Earnings call transcript Q4 2022

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Operator

Good afternoon and welcome to the Sarepta Therapeutics Fourth Quarter and Full Year 2022 Earnings Call. [Operator Instructions] As a reminder, today's conference call is being recorded.

At this time, I will turn the call over to Mary Jenkins, Associate Director, Investor Relations. Please go ahead.

Mary Jenkins

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

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

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

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

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

Doug Ingram

everybody may and Sarepta's Good thank for Therapeutics be you afternoon, in eventful Mary. and XXXX conference most year year event-filled full quarter Thank financial call. our the you, fourth joining Sarepta history. results for XXXX

first, in points a might luxury be waiting for for the a therapy at of families for who degenerative in of future distant promise vanishing the will time to are Duchenne patients lives deliver class arrest decline a dystrophy Application, to now. patients for next License therapy Our in Duchenne meaningful some disease; or SRP-XXXX BLA, gene associated brutal rare diseases, this that gene not for as do the the Biologics significant the good therapy bellwether moment: and without muscular improvements of with the with but our

the to And submission, on move yearly BLA interest I'm performance. quarterly I the in going comment and review BLA XXXX before given to keen the

in XXXX, the of the last year, granted treat BLA As ambulant filing, recall, for review our patients. quarter, in you as SRP-XXXX the date. for action BLA will the and submitted our fall Duchenne accepted FDA we XXXX May And fourth XX, set priority to

prosecuting We BLA. have diligently been the

the At have no well division formally completed provided division. We now by productive at that questions. those have they the that for and for is have we committee as the has us requested meeting all informed additional mid-cycle, a CMC answers division determined review identified. with division need information The now mid-cycle that issues mid-cycle very of no there to the advisory significant And an BLA. SRP-XXXX been meeting by see the safety

Products, Therapeutic to the expertise. a of OTAT, As have OTP, increase and office announced reorganized The you and has or resources being improve Tissues read, goal Therapies, enhance Advanced is and established. super the or entitled that Office is alignment, and is new may of capacity FDA being Office

couple A thoughts. of

of the serve delighted is therapy. and Sarepta establishment OTP. believe cell enhance accelerate to publicly and gene by in stated will it the lean and of First, Peter We reviews vision Dr. potential Mark's transformative

establishment great disruption delay a of positive unequivocally OTP that Indeed, as And establishment reorganization stated as not of not experienced we benefit a lines. result of that OTP this will any patients. see any and the or Second, time can potentially of and kind any we have FDA reorganization. confirm has we to impact the

second, those file; inspections and with the questions are launch to forward, along manufacturing inspections. X preapproval make going launch; The readiness. preparing first, on to success; following: Catalent, we we're completing FDA focus answering remaining of our third, the partner, a and the building inspections scheduled our any managing already fourth, FDA has for going preparing may and course, manufacturing inventory preapproval on for So have

Now I in committee. on today the for has as see update provided would division an amount been significant an of the advisory a there need interest mid-cycle whether the

will the we'll and However, the on XXXX be after action from BLA here, prosecuting date. or an update we focusing provide on

Moving now to performance.

AMONDYS. launch Duchenne that consistent the community ask team's given opportunity, one would year-over-year, EXONDYS XXXX an how services, of patients. commercial, where end-market manufacturing name medical approved serving to quarter-over-quarter our and access X VYONDYS XXXX, I and the with consider including the and therapies: Sarepta delivered executed chain, ponders our team, supply and As may cross-functional our perspective, reimbursement you execute to few, patient year performance From was if for and another a together and yet affairs,

at in net a Fourth That year million, $XXX a at of same product $XXX.X revenue is for XX% revenue prior million, at the quarter product increase over $XXX.X revenue total the year representing year. net while Full at and came increase. XX% in million total stood the quarter year-over-year in million. revenue came $XXX.X came

of comes For the serving the none increases. X and from rate, community we a which years, price which of at XX% performance consistent comes compounded grown annual Duchenne last have all growth from

product JPMorgan approved XXXX or a the million January, XXXX, conference is approval, guidance revenue excluding we impact X therapies, for Now PMO net of greater. our at $XXX our announced for the as currently in

a moment. Moving back for SRP-XXXX to

We with year are commencing possible, to that we ensure the SRP-XXXX science. broadest for this have consistent label is studies the as

commence in months, limit population be coming called commenced Study already as starting well. NAV-positive to the will study to patients studies exclusions. separate or the approaches removing make mutation-related have XXX. SRP-XXXX rhXX And X ENVISION we alternative to a will non-ambulant preexisting we our in available antibodies Further, with We study to

provides making Louise research updates with she activities. in progress as Additionally, and discuss significant our are across as moment limb-girdle Dr. development our will we pipeline Rodino-Klapac a

MOMENTUM, out the quarter. is this study complete On are if PMO, successful, our XX know, this MOMENTUM amenable. our later the also PPMO, next discuss who will enrollment filing SRP-XXXX as study and as read next-generation MOMENTUM we exon first designed a known for you for year. peptide-conjugated to of RNA division neurology platform, year Duchenne treat the will we will this with SRP-XXXX patients

am the proud years in I we coming in can yet of the do to years. that X that And made it comparison we the these together. last progress have pales good

of that We to and and as have breathtaking. is front invested that execution laser-focused to but opportunity and tenacious again, need the lives countless greatly of knows team to time mission. committed execute. we this us will has who is reward proven improve in to have been how Indeed, Sarepta the patients those potential the opportunity, this To and realize in a

that, with Head call the to Louise? let Chief Scientific And Rodino-Klapac. our R&D of turn Louise over Officer, me and Dr.

Louise Rodino-Klapac

are colleagues work where and editing an beyond RNA, before of therapy deepest promise impact XXXX we Thanks, the and an of we world. patients to the important for Sarepta. and in speak us important the across moment gene moment and And opportunities extraordinary science XXXX R&D where genetic medicine us of gene today. for represents to gratitude to are today to their fundamentally get our My around in accomplishments Doug. The the and change lives

dystrophy. review thrilled gene based This for Accelerated therapy our Firstly, November to via ability patients was Approval the a show of last a robust SRP-XXXX with benefit a on as decision lead to the FDA muscular to expression BLA version protein, shortened that endpoint we reasonably Duchenne dystrophin were dystrophin in likely of functional serving SRP-XXXX. candidate, clinical surrogate for learn pathway our the our accepted predict

mutation a protecting attaching the our move, dystrophin. lack in and become Duchenne gene and with muscles in from the progressively as a membrane force as distributing a codes for shock from to damage. thereby Dystrophin muscles their as dystrophin acts results Duchenne result, our that absorber we muscle, Individuals muscle worse.

by the change cause to the therapy of underlying is a course to with treating dystrophin disease SRP-XXXX functional with muscle. goal that gene the Duchenne onetime delivers of the fatal Our

What well-established therapies and most endpoint. has and dystrophin biomarker compelling generated FDA functional X approved SRP-XXXX at Sarepta construct, scientific about surrogate clinical the has to interesting positive therapy. our surprising but is on the of not early read-through using the to precedent, is date. completely short particularly clinical date provided our to functional preclinical Further, with as based on SRP-XXXX data results experience a the the strong underpinning Based

we and promoter. to was able functional into protective AAV, that along the years, course Dr. protective. gene an Mendell rhXX thereby identified retain be eventually researched tested our choice, I as and elements gene of its optimal chose numerous AAV were protein constructs with cassette cassette delivery. of easily fit MHCKX XX-plus This enabling the Jerry and areas Over built determine what and of We will packaged our and functional to into

early skeletal, a pleased demonstrating expression and significant muscle. target function we as are of of functional result robust restoration protein diaphragm saw We cardiac dystrophin expression showed with which clinical well of as the across benefits, that And dose. the as the data

the To DAPC. understand dystrophin of or it's with these viable collection Duchenne, functioning protein DAPC which critical don't In understand to dystrophin-associated attaches. and Duchenne absence, for importance dystrophin-associated proteins disassemble. a therapy it's protein their of a the significance Individuals the in of context proteins a complex, results the have is complex. to

saw clinical enzyme of DAPC saw SRP-XXXX CK of was we the or provided we when up-regulation models. inserted muscle an was an dystrophin, confirming Further, the predict protein. when that in in this, up-regulation of significant we benefit. further The DAPC the CK reduction we CK saw specifically, functional proof associated properties in likely X-for-X the protein, to dystrophin animal SRP-XXXX with is levels. reasonably Understanding reduction kinase of expression a increase there More almost protective damage.

the gave dosed of and advance strength XXXX surpassed clinic. any XXX gene suggest studies, Duchenne dystrophin of we've above this demonstrated patients, over more expression early SRP-XXXX necessary to into The protective have SRP-XXXX therapy what and conviction confidence would than the our literature of clinical results to far Since us being other across expectations. and muscle. multiple developed is be for robust work the

properly shock membrane sarcolemma the of as All localized is acts muscle at it or it a where absorber.

that cell-based assay reduction is muscle and saw potency SRP-XXXX, animal that the as robust shows the of expression we functional CK. a in significant SRP-XXXX in And developed models protective also at membrane. active, We with

patients of Finally, of to the in leads up-regulation DAPC. SRP-XXXX expression

versus X-year or able what show benefit predict. show functional history X- are addition and to to evidence, endpoint. across North natural primary would were able all is Assessment, X-, NSAA, our In this the points. Star We to benefit of time Ambulatory we compelling functional also

totality vast benefit Duchenne. based to likely reasonably based clinical and of clinical summary, evidence SRP-XXXX expressed we dystrophin levels with patients at predict able as the a the on In of that evidence to are on qualifies experience were provide objective data, disease-modifying and the agent in

on planning responding not As an for remaining Doug is is holding mentioned in the opening requests. focused comments, since team to on the advisory his any committee agency SRP-XXXX,

As with track a of reminder is fourth not and data review we SRP-XXXX EMBARK from up for cuts because publicly leading readout the year. on quarter data the we FDA, XXXX active milestones the to regulatory the studies sharing in and the this are additional which anticipate do

us and data XE to study SRP-XXXX-XXX, VOYAGENE. using from shared as also We for Continuing using a our in gene finalize Phase dystrophy the from initial study, muscular the now intend positive data functional this into limb-girdle adult evaluating SRP-XXXX VOYAGENE material Phase plans with pleased clinical earlier commercially announce a Combined material. patient that process give was of and with the we expression patients SRP-XXXX-XXX, dystrophy SRP-XXXX month platform our representative known later our a global begin patients treatment will year. broader we as type study III were programs. is limb-girdle population in first patient we VOYAGENE to and this believe I muscular ambulant insights study process for therapy non-ambulant dosed

study XXXX suited the we dysperlin. the for muscular for platform believe by to treat a dual-vector rhXX-mediated to protein dystrophies. therapy medicines is -- the addition, plan to gene In pilot our absence generate well of We systemic LGMDXE SRP-XXX characterized commence limb-girdle therapy our gene

Sarepta. for this in and pace priority a work continues Our represents on area key

Turning for casimersen. enrollment our half making MISXXON track and announce on our lastly, ESSENCE to we complete it on SRP-XXXX, and our our and of platform. MOMENTUM continues study remain study are is we ongoing post-marketing RNA next-generation now pace. for to toward with trial, the golodirsen XXXX. PPMO, data were And the good progress in we Further, requirement to back pleased

time until to we disease. at recognize my individuals and part In closing, I rest this along thank colleagues commitment not want and around like I million to in the patients. We families moment of their the to to a world advancing the with science Day generously take the do without therapies you for work XXX say gift Sarepta will and life's Disease This trials. important their with my whose work the also living patients a waiting could undertake Rare our developing is to I our would not you. and and over rare

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

Dallan Murray

Thank and X our year afternoon. therapies. strong good team all of you, the of Louise delivered XXXX, across RNA-based growth another yet In double-digit PMO

XX% full As XXXX Doug growth over This year guidance $XXX a approached $XXX.X upper year-over-year. XXXX to which million. of our upwardly noted, million million represented our product and $XXX than beat was more net growth revenue in the of that revised million $XXX rate end

start, PMO our strong led beginning by rates started product moment take XX and high throughout franchise. of the mid-XXs net year rate foundation with growth reauthorization full of a roughly with of XXXX. to to low year product strong to insurance the the changes navigating revenue EXONDYS full team This U.S. revenue served and and a of approximately in reauthorizations. coupled as year million. $XXX the for The generated for ex achievements XX% a revenues, successfully highlight This robust in some position I'll net year continued adherence our the XXXX

growth market ended year million we position with least, share. and a of XXXX net leadership and AMONDYS XXX% over And last over million. ending XX, XX% product total product revenue exceptional on total with produced million XX revenue in not VYONDYS XX $XXX XXXX net exceeded For in the growth compared execution $XXX.X strong in to $XXX.X of in continued revenues, team's the XXXX. but VYONDYS

million accelerate continues roughly our As XX% we've represented noted $XX.X net net product of U.S. revenues growth to U.S. was year full XXXX. of in previous the calls, revenue product Ex our ex in and for XXXX. overall

XXXX metal nearly XX% of result of with is in and on requires. focused team a that committed the performance urgency Our patients effective highly Duchenne therapies the of level serving the on wire

XXXX. business year again, performance over to with executed the by We XX% of ended and over on our now revenues Turning in million. the prior $XXX.X RNA-based product of quarter XX% XXXX net fourth more Once the note and the PMO grew fourth the a team quarter high of the quarter. than

for PMO revenues for individual fourth the of our RNA-based X outline now approved product quarter XXXX I'll net therapies.

revenue over And XX representing versus XX% of fourth roughly XXXX, net growth in quarter VYONDYS was QX the net fourth XX, million growth XXXX of For AMONDYS product revenue represented EXONDYS revenue XXXX. 'XX. of the representing of $XXX fourth $XX.X of product quarter million XX net XX% totaled QX finally, product growth XXXX. million, roughly versus nearly quarter $XX.X XX%

revenue and throughout mainly business patterns. variability grew evolved our due U.S. ex to numbers XXXX, the see quarter-over-quarter we ordering As more in

seasonality We U.S. with have quarter-to-quarter observed lumpy and fluctuations our orders. ex

forward. going trend expect continue to this We

$XX.X quarter. us million. to far sales strong example, For expectations the the due in were of strength sales This for very of exceed quarter U.S. to robust fourth ex XXXX allowed

the do you our of want first this you same the for XXXX. We not the model level to dynamic to persist expect provide However, year. quarter revenues into to visibility we of help

and Our January. is than million we our guidance anticipates this PMO forecast issued reflected net which product XXXX in of dynamic it greater in revenue $XXX

upcoming as to operational community the lessons the community serve. learned will commitment proud of I'm confident commitment expect team's a us we to what excellence apply I'm the our Again, patient in most guidance. of XXXX. Despite Duchenne SRP-XXXX. the we and grateful the we accomplished overall, strong This the are our launch serve year importantly, patients in quarterly And and fluctuations, enduring in well we to for

team's complete. organization for our the In the of build and track customer is existing in with XXXX, business SRP-XXXX parallel on execution nearly

the team Importantly, is community. to space prepared engagement with capabilities therapy hard-fought first patient with underway. sites Duchenne the and well fourth once payers into their key If gene our therapy expertise the SRP-XXXX gene U.S. and approved, to again launch serve is demonstrate the is in

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

Ian Estepan

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

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

XXXX totaled $XX.X million and For other reimbursable million arrangement for our for to to and quarter $XX.X quarters ended $XX.X with under million the agreement Roche collaboration XXXX, revenues, December costs co-development primarily the the compared same recognized we Roche. The fourth the period XX, million XXXX. and $XX.X XXXX respectively of relates of which of collaboration

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

December in same the batches months period months not the of XXXX, due to in with is to write-off meeting similar of of XXXX primarily XX, royalty in million period for our XXXX, recorded of $XX.X XXXX. XXXX, changes cost quarter a of during sales of we December specifications million approximately certain payments and in decrease activity our terms. to products same in The In cost ended XX, of in quality due $XX.X X no royalty the the compared the ended X fourth the BioMarin our sales decrease

same of a increases the year-over-year R&D $XXX.X in compared upfront On for increase and were respectively, R&D expenses quarter and of The recorded $XXX.X million for $XX.X million expenses. the increase due non-GAAP GAAP a XXXX of expenses for to the in and of primarily XXXX, basis, million. basis, of milestone to million. million increase fourth fourth XXXX, a quarter we On $XXX.X $XXX.X is million XXXX an $XX.X period

GAAP to turning compensation a million and recorded expenses increase agreement stock-based of $XXX.X for to XXXX, quarters respectively, increase of of due SG&A. grant by On Now increase $XX.X recognized was primarily basis, the CEO an XXXX. through and additional we million. The $XX.X XXXX primarily an in in executed approximately driven expense the million fourth expense modification

$XX.X December XXXX the XXXX same were net for due of for as investment primarily for of The X months expense other portfolio is change period repayment quarter loan increase interest million. as of of the On income incurred non-GAAP an $X.X to fourth ended the we the quarter basis, in XXXX, compared for fourth XXXX investment net the as period of to million GAAP income the XXXX. December the reduction the expense expenses XX, interest result XXXX. million million recorded well to compared our term million other due basis, mix a a $XX.X of of the same to a of for $XX.X in SG&A increase in of On an $XX.X our

XXXX expenses we by in the be prepare SG&A SRP-XXXX. in offset revenue. however expenses We and and increases partially as of that be both will inventory for will build launch the expect growth our in higher R&D The

that we at we we've year plans upcoming course execute previously the to restricted in call. that cash accomplished. billion cash, tremendous the and capitalized potential And over all and our we've progress long-term on $X approximately the had to are XXXX on well of equivalents of all We particularly I'm made December outlined XX. cash pleased this today has that dwarf have

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

Doug Ingram

lines you very Valerie, let's much, answers. and the for Ian. questions open Thank up

Operator

comes JPMorgan. [Operator line question Instructions] from Our Anupam of Rama the first of

Anupam Rama

the Congrats progress. on I by the review any press say no all FDA? on analysis commentary as release then inspections major external Doug, FDA manufacturing. was Doug, concerns XXXX. scheduled? Was the that filing quickly safety been the on comments, and did control your noted And If comments site the noticed correctly remember in in there that, mid-cycle a in your you I the for prespecified well that arm that the and on opening the have in analysis? that

Doug Ingram

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

would little that -- you on explicitly no XXXX. division mid-cycle and review the So in those saw no to encouraging significant to with us first, our profile that the the say they I the -- it should filing issues a who was, saw That informed gratifying but surprise, it know noted, suppose, way the the safety, at they come we that as as -- least, the it. writing safety FDA of saw with see

As into in -- already review. what to on beyond I say things, side detail said. I'm the will of on a not of we're clinical the going lot active an I've get review

know, division the we've did or mid-cycle not can set clinical deficiencies done questions least at material the there at say review, significant you data with questions we've issues that analyses at all. answered. the identify of and are I As lots any and

the that's And have manufacturing as of mid-cycle. where finally, as we site So to inspections then of yes, the are been inspections, it all the scheduled. relates

Operator

[Operator Abrahams Instructions] line of of RBC. comes from next question Our the Brian

Brian Abrahams

Adcom required. specifics curious, your know there any manufacturing might come I'm I sometimes there'd time specific post and they then that and the give Congrats any comfort quickly, overall And anything that. to least build sufficient sufficient up be why can manufacturing reasons would of just on still as supply. issues on nature CMC maybe rectify to Is on the to all level have I'm time minor you more broadly, be preparation I confidence in inspections FDA also dialogue of inspections? the the provided for guess no that wondering And say, more the if these at progress.

Doug Ingram

very Yes. Brian. that, for Thank much you

think on the we've part CMC First most CMC, there the significant Q&A everyone of in certainly is with think, I and gene in as BLAs the -- and about respect the with submission ones. before of particular, talked knows therapy, to some I

before, I of all to answered it the questions and relates were have as had, of the mentioned As we that their questions. all agencies current

we there. those So like good in feel shape questions. we're like shape feel good in very We very with we're

As timing very it relates of the the to preapproval the very, inspections in themselves, yes, confident we're timing.

So about date going the detail any PDUFA We schedules confident reason on any reason go ability we as least that but schedules now, delay into -- not sit to as to we for our I'm timing this right at the a or here there be ourselves. would and the today, other in the to believe have we're don't, and now. adapt of

issues -- confirmed and They other that program. really Adcom. where for didn't then advisory no additional we safety any significant the had with significant clinical saw or identify we they committee, it there are And didn't isn't major That's wasn't mid-cycle. then deficiencies as need They color determine right need the that now. issues an safety any issues relates decision there a an they than the to

Operator

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

Colin Bristow

progress. the Congrats on all

the on or thereof. lack one Another Adcom

able something its the is dialogue review launch around and you you supply -- surrogacy at Or Did can And talk of the the part about truncated focal maybe of FDA had one would this points of over if how this you your this? side, be the be that's process? outcome just part of comment is you expect stated -- on just time? anticipating are comfort You that the on PDUFA, number the just manufacturing dystrophin. approved previously, supply of ongoing the patients build then to to will an that you're on of

Doug Ingram

Sure.

say benefit. entire is the in dystrophin fundamental a to kind of I clinical review the First, of predict reasonably likely is use would short issue and that the functional that's discussion,

a to probability speculate relates this going the to not given Adcom for no of know obviously We it preparing mid-cycle on this isn't focusing what preapproval we on significant would that see see We We're launch. positive. But time and As as executing the say very that that we're there comments, is the can we success. any from to then review. lead -- spend we had our going productive inventory that remaining a Adcom means making what that there we inspections to issues, that and doing answering and are an safety successfully, our be sure Adcom. building for decision really launch-ready questions

do that and want relates community we soon we've We by back as they and in position with exact given patient to access a and orders the therapy all to is as we goal And get it patients be. will manufacturing numbers able to getting is launch are as to what this it an haven't but to stand therapy said from we this perspective. be and it, reimbursement our that without serve

Operator

Our Matthew comes next from Harrison of Stanley. question Morgan

Matthew Harrison

build major these the in I extent inspection used? touched facilities you first, being the maybe and that any now what could building that inventory then not build. the expecting be might you you've started reason were now you on continuing versus talk you to chance just to same to just lines know we've on more or in comprehensive supply X for Is bit the guess Can there just the follow-ups a just thought the multiple Are Could now. you you any just detail? supply have about on supply manufacturing. secondly, see? just issues me. or I is you're here. facility of this talk, But are And can, that you supply versus are or inspected? many How

Doug Ingram

Yes.

So as are facilities. separate X we are of there all, inspections, anticipated. first on They the exactly

at notices say posed inspection have surprise. been have In very them. that questions we've been and We've all none fact, the a of I prepared well would that none of the been for received

to So or be issue. very significant any do build FDA that lot to ensure remaining we're significant an any perspective, BLA this organization. Site we're or and review that comments we questions focused no, is used tracking is there's and to we Building important on very, we working Completing as overtime the in the work to there. so commercially; well. supply good shape there. we're inventory think to track. readiness a significant focus analysis From of that A and successful have a need we on able will this on all is review. a But team risk don't of satisfying that us an is as and inventory we're extremely be And inventory issue.

Operator

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

Tazeen Ahmad

you Can sense demand or the have the patients for be that to love what high already but potential your a to for hear to demand adjustments? there Is make of doctors' the expect an give don't I infrastructure would to any might how offices telling the that have to checks go facilities I'd physicians the upon would some volume we able color approval? to that. on meet know are you us

Doug Ingram

good I because thing Zolgensma. start Yes. very first I SMA probably and is place say in in a a think very of I'll place, of that we mean, the privileged because

zalgentima. XXXX be muscular would of have patients the a experts with previous Duchenne treating with experts dystrophy that -- experience percentage So with significant neuromuscular

So a good starting with we're at place.

to and to provide specific that to that need sure properly certainly go, a success. I'm the a going to to do but XXXX, to ready are of over there's to therapy turn I educated sites Dallan ready additional to color make that missed we certainly, say Now very still this this lot work make up, would I any

That community at that. as be is of would sites XX% up of the quarters to over high over then XX of of goal time about those Our course about as XX about up and the just Duchenne to couple have next sites running. the and launch serve beyond

But Dallan, I what there? missed have

Dallan Murray

No, we're I sites I whatever are a medicine. they're and covered heroic to really And think that Doug. educational with appropriate. working an you approval absolutely And plates it, can we compliant -- prior on the think from and standpoint of precision at genetic forefront but is have they these their they are do lot already

different. is of the the engaging already But muscular out patients, -- they treating are Duchenne there with dystrophy is So these that site the are it's Every team -- today are excellence sites. patients. these centers

So they'll go. and to ready we'll be ready go be to

Operator

Guggenheim. comes of the question of Robert from next Fink line Our

Robert Fink

is the to following in This agreements Debjit. launch? on place How for can quickly in approval reimbursement race Robert Sarepta

Doug Ingram

so to over here, get neutralizing is answer day on well. to process X. doing and finalized. infused antibodies we're amount and I'll of are an work assuming position and and for but right serve approved. to a short sites process community we're having approval, to payers The enormous only mean doesn't post to we're team But the that X already not get to going reimbursement to the that policies the is be have day with There's X. the get access turn to that tested going now get Dallan kids ready begin a this as go in day on It fortunate

Dallan?

Dallan Murray

ready terms And be day engaging we're of we'll Yes, payers. X. Yes. in already with

eligible launches. patients for the can we past we're learn And do accelerate X that going to and so on day I lessons from to think everything

Ian Estepan

reimbursement One to on steps, that you've precedent But very, our seen the our there's for to is have uptake. and through couple franchise. Medicaid the patients time board then getting and obviously, utilization And to through reimbursement that see going take access quarters to it review of work to system. and point, you're state we thing really I it I -- Dallan's right? strong a that guys to takes So add going those terms launches think before. to you very of what drug patients that skipping process, thing obviously, once and through work And to just good PMO think get on for add is Doug's exon you've and seen to therapy just through in one

Operator

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

Gena Wang

I the on questions from the Do established And X X these focusing Doug, mainly quick any And late-cycle mentioned have of questions these do sites. is these are commercial new manufacturing? X remaining regarding iCELLis remaining remaining? and question will that questions. of with the experience there sites have natures FDA. questions? What questions no you the are review expect second be you still

Doug Ingram

not not doing is answer is first what question a that I ton of first. I'll there's remind not there's the lot nearly we're a a you first. a Well, history. last --

my of organizations lot they we questions in because around now say iCELLis will ongoing to committee So an having interest detail of with that the you're going the happen so find the about we're amount frankly, one the advisory commercial Novartis. in an review in is make there to an the dialogue whether going are when review, answers. this or unless significant a to active in point was It's experience an not enormous What were we world of not. and be We're have I itself. and -- of provided is active mid-cycle review, I to things script tried

the have discussion we And with detail review certainly completed. think patients late-cycle agency inspections to certainly positive then in one. see The goes hope it's going by of I'm all dialogue a be go We way. the not are. then a if of you'll ton be item, answer the an into And of will And There deserve that respect well, we course, and along XX. where we'll it. and May will a

Operator

question is Cowen. of of from Next Ritu Baral line the

Ritu Baral

they around their to Adcom is at they you've around completed to sort put others, as had Harmon questions sounds just FDA were analysis biomarker plant all or have -- own back CMC. out the inspection-ready? they for of figured just mark Doug, ongoing the additional follow-up this The discussed? the a their the minutes, X like at It or don't. on And XXX that they've When inspections in around on just you that quick plants, it them? an second. one answer saying Does really they're and submitted? a have a you've any they you then sets or data Are and least XXX now revolve at done Or question that going can the Adcom point. that that they for requested of

Doug Ingram

last the answer first. question I'll

an surprised. just We're inspection-ready. saying that. We're are undoubtedly We organization get that doesn't want to

the position. very -- to we've are question first the get the ourselves the we So well. first good as put done inspection-ready -- answer that first work gets and a much to And to the to in all

that assume submission perhaps make you're Adcom. we to did all said occur connection is And me There What misunderstanding. need dispel point and the in would time, same Why? that committee to an into going an a the determined BLA there's going we given then needed way committee the that preparing for didn't to work that advisory to this Adcom. time. be goes we that I sure do do I to the opportunity we Adcom in mindset meeting an an enormous we wanted with an that that an that wasn't said us a good had ready an should advisory position. in agency to make second sure exactly for Because amount writing let which us we second and have later a So There's had be of in a meeting. a

well told -- they that agency take first the to need in the Adcom. prepared scientific It advice FDA, the the external division it for extent to had the that us for at wise and fall. that So written at be agenda review, was the an that determined to was time, we'd in Adcom mid-cycle that the participate this mid-cycle has, don't the both necessary or that that

we to that's where with So are respect that.

Operator

Wainwright. Joe from [Operator of Instructions] the Schwartz line of H.C. Our next question comes

Unidentified Analyst

Joe. This Beth is for on [ph]

that we're how Specifically, on you've are wondering complete expression skippers produced able provides micro-dystrophin quantify and by if considering you lower wondering the is been compared this of dystrophin for We that throughout the of dystrophin been has Accelerated complete the a much believe also exon a to benefit more expression less shortened expression something XXXX? EXONDYS' FDA review. the of basis to higher if of Approval precedent

Doug Ingram

Okay.

Let yes. me --

this question. Let answer me

is shortened. competitors, shortened, second me start when because XXXX the or case a Bill, that but the of dystrophin VYONDYS of dystrophin or fundamental by made with edited. of the of vastly the is assumes And EXONDYS it then misunderstanding then it's to one science very lightly a the in work AMONDYS that's you misunderstanding there's of understandable underlying part first which of or and there's that Let our the it the sort part one have misunderstanding is

there might the smaller. I That who isn't case. dystrophin that are resulting think those one is the imagine exon

the That a removal of frame parts that naturally The make removal exon associated are the messenger that place exon restore frame occurs restored dystrophin. also of of to it the the and reading allow to is RNA skipping with and to the reading the then will by mutation. include gene

impacted, like versus be And Becker-like same with smaller it can with a it you EXONDYS dystrophin, then functional. the dystrophin This get tests as [ph]. and of right dystrophin. the the the wild-type repeats they'll But hinges Becker in reading the functional and can make any and XXXX can VYONDYS for to make anchoring XX% reasonable another Becker-like places, so resulting approximation natural long that's other so the just And dystrophin dystrophin that's can and instance, and make And very be more -- retains full-length thing is the and from XX% right it's history and AMONDYS shortened shorter, case frame dystrophin. that or dystrophin. same is dystrophin.

use AMONDYS at get and for but perspective, and VYONDYS And there it's precedent where that EXONDYS and our to very while patient it's Those here. the is it there's of protect skipping exon anthronic least the -- concept, relevant a is doing therapy, dystrophin so therapies, providing which same -- so to you're shortened and functional what from different Bitexco underlying because them. a the mechanism will is the

X action dystrophin. make difference dystrophin in of making. difference here, to we're The being the is to biggest of different, gene between mechanism the biggest these a using we're the amount addition The cassette

the make well can than and multiples We magnitude there. over dystrophin limitations are given PMOs with delivery of with our we gene an making cassette order today of more the

do. what's biomarker upstream couple looking a number we're say, is then kind the what And of significant making. benefit. precedent? think so I amount the PMOs but for on predict that functional should there's amount an of idea then of think there's a I with And of kind with the happening there clinical of the we second dystrophin of happens so right than significant difference all And to enormous we right and is likely for. here I dystrophin that precedent not shortened things. And one, XXXX recently going It's be do of kind amount absolutely a other in biomarker all, the of

then, thing the is as long say of that mutations. remember box final must my better result one dystrophin as on And topic, I'll nature, soap one this in I'm on

case accident. do do average her a Mendell, expression Dr. in the but do So that, of partner, were mortality Rodino-Klapac the is dystrophin XXs kids a able in decade to shortened, construct Dr. fact that that can Jerry our worked the it still of or well amount happy a a protection. the make is would they position they be safely can deliverable make build in with Beckers dystrophin. a only -- adults they purpose and dystrophin and kids end, for the to They along average, not as creates can reading way Becker, functional. a over have robust frame rate the And it of average fact these into -- different. on well think that age is I and XXXX make

So question. long-winded my very simple to answer there's your

Operator

Our Suisse. next the comes question line Frommer of from Judah of Credit

Judah Frommer

the of in up payers. for question so, conversations same curious EMBARK Is as topic kind coming mid-cycle in if And and with way any Just EMBARK what a in conversations, of conversation up came context? pre-commercialization communication if there?

Doug Ingram

the As the EMBARK. the program there discussion pre-commercial it explicit in we confirmatory discussed mid-cycle, the trial, entire discussions, to the mid-cycle. and it includes relates no On was market, certainly,

So yes. anything know. if me let missed there, there's Dallan, I

Dallan Murray

exactly No, right. that's

Operator

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

Gil Blum

set do is going the XX any And effect think One by the other is, kind have Zolgensma? the closely road on Doesn't you to holiday? question a payer technical. filing? of map Isn't May that pathway have I quick the follow

Doug Ingram

is of, holiday. a is first it on yes, the Officially, date. PDUFA May So XX our

May be But we then hear which mistaken, because not business Day PDUFA day is XX. XX hear Memorial after I'm before we XX. So the would May assume we or XX, could we day that we'll we're the May would May on or if hear indeed a the date. would actual is This using

approach, on question approach, probably think Zolgensma the think payer the the one I I construct. payer is So

approval amount the earliest years I experience X supporting community. think over happened we with ago. an payer therapies. we've X Obviously, enormous The been Duchenne-specific of have now

So good I we're with get think about XXXX, payers access shape to and approved. to in dialogue able productive reimbursement for assuming very have we're that

Operator

Our Brill next Instructions] line the Danielle question James. from of [Operator comes of Raymond

Danielle Brill

Doug, of IP we see XX-week point data Study saw any you claims? random or data expression XXXX I that do then data. follow-up data from believe have X have Part time a question Do on question, expression the legal at later I micro-dystrophin for XXX. risk additional time at any you REGENXBIO And from points?

Doug Ingram

On the second say question, I'll answer and we no, don't.

understand what if didn't On the it first Louise, question. question, over -- I Apologies. to the understand you fully I'll turn

Louise Rodino-Klapac

that points. the points we in just additional time time was biopsy have. Yes. It no There's about additional

Operator

Our from of Instructions] of next line [Operator question comes Hartaj Oppenheimer. the Singh

Hartaj Singh

very a step big on forward. Congratulations

many Looking more. to forward

actually Just a Phase you've SRP-XXXX. starting know the I got I. VOYAGENE trial on the question

additional material, You're -- also lot material Doug, a doing cetera. et clinical the characterizing work. doing of you're around work commercial, the

little about III Phase believe a look I could If us to later but also you've the talk that bit this design talk that could year. like. to you just just indicated could start

Doug Ingram

you Louise, want take Yes. do that? to

Louise Rodino-Klapac

all at have we're so for that the indication and and ambulatory new looking spending date and population non-ambulatory. the -- looking this Certainly, is This that we population question. which in this Yes. now closely. in older at ambulatory Thanks we're an ultrarare is the both to we're data

population of III Phase be could And that design. the totality in captured patient so the

So But into Phase results be study. yet. will finalized these study design X for the that all we have trials ultimate consideration have taken the don't of III from it we when

Operator

Yun Our next question Zhong of comes BTIG. from

Yun Zhong

study? enrolled don't saving future of what study. prevalence And small maybe thinking information I you is you is Phase size pivotal to on I of program. of patient the disclosed current And So given the limb-girdle trying also wondering, what my question are the study get? the into believe the the of the allocation versus actually your And have indication, I was kind on study? what's the purpose patient for patients the

Doug Ingram

on then Louise -- done this the it when touch reasons wrong. study. can are and doing Yes. clinical there that me and we've briefly X mean I I'll I correct experience reason I'll why this it get answer we're --

to first it expression The typically pivotal gives the therapy for the safety itself. that population would than of our patient us and in trial an is opportunity we broader explore a do

we second we important did that had it reason us. that glean available we to information. will clinical So is material And the

have material. pivotal material that commercially the be has would for That and So need appropriate to be we to appropriate. trial,

of frankly, it to get now have launch appropriate provide insight at seemed right to to very the of time. therapy, appropriate the and clinical additional So we make and also, material the same good benefit a it

Louise?

Louise Rodino-Klapac

it characterized well. you think I

is this emphasizing in component patient than older a population efficacy in we've and Just where patients of previously which safety we at can is that preplan non-ambulatory an different look ambulatory and important studied older this population.

Operator

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

Richard Miller

is how Rick Catalent agreement the LGMD structures In release, on Catalent that could gene multiple press pipeline. support for you mentioned therapy This in the Kristen. the candidates

into whether deal LGMD? in a material what commercial-grade clinical-grade terms go later-stage maybe mean of with you trials could in material this here might little this for could on or So potentially detail

Doug Ingram

material representative to with a with the be us from to forward Children's pathway beginning know, with goal is the study is potential But you as our our with Likewise, and a Relationship from Hospital, try XXXX to Yes. Historically, manufacture first that's to Nationwide is have Catalent goal. evolve doing forward fastest that commercial Nationwide. the -- represented of started as to material trial. going one. clinical is started the clinical Catalent potentially material by XXXX would with have goal our inpatient for material clinical limb-girdle. significant to provided they the the going opportunity there. And Really, commercial from to first we have

Operator

Evercore. Our from Clark-Gartner next Gavin question comes of

Gavin Clark-Gartner

in And exons represent Phase in any chance something that on patients the a major as some you But XX. excluded included that range. dosing X Have here noted, of amendment? FDA II inside discussion through any exons you could you've label? there initial been is getting had those

Doug Ingram

that enrolling mutations. the BLA that have initial to we've mutations. thesis our going be It our started needed this those in proposed with being in the then now. we well be explore would done narrower, exclusion. went initial already we exclusion overly with a we that a same and a It's in in that have we're support right exclusions study major assumption for connection And went won't in the We amendment a narrower nearly those assumption. labeled were submission rationale should our had request label But BLA good with made because narrower exclusive scientific to our quite that why BLA

Operator

[Operator comes Richter Instructions] line the question next Our [ph]. Salveen from of

Unidentified Analyst

on for Salveen on progress. [ph]. Congrats the Tommy It's

eligibility? get you're tested how can or just before for to wondering long after the in between about how out. us Just prescribe commentary wait payer uptake that reads but to Any thinking EMBARK? Approval takes physician to Accelerated period patients might EMBARK remind that you some it for And see

Doug Ingram

we a and Duchenne I call, our go a current both discussions not of this groups would So thinking another end with people -- will note of around patient be for to wheelchair, going our another world, will out on on event. kid somewhere kid degenerative into this get is don't with the with never back wait by And wheelchair. the with that viable that is will died wouldn't based waiting a have kid option certainly a with and of and families thought leaders people all disease. --

So these the luxury of families. for patients isn't there

for So would those current we XXXX at do who Certainly, the their want people nor readouts we wait -- the assumption put our physicians road think that's do, not kids we the intend of risk, to assumption don't can treating seen down at don't wait. think have what would don't least imagine either. it's that and to I other

you to As the process far as the touch want itself. do process, Dallan, on

Dallan Murray

for to Yes. just there what the an well. out of at the fully what of be team I enrolled the And have position is think -- is process, to time approval in you what trial Yes. such incredible urgency, about a That's as And the in. data terms has seeing just confirmatory Doug. said to the underscore is we're

And inform so of that's on going forward all going our to this. dialogue

Operator

no like any showing to call time. to for turn questions this I'd closing further over the Doug back remarks. I'm at Ingram

Doug Ingram

and us Well, questions. thank good joining you for point very evening very It much, is your any. this everyone, a for

today as is think back Rare indeed Dr. Disease I that Rodino-Klapac noted Day.

other to forward bringing patients a to and moving as committed Duchenne beyond completely to And people are disease the patients fast We possible to as life updating rare using great I science way. Duchenne look and patients. along to better

as our action that XXXX, it is substantive again the yet BLA which update note will on I for you'll is the May relates XX next to Although or date. get around

So, thank evening. much you and have all lovely very a

Operator

conclude and you. Thank does Thank Ladies gentlemen, you today's for participating. conference. this all

disconnect. Have a day. You may now great

Sarepta Therapeutics (SRPT) 28 Feb 232022 Q4 Earnings call transcript