SRPT - Earnings call 4 2023 | Sarepta Therapeutics, Inc.

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Unknown Executive

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

For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10-K filed with the SEC as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today based on subsequent events or circumstances. And now I'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?

Douglas Ingram

the it By yesterday. France Fran. you, way, was everyone, birthday Thank

the Results by growth happy afternoon, and VYONDYS Therapeutics AMONDYS, of joining Financial good Fran continuing quarter Fourth another strong we quarterly Call, and XXXX So growth patient approved and as and for performance everyone, of birthday community. PMOs of exceptional we an launch thank and our you serve Quarter announced year Conference elevates afternoon, led EXONDYS, full Sarepta this

As at the the product total net year we prior year. the JPMorgan conference, net product prior in January over year preannounced at growing revenue over fourth achieved revenue $XXX.X XX% of full quarter came some $X.XX quarter in billion, same and XX% million, growing

will exceptionally proud to attention level all Duchenne. Murray, bringing for speaks to detail, to living here, of life performance in among representing continuing label those speak X or about million and was for Officer, therapies of this $XXX launch to ELEVIDYS performance reflects performance $XXX.X over innovative our was X-year-olds impressive preparation a with aspects Dallan course, so the excellence. team's year. expert for understanding net the Notwithstanding Duchenne addition launching million quarter, rare our passion which full and therapies, to only of strong particularly and his X- the Chief population. approved and ELEVIDYS first-in-class of total remarks I'm disease product our of the X% and, a revenue shortly. of to our Customer limited better In

And organization. sheet. were profitability equivalents, fourth having fully GAAP on cash balance $X.X to integrated profitable XXXX. third continue our we exercise non-GAAP of basis also the exited XXXX in investments commercial approximately stage on cash, billion the achieved with of cash a of biotech restricted We quarter We and the in quarter, a discipline

on color will provide Our Ian financial more performance CFO, Estepan, shortly.

for SRP-XXXX approval our the we We of In ambulation XE. commenced removing expanding fourth we January trial of for use also XX our fourth conjugated both peptide the on advanced that EMERGENE, Head ELEVIDYS and year, Rodino-Klapac, with next-generation in Part PMO, pipeline the amenable. the by quarter. the investigating our supplement goal age label results and Development, provide will restriction to exon quarter, also to progress of Also MOMENTUM BLA a submitted review BLA the Duchenne June our our date. from we this set the to 'XX Type And treat Research for target our SRP-XXXX of quarter, our LGMD supplement our FDA year, Dr. In of Louise and in treat February accepted our color are and positive patients trial shortly. review announced B, transitioning pipeline as in of fourth accelerated the more traditional. completion this

Officer, our is In multiples. Murray. will incrementally short, to be we grow internal our patients with turn short mature relying than starting approved and Dallan sustainable, financial XXXX, on improve strength, pipeline, very will build to year. lives the sales, from and and million our intend therapy, great ambition in we advance modest we grit, but to pipeline to set that X but to a Chief also RNA Duchenne in our scientific the external in are From in organization, and expand XXXX to important leaders to use a this along therapy I the ambition we Dallan? here, the in and plans of become of number year, of we successful way rare the treat greatest little will more And achieved cash out our biotech can innovation have with In going not there, Customer we possible If disease. to runway a a and genetic that, a gene call the bring vision. and

Dallan Murray

afternoon. quarter and represented $X Thank for product the revenue, The strong billion a fourth as good generated of in you, a already finish XXXX net team milestone over Doug, impressive Sarepta. to year an

noted, In product nearly revenue XXXX net of consisting XXXX. the dystrophy. the roughly of patients grew net $XXX own billion, $XXX the and PMO PML from year $X.XX of performance franchise, our our merits from our we Duchenne revenue and million previously product Each projections therapy of million net approved in As consensus. for and product stands for accomplishments million these totaled with from by of digits both revenue from their on surpassed the Elevate, launch franchise the muscular internal external gene $XXX first again seventh double

years, previous with any taking organically price of PMO on growth we approved our increases without delivered As products. this

such, we the an to community. commitment serving, this As in represents increase growth Duchenne number of the our reflecting patients are

Turning to launch lofty ELEVIDYS. We're execution, extremely with own exceeding expectations. pleased of our the

net success the million hope $XXX the of with gene given launches in for and occurred combined remarkable X therapy all Duchenne past those commercially therapy those the months, revenue with for of ELEVIDYS unmet be XXXX shows other XX gene the ELEVIDYS conditions patients approval summer. that The need. past genetic just from revenue can this viable, with surpassed providing fact, product the In

we launch support how ourselves on how externally, is patients. success we quantify this revenue we of the While measure

ineligible patients who challenge was was team and motivates narrow deliberate incredible risk commitment all preparation their our role to process significant becoming of are therapy, supporting ensuring and team who no knowledge fourth sixth X- eligible those and that intense Duchenne to approaching are to together for are experience with to The worked This birthday and eligible and rallied, is label. and behind played at the our test with patients. supported what put rapidly patients a was left how launch, in us. the an responded X-year-old the Our our

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are and penetration population. small Duchenne forward. very challenges unique relevant moving our has are narrow the presented We of the of number X- segment with that a This X-year-old label to executional pleased into

of Firstly, not are the patients a proportion this age in group yet diagnosed. significant

that of around the average age is United the States. diagnosis X Given in

who antibody involved and secure this must means top fully become a the being very diagnosed about have those a patients process access And One of window. PMOs, short also on to of therapy and this, example families be go ELEVIDYS aware the diagnosis, this more which patients time not Duchenne, had longer to educated that to than lot the Secondly, pretreatment of through time are need options understand of testing. in

race help these to in themselves patients. found has against team a time Our

quickly number situation relatively the of to finally, in small X- we working this results the will patients And diagnosed year. the X-year-old prevalent half of population in through which first within a be

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now take as look whole. at franchise the Let's a a PMO

net solid VYONDYS brands, of million. full XXXX both double-digit all in of and $XXX year growth performance growth product across X our mentioned, exceeded continued revenue XX their XX previously represents fact, revenue trajectory. million guidance AMONDYS $XXX This As

million quarter is the roughly This roughly of product million. of Looking net XXXX of now revenue revenue. $XXX $XXX product net QX the fourth versus at XXXX, in flat delivered team

XXXX As cautioned recall call, an QX due year quarter-to-quarter the in around for from million that $XXX you lumpiness at our we to were the earnings of observing keeping may we the increase guidance that time.

cannibalization finally, given the minimal incredible age Looking has clear was business impact discussed from effort now the we and we range. PML performance exactly Notably, on launch. retrospect, And the on guided. just we in as elevates the narrow XXXX, the business despite in Elevates saw PMO achieved in PMO our expected our performed it's

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Louise Rodino-Klapac

of nonambulatory this removing of and supplement in global, randomized, accelerated our also double-blind, receive gene the and evaluate therapy all with time, placebo-controlled will the who indication muscular then accelerated X in genetic that to being gene. science X Doug accelerated we that DMD Duchenne moment XXXX a patients approval mentioned, treat a disease. filed medicine. we've confirmatory approval. efficacy converting our of as our we age remembered older being aged a ago, by ongoing United United for the is and thrilled mutation with in announce ambulatory will confirmed ELEVIDYS defining years ELEVIDYS restrictions was ENVISION the and remaining moxeparvovec ELEVIDYS. to XXXX, FDA to for the outside June gene now ambulatory a the dystrophy. therapy the is accepted a living X for rare Duchenne and with broadening be were will the the aluminas been States, advancement treating well-being about non-ambulant Duchenne. Envision patients ambulation traditional delandistrogene in called of for FDA States. Should the just Since to XXXX Dallan. first weeks Thanks, patients study, study accomplishment X-part This through year And for successfully population and as have the efficacy and and pediatric and individuals health great of approved safety ELEVIDYS, In study Sarepta study, enrolled of whereby SRP-XXXX-XXX granted with also approval for in approval population. as evaluating serve they

enrollment completed U.S. of in remaining With XX% U.S., complete ability occurring we trial. ex confident this are our recruitment and the to

announced Moving to LGMD our January study. we underway also limb-girdle SRP-XXXX-XXX, now On study or muscular that and was screening known programs. as dystrophy the EMERGENE XX,

beta-sarcoglycan, XE of is program. that the The and EMERGENE expression LGMDXC circiclycenopathies, is successfully including or that are share limb-girdle explain beta-sarcoglycanopathy. to for and patient for dystrophy We the this SRP-XXXX a endpoint Phase trial the gene extremely the open-label therapy. why. pleased has primary muscular you, for type other study. clinical is dosed and field treatment of not an in important of remind emerging LGMDXD endpoint of for multinational of been To I'll first which III

of were or or we sits of the proteins as defective functional to subcomplex among as LGMD the officials sarcoglycan by in LGMD damage designs restoring a the as and opportunity sarcoglycan well of must as workshop Mark which for complex others. able higher workshop, restore is replacing the the Speed therapies. when called such cycloglyconopathy well the dystrophin. a beta be success, and from have and chance the is the sarcoglycan to muscle. as function as [indiscernible] in sarcoglycan Beta you're reduced of The protein ensure such of evidence loss missing thereby support that Foundation's I best their function the [indiscernible] expression that to by other for expressed of had scientific a types restore the LGMD therapies this takeaways important other and Mark which for as month, complex sarcoglycans featured at complex strong proteins, participate not earlier totality DAC. for and well are support for complex endpoints the FDA, membrane native dystrophin-associated gene, suitable the Therefore, to during of the muscle in and preventing for mutation tolerance traditional flexibility membrane key uncertainty rare beta-sarcoglycan, clinicians contraction. Further, complex gene is A complex a the as results regulatory protein protein Doctors and for considered. as The perspective that certain for such diseases, the also a including in surrogate included trial these from and patients, Dr. characterized also caregivers

treatments the which no are a from shared XXX, study, treat effectively as Medicine VOYAGE ultrarare foundation will initial and of suffering experience These as to and supports across LGMD. that also only programs ambulatory study, which X safety broader who the earlier which nonambulatory, from Currently, exist LGMD patient this The participants or a our other and pathway but LGMDXE regulatory XX EMERGENE and for LGMDXE for establishes it rare holds great Nature provide promise in the study, viable data, and other will SRP-XXXX-XXX, lay development individuals LGMDXE year enroll diseases. older, with not is positive expression is SRP-XXXX data gene therapies combined population. published functional our for future well our

Third, as totality evident. together as

I and older endpoints and to in a ages non-ambulant population. and of Phase the in evaluating safety ambulant for treatment change patients LGMDXE population primary The in As VOYAGE SRP-XXXX study X expression. the a beta-sarcoglycan reminder, XX ages in is are XX the

have results year. to the expect We clinical this

date, study, also clinical SRP-XXXX we've RNA MOMENTUM Based we of an is We Moving positive SRP-XXXXXXX. Part to a represents platform. therapy now perspective. the our of B believe to dose to recently a from data from announce SRP-XXXX. results our global generated on best-in-class multi-ascending were pleased efficacy MOMENTUM the trial

exon amenable older phosphorodiamidate to were Our next-generation peptide skipping. morpholino patients treatment, Duchenne the XX with

with discussed these that will NDA on XXXX. an results, anticipate an and of meeting and we discuss to accelerated previously the We the planning As a this are have FDA forward we believe quarter to occur path in approval. based third meeting a

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

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

In addition, our EXONDYS. post-marketing for we in enrollment MISSION our to pleased have dose-ranging commitment are Study, completed

promise a was fully comparing study. mg October kg diligently the mission remain efficacy it Estepan exposure and disease. soon over mg study I Ian turn Phase the III that Mission XX weekly. update safety rare Mission is committed living double-blind around randomized results. a and sharing dosage and will advancing rapidly world are with our weekly with kg significantly a financial up missions as patients data higher with and We ahead on to months dosage XXX as provides dose-finding becomes advance Ian? our available. per the great now approved per of XXXX through an XXX call eteplirsen, in conclusion, serve filled is for to the X-part patients. to to It enrolled In

Ian Estepan

fourth basis. a for available non-GAAP quarter our to reconciliation to as GAAP details a as LRK. the GAAP for well Thanks, Please press afternoon, financial XXXX release press This refer results. of website basis financial results on full everyone. Sarepta's provided afternoon's release of Good a non-GAAP on

tax results, from Before or that The effect we get reflect based updated of has excluded and income on fourth of financial no to XXXX year company tax the for tax amortization purposes. just of adjustments, which been non-GAAP impact this benefit change the in I Non-GAAP to added to quarter represents income fourth income results in-license of the beginning wanted comparability XXXX the full rate. each non-GAAP estimated pretax expense income longer the effective rights and quarter have for adjustment applicable tax and results. are flag

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

were million for net XXXX of product million revenue of XXXX, XX million relates increase product net X increasing the million Net PMO for $XXX.X was December The In quarter exon reflects collaboration to for product product XX, the same XX. the net XXXX. compared we primarily from for franchise well quarter of million million. $XXX.X other revenues $XXX.X revenue of $XX.X with in products period for to associated from revenue million product XXXX, for in and of recognized revenue net sales sales skipping our our compared ended VYONDYS the $XX.X of fourth as This revenue $XX.X of primarily our for period $XX.X XXXX to was from ELEVIDYS. Levites demand quarter as Roche. XX, fourth collaboration PMO and arrangement $XXX fourth quarter individual AMONDYS for million million same EXONDYS the with the and

co-development We a basic XXXX share $X.XX net and per fourth for million $X.XX quarter income share the $X.XX basic per diluted and loss of reported diluted or of per ELEVIDYS ended For batches the manufacturing the Roche XX, associated multiple ended agreement to for collaboration XXXX. supply or non-GAAP XXXX and and $XXX.X with XX, net same XXXX, net respectively. basis, $XX.X activity XXXX XXXX, or the XXXX. $XX.X a with of the of $X.XX revenue to to of December reported in $X.X $XX.X fourth million net compared million quarter of company quarter GAAP for $XX.X $X.XX diluted recognized December of quarter per million fourth compared commercial a non-GAAP share or similar the period of The loss no share fourth under in million totaled quarter income contract million of On the delivered per of million reimbursable costs Roche $XX.X diluted of the we XXXX.

increase we royalty of our same our for certain and a XXXX, PMO year-over-year million quarter period compared in R&D XXXX not in specifications The in increasing reflects demand $XX.X million. of no recorded primarily $XX.X a In million basis, for similar sales months in quarter same period of other due of and meeting no the in activity primarily period the of $XXX.X sales of $XXX.X million XXXX. activity $XX.X to payments our basis, the X quarter fourth the decrease the cost of XXXX, to million products, an products of $XXX.X milestone XXXX to for were fourth the expenses cost XXXX ended compared of XXXX to a quality the of million. sales and approximately in fourth in recorded million non-GAAP a XX, million of decrease with for and expenses increase we manufacturing write-off with XXXX, respectively expenses. similar ELEVIDYS for December XXXX, On batches and in R&D for $XXX.X due decrease and upfront On GAAP a of is decrease the expenses same $XX.X XXXX,

The and in expenses, investments in of Now equivalents non-GAAP to to compared for an increase million of $X.X an XXXX, as expenses turning million $XXX.X basis, a personnel long-term and $XXX.X and a for XX, of $XX.X professional the $XXX.X and due XXXX approximately change $XX.X primarily other offset the of a XXXX million. accretion On the we billion We interest compensation. was in expenses stock-based primarily had an approximately driven of services a On the were to for the of quarter increase for other in we basis, XXXX. million respectively and compensation to same partially million. SG&A increase recorded an million by of XXXX. increase of of and fourth fourth increase SG&A. net same quarter in XXXX, cash period compared GAAP period and to income rates. $X.X million $XX.X by decrease the million investment an quarter of cash XXXX restricted The December cash, in net due GAAP discount was in the basis, $XX.X increase On fourth recorded for

our event first with year. of may we business. it profitability. said, apparent the noted, second an quarter profitable expansion the are continuing the pleased From in of the the the for to in in of we're impressive be continued quarter the in ELEVIDYS the our our conclude, the remainder launch as to a full label, investment there outlined, previously That half. achieve in of to Also, just GAAP highly manufacturing be for leverage So we elevates. some the Dallan and community serve lumpiness preparation reasons financials of to is to the bottom on perspective, quarter incredibly This the and expansion first reach I will be label milestone year. expect in ramp of the this the line first to highlights

turn the We elevates are inventory hand on have Doug Q&A. thrilled community. that, I'll to back needed over resources Duchenne to to deliver continue build with And to the to to to the call Doug? the start the

Douglas Ingram

open TThank you call questions. Ian. the very much, Shannon, let's for

Operator

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

Huidong Wang

so know think many how I ask. very don't you was I X voice operator, I Thank quick off, questions. your cut I ask just can questions

is one ELEVIDYS. So

second, label, from LingerdoXE. proposed it was that be And the in Sarepta? Phase XX the regarding would be it level protein FDA or For patients will FDA with confirmed the And the approvable? Was study was what that on sufficient? III feedback based emerging purely

Douglas Ingram

much Yes. your Thank you questions. very for

I your future, questions. for it the appreciate X once, question For is but

answer me them. Let

far, in will patients our request for policy right science. And want perspective of our right living I is hope progress perspective. a but driven for it's removing that. But so too give this science. which label all, of supports from be on answer label it's right We're all We're age right on substantive expand the answer I evidence answer it's pleased the you the the It's view be clear the our perspective. the review. the the that star the a asked regard. expansion both with Duchenne by North from by we've most view of the very districts, totality is That very basis review midst So from adulation to answer It's -- for, to in the the and about a of and of division's science we're that now for that. the to restriction. the the with the right our early the and

would With they were respect with expression type level the studies, discussions, reviewed sole end EMERGENE, prior the the likely have have of absence study, We've the I the LGMD say, the for study basis shared of exclusive protocol to that in provide to on date. the that by I is later of should predict FDA, the at and including note cause the to given a been very patients us benefit protein, the seen commence robust with our has the there clinical we've their [indiscernible] and got we'll and additional X cohorts. of X. information or -- EMERGENE of degeneration blessing that who which on expression in of and

Operator

the question next of America. comes line with from Tazeen Bank of Ahmad Our

Tazeen Ahmad

as reasonable more that the get I label of you especially COGS, the of time just will assuming? and wanted to presumably over older weight as therapy to and get a of patients the how go to patients? get COGS you patients should range on expansion sense What's we as model look be the higher for

Douglas Ingram

turn you. Yes. to Ian, I'll this

Ian Estepan

Sure.

been obviously from XX% So cost from that pretty we we're today. reported clear that financials perspective, targeting a our we've

our be margins which to more therapy around on that. inventory get can't that the yielding that to You some see and because decline much much weight. be time, to we start clarity coming have Obviously, range, would increase old heavier give time and a it's see or we've on patients the as you'll is obviously, offset which But exhaustive pre-expense higher the XX% we online higher complete patients with process COGS our obviously starts than to of over to their heavier and for which a coming guiding. been could suspension,

So modeling as see is we you currently said of reasonable. margin some the around where it but we probably are, over XX% from time, erosion

Operator

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

Unknown Analyst

This Joe. the great is on Will for on quarter Congrats here.

exploration -- the with thinking guys by the this for with terms constraints Catalent Novo question about expected at and forward recent in potential moving expanded So us are in acquisition the you one and for of How year, capacity, this potential agreement in of year. for your supply label any the end an manufacturing term? manufacturing the near are of Catalent there then of

Douglas Ingram

Yes, some essentially your I there might be sort. for questions. a of all, think first Well, of misunderstanding

Let me very be clear.

at the end with of this year. Our agreement end not Catalent does

They've a have been good partners of ours. long-term with relationship We very Catalent.

with then the and run Novo discussions them. senior as it's had rest with management So there Novo today. perspective, impact is would way had be acquisition be Holdings Catalent. understand our to of the Holdings had I and the business by no quite will I hold Holdings as the it'll from run will sold to be, it GLP-X-related be direct think, the it, Noble of from we've whole, as manufacturing, at Catalent Catalent And usual down the entity going is Noble the we a acquiring -- I'm Nordisk confident that

about over be nothing the will CMC miraculous would it it's manufacturing And have and supply a the very than perspective journey last done So perspective, work for driven. I of if fact we're at this hard feeling from good say and together what that. the from we XXXX a generally, science wasn't other in that beginning

we're a Catalent we'll and course where do the we to and As of brilliant sit our partner, we're serving of today, here of very over job continuing about the a community good be are. year this serving the we brilliant feeling doing with really job community,

Operator

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

Gil Blum

age knowable. the loss the median remind may know between for of can ambulatory And be I So is population ambulation? But kind you us divided what of this and what nonambulatory patients?

Douglas Ingram

neo every ambulatory Dallan, broad It of you about is So of XX-XX estimates coke. ambulation. I the you recent and do can And have nonambulatory give between the patients. the logically about

Dallan Murray

out patients? for Did cut there second. diagnosed say You the a you

Douglas Ingram

the age of is what of said ambulation. average I Now loss

Dallan Murray

ambulation, than for? XX% sound loss range. it of in different is Average to will better on me, XX% would the data Louise Louis, I depending of publications have -- probably think that age right but be

Louise Rodino-Klapac

Yes.

To Some are point, earlier. are differences. slightly some your there

Operator

Brian of line Abrams with the Capital from comes Markets. question next Our RBC

Unknown Analyst

is for Brian. on This [indiscernible]

questions, So maybe okay, if ask that's I'll so. quite not X.X

have can process and you FDA guess, a full the potentially is just to then separate you're I ELEVIDYS, prepared broad the I through thinking approval mentioned related before label I respect the X clarify separated? remarks time be scenarios. separate application label to that the And that, expansion, them? lines, reviews, Can the in questions around discussions with they guess, you So conversion to guess

you from those the an is preparing is accelerated you FDA? of guess, all mentioned Or for this in the scenarios? approval I and setting, -- part So possibilities? if the Is you've that heard something get non-ambulatory just that

Douglas Ingram

there's portions of accelerated -- then iterations. said the are So And couple on confirmed a founded believe course, there'll there many broader the our at breadth and view be about essentially obviously different will label the and a the dialogue much that science. access things. all should population of One accelerated traditional that so are this be label to as of and of to expand a what what are there label? have X for are at removal confirmed entire second for Yes, provide from because there. There's approval Asian approval that's approval of patients in the same to our then, an of is the the going restrictions the for BLA to remain supplements and the are And issues ambulation from scenarios trial related many dialogue we about agency satisfied requested the time. requirements modifying is we repeating approval. confirmatory the the portions traditional to of question label course, There that the we that be translated --

think on science the what justifies. strong We have we view a

So time identical. are from perspective, the administrative lines first, an

So for issues. that completion is of we date those have both target a

of June both address perspective. we'll end So those that And of had very know, XX, June we're with because pleased a issues from guiding people XX. with to you August from we been as

discussions more the thus are of or subject perspective. but and I the we're from we going at pleased be lot Again, be is with there the label. the what's to do On have also this. the days. going happen. different far, issues I'd least engagement a Priority don't review the very of to end substantive broadest we're #X I and say to the to and our will more in had of work want the to iterations say that's review overpromise And possible to before division early

breadth foremost So patients. but hopefully X that better possible discussing, we the be also on be and will June the a this to 'XX of discussions broadest of secondary first issue more number will have date us, the discussion. access of separate -- agency with our to that of work But and and potential of as important do work a return issues traditional is translating course, and to more And greatest the then, the it's we that. we to are and from we're between focused accelerated label, given on and the do life the all

Operator

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

Konstantinos Biliouris

Congrats on the impressive progress.

patients expect not, patients only used would is there and So are the levies unclear. physicians so ambulation that straightforward given status question to If or to ambulatory -- be someone determine payers for label XXs. ambulatory whether a X How quick that we state losing for all from importantly, where your clearly copulation be it is progressively will assume us. may

determine So physicians is and would for eligible or be it if someone payers clear to not.

Douglas Ingram

much very question. your you Thank Okay. for

the in is and standard is that. assessments patient which And patient we've guidance physicians, is is question It then in medical The synergy, good physicians, gold tests. left do the functional of there to the to of the most definition restrictions. ambulation course, your that inability wheelchair what alone, on First, well-defined is the the let are, been of are meters is that me based walk is able is removal to is the the of probably for the seeking that point, of because get use how verified practice there be ambulatory from that, functional the assumption by unassisted. this the the point, answer by medical approach a continuous ambulatory means? a most common perhaps judgment angulation to But we And but reported without then determine clear. today. There that at the ambulation. -- short I is is resist physicians approaches label what

typical functional that assessment be to determine would that status. ambulatory So a physician would a use

functional difficult enormously to an and by isn't this assessment make So conclusion physicians.

Operator

Bank. Our Neena next with from Bitritto-Garg line of question Deutsche the comes

Neena Bitritto-Garg

the -- filing last Just with what nonambulatory, on in kind are just follow-up if you data included the that Can of question. patients us remind a was to on just any, that. clarify

Douglas Ingram

have went patients. Yes. an study for with old years that study we We XXX ongoing which study also is in a had Envision, separate a data obviously non-ambulatory to from XX go it. We children up that from study

of placebo-controlled Now BLA to that available made all safety a supplement. but basis. is that continuous is obviously supports ongoing on the And an FDA study the from trial, that our

Operator

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

Tim Lugo

currently focus the DMD be any and I or related. are of on other you there ambulatory potential looking this that also the the summer, expansion and warehousing know ahead anything commuting discuss aware be Can in we of age occurring should the function else that we subpopulations in level maybe of. Is subpopulations? all at should there which we

Douglas Ingram

warehousing available have dystrophy, over muscular and we But you Well, prepared I stand warehousing fully build XXX% serve have expanded the label an community There other mean but families do demand from to honest. going on the thoughts for material don't that of when is lots course with I'm what be the the amount and question itself. XXXX. is of by to therapy to we the this be from both to physician not Duchenne exceptional sure Dallan, demand

Dallan Murray

don't warehousing see and not that, in to are PMOs any therapy. launch, initial PMOs people an we PMO that of Yes. are don't the If And the the patients about getting that gene or leading team in where we widespread warehousing it's making worried job delayed and related warehousing with sure were exceptional did the anticipation up to gene foregoing did of the really where happen. we of therapy the anticipation

things excellent for mentioned year for I pleased we for eligible the continued is with as this gene in were the the patients incredibly team script, for particularly of we're think, were the I one ELEVIDYS. who So execution therapy of not PMO the

Operator

our comes Uy line of Ear Mizuho. the next question from with

Uy Ear

Could quarter, just of help full us effect. growth skipping not growth just going that Maybe to asking to understand year. the was guidance I'm [indiscernible] or products. you relatively So in -- the half. strong first the first the back In prior half the year-over-year anything

how those wondering for we Just of first XXXX should sort of products. X the think about half

Douglas Ingram

haven't PMOs on We for the provided doing this year, but guidance very well. we're

that in still course I and P&L. modus is of August, won't at the amount double that a good seeing of we from X significant last see saw very XXXX fact, a ELEVIDYS over right not we're digits. of by year, the are performance growing significant X that's the on will PMOs you And things all probably think now interesting and have of of impact the cannibalization

very with in RNA-based XXXX. So we feel our good where therapies about are we

Operator

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

Unknown Analyst

This on is for Lee Salveen.

site So the elevates how first if broader -- up to which patients for context you opens label is of expanded the think treat in might physicians constraints. and population prioritize do that the

Douglas Ingram

have that physicians are an be the are grapple -- there going a to issue to will That of Yes. constraints. number with

great ensure safety need always for things revenue. the remember to we are short-term outcomes we to recall or One that is and instance, prioritizing have of that over, you that

physicians sure this. in going to that follow can that and consider make use and certainly we to up patients will but need appropriately. make they physicians sure So they're to need They know only this, not

both likely can't are and this compelling short require them to spectrum. they them the decide The is we for the to -- do. And so we There medicine as answer practice will of what on invade is a therapy. arguments consider prioritize across that patients that

damage hand, in is young well get done. and X prioritize physicians may be the children there who to very to On want very intervene before

a the patients patients. hand, later is and the for other for There those against time race patients. On nonambulatory

getting patients extraordinarily And it's stopping before damage those important. too is to so and late

work these thought topic. on are and to and to have there'll be that sure decisions among going So I'm physicians discussions to papers are difficult going be out, that leaders white

kids The issue The us the to therapy label, this one. different and be comes from is get today slightly they and on kids to the before that label prioritization is ensure today, from we physician a age available won't the have on we to that label the than the get more given really them. out

to they out here, to really So get the through kids look age to X administrative don't it and deal of good that The because be the issues to physicians. to a if -- you about they're other the of to with But and the news if have issue in if ultimately the process get like. we range is in to age this for old. medicine bias then be the can will expand trying to goes this issue years X be tight tends bias the that ages the of side, and the physician at decision X the label those later practice will who prioritization and we'll

Operator

Ritu the Colin. line next comes with TD from of Our Baral question

Ritu Baral

-- I just of sort to up on follow to guidance want the given.

just fact a first sort patients you many the at systems or Your elevates or center the comment half Are as when is rate how growth on expect is point of sort comment the least through as a Is a that reflection capacity capacity that on of PDUFA. throughput X-year-old additional that the can comment to sort on expands. you've that the run in on you a the until getting reached comment not of And at throughput? label year, hopefully what of through on for this of the of possible? to like

Douglas Ingram

Yes, now. this really right issue capacity isn't a

that it's fairly a commented have is So as his remarks, unique really I population today the label in we in this population. think Dallan opening

X alone are those that not years significant range, very narrow nature population of all First, a as age of to X doesn't because unique old. patients we beyond that percentage know, diagnosed. is a the really describe that, But it's yet there this

then diagnosed. unusual. are it X X in not diagnosed so Remember, that And they're years on when they makes getting day. And all

diagnosed Some getting deep into of kids Xs. these their are

exists come tests is that administrative And means does what has an far get and to process go news, it that It the data mean? antibody so of most very enough with their piece to family get so grips well based through approval, to what infused. to get the get likely that then understand that life with to news devastating then infusion to the is in

population. So unique this is really a

to revenue a year. of In to $XXX is that team, million shout this we it X impressive the out product when in particularly light that, just considers that give did

simply what narrower much really So the X%. this X% about just about talk this that when than you population Dallas talking and might population, is through is we the unique a work prevalent to we really as imagine

year, where of opposed population you in assuming get the some middle the be label population to to would so bolus the treating prevalent group. this to as place And by this begin incident a expansion, you

news a Now XX we label a our for the June date is for request have expansion. good that

So be and hopefully, by July, greater of we'll population. serve have the a we'll to percentage broader label a able much

Operator

comes next of from Citigroup. question the Our with line Hong David

David Hoang

improve that little bit your about is something I pension manufacturing you that just how online? more once a And in operationally ask like culture have How to the wanted the fast might did works. margins? that come

Douglas Ingram

where excited of from very about. we think a to a but couple are A work we're about more perspective. suspension So There's things ton of do, to still

we we transition done now X,XXX on with suspension we're actually right our in getting and really not another great on but with just runs but the -- enhancement from to so, suspension getting product are focus specifically making doing good results suspension. the not we're of our XXX XXX qualities. that start so really we're do far actually in We're perspective month there. We're doing, tremendous liter. When on work our We've We'll engineering to ELEVIDYS yield next or liter. liter. great only couple limb-girdle will a XXXX, progress A sarcoglycan very advanced are programs. a some We actually suspension programs in of

this all suspension have If is XXXX. our to process commercially available goes goal well, by

move to fast goal prove as plans our going with we we we moonshot the patients audacious part then to -- But keeping possible. a serve them. that's as up can to We're with that on the make have center. history front come try Now hopefully, and can and and

iCELLis done, work we our go from and be around really we're XX% as but world And and that more it do, what COGS as a we a with drives perspective, seeing So Ian which greater more both with that. yields with far mentioned, the require margin. than still we get runs perspective will Roche, going as about might as multiple times won't as that's an partner, process, excited are clarification be capacity to It get modest. right multiples. we now these suspension our

but goods therapy as we're excited our lower our I a suspension -- across approach. now, enthusiasm bring right is to this days, world we're So and our for we and at ability what in hear -- still very, mean rents engineering we're just very you certain voice, of the early for in a about and this amount probably manufacturing mean this my go approach the cost And of it is could across world.

Operator

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

Michael Ulz

timing on quick granted since X the PDUFA Maybe date months a just expectations. of ahead X was

now thinking committed an sooner? reviewing PDUFA the close to still a and is the get might the FDA if to answer curious Or rapidly really date? decision you'll get is probably Just

Douglas Ingram

as said, though of that to request we lot to target expansion we think -- 'XX, would to reason work for be June us no we the isn't we're still have planning label With June of to and try that's But well completion we're don't anyone which move I to I are for there's date we're a XX we're date on be done, as going said, rapidly on this. that answer committed answer. fast to get it going prudent assume to possible. the get of Well, our that the 'XX, around, reviewing thinks assuming think FDA when believe all June I as even

Operator

next Our from the question Raymond Brill line with James. of comes Danielle

Unknown Analyst

expect fully PMO data enrolled. us admission Alex the year? know Danielle. is for this are we This whether for on just I confirmatory where trials should -- remind you Could

Douglas Ingram

-- No, doesn't it out XXXX. we for read shouldn't

Operator

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

Unknown Analyst

Brian. for on Charlie is This

as come but time opportunity were well wondering EMA would expansion the to a you from any we if on with you've anything you're regions? just know regarding were Roche I that? as So from online, any the content. supply lines bringing there you heard resulting the do if have another between European be label of to whole limitations comfortable Do prioritization think with the very

Douglas Ingram

goal to Roche. Well, is Roche submit in relates saying supply plans include it as supply, publicly that And then partner, XXXX. our is to their our

Ian Estepan

just And question the and just around X timing. quick follow-up to the previous mission

of Just to doses is a X the that to thing compared this drug. is everyone remind dose

the mg to off we'll read the risk mg from compared market. And the of so kg, XXX no which being per or XX comparative and XXX either kg the out there's mg the be per data pulled drug will

Operator

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

Unknown Analyst

profiles Rick of I you imagine able get Or the that you for are launching, is put X. DMD for are on Since if This patients you're ready to about talk on had looking changed? Kristen. and to way, Miller day who that on how levies the ELEVIDYS highly on it in are have caregivers to another engaged

you few a So ELEVIDYS? to of are experience caregivers forward after months more for or get in commercial child coming seeing patients line now their

Douglas Ingram

substantial Yes. broadest be strokes, think this I I going a would say to that's don't of is therapy. demand there changed. in for the

offered think opportunity by an important it's launch, we dystrophy one. changed Duchene I anticipated muscular launch. it's ELEVIDYS don't disease think devastating before that's I a the what is after and

well. any as muscular color views, that physicians understand that understand you dystrophy think I with Dylan, please have share Duchenne I on or But them. additional way the this your families if

Dallan Murray

exactly speaking change we're population. terms on set It's real in. coming of broadly It real is patients Doug. a here, a the small up think I major -- in we a seeing right, not No, as

And through forms. the diagnosed they're so discussions their enrollment docs, the in as had they've coming soon and as they're getting with

And no It's -- example, came different. for them in bolus of just we so differences big initially But in, that those major they -- we're steady kind now through aging just a wasn't patients as clip as been prior that's can a working essentially. of can to fast -- it's as patients out. there and

Operator

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

Anupam Rama

those sites you you Can had now about the about the in give active kind are talked At the first XX of where you sites us patients. couple of and of months sense being of a dosed year? conference half of in January,

Douglas Ingram

Dallan, I an Yes. give accurate if believe change But in remains can you us update that, that any there's that? answer. the

Dallan Murray

was more There's proportion all then. but at changed on new have and looking And have much. on we get actively this this flexible daily higher Anupam, model needed. that bring a that basis, sites, hasn't really than No, that them and obviously there's and dosed, can of sites there ready a we where we're trained as up

-- So we've got as support we've but ready and the and is to serve threw got than coverage, up running, team out we enough great more also out geographic needed there. and sites

Douglas Ingram

said in also earlier for important more over XX, But amount aspiration going do to trained be to this this was something far that just running. ensure to we that we this good great really been me direction. knows, as good and team call, goal are. well patient XXXX, in XX they've have sites someday XX don't the is kudos activated maybe this trained. by able to and know, in end which been And up not get or -- XXXX this team been to great because us important. that the XX there gene sites record And prioritize smashing an pride, sites for to launch don't ton got over to XXXX. be get as give that which this want they has to gives we a outcomes The sites is would These we done have everybody shape I safety a where and which outcomes. of that could to is, that -- XX for and got, I be in to would XXXX, and was enormous all outcomes there consistent in I of team revenue way try think infuse therapy are and outcomes get safely activated therapy would the you They're

we for have course, lot remains safety a laudable to the experts long and is that over case has infusion monitor physician done. around short not react ensure the so information term, inform want the only to of then, of ability way very gene kind an been they work sure kind programs not access the opportunity term. profile that in therapy, there's anyone that else And of to the We that, we've not full-body and other of the something that has has get unique. to patients every done to that the -- right I'm infused ever a

responsibly. in shape to right great and So now it serve community we're this serve

proud team in think and frankly, of is really -- I the I'm So it revenue where our also right XXXX. explains

Dallan Murray

Thanks, Doug. And as a direct you get that in XX% of just us team to closing the did and sites answer question, were Anupam's we're dosed. speaking, have on

exactly as go. you it's So just it we increasing just said, as

Operator

back I'd remarks. to you. Doug to the like closing now Thank call over turn for Ingram

Douglas Ingram

to think, a a or position which stuck was muscular us Sarepta. Thank and appreciate those and going We At who of I community all get do disease team you, folks can think be in patient heaven in lot year and lot where life XXXX good a in more fast-maturing you and put a and your significant of actually even a limb-girdles us for patients us the investigators we it. better a diseases. life other We XXXX an patients. where for this of frankly, the for with fully for of Shannon. myself, thank -- can integrated are invested for and insightful organization And we I lot a risk is, important where you to questions. have with Duchenne rare today, concert these Thank reward committed we physicians, dystrophy, patients joining biotech us extraordinarily going if are we with to better today. bringing rare that's serious to on successful. moved repeating is to

and remind for you, are brought can we label if this will year. I So successful our this in we goals year, meet the XX we

team growing We of even not and a incrementally both financial are and pipeline, after our acumen to the innovation, talent looking our have to through internal but in leverage this external levies of will goal the but strength and our going multiples. advancing we

we need we're I forward be a to look to and are said, for successful is going if successful and that in prepared ourselves to on to XXXX XXXX. big very that. do along updating well. ambition So focus And the the team way we build With as you

have evening. thank So a and lovely you,

Operator

for call. your concludes This conference Thank participation. today's you

disconnect. now may You

Sarepta Therapeutics (SRPT) 28 Feb 242023 Q4 Earnings call transcript