SRPT - Earnings call transcript Q3 2024 | Sarepta Therapeutics, Inc.

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Operator

Good day, and welcome to the Sarepta Therapeutics Third Quarter 2024 Financial Results Conference Call. [Operator Instructions] As a reminder, today's conference is being recorded. At this time, I would like to turn the call over to Mary Jenkins, Associate Director, Investor Relations and Corporate Communications. Please go ahead.

Mary Jenkins

Thank you, Michelle, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the third quarter 2024. available filed website Exchange with afternoon. is our sarepta.com, Commission release was XX-Q the press on at our and this The Securities and

us formal remarks, Ingram, open the are and Joining Q&A. Louise we'll call for Dallan Dr. our Estepan, Murray Doug After Rodino-Klapac. call the on Ian today

many Sarepta's and uncertainties, operations forward-looking control. results contains could that affect risks These this slide of any can we adversely of and statements. moment results statements trading review these our from will take like and prices Actual Please the which making a I'd are business, a statements. during and to be materially for the our webcast, beyond involve materially Sarepta's risks which on statements, such note forward-looking stock. forward-looking call, forward-looking number of to differ common

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

Douglas Ingram

last of net XXXX million, Third Earlier report was net Mary. million. ELEVIDYS at you Results about revenue Good another Therapeutics performance. of joining Quarter same Call. versus Financial quarter the growing reported today, revenue commercial you, I Thank afternoon, $XXX total quarter XX% thank pleased strong year. and Conference product $XXX product Sarepta we am to for

in the prior exceeded made revenue million our -- Roche, guidance we Our quarter. well as includes analyst royalty revenue, performance Roche's quarter. the U.S. substantially Even our partner, consensus. ELEVIDYS And our achieved considering from ex-U.S. if $XXX.X one the performance, without good progress ELEVIDYS royalty has in as ex-U.S.

guidance. and We well QX tracking are to consistent prior with XXXX performance,

comprising approximately substantially XX sales analyst in XX, achieved PMO VYONDYS again, consensus. quarter, the AMONDYS and XX exceeding EXONDYS million Our $XXX third

As report on that provide expect again we meaningful material through pleased we of Chief see from basis Customer Dallan cannibalization net profitable will our in yet not GAAP XXXX. I and a third were more do ELEVIDYS non-GAAP franchise performance commercial to are we and color Murray, performance, to am anticipated, cannibalization our the not seeing also on a moment. Officer, in quarter. PMO

to R&D. Turning

prioritized made and programs. quarter, great we've advancing our this We pipeline and have progress rationalized

let I program. discuss me Before progress, our PPMO discuss our

the that was will risk-benefit the FDA the ELEVIDYS. seen program analysis landscape consideration the development You done evolving decision and made for after This release to our the have approval in after of dialogue discontinue SRP-XXXX in for Duchenne, for have requirements and our the own with we their program. including

one for never decision to for a community our one Now decision confident the stakeholders. are patient the right we our discontinue that program, but takes and is the lightly

our to pipeline progress. Moving

to both for fourth our the treat the of of quarter approval XXXX, accelerated in this BLA planned XXXX treat SRP-XXXX year LGMD, to or XC, commencement muscular SRP-XXXX into of early trials support in in Type limb-girdle trial a commencement which our the dystrophy late-stage XD, Type to and beginning XE, clinical trial LGMD, SRP-XXXX intended rapidly are approval. for We treat of with for LGMD, move to to Type are

clinical ELEVIDYS with activity ex-U.S. to for the our to intended clinical and made those with Duchenne to clinical for the We development our data meet including population and value ELEVIDYS. to efforts have living post-marketing commitments, ELEVIDYS, speak approvals support presentations also work progress expand development that of

proceeding program very to ELEVIDYS move to is manufacturing suspension our Additionally, well.

and we FDA, We of with have interactions a engineering study continue had runs XXXX. in commencing in anticipation our the bridging very encouraging

pipeline, to concerning progress cardiomyopathy made CNS neuromuscular, have progress and of good with more We rest our share the in pipeline XXXX. our intend also and its

of Rodino-Klapac, with moment. And that, and detail for our in our discuss on decision Louise Officer, will Our turn Murray Head R&D call progress and Dr. this Scientific commercial Dallan? more performance I R&D the Dallan Chief a will plans. to and

Dallan Murray

been as and you, and patient and the Thank of to X-month driven demand The be ample impressive gaining and continue ambulant led site positive timeline. Doug, access by conversion within performance report approval conversion continued to afternoon. we've launch. to quarter, the from strength rates, We're we see achieved non-ambulant and populations, success ELEVIDYS patients good an to pleased the robust consistent reimbursement both capacity, access third in since and the by has trends continues X-

older Additionally, as important works to such broad population. model, flex the team to as built note on we've the needed label, execute non-ambulatory accommodate that it's patient the which to a us allows from segments, demand different as thoughtfully to

product of growth our remain revenue and XX% Net $XXX for third revenue ability to note exceeding the looking quarter-to-quarter and nearly the guidance to numbers. million our XX%. Of impressed to team's reached by forecast. quarter, Turning quarter with prior of grew compared ELEVIDYS I forward,

made QX achieve on in clear to all continued for current a We've and the team expectations, all position factors necessary XXXX. expectations and performance that on in we broad Muscle, Further going The in is As as our good have performance execute the of are guidance is momentum QX meet on at World line enhanced with discuss, we promised. by fourth strong the point in consensus. Based remain we this and anticipated to unprecedented confidence of guidance, curve. the by at this to reiterate wealth industry. ability have progress of launch into data the factors these we execute Louise with visibility our we strong which And stated previously the quarter, predicted, the on our of launch inflection presented our shown a have quarter now QX will our provides as label, our the evidence and dynamics. meeting

we are at patients So to the high muscular to an impressed sites of Together, rates, United unprecedented conversion in see rate. and changing treating demand, are strong we States. successfully face continue of we Duchenne dystrophy are with how summarize, the care

our to which franchise, impressive in quarter. also achieved third results PMO the Turning

R&D over the of serving way solid quarters Louise. $XX.X compared $XX.X product million The to of Duchenne all the revenue with expect compared revenue we strength the revenue team We us million the nearly approximately to have for whether we every of in in continued XX% a third way. community sites approved where that, to be, X% XXXX. In product the net XX like QX on in I'll we And the million like the this journey Rodino-Klapac at pioneering our $XXX.X VYONDYS the and or delivered of net most call closing, We're revenue. quarter. that majority growth and PMOs PMOs the XX with of grateful hand vast see And to with to healthy Importantly, be net thank in therapies and QX patient in and QX, I'd patient update. been Louise importantly, AMONDYS years. Dr. community. And delivered coming with of for Duchenne of the representing net our roughly the partners, elsewhere. quarter led franchise. EXONDYS growth million wanted had delivered $XXX in product in thank the I'd represent the and exactly who with Sarepta is always segment step care XXXX. to XX in for to product of third to

Louise Rodino-Klapac

my begin I'll remarks Dallan. Thanks, and our with on provide then, an update pipeline. ELEVIDYS,

high-impact program clinical as and in sets We a they recently the primary published share Medicine, continue EMBARK to results the new available. ELEVIDYS We become advance data X-year Nature journal.

Society SRP-XXXX, in EMBARK EMBARK slowing at data, changes of with early outcomes October. multiple while no protection post-gene MRI and the MRI World therapy, longitudinal that Part was X, consistent had we stabilization of This occurred with cardiac placebo-treated muscle by additional MRI studies Congress demonstrated harm observed. MRI. cardiac at will showing functional fibrosis. Cardiac addition, of disease there of progression were from cardiac Muscle in evidence Muscle long-term In presentations X-year assess accumulation fat muscle. and patients, or MRI evidenced progression included Future

described, an muscle. that the previously to in ELEVIDYS MHCKX protect promoter contains cardiac we've heart well expresses As

shown Duchenne overall cardiac in improvements as well mdx function the in survival. as of have rat model We

XXX the addition to also ENDEAVOR, ambulatory across EMBARK demonstrating Study In data safety expression data, or consistent we've and patients. from expression and presented non-ambulatory data safety and

continue October see to end late As patients and consistent of of our XX safety non-ambulatory over and within XXXX, we profile. program clinical the a have dosed ambulatory

No new analysis natural by we Finally, divergence slows DMD or signals demonstrating shown identified. XXX, by the were with -- control data in Study external from that history increase SRP-XXXX longitudinal supported over time, analysis. as diversion cTAP progression independent stabilizes and from an shared safety X-year

includes via our approval As patients, call, to and addressed post-marketing a the commitment benefit, mentioned clinical for ELEVIDYS on confirm last be also our which XXX, accelerated will non-ambulatory known Study ENVISION. late-ambulatory non-ambulatory

a ENVISION is global XX-month the our in following complete completed XXXX recruitment remaining occurring expected randomized, expected with double-blind, progressing to placebo-controlled reminder, Enrollment study. XXXX period. placebo-controlled As ex-U.S. and is X-part last be in an a ENVISION U.S. visit enrollment well with patient is last

advance to with We studies long-term also clinical monitor ELEVIDYS. that continue follow-up

ENDURE studies EXPEDITION. include Our long-term and follow-up

a will IV for study ELEVIDYS As with up reminder, that to years. a treated Phase individuals follow is ENDURE observational XX

and that follows were approximately previously a consistent for safety years. Phase measures is up and addition, trials enrolled XXX in for EXPEDITION In to enrolling clinical III X efficacy ELEVIDYS patients study

to receive label, Regarding the expanded ELEVIDYS continue ineligible studies. advance patients we currently under to multiple

we cleave the second antibodies XX% of to remove imlifidase For out with plasmapheresis screened X and pre-existing commenced are who patients antibodies. one anti-AAVrhXX studies, antibodies, a to for with approximately have

patients safety have We data and XXXX. expect expression in early sentinel from to

as are under treated patients for experience together as dosing and Roche, ENVOL Study young Study with we X, addition, X under our to months. executing X age as our gain the patients in partner, we patients of young and XXX XXX, have as or X In

ELEVIDYS We a trial experience with X range continue in disease. communicate of advanced more patients from to treated that those are with under those to

dystrophies with our to LGMD, limb-girdle programs now the or for Moving SRP-XXXX. starting muscular

to dosing we treatment XE, for our year call, III muscular disease. Phase as in end multinational be As mentioned known dystrophy, point track absence the EMERGENE, Type SRP-XXXX-XXX, we the expression which beta-sarcoglycanopathy. The trial of study sole the study quarter the agreed on primary is limb-girdle of beta-sarcoglycan, SRP-XXXX initiated by EMERGENE open-label this first is end also clinical early of the of of fully for the cause enrolled XXXX. on of The is

positive will XXXX. filing pre-BLA a BLA we anticipate a Assuming in meeting,

for the progress ability together debilitating, a with pathway in an is Phase small but suspension which clinic. Having our pipeline, by that other program, this is this IND and to viable results update, and are LGMDXC, beta-sarcoglycan through encouraged willingness progressively our a condition early functional sarcoglycanopathies are mortality. a progressing The to in LGMDXD loss the we both ability our ultra-rare submitted study I delivery of We to process protein SRP-XXXX, year to our not biomarker leads genetic with support advanced reflecting of agency's expected important, extremely for year's initiation by end. ambulation just NXX of SRP-XXXX, demonstrate for including SRP-XXXX the successfully

SRP-XXXX the limb-girdle dystrophy, reminder, XD, of a treatment for alpha-sarcoglycanopathy. muscular is As or designed type

we limb-girdle our also we XC, progressing months. for the for of our and with have LGMDs We to clinical type muscular less QX dystrophy, X summarize, the gamma-sarcoglycanopathy. rapidly plans a year to or very initiate progress Finally, SRP-XXXX are of our clinic with in QX than of expect with in programs to engage FDA in this in are the pleased of plan XXXX. To X treatment portfolio LGMD study

the as look clinic. Continuing platform this of remainder mission the the We perspective, trial, on across with as are both our and platform firmly PMOs, the sites ESSENCE and R&D assets an on sharing forward RNA the remain manufacturing casimersen commitment and and set for synergies for well are for both complete. enrolled golodirsen data fully to to post-marketing post-marketing the soon are or as beginning our We with LGMD and requirements EXONDYS as our accelerating maximizing is study our track.

to now Turning PPMO.

is also to our means discontinue patient and As decided PMO of to today we safety the SRP-XXXX, with dosing to participants in does development. a encouraged MOMENTUM And not study, of we while expression long-term support setting Duchenne. peptide known or line investigational development conjugated as the highest commitment dystrophin first, are SRP-XXXX has XXX results vesleteplirsen, our with priority. treat study stopped. The in announced chronic unwavering in SRP-XXXX, our safety treatment PPMO further by have This our

was hypothesis initial manageable Our hypomagnesemia the was and monitorable. that

us of analysis Although study. in led small treatment far medically events to persistent thus our risk-benefit remained hypomagnesemia, saw manageable, discontinuation, a and patients, the number end despite we

around information the clinicians Duchenne, grateful study. in to families increase are our patients, important participated who use The we RNA-targeted production MOMENTUM study the provided in and dystrophin of therapies extremely to and

discussing and future spend a our I'll moment pipeline. Now current

expertise clinical During also portfolio advanced antibodies developing advance trials We've the pre-existing patients as new the rare since advanced for regulatory gene past of Sarepta, and therapy platform. been and medicines joined precedent capabilities to a for and I for rapidly we've assets. identify the for building X genetic diligently with years advanced significantly research field current our and redosing. We've by potential evidenced disease LGMD through

neuromuscular On side, developed system and potential capsids therapy change will and disease the we landscape AAV areas. have unlock optimized, gene in research characterized for new and central that cardiac the nervous

data. preclinical AAVrhXX the are Charcot-Marie-Tooth, As now example, and to rapidly construct we've optimized an clinic CMTXA, or XA, exciting using advancing the for following Type

X R&D approach myelination product approach the because pipeline other who highlighting as gene colleagues results. demyelinating R&D of patients impressive in CMTs possible. over to using As has make participate to indications. their well an a on this all update as Day XXXX. I'll in or neurotrophin applicability for financial incredibly The nerve We a a our our Ian? reminder, Estepan and NT-X all CMTXA. is regeneration thanking trials the turn for This delivery my our in work. I'll improve future and in of forward to call look in now talented we're other Ian surrogate bright to who the by of an close pipeline

Ian Estepan

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

the which company quarter in of beginning tax are fourth estimated Non-GAAP impact for XXXX, in-licensed of income The comparability reminder, of income change effective the XXXX for no effective has to applicable non-GAAP and amortization pretax results. from tax expense quarter third non-GAAP our the income added a tax represents of excluded each rate. based adjustments, As longer this income results rights of adjustments updated the been the on purposes. have financial tax reflect

the million X of revenues September an third company and period same net for XXXX, of recorded revenues of $XXX.X revenue the revenues compared million of of million primarily ELEVIDYS. of total million, product the $XXX revenue period net product for other the was of to net our in ended compared $XXX.X So of the reflects XXXX. product third to million sales was product ELEVIDYS months XXXX XXXX, from $XXX.X Net collaboration increase XXXX. PMO $XX XX, product exon-skipping associated for quarter million revenues consists the for and increase same to which same revenue $XXX.X from $XXX.X of million. Net The quarter of XXXX for compared revenue the franchise period with

the ELEVIDYS to the XXXX, recognized of September the which For revenues revenue months relates revenues, commercial primarily Roche the reimbursable under and agreement million to collaboration XXXX, royalty $XX.X ended period for the increase from for million the of to other compared and million. The million delivered quarter supply Roche of compared of totaled XX, Roche collaboration X co-development company an third period the $XX.X XXXX. to million XXXX of $XX.X costs $XX.X for $XX.X same of same

by basis, GAAP for of and of quarter income of per per offset expenses. On XXXX diluted $X.XX non-GAAP Roche per XXXX, third of reimbursable increase eteplirsen a respectively, income of related period PPMO and $XX.X quarter million program. reminder, in cost third termination year-over-year to share Roche, decrease diluted net GAAP XXXX, The and the associated sales XXXX for clinical in cost per these increase label offset to of the months million the the during cost XXXX, of recorded reflects million $X.XX in ELEVIDYS respectively. We of million million a a share costs an partially compared and in we manufacturing manufacturing $XXX.X million cost a or we loss XXXX share net and September XX, to of of our $XX.X net sales $X.XX third $X.XX a expansion agreement, $XX.X million. non-GAAP of operating income third the primarily of the of or $XX for of approximately supply quarter expenses $XX diluted the to $XXX.X $XX.X XXXX. In R&D million or of million and and an net ended $XX.X share XXXX. a compared quarter and XXXX, the in increase per reflects reimbursable diluted following primarily cost June. On share recorded a million same the we in a $X.XX $XX.X in inherent co-development net are sales by the reported in our quarter or the basic third a termination X The of commercial a basis, As activity reported with basic and

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

$XXX.X and of we to income to compared XXXX, primarily basis, to related headcount. On of same in to an an no strategic basis, support primarily in $XX.X for GAAP continued cash expenses, third same or cash increase XXXX $XXX.X due X million XXXX. SG&A the third investment expenses the cash, equivalents, the with ended impairment driven the to as of for quarter XX, $XX.X of period compared quarter to commercialize the million had was $X.X other of We period the The personnel SG&A. increase net a the an $X.X non-GAAP increase approximately loss XXXX. basis, $XX.X efforts we XX, similar $X.X XXXX, were for for long-term activity months an and million. in the million in GAAP change changes professional other the of by billion during a is third an a increase XXXX to restricted other net recorded ELEVIDYS of compensation increase and primarily services expenses XXXX. On XXXX, million million. On The and recorded for of turning in used of a of Now million approximately in $XXX.X and respectively, September September XXXX quarters million investments

commercial strong We conclude, for on execution to to positive the the Doug the a unsurprisingly, profitable management in focus in XXXX. And financial sustainably continued call over expect turn Q&A. early back be for and now and Doug? has flow perspective us and cash strong position. our non-GAAP GAAP our start So I'll put both to turn expense with that, will

Douglas Ingram

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

Operator

[Operator Instructions] to the Our Securities. BofA first question with come of from is Tazeen Ahmad line going

Tazeen Ahmad

the I rest wanted what bit guidance maintaining remarks to the you your said year. of clarify prep in for little a earlier about

So upside number the what that that number when original now beaten you us for is? XQ Can the gave just based XXX% Is guidance, you had said tell what results XXX% you expected that from guide you on us base that would upside reported? to you you expectations. XQ you've in it

Douglas Ingram

the XXXXs about population very we guidance and XXXX or very also guidance and QX. gave approved with note sales are of gave for we Yes. had for have to the before, -- X so, be we'd in be around for our into continuing billion, linger XXX% XXXX, of which projections, X/X number we're which treating above ELEVIDYS long-term over the comfortable which the of growing comfortable X/X was and or confident we'll we that be years. PMOs. And that the $X will at feel which our the course the would We would long-term -- some be be billion also therapies guidance, across continue very, more XX $X peak prevalent ELEVIDYS with with would year we we be very will of I that guidance and we the

Operator

question next with Barclays. from Our Gena comes of line Wang the

Huidong Wang

one ask only the question, will can of I I discontinuation about XXXX. PPMO ask Since

we FDA with color at at it XX discontinuing regarding what more be -- raised? that a XX also maybe bit what you bit Is data you over in the cases? And the was Is Can give So the at PPMO the back, mainly still And line give the level. could little planning you that with X.XX% the protein severe you that time, little XX% and you a old franchise? XX-milligram safety? protein And projected when more it you completely it hypomagnesium? weeks expression. main are point projected dug is since color can at see concern the the to weeks more

Douglas Ingram

Yes.

-- of question the the question, Gena. So over comments about PPMO to I turn decisions our Louise first thank all, for some make will you your to program. I'll regarding

Louise Rodino-Klapac

all try parts. of the Certainly. I'll sure to make I remember

entire So other franchise, use we this -- on was the of all through stop discontinuation penetrating PPMO cell discontinuing to also multifactorial. and on the are the the so, includes program peptide, same franchise. PPMO based They the the programs. The decision

that At So the approval of evolving cases. the landscape of And was prolonged this that of was said our FDA hypomagnesemia which accelerated the current some it we've to of in was that. date. the included then ELEVIDYS. based benefit-risk and the on had approval assessment, we on including the profile -- the open development Duchenne, hypomagnesemia not based that was with pathway which safety, consisted which own primarily the our It point, included including discussions saw, FDA, the on

together So to our of led things taking all decision. those

Douglas Ingram

this, folks. all remind to just Let contextualize me just

also of rests I the things. oligonucleotides, in X one safety obviously the agency you precedent but morpholino safety So And muscular of February accelerated well is of then, commencing analysis use and of that value very the had that dystrophin did change. the safety as really a for for approval FDA concept none. When the And itself really the will second PMO, and on almost of continue. the had that rested with then XXXX, analysis. change a PMOs, It's production accelerated risk-benefit and profile a really light to for the approval was precedent pathway delivery to in on it independently that course, of that rested on it dystrophy, wasn't of confirmed we in the potentially program a profile PMOs, comfortable concluded laudable, the risk-benefit attaches that think should this the mode Duchenne

we first in I as the where foremost, serve. is stakeholders the this that's think our we well. And of -- benefit it and patients is course, are. So that it then in the And benefits other secondly, of

Operator

question And the going is of our come line to from next Andrew Tsai with Jefferies.

Lin Tsai

Congrats on execution. the

the worldwide X ELEVIDYS XXX your far, partner, treated XXX made Roche, about So how ago, weeks launch, comment a have in patients including U.S. about so been the

even So out sales can are jump QX a be implied reconcile patient QX you we us in help getting could good Presumably, clinical But that XXX your could U.S. guidance. be patients, chunk in above trials. a those that number? backing nice,

So us that. maybe help reconcile

Douglas Ingram

our we I'm enormous guidance we're Andrew, metric, the to to those numbers stands revenue it and comment not that it on previously. the going as Yes. today, that. or as dosed We're that provided use qualitatively is certainly an case mean of that have -- going we like number confirm we patients. standing I haven't provided

We a seen late-ambulatory other either patients dosed. difference metrics. are of that now, mentioned already in dosed, have to with non-ambulatory patients you of and an And by about some will the XX more commercial, any all probably or have amount safety than have we've XX clinicals that we ELEVIDYS. Louise experience we've that in between not extraordinary so, as you to addition know, or

and great. things look great. profile the looks the going The launch is of the great therapy So

excited where are you So now we update with it. And to give that's QX. an we're right after

Operator

Markets. RBC come with of going Abrahams Our line the next is question from Brian Capital to

Unknown Analyst

This on for is Kevin Brian.

you you've can in speak ensuring progress remind conversations payers and in coverage And Medicaid with Just coverage policies on to estimate on to to made you maybe what policies? proportion be ask, more DMD us broader efforts your you patients if what can with speak Medicaid. of can wanted to you

Douglas Ingram

Yes.

if to the are following I've me. it's anything well. we're have patients things commercial right going of data add couple Medicaid, a So a remarks, perspective of a error, the to fundamental it's a commercial, productive. And made and that Dallan, if we is group of -- one, Medicaid from evidence the both one correct perspective, about you benefiting very about or last things; decade, nearly in increasingly fairness, kids payers really in great. XX% and there's years, my in broad supports things amount XX% access that gives we a It us of Duchenne the Interactions, are manage Broadly conversations on The process bringing At all having now Sarepta on you're can ELEVIDYS lot and that working very payers speaking, our is about for to a of therapy. down this. If talk that gaining physicians get we're become expert time, absolutely about. with have and through quality the X therapy. depth patients benefiting is one focuses to the and same with a conversations from to very become of have those more sophisticated over how fantastic, to we're itself, the

works Medicaid also entire you're launch. how would and course, to we this that they good more resulting of all launch, had prepare discussions and the that very, imagined we dystrophy ourselves the that the may the the that today, been are which for trajectory is quality productive years matching so experienced far it's disease is, seeing with commercial, both which in And become payers, have with did that of performance work and have very the Duchenne more muscular some like X than of ago. have far And that we

Operator

with Sachs. Our Goldman of Salveen comes the next line question Richter from

Tommie Reerink

on Salveen. Tommie for This is

infusions XQ And there end that possible the the some the tail that think on believe on that just XQ the the call, quantify were launch to in to was to how So thinking it broadly, more year? rescheduled XQ. of what's I mentioned Is peak it latest from were of impact?

Douglas Ingram

going first -- question, part Dallan. I over to the to I'm the of will turn

growth part over if prevalent we're XXXX. will I right more say by will to is decade, that that go a to want of the a through and going now is the of comment this on to With QX? second you get we question the through bit said, significant Dallan, The population little of that what period course you

Dallan Murray

the and so off and that sales our if remember, QX impacted that guided or way, already. we guidance question Yes, off which of was, guidance sales, in -- expectations QX our for was factored our performance into growth of QX QX any

there's real of of So that. the quantification no impact

Operator

Our from JPMorgan. of Anupam question next line comes Rama with the

Anupam Rama

Just a me. quick one for

kind like near a It in XXXX. Day looks of in at -- hear we're little on more your term. bit pipeline here more details R&D bit an little going the a given You gave to internal focus

think Just about And should know you time said external earnings, wondering I you one you how quarter at but would of the a guys on ask sort I some January, might preannounce its internal cool. small question, development. resources since conference business in the healthcare pipeline that guys spent only third versus could doing and if if we just be

Douglas Ingram

Okay.

So first question, of for all, of your very let me thank much all, you -- Anupam. first

enormous that of bringing of result hopefully, and in now a question, to any a very are we of you right -- impacts so significant of is is your going doing ELEVIDYS. the seeing not things. that performance years major not the really from far, X on which can and see which course, of launch work durable, amount kids, launch first first important right ELEVIDYS, let and that's PMOs, swimmingly, very also lot themselves me the continuing it life And we -- folks. better that's a have As One, say part well, to as is relates the by of an over focusing very are many of now,

there. fantastic all So

program. stage, are some But late be trials few that can very in near in deeper we we'll and clinical next pipeline term the things organization effort months even of advancing of in lot a you -- right really of in X a We're getting of traction now internal next there. big do of raiser the kind second pipeline. year this you is We're of The some to There show a going excited about. great of our we're the our really excitement. lot curtain on and is the see limb-girdle the

that's what an that. very beginning as right we're focused accelerate, So excited to about That's organization we're really on and now. very,

to going are we Yes, go.

internal Ian basis flow we're going a said, non-GAAP have we now, opportunities basis be early prior year, some and think it's that which our even been that things starting be we're positive therapies, gives of going about. are As approved you're of including the from accelerating profitable us our excited we're and very success we right X more But right broadly. really to and ensuring a focusing if go-forward the quarters on to next GAAP this cash ELEVIDYS advancing on really to minute, as the has pipeline, launch and now, a about us and in asking

to second commitments. make the I'm on any not going one, Now,

where at we January, here history we there, have As do right we and we pre-announcements what as but doing of will in That's stand know, a course, some we likely but are. consistent that now, conference with had, we'll January history. wait in you of be see

Operator

to with going Raymond Danielle is Brill James. from come question the line Our next of

Danielle Brill

there purposes, launch is like going XX our sounds for and it XQ, is in and additional treated by patients the great. XQ So all math, versus intents about

just are So kids treatment these I getting the the thought bottlenecks step-up treated? pronounced that you expansion. in to guess Can more and what in label could main we what seeing bit more little you're about market following the be talk just a rates the

Douglas Ingram

say Let me just again.

other we're exactly excited quarters spoke trajectory all, use in back. few seeing about metrics. it's we're as launch, And going we progress marker. We're performance, the when -- we're revenue First very we going a And talk had of envisioned going to and to we about and of to we're plan. not that our about the talk our to going

X is that going growth fantastic. The everything broadly is combination of a and of shape always will things, So it be.

ELEVIDYS Some that's not simply a is that post kids It's you dosing they are of of making up have expert safety of ability today it and from and efficacy sites it always -- and so positive sites is have more that profile a we my running. perspective. matter sure getting and manage at that this site have monitor have we the that that to well-informed, well-educated, piece least with and extraordinarily capacity,

going well. going perspective. both that one, third great, very and is a Medicaid perspective release about are and just the Everything of sure which payer we're that. And from making is batch interactions, commercial course, is thoughtful The one a manufacturing second and

growth as QX and and both will of years population that, now the through we reiterate long-term prevalent able we So about decade, treating population XXXX, mean treat multiple things and tracking why are provided a also exactly forecast incident to of and had hoped, as over even we our be into that the which the significant guidance the is the as period the for going that's we course the we, fact future. previously, we're guidance and our deeply this $X of are of is that billion,

Operator

comes Ellie with from next UBS. Merle question of line the Our

Eliana Merle

Can mentioned on sites? many site guess, latest just elaborate But site guess patients you're how the dose bit more across on just seeing dispersed I And your ample been just that seeing? capacity. this has capacity a I You comments concentrated you sites the how in now, XQ? dosing or you're

Douglas Ingram

Yes.

-- just we those have sites. reactively, So might actually we sit They we as what increase to an of modestly broadly, answer have have about Dallan today, sites on, time can here percentage over been us a so sites. dosing XX I'll and say provide

Dallan Murray

and sites specifics quarter-to-quarter the really majority centers into don't have these Yes, vast dosed concentrated patients, how of on we get are.

revenue, we're our So, guiding on we're product focusing said revenue. as Doug had earlier, net -- guidance

Operator

from going & Gil to come Needham the is question next line Blum of with Company. Our

Gil Blum

progress. the on Congrats

away for that is from life thoughts management moving strategically like of of now feels PMOs? cycle are out, XXXX Sarepta So it exon skippers. seems any the there And kind

Douglas Ingram

want to their are of constantly First, we good that but about both we're benefit well. as we're the exon reason moment now small really -- discuss PMOs not emergency that's we an EXONDYS, are The -- evidence patients. The skippers. lot right to other cardiomyopathies time looking and not of also room therapies but are right in excited, so multiple multiple is CNS a ELEVIDYS suggest visits right pipeline, from consider. The We're to what doing that away position deep important away the to though years which have other VYONDYS our are now, same And and thoughts will amount meaningful the recently that they're but at a provide dystrophin, moving opportunities a even years and excited on that rest and is very in the we reason for moving enormous tremendous. the like. we a from make skipping to enhance do, and including very of experience skipping. now, exon our ELEVIDYS a a can neuromuscular I in do and detail, AMONDYS modalities seeing for for thing don't We're years any of exon be as them have PMO about presumably and real-world dystrophin off answer amount reductions a of over okay? multiple as mortality, of published So wheelchair, an on small out better amount vent, we

we a that one providing an even while safety that to PMOs very remain think about very as but to to And PMOs are great the for and exon-skipping alternative about that the is significant we high. And think others, benefit why they should any that's patients, the our So long. really on provide excited road that's always approval only really our side safely PMOs not remember, beating of the that about great. is bar things that's we ambitious, patients, one profile very and for is we're own benefit and the can our the the about is the to pipeline PMOs

think benefits a one the to that for PMOs, either we or or I less and there the road they're VYONDYS ton will that about is gets particularly in way excited that way. And during think And patient remain our we're So a seeing the every we're I I durable. right cannibalization, be about to of fact the PMOs. for not don't long EXONDYS that a very excited want excited that that week launch, and I'm things suggest from now, is minute any AMONDYS. and going We're of a an infusion

Operator

Cowen. TD of Our is line going with question Ritu Baral next come from to the

Ritu Baral

-- guidance, kit, Doug, and make you I especially wanted about when their insight and just mentioned et guys to seeking patient where forward you the you time -- to we reimbursement are X-month kit, given? And should two, guys X granular to Ian's swing months' the And notified is fill. the are as despite sort beyond to going you thinking of you've we ship ask be when congratulations, have logistical what on they're about about to to basically insight, some this guidance given X given revenues. a to should going factors X- cetera, have swing when we one, tendency to conservative to continue quarter, guidance? factors beat, get healthy pretty are give given dosed, that

Douglas Ingram

with. makes that. we always if it's with ensure to to us that our are guidance, we but provided. comfortable that accurate provide course, we goal of will we're proud of very continue that seem the that Look, I'm And comfortable reasonable guidance And and probably guidance conservative,

muscular lot positive, of our notwithstanding down going at preannounce about provide and like, does with very amount and start like. And not guidance forms prevalence, -- JPMorgan, about that talk my sit in you moment, our the with to at view, as significant but going only not JPMorgan again, said, at to beat, update probably Duchenne in broadly us if know, a the to about think all that we and enormous forecasting as like today, and this here do we We're XXXX. guidance and of we'll an we're and have insight, dystrophy the way we We as and about epi confidence things

Operator

of comes Joe question from with the Partners. line Leerink next Schwartz Our

Joseph Schwartz

enhancement a you will characteristics, at evaluate strain? you And do. will edema study plan wondering to And how tissue longitudinal you have evaluate the peri-dosing? gadolinium you that presented I question about global the WMS? will to fibrosis, differ next using Great. this MRI I'm data be like MRI from and

Douglas Ingram

Louise?

Louise Rodino-Klapac

Sure.

time, from natural now. dosed longer-term EMBARK following that and been the history So natural be patients patients we'll data of since the data, if be a compared, over then, the wealth compared there's history all have to study, will

history. natural then and be compared baseline to their they'd to So

early gives the So inflammation TX. we'll edema had MRI/MRS noted. and do to continue of signal TX that the you and

would a have and to MRS. you precursor seen that's by So changes that MRI

those to as continue the of use MRI a and measurements we'll results. X portion So strain

Operator

with line Konstantinos the of Biliouris question comes BMO Capital Our Markets. next from

Konstantinos Biliouris

quarter. on the us Congrats on One manufacturing. question from

the Given potential do further and approvals comment and follow-up high the your manufacturing And manufacturing sales or on only a demand? optionality level this Fisher with in with there can topic. Japan, that the of to be upcoming acceleration suspension is XXX% whether of quarter-over-quarter Can meet ex-U.S. ELEVIDYS you well? to growth Catalent you Europe with clarify on will capacity Thermo same as

Douglas Ingram

Yes. Thank questions, your very for Kostas. much you

forecast We're our the to very relates position question, from in our manufacturing with supply. it your first part we're of a needs. partners' to manufacturing ours serve and and to as our comfortable good First, very a perspective

As it made suspension we well a and will. to stands going in as have it believe to use commercial well, And supplier suspension, We're today, goes relates everything very assuming we with suspension. going X,XXX things to both year a not on it next who for engineering study as we're we decision, will in XXX-liter right are bridging and everyone, start runs, our liters, remind now. and

ponder continuing So evaluate. and we're issue that's an to

Operator

question Our Brian Skorney next Baird. from line of with comes the

Brian Skorney

an who or serology? access in higher HNSA-XXXX Or adequate target had prior have study cleaver question that? on your Hansa clinical received you're of do here? you Do on kind guess collaboration rhXX Hansa ELEVIDYS I either about? focus redose, how had and And data study development imlifidase should in naive to about just with is dystrophin you think do but go And apheresis patients response? we Do think is you to included initial when the seeing next-gen here My you from deal? the planning about patients or

Douglas Ingram

this turn right. to I'm Louise. to going All

a I Before for I have clarify. very Brian, much clarification And, the here. opportunity you make and do, I question sure thank want to your to

road allow cleave we're or either studies. the we is our that today, to just clear, modality down sit redosing. opportunity some so as the in use goal near-term So definitely about there that of manner But not excited neutralizing actually a antibodies we're is might clear very potential. for that to here an And

that put cleave through Duchenne color are exposure screened is to could studies acquired to or ELEVIDYS, who would and we or actually right to near-term for with to them which would by different, a of in Our get patient intended do opportunity something if dose have on provide Duchenne them antibodies patients where environmental be out ELEVIDYS. And with so provide Louise, another questions? some position a that now an Brian's opportunity have that, XX% to been clear to want the slightly you

Louise Rodino-Klapac

Sure.

on there's in So studies. we when a data question about a us We'll and early expression initial the for will to regards redosing. data potential data we'll XXXX inform safety with cohort. in study anything both have for the And have future do really, for that

data do. As case nonhuman Doug to mentioned, but good primates. ready that's not shown ourselves be right positioning now, been We've the in we we've goal in

for much challenge levels. antibody the higher redosing So is

these go from with we a design data preexisting antibody how if to and charts redosing. might can study seeing this do critical so we were we so how far will And be in

Douglas Ingram

negotiation any of to a And answer question, on therapies. first have final then, your Brian, right next-generation we

Operator

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

Kristen Kluska

Congratulations beat. on revenue the

of credit lot whole a for getting limb-girdle the be not to portfolio. seem You

how So opportunity. these wanted market the could ask the about us launches should we to patients around identified be be that and can of you I time expect remind of about if number you thinking

Douglas Ingram

And about opportunity at is the to of in so portfolio. dystrophy, quite Duchenne you investors traction particularly do the today if and of now fast probably size poignant Duchenne limb-girdle our much. very make we good in of And same XX,XXX XX% you on muscular to XX,XXX, massive portfolio me and you we're starting Yes. States. limb-girdle XX,XXX because question the say speaking, this think can about That's a great significant. time. range opportunity more by United Thank the Broadly let have question. it's well is the it's move like see muscular maybe And to do that really that dystrophy, the our a a

in the of muscular programs of late-stage -- the actually program of be are and the range X to near-term development significant. quite going in few in X themselves Duchenne something with sarcoglycan Our They those all dystrophy, more we're are months. XX% coming or

on big hopefully, think opportunity people this more ELEVIDYS, do that the confident a is get more that. helped clear and launch it they about start will excited as quarter So has about focusing I to and of and

Operator

Our line Biren from of with next comes Amin question the Piper Sandler.

Biren Amin

what standpoint? from access just both you of about non-ambulatory the And you talk across that type saw seeing of versus are split ambulatory quarter groups? a you Can patients third maybe across the in commercial

Douglas Ingram

broad of that is launch, to be anticipate, and I very favor in for as that, but that late non-ambulatory things anticipated as may mean, the we going non-ambulatory not the qualitative We're seeing one a of granular patients but isn't some Yes. bias breakdown ambulatory, forms fantastic. a that would launch. I that there patients case, of you at may others some probably anticipated can significant it imagined. is have patients, ambulatory one a over certainly, percentage of have And is color. significant give We're at start which provide

it's advance XXs, to very for been out ambulatory forms Duchenne that in start dosed we late think that that which as the safety dosed increase is We've and patients, in that the men and of the non-ambulatory in there, as only obviously and the information mid-XXs, excitement. profile seeing the more going awareness are dystrophy. have is already late non-ambulatory, I very, kids who ambulatory, have had which comes muscular their the about same setting of we're patients number

while very it's percentage -- is is over of some non-ambulatory, very that's to the to significant is versus missed? increase a towards bias about and else early quite anything is that, have non-ambulatory, that continue anticipated, confident time. Dallan, we days. So guided I've we're to ambulatory there there as there and There going

Dallan Murray

just at quarter-to-quarter. to to not going fluctuate No, And really not add, all. it's

that you bias quarter. of the of going said the from don't launch, in in early expect is fluctuation kind we As but towards that breakdown had the quarter population stages ambulatory much to

Operator

to Our next the line Evercore Gavin going ISI. with is of question come from Clark-Gartner

Gavin Clark-Gartner

week, XXXX? confirmatory trial the primary just I'm and for trial, in still still latest just guidance wanted Viltepso broadly results? I following any it is actually to at page, really completion heard the next date trial, data do anything may just notes pull for on of you looking the ESSENCE ask actually. is more And market drugs the the what haven't off be, especially these we fact the this have light in sense requirements to the the

Douglas Ingram

first in correct Louise, ESSENCE if all, XXXX, wrong, the I'm readout correct? Well, of is for me

Louise Rodino-Klapac

correct. That's

Douglas Ingram

standard. in to and Yes. should a evidence the evaluating it And on as then at the with of associated When that market of been standard isn't be the including then, with relates look evidence. based that the evidence it therapy or trial totality results, positive whether yes evidence will real-world for of totality has study standard, therapy, we've results, the that far, information the like FDA which won't to the the we'll no all be the make the study removed in PMOs, presumably and decision. It all therapy, including including single gathered on from trends the on the respect is is of the very a one a based so

Operator

question the with from next to Our is Mizuho. come going Leo of Watson line

Leo Watson

How skipping 'XX, on exon you're the dynamics PPMO? discontinuation This cannibalization how expect through data on just is we're going on in in Leo And you given recent competitor Uy. term. quarter. strong and cannibalization material are of long thinking the competitive you no about the the while curious about for thinking a readouts the forward the business Congrats

Douglas Ingram

first. question second the answer me Let

net after So we're won't at subject if How entire those will have of in reason the basis we We the to term also to we and going any cannibalization watch see XXXX. be ex-U.S. of all, don't have goes believe do very are but over significant, cannibalization. some least we'll but to the on near over these sales this we'll something to be certainly that that course post-'XX. think therapies Remember, cannibalization is see 'XX. monitor, we PMOs, how net model and that a going be decade, durable, at

right we're that other on I is on very should them either and relates us PMOs competition programs, I certainly today, near well. it not on one comment competition, and As will the focused now. folks' or not I on wish ELEVIDYS, note There no to

about of open the eteplirsen, want doing the much when been out, the today. I PMOs, to late to issue those was that is supportive. PMOs that real-world One do an There in the as are point but approved, EXONDYS really has relates the good it perhaps things how was XXXX evidence

with the doing a can in amount these enormous You therapies. it see laudable They're profile. of good safety for compliance really, an rates really

think getting this bar. -- long thing and perspective exon This for would about ought over And a we is go the say think time. I so PMOs and And data that for long road exon, to to both when a remember to the competition, safety that high one allow think to one overachieve clinical PMO of that -- the one. requires is an we about approval even very period is efficacy one a is not very the buy about the bar very to one would from

wish these well. folks I So

long a have to place. I that get the think road and right they bar a high to

Operator

come Our next Corwin line going with to the William question from Sami of Blair. is

Samantha Corwin

quarter. the on Congrats

then limb-girdle, do those used in there savings be limb-girdle any development any launch any synergies reps you the sales of XXXX, and to LGMD And programs? discontinuation to expect in ahead between ELEVIDYS cost centers? with treatment a the or Looking accelerate could potential of of of terms

Douglas Ingram

Well, access group make with done of the to that and limb-girdle nuanced reimbursement folks we expert, that fascinating those rare and we there's very service a here focus Sarepta neuromuscular have in we've a successful. advance that gain and have EXONDYS that going to XXX%, very, a to It's your consider things we're late community There'll seasoned there that sure But between to of today. very ELEVIDYS? yes what back be synergistic. so to synergy become launch way group Is need and to and to both be in launched a very disease yes on thing the remain how of and a the absolutely. folks do to XX% the XXXX, community in distribution. It's with know questions.

same at of of So and really, well. level frankly, ELEVIDYS safety they of And yes, talking synergy limb-girdle synergies. we're will there'll that folks nice shoulders really standing really I these capsid, the would as therapy the the be on as same shared the the lot programs that ELEVIDYS. so remind a about profile be promoter

it that So to because don't these can the possible then, based fast plans suggest the of move productivity the on XXXX, can of cost we and only these I as want to shoulders accelerate savings standing the you're we're from as ELEVIDYS. And already relates doing on is to therapies as everything XXXX we in our be focus to from earnings development announced it back into call. next pipeline, made and which the to talk progress seeing to in that But XXXX year. savings some down research certainly, cost we advance this program, get we've that will and and very including we are there about excited use our

Operator

Mike of Morgan with next line Our the question comes Ulz from Stanley.

Unknown Analyst

on Mike. Rohan is This for

tracking any versus and sales, provide how trends the on color they're inflection for can you XQ about expectations? ELEVIDYS point early First,

Douglas Ingram

right do to that We're not going now.

the XXXX, provide would which for of X ELEVIDYS -- will very We approved come standing are Notwithstanding billion, our is that guidance from I a $X from us we're previously. will for have by our to we -- our come we've appear which and criticized some conservative, you, sort are QX, guidance guidance significant for X/X X/X and which of reiterated we PMOs. that X our being reiterating provided of

over very with we're of or last internal forecasts very as are organization so. about feeling we our good and consistent an So where year the all

Operator

the to conference hand closing remarks. to back for like now would Doug I Ingram

Douglas Ingram

quarter. lot time PMOs starting are a approval getting very your excited what evening. -- comment I don't one we're you perspective, the I into that to be we We've a is really spending of this for there made questions moving support clinical the about about today, as launch with, and From thank can well, trials performing those agree are pipeline. very which think going positive swimmingly, will there's therapies well. And the insightful had pipeline much by of been with traction very our and programs very late-stage And standby has for ELEVIDYS us. well our and we're for we're Well, limb-girdle the enough but our to the really us see or need on very and excited in well, as very generally, all our who good girdles there, the spent attention investors opportunity the do who do limb us both patients some

I all to performance about next fourth you date when quarter much. all you announce Thank the year. performance and to quarter to fourth very keeping up you we look forward talking

Operator

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

disconnect. You may now

Sarepta Therapeutics (SRPT) 6 Nov 242024 Q3 Earnings call transcript