Earnings call transcript Q2 2023

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Operator

Good afternoon and welcome to the Sarepta Therapeutics Second Quarter 2023 Earnings Call. At this time, all participants are in listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions] As a reminder, today's program is being recorded.

At this time. over Communications. Nolan, call Relations Director, the Corporate and turn to Executive Francesca Investor I'll ahead. go Please

Francesca Nolan

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

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

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

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

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

Doug Ingram

conference second thank call. joining results quarter financial Good Fran. and afternoon, our you, for Thank XXXX you

long in Sarepta. history perhaps the The second quarter of this of most consequential consequential period was the year

member possibly with approval more eloquent with on the than could still. June it muster. community the I shared Duchenne, Cassidy committee collective in an then than living more words living that ELEVIDYS of view Duchenne far advisory families the For Duchenne and Buddy was far

is there us in now and more let to bask do. I glow pause As quote, and achievement. Buddy said, the our But of

gratitude. in bask us Let

Let in us celebration. pause

as quote. and watch in us the dawn brings Let rises day, close

this the advisory our of of ELEVIDYS year, recommended Ambulatory vote, now gene With majority advisory May of In for FDA meeting the held approval called committee dystrophy. with committee the therapy a an patients the Duchenne treatment on the ELEVIDYS. SRP-XXXX, an muscular

if objectives, Peter treat XX, the On broadening review any the dystrophy, the FDA its EMBARK minimizing maximum granted data of label any to the currently and leadership and June agency, restrictions. five the removing for of FDA impediments approval, by in meets will that Sarepta words with the age old approval Dr. Duchenne time results four rapidly labeled to speed to confirmatory accelerated patients. the At ELEVIDYS informed submission for trial our FDA year the and the community patient muscular Marks following move of and

our have we path. So

trial benefit confirmatory well statistically designed to studied and powered is a Our in the population. show significant

will in have that's Duchenne we For all applicable have seven years we mechanism four in with And studied action ranges. this of old. to to the patients confirmed benefit doubt, show a the avoidance population that, age study the of powered to

We quarter top-line fourth year. have the for this in will of the EMBARK

shortly the Duchenne of able to top submit first be our to should as include If majority announce the them patients. the label in to followed results will XXXX We half line supplement. we of we division a successful, expand by label

alternative commence clearing non-ambulatory commenced Study-XXX. cleaving have trial trials intend Additionally, antibodies. or we to with we approaches two And already ENVISION or pre-existing our to

Duchenne goal XX% to as patients. Our label as expand for the is our much to cover ELEVIDYS of

our been on Officer, well. fourth Dallan have consistent is ELEVIDYS all very of approved Duchenne and with provide launch is will In the track ELEVIDYS the of going Murray days details therapy, record, a our launch. we our launches, moment, early the prior successful Customer our with Chief ELEVIDYS

A significant amount a initiated have of been physicians and ready and families enthusiasm and substantial we prior from start of more and negotiations significant payer positive as And discussions infuse, to successful number sites weekly. now have forms, a more therapies. are of there's number coming than our

In announce to am to faster infusion execute. and to today, we pleased projected occurred than team's that reimbursed first that fact, ability earlier I the speaks ELEVIDYS is the

this now year. please to that both logistics each the FDA the lot. bear the protocol release primarily in that due we begin will positive infusions this mind as ramping payer and This which an And and is, to as significant released requires is for ELEVIDYS later

therapies and Shortly our and activities with we the next on our overview Louise side number in and LGMD with SRP-XXXX, generation our provide remind a of ELEVIDYS of to therapy have an will a Research Rodino-Klapac gene Head development XXXX. To you, Dr. progress portfolio. PPMO. advancing including Development of are pipeline in Chief Officer and Scientific addition our deep

by XX trials end we clinical the year. have fact, this human In ongoing of

continue to cassette, next in advance be tropic models, XX-fold much AAVs. to early as MyoAAV we And than which generation as more our current looks viral animal

From focus with XXX a partner all launch manufacturing on and perspective, our our support our Catalent launch. technical releasing operations professionals more our to producing than and

ELEVIDYS. work approach for to advancing Fisher future the Thermo also at then establish support Looking plant pipeline. our our to we comparability to to midterm, to manufacturing the And will entire are looking to we continue our

In potentially across LGMD fact, next made portfolio, we generation the therapy which in including have ELEVIDYS. our even we use and gene suspension great programs, for will technology, progress

In our year, X,XXX to this with with have completed later run goal fourth ELEVIDYS. we largely based occur to the near three term. of runs the in leaders, scaling with process suspension- made leaders development a for fact, And XXX we've

the seen current standard. greater have are than multiples yields that We

will the improving delivery the As gene leader therapy rare and on focus diseases, our we in for the science of therapies. continue to

in quarter $XXX.X approved warehousing PMos many net came internal exceeding announce not revenue and revenue to our over AMONDYS million three in pleased strong related second total saw impact from substantial and with ELEVIDYS the the in PMOs, to am In course estimates quarterly the have second do for our analysts that approved at we consensus. quarter year. we PMOs had Moving finally anticipate I at product on another performance, approval, quarter impact EXONDYS, came VYONDYS. to the and no negative $XXX of the million

to We too will remain guidance comfortable is with $XXX net of an million full for in our current revenue, PMO, year guidance. ELEVIDYS, the exceeding a total but that to for continue evaluate that. accurate with guidance modestly It product to launch we provide early bias

backed our distinct patients easy welfare is a action. It have say patient words considerations scientific use one put and tools the to them. when and our include by fight necessary, all We commitment other we patients to And for are the we Sarepta, to represent is who we above courage upon of the culture. our excellence, focused At but that us. depend fight the for

not off. may it is be easy, fight Our always but paying

in We have therapies and four enhancing a approved our wealth pipeline. life potentially programs of

$XXX the this and four is of when achieve approved far approval our on will more three year therapies first considered. We million year our next newest

successful, high quarters. and of Duchenne. have if broaden probability plans for profitable we few plan are could gene first next only near to success the We And be in therapy the the our label a term

a we have our already. and only But execution, done We have dogged with pipeline just our plans, to begun. our lot commitment

an on call to research progress. development I over will Rodino-Klapac, update Dr. will turn that who the our with and And provide Louise?

Louise Rodino-Klapac

represents towards accomplishments pathway The the many highlighted the approval approval the approval over ELEVIDYS a the view community accelerated XX, individuals and the broad within XXXX. important with the and for or regardless last ambulatory approval Doug. step on are Duchenne of living for age Duchenne R&D Thanks, ELEVIDYS status. by an quarter June of The win

muscular committed our and to we present supports values to remain the to months dystrophy. change evidence an firmly the As ELEVIDYS's look objective to science follow and we of forward trajectory ability that ahead, weeks the Duchenne

results. dosed XXXX, shown Duchenne studies the and studies, we results functional and other gene three for consistent both across number biomarker to development across of you, this Since remind To than any expression we've with largest therapy more disease. in clinical patients, respect multiple

results multiple data XXX, for safety XXX well to as these analysis The as and ENDEAVOR, propensity trials, time integrated results external including after an score demonstrated functional and to matched studies included clinical positive profile. efficacy XXX, four safety two across ELEVIDYS In consistent points, for comparing years ELEVIDYS at treatment addition control. from in three and studies, one BLA clinical

to Importantly, rise run. functional key and reinforce data NSA time walk independent across endpoints, improvement the these three the the improvement showing XX-meter consistency trials as such secondary functional of across and

measured in EMBARK by ELEVIDYS The study. the data the patients peer-reviewed X seven one publication XXX remind at from appropriate and studies of applied to placebo-controlled Phase have in Duchenne control ages ELEVIDYS is XXX, XXX design, and journals. a MSA X, score for execution has the analysis been or XXX which shown trial statistical to learnings a from studies year. four We published is and in plan and to ages populations, XXX as accepted EMBARK, seven. double-blind now for randomized effect four you, To treatment consistently either compared Cohort When change XXX,

entire enrollment completed shows significant will We compared if placebo. one at a treated trial positive in The statistically the in be difference year population scores fall considered to NSAA successful of the XXXX.

sources. our external conviction XXX is extensive took We our following. controls, success, natural founded on measures weighted ensure and the was history experience XXX based with and data and designed which data Our in XXX, around EMBARK with studies to along propensity, includes

measures XXX a at baseline and improves screening. ricin study including on Study be five EMBARK homogeneity, with for than increase on seconds requirement a less The NSAA to to design ceiling floors and floor a

We factor treatment also between NSAA for ensured by a a a baseline balanced including distribution stratification arms, score.

the patients. XXX power target sample to increase and enrolled also increased to XXX size We

a XX% study measures well of over All in these results powered

As always points, to discussed a assumes XX% X.X EMBARK than it That's the in XXX confident the deviation analysis study. at powering done X.X that also patients provides the and confidence when EMBARK. of on extensive success NSAA. committee in been and better additional was of modeling point powered May, of see in We've we've difference greater the advisory

in First The increased NF who which efficacy through goes meet to one was homogeneity. when narrowed those down, confirming [indiscernible] population the for an of XX. integrated deviation an included entry BLA, summary that's criteria the standard

simulation that greater data perform all from than drawn for existing scenarios program models also have We XX% power. yielded

we're effects. lower Given detect even able the protocol, treatment entire and to

points, X.X analysis at significance X.X of deviation points. standard treatment intent overall our assumed as an low would with the treat observed population statistical predict effects to example, and For as

Taken in EMBARK. readout have Part X confidence the we together, of strong

ELEVIDYS patients our Duchenne. X older additional and ENVISION with to we've ambulant in non-ambulant regards commenced study With in dosing studies, placebo-controlled Phase

Duchenne We goal the with on of are ELEVIDYS AAVrhXX well [indiscernible] study population. an antibody-positive as the patients is this serving in study also Duchenne all This an to as track commence year. entire

LGMD our advancing for oppositely ahead. to XX programs limb muscular across patients we XXX study, of history to girdle updates We're forward portfolio providing the circle We LGMD. important months. moving remain report enrolled to followed been pleased to months or have look and these on Now and enrolled that will LGMD a for fully dystrophy variety committed journey subtypes be with in natural glycan have

We've the patients our using material. clinical study population. SRP-XXXX-XXX, data also our Type broader initial limb in made excellent Voyagene expression the progress shared treatment enrolling positive patients for give XE, evaluating will process SRP-XXXX muscular SRP-XXXX dystrophy insights data a from with X non-ambulant ambulant of adult patient functional Combined believe into Voyagene, we and from girdle study, Phase us and

using initiating include our enrollment of Our in and representative year for process commercially milestone material year. Voyagene next study and completing Phase later X QX the this

dual we've us study for associated of by therapy, gene, NAVIGENE characterized absence dosing allows mediated to SRP-XXXX length [indiscernible]. LGMDXB the strategy systemic of cause dual sole of vector gene to LGMDXB. a the vector Finally, the RHXX be deliver innovative full the to of pilot The commenced treat Ferland

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

the we to enrolled enrollment MOMENTUM to to we've PMOs We SRP-XXXX. regards announce year. studies made or to progress now as our make is study progress track XXXX. mentioned XXXX half pleased quarter, a last track Turning fully good back marketing our on post study the for And on MISXXON data remain enrollment from to for in in of the were the which be RNA study In of for continue for ESSANCE platform. to golodirsen our requirements completed in and with and post-marketing the the complete with quarter trial, this we casimersen first

The see the we before world. to accomplishments impact progress the can excellent promise patients science to fundamentally genetic opportunities side, gene research the XXXX and of make to work of lives pipeline colleagues and we are for In of the exciting seek across us around of change their an with where and to get to today. our candidates. along medicine editing, platform, My MyoAAV the on us extraordinary gene gratitude R&D RNA, deepest therapy the

community, the expedite to we up, as can the as to patient medicines can to and ELEVIDYS urgency science Finally, understand we rapidly We're transformative forth expand allow. the will the EMBARK we that tirelessly we're genetic read study working doing label. bring everything

data the or four next three be in expect the We will available months.

will for an Dallan? to I over activities. update Dallan our call turn the now on commercial

Dallan Murray

this you, Thank were good KOLs and our patients gratitude colleagues to And a XX. point. approval ELEVIDYS the Louise, receive and owe trials, We who for the R&D to in June to participated for afternoon. the of thrilled us tremendous we debt getting on

the at this for access working the old that Louise access Doug earliest day who and to to are swift have to patients. ensuring is is for four diligently has Duchenne committed transformative point year those during from And eligible night time label five expedite the and benefit team said, we Their to time, focused As potentially feasible. deeply and therapy ELEVIDYS. dedication all on can this broadening seamless

rapidly is for broader our eligible the time sites groundwork stages, of patients of old. also years when and boys those who same treat early population to all a preparing are In time, we focus At the on six the today ready turning patient sites are who much the laying to be the comes. these supporting are

educating executing reflect teams sense in commitment Duchenne are gene potentially medical of to centers journey. intricate remarkable Right the as of managers hours three timely XX we've and treatment our are the our from one, launches. our team's launches and for patients within navigating fully providing progress assist transformative the The approval, therapies commercial -- and of done to The patients' commendable and for case patients. truly in prepared day were previous therapy access aspects now to unwavering urgency began

focus of a launch, building antibody highlighted which capabilities must The previously of we the has components site peer serve get readiness, are the community. order the and Doug right in testing. unique gene engagement, areas therapy for to three

In therapy site terms readiness. gene of

over to sites As product. and trained, of today, we have activated receive ready XX

this ahead achieved own have We our of timelines. aggressive

front. payer Medicaid team million Since the has and On both the approval, lives. XXX representing engaged payers, approximately commercial

formation clinical Continued progress has been support approved to made to label. them our the in data educating FDA and informing policy on

of States, in for Healthcare United policy published ELEVIDYS to As the their one an the insurers yesterday, example, therapy coverage FDA-approved United largest aligning commercial the its label.

kits continue touch of two a discussions community. within them the broader for we We encouraged strong receiving to our sites finally in the And are and by on smoothly. began approval, antibody very testing, and is over day KOLs, or demand working forms and process are is seen basis. with of ELEVIDYS and the hours Testing We've XXX within hands enrollment underway, on in to payers come see the a key approval. of currently daily

has had referring team early these XXX discussions. sites. In treating XX, and over both Since the June interactions with

on and procurement, a eligibility, patients has for the approach preparation patients this Launching level timely patient therapy. sites, also HCPs been only with of with access Duchenne and and therapy Our ensure not to safety, to first first dosing communication high groundbreaking reimbursement. payers and gene a and requires families foremost patients, have but focus multifaceted

now are confidence. at of efforts, result patients the diligent our can preparation we receiving a ELEVIDYS begin with where As and point

are policies. previously KOLs the note reach highlighted, out optimistic experts to seeing towards making to I Of of has goal their been timely As local payers access. our and inform our progress ensuring states constructive, is and team with engagement we

the basis for to to as that six patient working in To with multiple plan. three have to expect today. received the thrilled we are end, dosed depending In was were access months a on we obtain we And place announce within on payers payer to interim, case-by-case the mentioned, Doug policies have We that authorizations. patients. first

half, Medicaid a with infusions of week the and combination the next scheduled of the insurance a course coverage. country of patients representing have Over a handful and across we additional commercial

country patients. by patients are all sixth patients are been been around humbling. truly particular, The involved done support being a their since those rally tremendous there the approval who In those the birthday. We effort concerted across incredible to from efforts to to work gratified support has has approaching

accomplishments by date, While guidance progress, we this we are early revenue that launch. stages providing it's and encouraged to our to important reiterate are in not the of one-of-a-kind

the are confident some call, take early in before As launch expect made we on we clear ramp can approval stages dosing time generate will earnest to the begin Doug to come in the this

business to a Sarepta who want within reflecting PMO partners from right reality worked make prior one. to our key ready serve thank on have I to take patients moment to these so this a us and allowed everyone hard our be day to and to launch

the launch. Now lost not our challenge a it's regarding PMO patient community, business, first to therapy gene the our highlight the of have Duchenne we've fact step critical risen PMO while to supporting that we

Doug revenue million second in growth QX XXXX. X.X% over of mentioned, $XXX the growth XX quarter the of over 'XX. we quarter net EXONDYS XX.X% $XXX.X totaled As million, delivered in product representing second representing

of $XX.X For growth VYONDYS were growing versus AMONDYS representing $XX.X XX% second of million, over for X.X% QX XX, the XX, more sales XXXX. than quarter totaled sales And roughly XXXX. million,

guidance second over was the the Total an ex-U.S. and in prior was forecast. million. This reflected represented net fully and annual product which roughly quarter, quarter expected revenue in $XX increase our

expect to the ex-U.S. mix. product in bigger even a of overall discussed see the quarter-to-quarter more as fluctuations on becomes percentage PMO As call, net quarter's revenue last we overall the

in all have Importantly, been with annual fluctuations along of PMO and these base anticipated the mix reflected fully guidance. revenue quarter-to-quarter our shifting the

at reiterate our we And line of completely of PMO for year. point in the the out in in what we therapies. coming our QX are million with Overall, year PMO revenue expected the $XXX net full guidance of this fundamentals product business

fully executing with of second patients support in the business PMO engaged In closing, pleased all to to our the see four today teams I'm particularly of success our are continued quarter. and approved therapies. our

muscular the transform for In potential all three space and medicines genetic future dystrophy. should confidence and Duchenne populations back such PMO fact, of limb-girdle level way of patient execution of in pave the with the high on ELEVIDYS to looking of our precision Sarepta our rare a as launches for other the provide

call over update with the for I'll And turn Ian? our Ian that, financials. to on an Estepan

Ian Estepan

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

the period XXXX, product of $XXX.X of revenue the for demand the primarily revenues an $XXX.X consists franchise months total from million, of product was $XXX.X revenues the same million. for the which for product of million PMO net $XX.X quarter period June and revenues of For second The collaboration XX, revenue for Net to our ended reflects three of same revenues increase $XXX our to in net increasing compared XXXX, recorded increase products. skipping of company million XXXX XXXX. million compared exon PMO

recognized same June for the period XXXX, quarter relates revenues, agreement the $XX.X of XX, of each costs to XXXX. $XX.X $XX.X of XXXX the under quarters totaled compared of million The Roche the to collaboration for co-development with reimbursable we million million Roche. collaboration XXXX second which arrangement In our ended and

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

demand the a approximately same second the sales million $XX.X XXXX, cost in The specifications royalty BioMarin our certain primarily by three royalty in period of three our the of decrease XXXX, meeting million write-offs a changes months in for payments and to June the quality during as ended in due of products an of decrease quarter XX, of to for we the decrease PMO not partially of recorded XX, months same offset in ended to terms in reflects XXXX. products. sales XXXX, the XXXX, compared increase our compared June period cost batches In $XX.X our in

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

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

primarily by On GAAP respectively, of basis, a $XXX.X second approximately increase turning of for compensation decrease $XX.X Now XXXX, expenses million of of compared same for million. driven SG&A quarter a we million decrease were XXXX basis, to to quarter a in the SG&A. $XX.X period expenses for million XXXX $XX.X stock-based was non-GAAP recorded an million. XXXX, the expense. $XX.X and On of million second a the decrease and $XXX.X The of

due in and as loan investment the December to investment net a the mix XXXX during GAAP increase in expense our basis, to compared income for XXXX. other million other the same a $XX.X in XXXX change the primarily interest term accretion incurred we On of quarter income as is for a net discount result of The $XX period of investment recorded XXXX. as of to of of million our reduction portfolio of an second repayment expense interest due well

entered were FDA ELEVIDYS consideration with in $X.X carrying had the did restricted $XXX finally, the in the an sale. time sell received of priority to recorded agreement In long-term and And cash, commission approval no of from with rare not the value a million gain a have at a we June disease proceeds billion of pediatric equivalents, for voucher quarter, of as approximately net cash connection The review investments the of second into from PRV XXXX. we the sale XX, as as cash the ELEVIDYS the cost.

closing, the in And patients to the joy Sarepta first dosed looking and the it serve, to being forward biotech the how actually patient time of profitability. to of like all from just reiterate we today, hearts. then a companies financial So true rare our I'd perspective, one a is exciting for seeing brought transition we're to make to

becoming fact, the In anticipate quarters. non-GAAP positive upcoming EPS in we

So this has truly been been quite an accomplishment on proud all fronts do. for what of us to and we're we've particularly able

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

Doug Ingram

you hear, and let's Thank comments. questions. call the to hear Ian, very last much, those Jonathan, for open

Operator

Bristow question Instructions] Colin [Operator Certainly. from first of the from comes UBS. Our line

question Your please.

Colin Bristow

Hey, any And EMBARK, good there would part this patient another at. hitting you is question And X.X is comments powering with X.X of congrats two what the whether I be first been as on low milestone around dosed. has the clinically the sort delta. as guess considered with discussion around delta I'm made with FDA afternoon. curious point meaningful.

to then that, given be there's status? maintain no sufficient of FDA the on any the to in this has shows population, EMBARK to is Thank approval multiplicity And getting in benefit you. the we're is this X- is X-year-olds, primary that the strategy, would a I which think common your directional formal overall question whether given indication sort

Doug Ingram

quickly, signal the particular isn't real to X But a be -- we XX directly question, you I what with a number. off. from of time that had effect. meaningfulness with here. the at agency I'm and time. discussions degree Using so that a to haven't issue. we weeks continue X one clinical on miles sees effect dealing over is we'd is of an the the going trying It's metaphor, Catalina This if to about if XXX-XXX that effect. the want we're powering from just Tokyo. disease remind turn be the to far the latter question wouldn't But on XXX, So away degree, degenerative we over -- to But Louise the off with That's were Angeles, we the by hit that Tokyo, artful get off took in we Los we Island, long-term

in a clearly see of a very in significant we the a benefit weeks, changed statistically can positive trajectory XX we have If this mere way. disease

So question over let that calculations me on powering said, turn the Dr. the technical to Rodino-Klapac. with

Louise Rodino-Klapac

Thanks, Doug.

So that did, isn't we based on this a traditional the modeling power calculation.

size hit a that simulation in to model we stat order could was to determine effect sig. this order see in lowest So the

following our in So model, we've not patients, we which dropouts which any put is because in the patients enrolled. of was No assumptions into XXX number dropouts we EMBARK. the seen

to We low X.X still And assumed at significance. four modeling significance. we then effect to statistical show standard the as greater And of year-old than will the that's order or as is the to in equal go This lowest seven assumed us how X.X% population. we see arrive to how to as number. still across isn't the the could size entire a in effect we deviation the This size. order statistical varied see low hit

Doug Ingram

see And goal and we would final meaningful at the at day, thing. look significant the agency the the primary are totality At had you therapy the Colin, evidence in way XX asked a to success of the and EMBARK, to look is end then powered agency for Again, and the from is one our statistically of the about secondary. data. the across weeks. the benefit standard

population. look at broader of we significance, -- the we label of look So evidence to mechanism the the the to expand and sort action the justify totality statistical presumably, of at

Operator

question. next Markets. the comes from RBC of from you. our And next our for Thank Capital Abrahams Brian line question moment One

Your question please?

Brian Abrahams

Hey, guys. Good afternoon. Thanks for taking my question.

ground you're several see late-stage So what data I'm DMD from gene in going seeing we're based curious, the on I half year, therapies. far. back so this to of on guess the

assuming as views the your therapies If as or on based with or choose you are multiple a approval ever that that there's you patients to the with be are FDA? a therapy what physicians guys interim gene What to explore looking paths they to take, sense going potentially Is potential have for and contemplated available. explored something analysis? on functional

Doug Ingram

there's one honest, -- therapy that's we We about now. a other muscular think for that's little be gene -- dystrophy we And Duchenne have very are not to organizations right therapy. approved

have We a path of front us. in

things that and patients have to organizations on those the We other tertiary therapy to the the are today, we're are our us. label. serve of benefit and of need the about all than top is And from right now, worry we tertiary about And their to available priorities focused now. that less -- worrying we that's to and or plans broaden what right that

Operator

from moment Ahmad question America. One for the from question. Tazeen you. line of Thank our Bank next comes our And of next

question Your please?

Tazeen Ahmad

afternoon, Good for and taking dosed a bit just the patient it age hoping you took also thanks you as be identified can today. of one representative guys. for time Thanks. I that drug was on And appropriate months, patient get the long to at the if how the do next the my to question. to expect go-forward Maybe time basis, patient on for the Hi, the few the uptake? a detail doctor more today. from share little that least line was for

Doug Ingram

patient away it's question patient -- the not from but patient, patient, day I sixth normally already would privacy, believe social was because I birthday. on of the on think I media. The So their was out one this answer this

to amount So but of the speaks out. patient sake, ensure I access, execution that the patient as to their received this physician an which team. staff's kudos for part, frankly, on team's the to this on ensure age merely to enormous speaks appreciate, of payer and this think it can there And not was didn't you prioritizing work, the our -- frankly,

access to working all think of the It of be willingness a and thoughtful with. And the speaks to I of provide we're physicians speaks for which patients, payers ability the to passion us. is about wonderful and it answer frankly, that to

it completed. to that. Payers, and get And policies then right dual-release We product have releases then have well. release to process like a the Looking year. it forward, think as I is reiterated that going later remarks, and I've said in his obvious should significant Dallan in this reasons the release as therapy and now. that's and There before, occur administrative to FDA are ramp for you the we administratively, assume this

as that infusion. really want have handful that noted, do infusions for I later literally to year. are reiterate, currently see Dallan the we few look patients of this in should a But to coming next days you ramp scheduled really But out over

Operator

Thank you. question. our One for next moment

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

Your question please.

Gena Wang

ask also questions. will the I peer you. Thank

DMD So And policy treat interaction six patients other how based you can initial those a like in payer tried old? place? book these your have with before way of payers' is policy to there revenue years many the hospitals, on And what you place? patients strategy in before approaching

Doug Ingram

these you have get your question. apologize. so this of the Dallan, But to is physicians they with children it closure last is I boy payers, of you'll strategy, going passionate the on to of that with that? received strategy I access prioritize over for working answer, shared you sixth to can other I'm strategy his which access the birthday. but that all alone, And part [indiscernible] today fairly to our turn day do any but who Dallan see today possible before with missed is own the label. not in saw age thoughts out one that

Dallan Murray

while. thank incident of you call, -- the patients on those the In year. in are the been in born said -- turning earnings an next question. we've Yeah, the as XXX in We for Doug term, Gena, each we population short six there's boys that Duchenne focused

there percentage good of But those is of diagnosed terms actually focus a the six in in our all by execution. diagnosed age are not and a by of that get There's of their number sites are don't the birthday. turning Now patients that big birthday. their a pretty sitting sixth patients good sixth

guiding launches. focus net Doug also the aren't numbers. on done we've going revenue as We're patient before, with to and around we've as other we said As said

Operator

Thank you. our moment One And comes for from our question. next question Goldman Sachs. Richter from Salveen next

please. question Your

Salveen Richter

is stratification, here you're you're be in and else that it's using Thank Thanks XXX. what anything here. guess, taking assumption? congrats afternoon. treating for you. heterogeneity I support even in to recognize there question. that Good X.X well assumption, the about the on With study saw, with -- thinking first regard to you the the how that's deviation what I context higher? if of as And would And standard patient believe, my that, impact

Doug Ingram

actually the presume potentially one actually, Well, opposite. would

that occurred the protocol So create the tightening original but deviation to more that homogeneity. X.X standard and take considered XXX, historical XXX EMBARK consideration for or EMBARK of the with the heterogeneity didn't respect Study the various powering was trials to into reduce

we recall You'll have that a trial. ceiling very and in floor strong

You'll year-old but four seven baselines range. recall five to to that of we the six have stratified on not only NSAA on age, the and also basis

to patients all we restricted that the time five recall of seconds. have rise under You'll

points team Louise modeling across modeling, standard and should more But least change And research purposes. create homogeneity that didn't at reduce of population their standard the those actually they considered for take before the It deviation, into their reduce did that deviation. X.X the indeed, that so they was didn't amend didn't All mentioned theoretically heterogeneity, those. consideration. when the

absolutely we of where then sit And overenrolled. patients, So a last of -- had some with and before remind the off today, and which occur we very was patient anticipate we are mid-September. study good XXX right feel last about to was now. here will XXX we've presumption the powered dropouts We study visit, any the now in you, frankly, patients X dropouts It's don't dropouts.

now. where very are about we So we feel right good

Operator

our One moment you. Thank question. for next

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

Your question please.

Gil Blum

from the taking Good age, bit on in from data question a feedback for situation the think agency? there potential what afternoon, given Maybe question. And thanks an a Is you up on of will you. based earlier everyone. Thank our broken here. different be rephrasing EMBARK a

Doug Ingram

data with the confirmed very age rapidly and the FDA submitted a conversations And which FDA seven. study successful will if It's review No, population, has in no, our treatment they leadership, we're powered to that. has the powered four is once to limitations. remove effect data we've trial, we've see to and don't. to in we that move had that been well see which The them to study to

Operator

question from from Ear of line Uy moment question. the next next One And for our comes our you. Thank Mizuho.

Your question please.

Uy Ear

Hey, guys. Congrats on the dosing the first patients.

just So remarks. the my said referring in Doug to is question prepared what

mean half older non-ambulatory think that much to would reach the you're the kind expand the be the by You elaborate included? to that? XX% Thanks. Can you the said the patients? come label those you is Will patients, majority patients. and patients what I goal in of Or first of your later to by would where of going

Doug Ingram

Thank very Yeah. you much for your question.

this. So steps let's go. are sort to two There of

to immediately review label around we So before EMBARK a a that data as will success we the we agency with that of supplement and we've lot them today, of as and even of EMBARK, submitted submit the four as five-year successful, old patients. and the a soon goal here BLA is beginning they stand have When conviction the data. have EMBARK receive for we

Assuming of will non-ambulatory that that is age from and ambulatory, would the patients, that all assume EMBARK gets successful, removed age label the us and limitations all both we ranges. be to majority

said, that as we've aware this all removing a As not logical wheelchair ranges. had no be be a is with limit There to status we've protein ambulation the to if of a about would or would is conversations in assume age reason the It's agency basis not. patient access. that

very feel So confident that. we about

have on have as data older supplements cut data take current one study is the already well. a non-ambulatory our the to that thing from which we'll non-ambulatory we'll safety safety additional patients There we additional is and do,

goal have year label our broad first a that covers next that's So to early all patients.

to will in the receive therapy. there limitations -- two remain two ability are still There that

that for that We're time or exon this that be going the will or is for hopefully contraindicated some and remain and time. to those the work do X% of for to That them X of in continue that X label. One But mutations. mutations will cover so are that. being, narrow on

be antibodies, dosed. patients And looks preexisting for patients Rodino-Klapac RA prepared those clear something we're Dr. on alternative clear as our then to environmental About to as XX. that exposure that a her two like Those and the XX.X% them starting remarks, have antibody cleave But based currently telipidase antibodies. approaches some antibodies. different to second an alternative one in represent to is cannot second seroprevalence in mentioned apheresis one study

that successful, start the effectively as dose have term, antibodies. presumably majority even just That And patients in or patients the of XX% much if as potentially to near safely and then of one physicians patients. those are us of empowering would than could pre-existing both more to get we to

our that's goal. So

Operator

of Thank the question. you. question for our Ulz line Morgan And moment Michael next from next comes One from our Stanley.

please. Your question

Michael Ulz

you In mentioned question. the Thanks are for the one ELEVIDYS another greater XX Hey, taking just than Maybe currently. activated guys. on trained launch. prepared sites remarks, your and

of where can total get or represents you're you whether that Just sites in currently the process? trying number a give if us sense at curious a of Just that expected? Thanks. to majority

Doug Ingram

number a XX tremendous sites. sites is of

am I We team's -- ability. completely the are with thrilled

XX XX% about of patients covered are in Duchenne So by United centers all of about the States. care

enormous an to ELEVIDYS. is sites of XX sites infuse So number

XX Our goal is to significantly get as over ahead we're to of goal do to many our and as time, that. sites in schedule

shape Dallan we're an now. site amount and in gotten where really, great enormous his a kudos of right deserve perspective really and team we've from for So

Operator

our question. next for you. One moment Thank

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

please. question Your

Danielle Brill

to powering assumptions? see guess to that other have blinded from data assumptions. allows on deviation. on data afternoon. you the able curious, a further to or your I you hoping Good standard can guys. I test the SD I'm stress your blinded you Salveen's Thanks. follow-up quick Hey, of question kind elaborate are confidence EMBARK Just any in

Doug Ingram

-- We at do don't very access to blinded looking insightful, to unblinded have wouldn't we and not the data. the be access have so data

very comfortable not in restrictions were has we EMBARK that homogenizing initial the have So powered the study that with that did was studies our feel we it. standard in seeing is with deviation that

the all and had time before, as when we kids they simulations powering not on did age floors take when five assumptions I into homogenizing group having account change and a making seconds yet under team ceilings the stats like, to of and and in stratifying rise like. did like, actually Louise and mentioned the protocol. the that the And did our aspects strong test her simulations, Things the sure and and the

to unblinded test on data sit So access about to we deviation. standard haven't those had good we of study, good feel But feel assumptions. powering today and the here we as the very the we

Operator

our Thank comes question And One from TD Baral you. of next question. Ritu our line for from moment the next Cowen.

please. question Your

Ritu Baral

taking for question. Thanks guys. the afternoon, Good

bit can a than the Zolgensma Just couple next if in weeks, that then different comfortable you of you back are expect be there about can centers to second. of little say faster and of move and how history you Doug, you patients they they that their with XX? for handful And talk are? to will given five the a the -- centers were referencing the centers at maybe others,

can if have been tests release squeak then another can one I know And you how you antibody run? And that? in. Do many

Doug Ingram

first answer can answer know I two. the So last the for I you. don't

of them the of different sites. -- handful so are at All all

otherwise faster, sites. different move good kids, -- from get first across broadly particularly the everyone's news any these possible is trying might physicians really as benefit passion the the are sites age And out. is to they're if all, these then wonderful. that as So on to kids kids amount of fast of infused And and

benefited fact infuse sites to really these with of We know have the these sites, most really -- that Zolgensma has therapies. gene benefited majority and how experience team the these have had of the from

initiating went we site process of and got So getting them the and ready. them in they were well prepared before

I say there's as move of enthusiasm reasonably fast as broadly would lot to get to ELEVIDYS. a patients from possible speaking, So benefiting

Dallan Murray

is going functioning and we're it's disclosing And in the smoothly well demand. smoothly. numbers but terms we And the of working happy aren't with now, right well, going the it antibody and testing, and

Operator

Thank you.

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

Your please? question

Anupam Rama

Thanks question. guys. Hey, for taking the

point. clarification a Just

this. the Sorry Thanks study starting if missed gating are later the I year? to pivotal this factors What so SRP-XXXX much.

Doug Ingram

you any if manufacturing, I'm over and to CMC me mistaken. just but not But this of all turn missing getting tell a think without nuance. wrong, Louise I'm I it's Louise, it if I'll

Louise Rodino-Klapac

accurate. that's No,

lined set. data most of patients them we're sure making release all great -- we and the and fully assays . history and have having we mentioned, So in we deal place just of up, Journey as well. natural enrolled But as are a

Operator

our moment you. line Leerink And our Thank Partners. for question. from the from Josh question Schwartz comes of One next next

please. question Your

Unidentified Analyst

Thanks Joe I'll is my congrats today. our This progress Will the for add quarter. for all. questions. Hi, on on taking this

are opportunity Is the that you for one us, but market. PMOs XXXX there candidate PMO, for potential SRP be a any patient PPMO? for what not why wondering franchise is this would Thank a is the a So a expander? on currently currently you. And reasons relative to

Doug Ingram

on it. So not

the benefit it if to see broadly successful, we're I later and data year. XXXX speaking, this going so justifies -- successful for we'll and we'll an year, the if later we're risk was So it, with next the year then data think, this seek NDA

things. would -- then a of be goal The couple there's

that it is would more goal first more The be and PMOs. our convenient powerful potentially much version

a it the and So patients therapy, on great transition already benefit would therapy them. to be for this for them

we the We might United multiple great would more have might You in States, we record the X%-X%-X%, could success had greater even with have -- folds which than envision dystrophin access. we've making with a be were if PMOs. even success access

United of is does it outside around which move always excited offer our which we're the the patients. benefit the RNA opportunity broadly do, technology more to theoretical world then very bringing to to is States, something And

lot potential with So if PPMOs, of the successful. there's a opportunity

things this if momentum B at across with some see So bringing we're Part looking forward XXXX. and forward to decisions the looking aggressively of and portfolio success making the then we later year

Operator

question. moment next next line our question the And from Cantor One you. Thank Fitzgerald. for our comes Kluska of Kristen from

please. question Your

Kristen Kluska

Hi, milestone. on everyone. today's Congrats

patients a month end Thank of year, to you steps payers us shortened? on broader half these could be all expectation line getting across now taking in you've next you're that commuted up first it you. the time is you've that Given therapy if terms three testing, communicated in the to your four label of sites, and that antibody getting

Doug Ingram

This generally course, And policies some to codes speaking, an with Yeah. place release is current The place. this the In initiation issue we answer of be have like. be launch and in the really to is places, process. have therapy, the in yes. have of then,

that but to So move had early I But said this before, lot over real the assume as we fantastic to time. ought ramp position success we've should begins a very . a be year later of faster we in

Operator

comes the of line from question. our Thank William next And moment One for you. Lugo our Tim next Blair. from question

question Your please.

John Boyle

on Tim. John for question. This team. our is taking for Hi, Thanks

the if if team effort seeing more the could about access age little making So approaching you're just years cutoff . from beyond just the demand seeing that I you approaching to six patient starts wondering to age, higher is patients. bit and of patients patients any wondering you're from that a was get talk those

Doug Ingram

over I'll to turn Dallan. this

Dallan Murray

Thanks age Yes. We're group. both question. four the the five broadly year-old for demand in and seeing

happening is so that the urgency skewing now. right the the early it payers of between have on, to dialogue more those and In terms teams just the KOLs, heavily and of birthdays because that's and access maybe patients

all are -- eligible, quickly seeing early of birthdays towards to So expect X X we we the skewed but to as group. towards work year normalize six, patients on, that that are six age start it very with the to we turning yes, that people

Doug Ingram

want older across So with an the start there's ages. that that demand likely forms to in dosed before obvious the we're seeing label. get kids to We'll the access issue age get skewing in reason range them the for we

Dallan Murray

Yeah.

Operator

from our moment And next Brian One Baird. you. comes next question. our from Skorney question for line of Thank the

Your question please.

Unidentified Analyst

question. This Brian. on Hi. is our for Thanks taking for Luke

commercial progress availability might products up getting the running, population? trial pace headwind and of the in you the signals to Regarding have seen the that of a enrollment any be ambulatory ENVISION

Doug Ingram

to going answer be that no. The is to

the thoughtful protocol ENVISION. we about So for were very

the we as of patients have progress out the we've significantly we limited come ensure that fact, to number States of that broaden label. that of In an with United therapy don't going are issue the the to

we're Now enrollment this instance, is of an the rapidly because will seeing States. part recall. as everyone to slower United majority on for may But the that enroll impact we're which going for from outside than mean and EMBARK, subjects it does not that the enrolled basis of of ENVISION move that ultimately, approval

Operator

And our for final from the for moment today Debjit from question question today. final Chattopadhyay of One our Guggenheim. comes you. for Thank line

Your question please.

Debjit Chattopadhyay

current where scale? either What will the gross successfully to for do margin you afternoon. on it peak is And could good Hey, you migrate if ELEVIDYS? capacity the think migrate or X,XXX-liter

Doug Ingram

to this I'll over Sure. Ian. turn

Ian Estepan

Sure.

kind of PMOs. similar range in targeted the to like expect So we've margins our would to what XX% so high-quality seeing be we're that said agents, with that we

point, patients could when going obviously, earlier are then it's that into heavier the but patients to of higher kind enlist being And big then change. XX% good range. obviously, the be your as a Now see basically we dosed. And down, trend in to label

not We're -- has perspective a impact position Doug's our manufacturing But from process. where we on from in that it a big are on obviously, point, suspension a quantify our to to yield.

not that But so driving higher. And could we obviously, we're to willing number to that that see commit yet. just

Operator

management This any for the you. the hand further back Thank of program. like conclude session program does question-and-answer to I'd today's remarks. to

Doug Ingram

you, with everyone, questions. serving your evening approval year, Thank progress it. from thank us for both for I ELEVIDYS this and and appreciate great the far very we my obviously confident about so perspective, both current this and of joining We've our you, Jonathan, then I made, patients, done with PMOs. has our will on ELEVIDYS feel and existing a to the we are serve continuing the our us community with guidance the job -- PMOs. that team PMOs. remind brilliant of And with

to at broaden Duchenne with about condition, living and of an ELEVIDYS that essentially lot a FDA. to share patients families. which both patients readout the amount reasonably continuing with with have year, colleagues of we as to this as label majority and at and at we rest our EMBARK serving to their the possible PMOs, time course, do patients, enormous the our the for will We the soon you we the goal, And of is the these share vast the getting then with of that bring have serve same

evening. course to With over that, I the everyone quarter, everyone ask would a look I of forward the to lovely have updating and

Operator

Thank does conclude program. today's you, for ladies in participation conference. This the and gentlemen, your

Good day. may You now disconnect.

Sarepta Therapeutics (SRPT) 2 Aug 232023 Q2 Earnings call transcript