. was the result in over net as of same from revenue a quarter a our business team's representing $XXX increase whole

each product was XX% QX quarter This was million for QX of at VYONDYS $XX.X revenue And in revenue product the X.X% over individually, million, QX net of of PMOs EXONDYS was XX% above QX, XXXX. above same roughly of XXXX. revenue in XXXX. generated of product our net AMONDYS XX represents now growth which XX $XX.X XXXX. million over net XX $XXX.X in Looking

now As market X we of mentioned in our previous PMOs. approved phase for calls, we the all in the are mature of

from result, quarter-to-quarter to a in and we basis. while growth lumpy difficult and continue phase while revenues, to flatten As expect to U.S. thus the a the remain to the quarterly on project growth U.S. still ex

PMO our than for of guidance together, annual in revenue net can Taken in business $XXX product million we our XXXX. reiterate greater

the been of by stands and years. while the the achieved I've I'll the in been has what continually years those my teams end of execution amazed by the and over XXXX to I've that our success anything commitment years the Sarepta quarter seen Duchenne beyond third resilience, in serve XX and teams working impressed saying And community. have above impressive, over

numerous and of too restoration this gains put individual have express we to in future feel any The team. who country how whole every can't for as bright much team we mention who celebrate the the a patient community can stories dystrophin across just towards and each to and Nor for here. been when of the from Words waiting therapies. am joy Sarepta Duchenne are access our we progress. our very is proud for The deserve adequately I

with so turn that, And Louise? Louise I'll Rodino-Klapac. to call over Dr. the

Louise Rodino-Klapac

Thanks, Dallan.

remains goal to with rare lives the of and diseases to steadfast science unwavering. commitment patients our the Our change

many for which limitless to and either opportunity for Duchenne, nonexistent. good with is or diseases do therapies other those Our limb-girdle are living inadequate

focus As the the our RNA EMBARK on gene Doug of has programs. I and therapy will detailed comments results, progress already my

remain the to We these ahead. a of limb-girdle important muscular programs continuous committed or months look subtypes providing our dystrophy in advancing across and variety forward First, portfolio to updates LGMD. LGMD on

our community International pipeline on our Speak the presented LGMD evaluating shared in Phase patients process SRP-XXXX made to using Foundation's past for dystrophy LGMD. the Conference I We nonambulant begin, urgency weekend of SRP-XXXX medicines ambulant our clinical the and patients limb-girdle forward adult for study treatment XXXX To we material. progress at and with excellent muscular genetic LGMD type XE bring this for,

expression we pleased and patient into our Phase We Combined from insights shared commercially with are in initiate broader study remain on will year. initial VOYAGE data track report this positive later from our we the We enrollment and study, representative data a give material believe population. functional process to to III that using us SRP-XXXX-XXX. completed

report systemic for mediated therapy treat excited RhXX to in We that individuals are we vector our to SRP-XXXX completed with LGMDXB. dual gene also dosing pilot study,

first the allows sole the reporting cause dual gene, of length LGMDXB of of deterlin. us from characterized dystrophin We the study to results absence deliver in forward vector strategy innovative protein the LGMDXB. by half to is The the full look XXXX. the

last quarter, XX mentioned study all natural As history patients LGMDXE, fully has the XC and we sarcoglycanopathy XD, will enrolled, and Journey of for our LGMD been follow months.

LGMD make in manufacturing and to possible. studies progress all continue initiating candidates for in to We of look as scalable our forward as as rapidly our clinical pipeline

were we've We in the XXXX. targeting for we're Turning readout study enrollment quarter platform. of with now and MOMENTUM pleased SRP-XXXX, RNA to the to a complete study made XXXX progress our of in for first the

our Regarding PMOs completed mentioned in for golodirsen for post-marketing we as enrollment studies post-marketing a trial, and the last the casimersen. requirement ESSENCE quarter,

is X-year out ESSENCE early due XXXX. reminder, a As is to in and study a read

efficacy have weekly our the kg and XXX for significantly dosage to dose EXONDYS. of a Study, comparing double-blind eteplirsen, to kg provides XX per a higher our pleased are up per weekly. is dosage a randomized mg we mg marketing dose exposure that commitment approved enrollment to study finding in Mission post safety MISSION Finally, completed

with mission was XXXX forward is reporting enrolled. RNA continued it becomes fully enrolled that committed progress our in X-part rapidly and Patient We October sharing as a with We soon look XXX the diligently available. III advancing to programs in data remain Phase months. and study coming as to patients

promise its on later, of note, have personal genetic reflect humbled, my much I'm we we a science yet my medicine. and more in realizing life. in as and I'm years for passion are and grateful career many today to help early and promise On the to where do. so I These others begin

for world We continued like patients, living forward the here the thank the disease. around team toward rare a science and I'd from today more and moment to In our closing, to mission. future passion move promising take with a to their dedication for individuals

our on I'll Ian now to financial call update the turn Ian? an over for results.

Ian Estepan

on refer Sarepta's Louise. the financial the everyone. GAAP to sustained to quarter said, XXXX press to quarter available our press Good results . We're reconciliation Please the Well or for on results non-GAAP of of achieve with this financial well population, profitability. launch. as achieved a website EXONDYS release details release basis provided tremendous afternoon, the This of we basis. And Duchenne on a broader obviously actually full non-GAAP for as afternoon's the for GAAP third a should start non-GAAP back We assuming a label expansion results. profitability. pleased financial an quite to the

thrilled to just quarter this the of quite achieve in the launch. We're milestone first

September X total XX, of same revenues revenues million. increase for of XXXX, revenues $XXX.X an company of $XXX.X revenues to which million, product months million ended For recorded the of net and XXXX, the the collaboration compared $XXX.X period of consists

same product franchise million. of the revenue quarter Net was product $XX.X third period $XXX.X for million ELEVIDYS to the Net XXXX. was the of $XXX.X compared revenue for from our from XXXX period for same million

$XXX.X for VYONDYS XX, sales XX EXONDYS $XX.X and million the were for AMONDYS million individual For for net million quarter, $XX.X product XX.

to net increasing sales associated the PMO as reimbursement $XX.X million demand recognized with of net In reflects of for our product compared Roche. products ELEVIDYS. costs The third September XXXX, quarter Roche which our the in million for we totaled collaboration collaboration with same XXXX. well increase million under of of $XX.X primarily for revenue XXXX, each as XX, relates the product of quarters of The revenue, and ended the agreement revenue co-development to period arrangement XXXX $XX

$XX.X per million diluted in share the $XXX.X the compared $XX third XXXX. million XXXX GAAP non-GAAP third $X.XX basis, or respectively. or in of basic basic $X.XX quarter income per quarter of third of or non-GAAP share a for a On and share diluted or $XX.X and diluted $X.XX reported diluted reported XXXX, loss net per share XXXX of and a quarter of the and to million of per loss net $X.XX net We we million

period in XXXX, decrease recorded of of The the sales approximately million quarter cost write-offs in third $XX certain we of cost $XX the of for sales In same to million of the compared reflects primarily meeting quality period for the our increasing partially batches specifications X offset no of not XX, PMO the our demand September activity PMO products, with same ended in for of by products. our XXXX, XXXX, similar months XXXX.

decrease decrease in decrease recorded expenses we clinical to for in $XX.X of XXXX basis, a by primarily and million. trial million due expenses. expenses GAAP $XXX.X a offset On $XXX.X and in of third the The million quarter R&D partially is XXXX, manufacturing respectively, a increases year-over-year our

of On to of third non-GAAP for expenses were basis, a for R&D million $XXX.X same $XX.X million $XXX.X the quarter a decrease of period compared million. the XXXX, XXXX

XXXX, a turning On an million. $XX.X quarter and million driven and personnel stock-based $XXX.X in Now XXXX expenses by offset we of and primarily professional an compensation. SG&A. by approximately the to $XXX.X a third other increase million basis, recorded and respectively, of The of decrease for increase was in increase compensation partially expenses, GAAP services

investment an quarter contingent of of GAAP change the $XX for by the to of of offset expenses the a period of of interest non-GAAP loan to our quarter as our On as the XXXX, is net recorded of increases the expense XXXX. of in increases portfolio XXXX. in consideration, in well XXXX during accretion investment basis, income due third same repayment and expense other due discount, SG&A our million XXXX impairment and on an income $XX.X loss of that mix incurred million other reductions investment of million. term $XX.X million were interest partially result for period the as compared the primarily of December investment for in a net XXXX same increase for a third net to compared basis, $XX.X $XXX,XXX On we The the to

with $X.X based build and no on had equivalents, our results evaluate capital DMD. to our have That in We there's with of those valuable. but XX, long-term serve the expenses. inventory pleased XXXX. the sheet, of EMBARK We're cash today, approximately billion to cash, cash amount we turbulent better September information and use cash as even of our continually We more balance on investments said, we markets, becomes cash restricted the than in know obviously

that, I'll the over with Doug to Doug? call to turn So Q&A. start

Douglas Ingram

let's very the Michelle, open for you questions. much, Thank call Ian.

Operator

[Operator JP Rama Anupam from Instructions] The with Morgan. question first comes

Anupam Rama

results, gotten to back going question. Just the we've this EMBARK

functional multiplicity endpoints, If your you guys adjust would key for significant secondary on key still have measures? statistically you on these outcomes

Douglas Ingram

Yes. this Thank . Louise. Anupam. question, you to over for that turn I will call

Louise Rodino-Klapac

do significant hit global this performed from to actually is a did you. test to chance. not do and that we multiplicity the just Thank show We test, and that by just secondary statistical

show combined that with fact, tested secondaries were, significant. the statistically NSAA So they and we essentially in

So this of way evidence quantitative test the totality in to a is respect.

you showed we on see our that the essentially forest think to statistical are together, statistically call, if pot these significant. of So test that show that's that a we

Operator

of The Tazeen next comes from question Bank America Ahmad with Securities.

Tazeen Ahmad

most For the me, getting inbounds last X just timing. is of popular the I I've to think one been triangulate try days in

the the what complete provide of traditional this X-month is complete? So involved calendar on for with kind steps like to filing that filing? extent Could to be more would an the this something color all Doug, in can agency it are a you order a like example? precedent time us And review, there any to this Or next it application? would year review be for that it could take be your

Douglas Ingram

question and and than and Tazeen, giving to other well. thank let from you time as . to commitment rapidly going by then I'm division for the saying lines move not frustrate by a me to rapidly hard preface going we have probably we're your my question, you fast move

supplement and right The now. efficacy want do the Our very very is date, working we're but very, don't goal soon. believe is soon to be supplement. commit I team that exact to I will it BLA to submitted. going file that an on it's to a

I as committed the review, believe moving as may think reasonably not I to is -- have fast agency is believe this. X agency do possible traditionally, months review I to the to

And for this. there precedent is

see And a get of this, course, time You can in this like remember, isn't the for all areas supplement. something a where you turnaround. other oncology, BLA very fast but this like BLA,

So ELEVIDYS it the to believe is of and course, does that is totality fundamental life of better conclusion will focus question, And focused, justify important a on that patients. the extraordinarily the that is these does. bringing evidence, the we inquiry

quite clear. the this standard I is looking promise quite I statute that is now. The a substantial the music this know I is clear. on apologize. of guitar for evidence, It's totality at playing evidence I'm don't right not if

The reports statute severely effective no says that and also it's particularly regulations we'll used, like clear. Duchenne, therapy X exists. debilitating, very to fairly the when especially therapy alternative satisfactory the that Can conclude have for and ones the have life-threatening responsibly are clear

if standards. see and The arresting can FDA broadest out flexibility for that just functional is has as exercise in patients. the And evidence statistically adjusting is the multiplicity, and statistically determined that very that is compelling secondaries, to across do the in Louise you But you risk our -- statistics, applying statutory not only are primary these significant. it whole, look decline the pointed false of to everybody, the a the adjusting ELEVIDYS actual appropriate on for positive powerfully any

while agency we're a quickly all that to to working am exact successful and to winding the I give which soon. move time an BLA we're broaden review line, very to a us label. And So this original back very is is committed that rapidly. of submit to won't the going with this question going to confident I say supplement Tazeen,

Operator

Wang comes from with The next Barclays. question Gena

Huidong Wang

with like, protein you in the what be Would protein -- function in will you to what when level out package? was this supplement can follow that us? data will you versus included say, just accepted. kind be Maybe the announce And BLA package, data the will be data also the addition efficacy of also share of Doug, in lay will correlation these included comments.

Dallan Murray

-- relation -- line, review like. this any first still the CK transparent be to announcing going of we're request focus I is removing There to then, other generally in protein about will and age be package instance, the for decisions to the final other emulation. be and during the it's this and But more evaluated to be, don't the efficacy our by all things, We certainly But speaking, this we're on made for obviously, information, going pretty process. course, and including limitations limitations what label top and think in evaluating that. the folks. safety obviously, to we road tend we've the with of therapy. and foremost, then And

Operator

question next comes with from Colin The UBS. Bristow

Colin Bristow

driven you the were Can patients? we've interpatient from responders. the the results Phase experience, positive on in a II the just on saw talk maybe could ELEVIDYS-treated impressive congrats been compared small to of you and this ELEVIDYS. group getting another a And bit a speak data. the Maybe variability if And variability that you X by helpful. high is, how of be EMBARK Sort to would question

Douglas Ingram

specifically, but Sure. the to to to positive results, answer these flip not going on X.XXX. generally the X.XXX walk not it's that over close are on positive p-value the to at results on I'm rise, around they're time XX-meter would Louise I the this

together, the when it's X.XXX. and all the looks the analysis, X On secondaries global at primary statistical

Louise, want it's perhaps some of you answer variability issues. very So the powerful. to But specifically very, more on

Louise Rodino-Klapac

see our use Generally, any high at either variability high we to did not we anticipated amongst that was what did -- we power not the previous with or deviation patients see studies . below standard from Parker and . Yes. variability variability. we

Operator

comes question Guggenheim. next from Forsyth with Reed The

Unknown Analyst

Did provided include from on... the information data the EMBARK top FDA line to

Douglas Ingram

sorry, it but I perhaps? you was am question. unable I hear to ask Can again, that very

Unknown Analyst

Did FDA information Debjit. EMBARK line the include for data is provided on... top on the to This Yes. Ray on

Douglas Ingram

sorry, the the question but I'm hear beginning, itself. so I right? Goodness. don't heard I

Unknown Executive

on the he think asked expression. aspect data microdystrophin included I

Douglas Ingram

during I we'll data available have the with -- Well, that process the review imagine FDA. that the with

we've we're normally seeing the in is range expression Louise of the As seen. what in said, hunt

about there's it. So unusual nothing

very as value it Xs, is Apologies expect, that. the robustly. question limited about hear Robustly part. I on would X.X, a P you the on many That's couldn't you strongly. sight fact, In

Operator

comes Brian with Skorney Baird. next from The question

Brian Skorney

I'm I second not at closely guess under different a magnitude, that in favor sorry Maybe tell half that's it I difference nonsignificant a terms or want maybe tell, differences. of but me not. in very to you wasn't treatment, something think, but before, had if just I we right think, looked in

So wondering and you've I the I issue making differences acute rise case And know particularly talked gone to of X.X for XXX. the just baseline you've arm particular, evaluate and extensively if to imbalance same time back here to looked it tons the with subgroup was Study active in? at looks Phase imbalances adjustments seems baseline and at about the baseline the in X- X-year-old in to So was placebo rise was in if

Douglas Ingram

Brian. that Louise turn for question, you I'll respond. to Thank this Yes. to

Louise Rodino-Klapac

there compared control speak because it inclusion the generally when generated to take to we XXX, difference we that those and previous criteria we been patients a when our had I'm excluded data external and for did those going what rapid decliners. it exclude applied have to the -- found that was would criteria is by you we've Embark you that where what

same of a specific numbers the difference. So case, exclusion in we right criteria more kind and the a applied now, But did do we see but did significant difference. escaping analysis that we -- where that are saw

Ian Estepan

and XXX. we've a saw just in what good between Maybe in point, really thing X XXX observed observed Les' we that to analysis consistency Yes. what in we add to

Operator

with Salveen from question Sachs. comes The Goldman Richter next

Salveen Richter

might formal label or With payers they feedback existing procedures with fails the about any just a data to accelerated preliminary use physicians or regard here? how your regard the that approval trial after And submission, and to revisiting regulatory then involved in patients from an endpoint there on the confirmatory primary with from in are regulatory how impact here? think

Douglas Ingram

of of Again, been entire data, including I may And confirmatory the strongly looking whether determining as the the data benefits not studies. an that accelerated in of have but would the on be argue confident all of set, not the the evidence totality therapy the clear, surrounding is benefits approval confirmed standard all question, the by first understand but of evidence at strongly the well supporting very exist, for just confirmation as I'm embarked, not that of that Yes. this would evidence other only I therapy. the confirm question,

patients to at and it adjusted of looking this around to angulation. patients quite of confident in artificial these think -- analysis is these breadth to it That with division is shape the deserves that, to forest to be at know, looking available I So focus of limitation evidence across a example age going then and The the be our this an the there. of the were very confirmed restriction be statistical is focus I quite clear the now going patients that and forest the this label. on expansion good you the of therapy at am totality of made decline as the looking we're and to this in as arresting results plot view them confirmed having review plot strong for is and relates multiplicity, our that is as review or

me launch everyone discussions with performance fabulous the just team a it will have. that job in of with quarter the our his and bolsters have As have team, agree and shows this Things in affairs, to additional hope payers, done medical well. Dallan payers. armamentarium this field this the evidence elevates to very and can I is reimbursement like. it our and relates that force, our gone commercial, supporting access our the

as X you we a a can powerful give metric with that have Now put kit now example. on on We really speed on which an it's the compelling therapy.

rise, time not period. we've on greatest talked am the as remind occurring the I value, seconds, if ELEVIDYS of I over by the in but mistaken the is and ambulation. ambulation loss to time And of using think, single shown loss XX-week literature odds to reduces in I P early you rise about of as X.XXX, X single robustly that So time will has is that often the above is and rise a in only -- EMBARK is a not prognosticator predictor of XX%. of greatest the

possible. an access. additional provides frankly, done as therapy to is possible forward label of soon this label should as this and kids provides this important get who, providing soon job I expanded This a this a expansion And So why really far, on good as additional compelling with to be argue so payers compelling be looking for as that point therapy argument as it should -- have well. really is would evidence

as well. everyone to So access it has

Operator

with Uy comes Mizuho. next Ear from The question

Uy Ear

more Congrats on -- they great primarily much would you And the many is, theme. dose and anticipated my patients be the approval those the question were how to pretty like they were in are to I coming El like just And quarter? -- how get be accelerated the patients able those dosed share approval, bolus. who this so waiting the them the guess quarter with continue many for first were on

I Now the early of read days. you all? to product that has any it's the shift patients Embark at data out, know any have in do sense receptivity

Douglas Ingram

Yes.

Let me quickly. answer those questions

for think don't shift this be therapy. in First, there any will I the desire

and broadened. are as to of in parents for want X-year-old patient that except I in going to label be in to even range not are this think patients start probably the more to impatiently the patients are have compelled this I think X- should that not wait they are

and success net as metric our We to don't revenue. going the for our measure patient of on success We're product numbers. share use

obviously, that you number on bolus get warehousing know, said, sort X the because gathering aged were preferentially dosed there of dosing they and they getting and of to of label, out patients, or patients of X% to issue we below were the together restricts that as the physicians certainly and X%. before some could ensure As

stream a forms steady had we've start the But approval. of new since

And done we forms stream on others been other done all looking team, are thing so a job a forward, the steady say have activation. site has but through of process. And working X the it have people start I'll and that asked, brilliant

but of end perhaps aspirationally, thought sometime XX I ready have to today. launch. the know, at year, to goal XX and year, the been all sites we the think we our you way sites, to And then our infuse get goal, as we're very XX can next at aspirationally by

the there through. ready has and steady a to up start stream brilliant done So making jump just sites running and forms and a getting is team work -- of

Operator

Brill comes question James. Danielle Raymond next with The from

Danielle Brill

a spent the X at efficacy recall that time to myocardis event I'd lot the EMBARK EMBARK at discussing discussed being to like including time. switch in occurred we data AdCom, so at over Doug, of safety. had I events

this Just study any other safety there or nature occurred the to events any hospitalization? are additional that of leading SAEs in curious if

Tim Lugo

I will Danielle. to to you your the call Thank turn Louis question, over respond. for

Louise Rodino-Klapac

bark previous of trials. the all Yes, the not in the of together about profile SAEs on any see we safety that taken was data a the have differences or types trials, safety reassuring base of things those X did we the SAES continued was most and the frequency of large that's now consistent

Operator

The comes with Baral question from Ritu next Cowen.

Ritu Baral

that scenarios are numbers through label Doug, are for the could for walk experts essentially in regulatory -- you see patient could and Dallan, different expansion. you play maybe, suggest how us

mentioned that like full by -- to but would that to of population, are my number are calls FDA your those got years if again? X- ambulatory would change how covered extends age. how you it And maybe XX of segment for You XX route the hemo wants down you do to which -- XX% sales per go X-year-olds now

Douglas Ingram

ambulatory? For

Ritu Baral

of Like loss average ambulation now Yes. the XX XX. that's age of

Douglas Ingram

to to be not second. X, limited from given I'll be want would very first there's That to correct. think looking may expansion be and scientifically reason confirm any I clear, data. that to mean, X of to we X, a Broadly Dallan the X I don't speaking, we're look, X.X X to label go a in for all,

seen before. modest seen is for Duchenne ambulatory ambulatory all we've question, never about and these once any never -- And you've in that sorts all But answering other of versus age-related XX%. restrictions. nonambulatory label therapy, the

and to the like so nonambulatory half So that, this on our mind, topic. there's else the is Dale, -- big course, so cut. is all say other ambulatory about -- of population But to half, you'd there anything and is patients the of the

Dallan Murray

No, it, covered I think you Doug.

all population, to of of Our age we question is entire ambulation. loss your in goal about to patients read get that the average can the you target

there's the of the But right range. definition ambulation you're a about think I know, too loss the KOLs lot talking heterogeneity. have in been you as of because,

older are cohort have treated as walking with years now patients have have been PMOs ages, believe, whole we and you at for ambulation that of who've much we older of So some a patients well. of the going to, loss ages

But a who by all label our believe be won't age. -- we targeting and aspiration population the is it Doug of will eligible. said, as broad be the patients be ambulatory won't defined So

Ritu Baral

that is PMO XX% too? And your sales of

Douglas Ingram

I think versus Yes. XX%. The that in it's range nonambulatory of ambulatory split,

access I think is in the patients. older the difficult more

average, a in on higher given younger So we've the population. penetration got

Operator

with The Stanley. next Morgan from as comes question

Unknown Analyst

give active. have of You sites of have actually that us XX far? prescribed you those sense mentioned a about elevates you so percentage are what sites Can now .

Douglas Ingram

level specifically I'm going infused time. We're We probably sites of number at or resist of going to on X are to either -- frustrate any going apologies to you. are more numbers patients detail that infused of

X we done have to product is the last the generally the net success for I for we've folks. time good like. with and use uptake and over years, As and success We think revenue . marker done

Unknown Analyst

that, apologies So Mike. for

Operator

The Lugo Tim with Instructions] William Blair. question comes [Operator next from

Tim Lugo

non-age-restricted It not like the approval you remember an AdCom? the in X accelerated week the expect seems But earlier we for discussion. much participants mentioned the amenable were to all than additional to guess I advisory can approval did more broadening AdCom, we You that when committee the request an agency expect label. why was. listening the didn't for

Douglas Ingram

Yes.

Okay.

the writing. historical that reason shared be I as one. those us -- that have going view factor coms I say by that, a the would is poor So might is it's will don't that going are that The I comment. record we and we that not And ad to I and sit that of that that outcome predicting in notwithstanding believe that believe, don't believe There surprised today, to time have we're have were an a a we last said think have here the need we good track to we by I'm FDA question, leadership.

discussion changed has the before, by And on the this with encouraging you where leadership pretty submitting OTAT for been through of had significant the the year. possibility data a to super the of gone said course particular, There's some a we've replaced would the it the FDA division, been and agency office. has changes we in over very a just remind I has reorganization productive on broad As and also that label. note --

took leader, Dr. the Head of. ago, not Verdun, long as And too helm the new Nicole a

prepared And So the I that in Obviously, would would well well we say believe is perform exceptionally that advisory I were not event evolving. to there there. we remember would be an for it. division was the committee,

fabulous outcome. his sit team recall, I us. that pretty did think we X advisory job But an expansion a get today, Dr. And we may little Louise can we and win did confident here just as as Rodino-Klapac a without feel representing ultimately we that committee.

Operator

question Needham comes Gil & The from next Blum Company. with

Gil Blum

Congratulations the progress. on all

ago. a back questions on Alan's follow-up about any a to Going question, earlier questions

X data looks rise. older especially interesting that Study Study crossed there you that XXX. -- regarding XXX some if chance the data. be any there comparison of time were EMBARK Is on part on It to that in the to were see and will to in patients over are be So how follow-up, would

Tim Lugo

thoughts any on Louis, Sure. that?

Louise Rodino-Klapac

the are . to to continued up Okay fall patients all for XXX X years.

didn't don't something over time. look at have certainly at we so So look they have, we We can can that. that's that that

Operator

from with question comes Capital. next RBC The Brian

Brian Abrahams

if launch. that might recent with time FDA full tests Congrats protocols on unblinding and on in your versus ELEVIDYS the expect this any you functional place quarter the in of in you the topic it blinding, remind there discussions? Embark have on up first to strong was might -- us came effect a protect And in ever of NSAA? differences against the Can the was functional any,

Douglas Ingram

can rigorous I to kind aspects the assume would if less about unblinding. to very time there well of very want was exceptionally discuss run. very say be objective allow But study I the in itself, think and process, the process that . blinding protocol protocol Luis blinding I that I actually also generally event should about wasn't that the clear can the would influence good subject of be X test which Yes. any we that. to was these was remember, the

Tim Lugo

any blinding on . the Luis, process? thoughts

Mary Jenkins

Yes.

Louise Rodino-Klapac

physical slide. and as to PIs therapist specifically the Just patients the obviously all as caregivers functional doing well completely are the blinded the tests

maintained So maintain blinded staff blinded. the is party. steady disrupt studies third It's by a

in process that blind to sure make intact. rigorous a place is the remains . there So

Operator

Fitzgerald. Kluska Cantor from Kristen with question comes next The

Unknown Analyst

One This cadence question going Kristen is for on laid the the than for quarter is Congrats ELEVIDYS. us, patients original you Jason on the strong Kluska. from treating Bouvier out. faster lines time

what impact broader wondering how So there? just And also the are launch might we're next this drivers the year? potentially biggest

Douglas Ingram

therapy, Dallan is Well, the exceptionally X I'm this going in therapy is team. course, want because the is to itself. from the unprecedented question brag know our launch going launch an significant think and to take the my success well of why gene even I this why there's cadence perspective, it, I first I wants which, mean about of to reasons of

have age-specific that stabilizing with been it extraordinarily have this they the need their doing patients on share families who or and things belief able these been to that experience therapy, an of and strongly is been kids ELEVIDYS haven't needed kids therapy do. that untreated that

mean Customer the led then and we don't of gone I now beyond we you folks have And the large and this is little going team. X if on by manufacturing an bragging have of execution is everyone execute well. me launched. exceptionally as Dallan, isn't our Every this distribution remind I for well, to want by mind just therapies a effort new exceptionally us. example that a And team have about those now their by bit folks great Officer, X that, launches therapies this to that this. Sarepta

just that P&L, quarter was late first launching XXXX. moment. PMO still a the our mean at in I brilliantly it's year, digress mean evidence at grew look approved growing. there. for we now even XX% from the is We doing if we last as we're I over same quarter are We're

we be have believe, the therapies, combination I great one it well-informed to ELEVIDYS all means are of to exceptional been majority young of Duchenne what in mean X that a as United this how disease. of execution. able our this about does the things us focused, and there's serve with think for to future, I irreparably know therapy bring And that and and States label generating we'll the So that is ferocious a and living granular, the community of excites from the the broader children men both

even as Thank And about more very your to bring really much I'm you doing therapy we're to this the now. excited question. patients launch so really for the opportunity with right well

Operator

from Joseph Partners. The Leerink comes question with Schwartz last

Joseph Schwartz

impact trade-off And candidate, are look you between to FAA, and a was how efficacy. that greater If that impact data gene would soon. therapy value have relative interim focused wondering thinking in clinical you'll how about for a see upcoming your the it Is safety do an on which produce you how able particular? I will ELEVIDYS? different proposition anything Great. there for be on were

Douglas Ingram

made of as all of in the many before, And disease. the exceptionally of beating decisions benefit Duchenne a that. brilliant experience, job have dystrophy is about I this doing our is focus pleased we and proud energy -- Look, are an question. ELEVIDYS. of many the competitor and in of. We're for There all times Roche's people muscular think capsids exceptionally the like. constructs performance you front And said years damn that of of and some many of ago, with the with we our team and years Thank I've and exceptionally for us were

given we're what their do and profile who have. protect required that intervene had who it they here rational is It's develop. to best it us. and infusions. that to fact confident what nothing particularly I'm decline would try copy that and construct it's of with a that amount able thrilled of children make muscles therapy been with standout. the quite And sure than anyone decision only, they can arrested opportunity And to do they safety full a would to we lonable body but particular these about the is capsid not that the frankly, a less an use shown these today and construct has would rhXX capsid

we competitor focused muscle dystrophy, Duchenne any to not committed So on beating and are disease. this we're ferociously -- we're besides

focus you But much our your questions. thank that's very for right now. So

Operator

for I closing Doug call over turn would to now remarks. back like to the

Douglas Ingram

And Well, your Michelle. everyone, thoughtful thanks, for you much, attending tonight, questions. and very very you thank for thank

Let me summarize.

a and quarter PMOs, opinion, continued the approval, launched face are the This future. in near growth been flow our one. of fourth an my an profitable, we a which are extraordinarily robust even marching and positive serve cash we has launch. toward basis, we launched ELEVIDYS we with in it important And in elevates With our brilliantly. now community on We've non-GAAP continue the to very

enduring a strong that We on have built organization focused is major X things.

continue precisely The first done. We the we is strong of move over the supplement to community and to things our and the will doing on efficacy performance label. is second And months. the conclude submit coming executing intend patient getting speed to be and is and science the serving ELEVIDYS to our brilliant that through broadening review our this community. with intend what We serving commitment

And

Thank a bring of lovely intend to all we the with has living of our majority restrictions, label remove have you. And I updating to you on along Duchenne look and to the to therapy with the our been this that, way when patients ambulation updated age in United forward States. progress and evening.

Operator

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

may disconnect. You now

Sarepta Therapeutics (SRPT) 1 Nov 232023 Q3 Earnings call transcript