SRPT - Earnings call transcript Q2 2024 | Sarepta Therapeutics, Inc.

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Operator

Good day, and thank you for standing by. Welcome to the Sarepta Therapeutics Second Quarter 2024 Financial Results Conference Call. [Operator Instructions]. Please be advised that today's conference is being reduced. I would now like to turn the conference over to your speaker for today, Mary Jenkins, Associate Director, essence Relations and Corporate Communications. ahead. go Please

Mary Jenkins

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

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

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

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

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

Douglas Ingram

wanted accomplishments of reason. through detail, Sarepta greatest X developing the life-changing years that of Duchenne XXXX Thank a over joined Results for successful. could and of I to therapies you, X launches many that X an of of joining one a Conference I those lives Sarepta effort. you for to making obsessive Sarepta Therapeutics Mary. last over afternoon, all Financial years, hope the I Second launching had We've Quarter thank had Good medicines be successfully and very patients, everyone, part majority that and attention little improve the believe ago Call.

We XXXX and of CAGR disease. cash we established We've And pipeline. of are therapy the beginning have RNA in revenue for XXX% end deep built the We've rare as have a this at about And leaders XXXX. gene grown ourselves become quarter, flow the from of profitable. positive. as through we

quarter of June it to Mike a second many of of we we me make and this brought we that [indiscernible] broad of ELEVIDYS so United from obtained Duchenne finally the reached with But say patients can only in that to [ objective Patriots and because to it was year have that that XXXX was that dying the and finally in in approval available Sarepta, over States. XX% living ]

X an FDA had for partner, ex patients year, of Roche also approval history, making good approval the ambulant all and U.S. those the Also aware, the progress granted accepted June, with on accelerated for also you older Roche, this for in approval obtained for As that in so treatment patients June ELEVIDYS of other and submission in the granted is announced Agency nonambulatory Duchenne has European consistent are basis. and review. traditional every communities. far approvals our serving Medicines

to largest patients. to bring the yet launching the Duchenne dystrophin of vast history are, first a with we here the opportunity in So therapy yet gene therapy majority approved restorative

access. developing great is disease, managing working X-plus perhaps manufacturing who are one last we watching above over we the to distribution. and we has we in world what therapies, therapies launching become But at great have good complex payers at. and exceptional process are to this at no the realize at and are Certainly, Duchenne get them the approved years wells, are and rare for been ensuring all that we managing at Anyone second with particularly will exactly

of narrow product in million. earlier that at forecast. few combined. launch, full That's and restrictive our in in we the with net $XXX.X in the launched achieved doubling quarter, Even million of the $XXX a label, with more approximately came first $XXX we other months gene achieved than XX% last therapies second [ second in point, same an the in quarter XX quarter, ELEVIDYS years To year. exceptionally line the approximately revenue All increase over million last ].

reward enormous. previously, this the be broader the label noted serve June in in opportunity with year, committed doing granted have we of will As investors and so to patients

Our positive between even patient payer demand X to are expectations. positive kit X form typically [ to interactions. ordering me To to and forms enrollment infusion and expectations, will our near-term that early process ] optimistic has All currently signals let take manage again launch the note enrollment to from exceeded from physician months. assay

strong growth year commencing this year, of we growth very exiting fourth but the quarter, year moderate So third will be and growth. see in anticipate of in revenue this XXXX we will quarter a the then strong

product X the our Officer, $X.X $X.X currently incident approved XXXX. and will be before is color that from over will billion, the We this population Dallan Customer prevalent will peak of in more come our a and Murray, course that decade net moving our revenue billion years Chief in the population provide still moment. year launch on to XXXX we treating just across Indeed, to between the therapies sales. XXXX entire anticipate away in

sets and presenting LGMD SRP-XXXX she clinical Officer, later also will Society R&D plans of year, this on will multiple from the Louise advancement Congress discuss progress the Our updates data Head including provide our Chief at World and Muscle our the program Dr. quarter, across R&D our Rodino-Klapac Scientific from of then for last programs.

launch across plans. Dallan updating Dallan? will advancement to the performance forward details turn year our more our on and performance and commercial that, Murray look I with And call the of on you pipeline. the I to for

Dallan Murray

good Thank you, Doug, and afternoon.

limited our roughly $XXX we on Doug net we for revenues XXXX. X- As will quarter on a X-year-old franchise label the given the in We we second from quarter to in broad XX. of last is product the June received outlined, that have generated QX label in Duchenne million ELEVIDYS, executed which

expected, quarters. product X in early net had each of note, was the the a resulted quarter. few completed the end revenue Of second third prior to flat the rescheduled ELEVIDYS being at compared for we of in quarter essentially when which As the those infusions patients doses

$XXX quarter. over QX Net was QX. million modest franchise represented for growth [indiscernible] revenue ELEVIDYS million, $XXX product the revenues Net for the which product in PMO for remain roughly

patients weekly ex PMO regimen team remarkably years compliant the mitigate their committed who expect Going supporting in forward, now. help to growth with been remains by existing the have PMO while will this the cannibalization we quarters, U.S. for to in And cannibalization. U.S. coming ELEVIDYS those PMO some

that opportunity date the ability The and the Now Duchenne recent progress who track current Zolgensma. entire overnight. are centers rapidly the the turning populations. We're of we're launch. in to SMA are patient launches, to fact ELEVIDYS for the to demand. their this, treating manage with What their and the became same neuromuscular the entirety prioritizing are is realize to and Duchenne all reacting key eligible front now us. centers navigated seeing ELEVIDYS on perspective, the these unprecedented given To launch We're including from Duchenne through to of put sites confident of demand population essentially in working pleased almost the into situation right patient the

should into reflect infused uptake this obtain gene assumptions to only several Your journey new the launch. weeks patient an therapy. We're

preparation, had to with report, expansion. exciting in some have progress of team In patient initial we screening first which hours successes begin, were requests steps of of important ELEVIDYS pre-existing we ] forecasting and the in insight. overwhelmed the market screening the label for for made kits demand has [ an nearly However, the time highlight expansion, the leading therefore, our antibodies. short despite To and assay we've with one the journey, indicator the is label careful the

well weeks Our kits first of assay the over in and shipped rapidly team launch. X,XXX adapted

is other robust that expect kit all very would you growing. our and assay As of show patient from indicators demand demand,

indicates forced time. of itself in research launch the a and demand days the in are that and interested plan demand expect we reaching key vast our only patients majority sites. patients to Duchenne journey. This of seeking therapy, unprecedented determine patients the early treatment support how with this manifested with to has center the grow the market Our to out of each This already for their flood to to the handling approach treatment navigating best in

highlight from have to XX support some hearing enrollment that we're years now patients critical X from X-year-old have year more into forms have HCPs of who than community the an sites. long of states, with in few initial HCPs over weeks from being for from to the gained ranging with also a right enrollment of just experience lofty exceeded last XXX advised XX% such, our that the average ] age XXXX our It's patients are population. own [ forms ELEVIDYS we expectations submitted approval, broader to wait the the the launch. of X- Enrollment just received times. Since we forms XX. for physician over age, community our XX including As have presently

Of non-ambulatory patients, note, they physician by declining launches. label see first and all exon the launches, following gene our And of ambulatory previous of as skipping our therapy we approaching can early early the diagnosed closely DMD days have patient this launch pattern are newly same expansion the that the populations. are on community prioritizing previous of followed

is hands-on XX% this drug. dosed the patient, grow sites in we with that had One of centers have factors experience at key end, that we a launch the expect now critical have X and do To treatment any of label. have our that to success broad the least over

to representing X issue to signs our policy payer date, We've the with most critical million XXX plans basis. we top than To patient it to started request the payers patient have already commercial In including each to success for We months productive meantime, of engage meetings medical on take teams policies. greater state turning front. and the anticipate encouraging a see will lives, access. case-by-case new payers with reviewing having establish patient the X Medicaid on And had and are agencies.

from pleased in have patients and X ranging nonambulatory that both both end, established be Medicaid population age. for to indication. accepted ambulatory to And are both with to to approvals policies and call, already Medicaid expanded, authorization from XX commercial that payers who there covering of patients we And as ages payers and highlight for expanded also commercial of I'm this years ELEVIDYS medically have

authorizations dosed first the week. of multiple expanded these in with this population hand, have expect be we for we scheduled dosing, finally, already then and patients And the from to

is see this, QX of as you'll all X/X ELEVIDYS to accelerating modestly Doug a nearly the we in by revenue to growth this indicated, result quarter-over-quarter see jump of QX quarter. the ELEVIDYS a followed doubling third QX. from to QX a As and from revenues of expect year, in of What

will only when the themselves of to in a to be But a fully the the what label. remainder prelude beyond, XXXX had sites broad adapt have and you'll XXXX opportunity to see

Our confidence around And this $X.X XXXX $X.X are show with net product is Duchenne revenue franchise guidance potential growth the heading billion market for where potential. we market to our our should revenue massive. that of absolutely you billion and

exciting are a truly Let just a we how out. perspective. from is launch There how illustrate opportunity picture me things moment for which X to expect painting spend the commercial play this this

with prevalent with therapy Duchenne no near-term remainder eligible key the foremost, centers decade. the expect competition, over this to collaboration gene and First population the in we of treat

Secondly, X a years broad label ability for past should record provide access you over setting. our patients Duchenne reassurance all the secure of in track our to

will eligible. secure While it take some vast patients, who patients are majority to the of we may ultimately access Duchenne time for

Lastly, has entirely fact and think of so onetime very don't appreciated this been patient along importantly, each dose with is for I describing the outside the fully was upfront. And Sarepta. revenue. And ELEVIDYS I that, earlier. is what is this

the to early that adjustment of revenue take off this year, expect robustly. we the period After in back half

the with built to opportunity full for this proud originally years. everything the for We've challenges a learn is we anticipated for team of do. evolve now safety patient the year planning at population. was that of that a What have and been have and had Duchenne many I'm is center full most We

everyone and our all up are both a thoughtful to initiatives, do proactively We success. support sets approach taking thing education integrating doctor enabling and between the discussion informed of a into is right long-term fully patient in for and

as and share any we deserving some with do of centers there As that our will see for us be support frustration work may launch, through seeing to take today, ups downs. and we'll we're these in time our the It demand to patients. the best

there a an us in is no argue even team demonstrated of disease, [ launches, the have could ] landscape in in execute we I As one biopharma is would incredible front who rare entire and better opportunity such previous right we now. in on our the

we So the and demand in as from had interest strong anticipated. summary, stakeholders is all

expect the Obviously, hard patients We the community time. are system with all can this and by will of we as demand now physician seeking working only treatment seeking who support of all increase are accommodated cannot patients We as be right immediately. to treatment. the the

realize support revenue them biotech. opportunity perhaps of will we near-term doing, the in So biggest in and so all

to our important their everyone and moment. all like thanking this in stakeholders Sarepta I'd to of end at getting by for us role

of [ dystrophin restoration every not these as families. today with matters to ] their As We therapy. the we the to majority we received dystrophin change Yet today, that, continue majority Duchenne aim and execute that and of have for for sit patients eligible yet realization the frustration therapy. are here minute patients

Now hand our I'll with R&D, Louise Dr. it that, Rodino-Klapac. over Head Louise? of to

Louise Rodino-Klapac

and label and expansion Thanks, then my provide begin pipeline. afternoon. the approval the good on update of remarks our I'll ELEVIDYS with an Dean,

study. to the and regardless study ambulatory these Following following: a traditional clinical present. X state long ambulatory universal, embark Duchenne thorough FDA of was [ review related And Therapeutic all based label after ELEVIDYS by expansion multiple data, granted research available and of critically of in the the approved as patients, considered our and decades the ELEVIDYS confirmed mechanism ELEVIDYS disease on that . The double-blind, ] of of scientific older. of Products was is on development action benefits evidence studies, of approval aged muscle the based of placebo-controlled is Office and as of patients across the all

reasonably to the result, benefit likely our clinical expressed in this nonambulatory a therapy is population. As in dystrophin by ELEVIDYS patients

granted to patients, confirm As ambulatory late treatment benefit, X known a approval accelerated result, commitment and our and study approval patients for we nonambulatory nonambulatory a the has accelerated ENVISION. The or addressed includes as XXX, older. ] been ages of nonambulatory AA for post-marketing clinical via also [ which

our well and global double-blind, placebo-controlled nonambulatory As safety individuals SRP-XXXX study, with is a therapy randomized, to Duchenne. XXXX progressing ENVISION in reminder, following occurring older a X-part is and in Enrollment enrollment XXXX, is ex visit placebo-controlled and last expected the complete last recruitment patient U.S. an evaluating of ambulatory remaining XX-month efficacy gene with the in U.S. expected period. completed be and ENVISION with

long-term We also monitor continued follow-up to studies with a advance clinical loved. that

follow-up ENDEAVOR Our third to be that years. and with of been treated XXX study will individuals EXPEDITION. ENDEAVOR is to observational activated for long-term include will One follow XX have enrolled. up IV studies and a patients ELEVIDYS up Phase sites

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

multiple expanded ] the under receive to label, ineligible studies. [ we currently patients Regarding are conducting

antibodies, cleave plasmapheresis. remove licit have a So, of study who commenced shortly antibodies within will patients and for with inactivate out anti-AAVrhXX pre-existing to antibodies the begin are we XX% the study separate screened to approximately and

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

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

XX late have program. patients As end nonambulatory and of of we XXXX, our ambulatory within dosed the June and over clinical

at sets to the scientific Muscle which additional World releasing cardiac data expression cross October. the and throughout data. the embark and X-year or MRI. presentations Among look ENDEAVOR and Study the available including accepted, Study ambulatory late patients nonambulatory our clinical multiple next in is multiple have form, [indiscernible] forward Society disclosures from early We in data, muscle planned longitudinal and safety MRI XXX, data XXXX. Congress program XXX, We

are Pharmacy at Care Medicine. American numerous Managed additional including as planned the of and our Multiple or the Diseases, the of and plans Neuromuscular manuscript submissions of to Neuromuscular International process Association release abstracts Electrodiagnostic Academy in Congress congresses,

report Finally, journal embark coming high-impact has the pleased in been are online months. the publication to and in that primary a manuscript will be we for available accepted

who happy thank approval. the the and families you we to courageous ELEVIDYS, living label our wish summarize of community expansion a chose To to I'd the the to are opportunity in for Duchenne ELEVIDYS. extremely support receive special studies our for those Duchenne very that participate this offer on like to that expanded with

and serve and community it studies our safety We available. of clinical basis will to an the through becomes efficacy on execution communication as diligently of ongoing continue data ongoing

now or muscular LGMDs. to dystrophy, our Moving Starting programs, limb-girdle with SRP-XXXX.

call, III on is first of the for our under as SRP-XXXX trial also mentioned of we As Phase study treatment XXX, EMERGENE, beta muscular for open-label the clinical EMERGENE of the known multinational dosing quarter SRP-XXXX of the of cause expression agreed circicaglycinopathy the [ is XE ], is of disease. sole absence [ endpoint which ]. dystrophy type beta-sarcoglycan The primary limb-girdle

We willingness for leads ambulation pathway which progressively of early treats an viable loss agency's mortality. condition genetic are the that to results ultra-rare debilitating, to encouraged a and in create by SRP-XXXX is

meeting, ] Assuming XXXX. BLA anticipate [ in would a positive we pre-BLA filing the

also treat review of pleased process we SRP-XXXX. for needs. received Fast designed unmet the Designation Fast that fill to a very Track conditions and and Track The drugs announce development to We serious Designation are facilitate that is expedite medical

SRP-XXXX from as disease designation orphan Agency. Medicines been has designation and designation Track, and as previously from also Fast drug pediatric orphan European rare to well the drug addition granted In FDA

] progressing of this a program, delivery the not our ability our small is functional to for study, [ which NXX and end. a clinic. Having initiation in progress [indiscernible] we reflecting but year's process biomarker to successfully the both suspension LGMDXB, with I start are for to this will important, of other SRP-XXXX, cerviglycin beta extremely pipeline, demonstrate by including protein ability The IND update with submit just expected to our year advanced Phase SRP-XXXX, together our LGMDXD

that XD alpha our treatment reminder, of [ clinical dystrophy is for gamma year [indiscernible] of also a muscular the a initiate meeting. type with XC the or type designed for QX We limb-girdle engage of muscular of we in the plan for to SRP-XXXX pending this or QX dystrophy sarcoglycopathy with to outcome plans progressing treatment As SRP-XXXX program XXXX, limb-girdle Finally, are in rapidly FDA study ].

are to the of of X progress than in portfolio, X pleased with less months. we summarize, LGMD very LGMDs our To our and clinic expect have the in

our advance casimersen post-marketing future current RNA ESSENCE The We mission set the matter firmly our to perspective across our for pipeline. LGMD well subject track. this beginning synergies assets of Continuing built our we've post-marketing an positions commitment are from or expertise both golodirsen manufacturing requirement and with on and R&D remainder us fully on as well rapidly and remain the maximizing trial, and our EXONDYS platform for both enrolled with clinic. the accelerating platform that are The PMO. sights and and as the are to

a as Phase sharing up diligently eteplirsen We XXX X-part ] As and read ESSENCE significantly is out it a per that with study. committed placebo-controlled to The safety XX was provides XXX approved XXX weekly. XXXX. to study MISSION a double-blind and dosage early reminder, golodirsen a fully III X-year missions patients. is soon randomized enrolled becomes is a to was of finding to both higher available. patients. and remain fully due [ randomized study the and dose of efficacy data MISSION It as study enrolled dosage advancing mg is and comparing per casimersen mg rapidly weekly in X:X exposure kg with kg

Part of PPMO. announced quarter, or from PPMO. our XXX. in to of B SRP-XXXX, Turning our an PMO conjugated first the positive we MOMENTUM study, ongoing is study peptide or study investigational SRP-XXXX Early results MOMENTUM

an requirement could on the is Cedar serve prerequisite approval accelerated engaging PMR study Agreement [ Phase for actively PMO are as the accelerated study approval. a a We ] filing. Phase or post-marketing with III for potential that a III regarding study

study to and considers completed a be that Our enroll intent time in patients landscape a current for gain is to trial. the an reluctance a placebo-controlled frame can reflects reasonable agreement in

reached Our year. is a an design this on trial agreement be hope can

take world my to a to thank future dedication, of devotion our for to our They disease. around advance living for colleagues I'd team summary, In demonstrated rare entire a patients work looks R&D a I'm to and with that goal mission moment helping forward unwavering we diligently advancing proud ahead like serving as have to the portfolio. with their of. admirable our bright integrity extremely patients the scientific passion

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

Ian Estepan

afternoon, Thanks everyone. so much, Good Louise.

discuss Before I I review quickly topics. to financial want results, X our just

to today get help some think plan revenue I launch add just but our into launch, everyone what we curve appropriately. We guidance. as the First, want will to the we've on color to further guidance approach provided I ELEVIDYS more refine the model

cannibalization very launch that strong broad some likely is the we the PMOs The guidance visibility how It's upcoming in ELEVIDYS year? PMO and expectations our that takeaway financial us will franchise. expect So provides of impact we as put anyone the guidance and apparent isn't in our a very, expect surprising position. will over all, is our all trajectory ELEVIDYS launches the from CMO consistent of our the guidance also modest with the previous

as mentioned, for our fact, first In time we were cash flow in positive the actually Doug history.

year. positivity We the expect middle to in sustainable next of achieve flow cash

reaching utilize revenue we've how end growth at our strong almost with the we decade, cash. to questions of several the So on had plan

overlooked gets want we business successful I capital of about BD track I development. thinking ] record think a how that briefly it, deploying I into before proven [ So it our in about to went strategy. And have we're talk often

as an a acquired today it. had we be here differentiated identified treating fact, In DMD to approach wouldn't if and we evidence

core neuromuscular ours. We'll in both focus remain a area and of our will space, leverage continue capabilities rare to disease of this and

of in match studies, noted efficacy of in the can rightly valuable the early probability ability commercial determining which insights controls that have success disease. Doug use also We propensity provide to in are better not our capabilities evaluate program. rare the to

to evaluate continue see deliver as capabilities. cardiovascular positioned to muscular for patients to opportunities that tangential CNS or are look expand need. also therapies to such in We'll we'll So expertise transformative at if areas best our look we're to or and

and the all to growth in no with front with we us, well transact, have have that said, we being anything of do positioned we're to imminently. need this Now but

We substantially strategy. have the the luxury here into today. find more squarely have but to of discipline able that We'll assets to being us deploy, resources fit use has same our gotten we'll financial that continue to

we've change week, dynamics quickly. over As market witnessed the past can quite

Our somewhat fundamentals appropriate. deploy the should strong from to environment strategically capital us insulate allow where us macro and

the So notice ] helping development continuous [ related manufacturing to finally, gene wanted one supplier our of in we've second our of financials, a to our programs. of issued to for to I the terminate and highlight Street supply therapy that our agreement model period adherent our process

analysis invest the we're this the we've currently will with of of and adherent for a August in continue The perspective. place a several suspension based that factors, we process to or not manufacturing sense of made the we're do mentioned, progress termination. the it performing believe makes strategic from impact on effective Doug be XX, financial and our to As including supply termination financial as in program. good an process,

the is and to to months termination will a This operating termination ended September reduction be of XX, approximately termination XXXX. for result applicable I today, $XX expenses the no reiterate has expenses and to further from the of analyzing reimbursement expect costs on X manufacturing months X September range development want XX, we agreements. impact We're net the impact Roche X $XX write-off recorded that to additional million million suspension-based ended research agreement. XXXX. any the X expenses, and under as for further As this of ended expected still any record our impact in and that months

tax basis on moving well financial financials. press amortization as of for income tax for results Now to of tax the longer applicable adjustments rate. reconciliation based a results. non-GAAP for afternoon, GAAP to basis. excluded our effective quarter Sarepta's adjustments, are in which a been have financial Non-GAAP quarter provided to income refer the each available financial quarter expense as income XXXX the our fourth results. of release non-GAAP added impact XXXX, second from a pretax for to company The change inning press Please second results on of in-light the of This the and has of represents estimated comparability reflect purposes. tax on non-GAAP XXXX details effective GAAP full no income updated release non-GAAP website this

was Net from our second the million $XXX.X for of compared $XXX.X product months of For for the $XXX.X total of period the ended and for the of revenues revenues revenues X of XXXX. XXXX, revenues was product and revenue for of quarter consisted million. June the the [ $XXX.X skipping EXONDYS ] compared million, net to ELEVIDYS same XX, which revenue PMO increase company million from recorded revenues million. $XXX.X second other million of XXXX product quarter XXXX collaboration to $XXX Net franchise period of same an XXXX,

of across collaboration compared the [indiscernible] $XX.X totaled decrease quarter of of $XX.X million XXXX, for of XXXX quarter AMANDA XXXX, $XXX.X primarily [ the same ] For second recognized and to period product product royalty agreement June increase collaboration $XX.X of The from individual for sales for for company development of ended XX revenue were million relates via $X.X BIONDI product with $X.X Roche second the revenues, the million sales and million primarily the the million net in for of to a Roche revenue the XX, reimbursement months of the net associated which revenue same reflects XX. period million -- XX. XXXX, X revenues for compared $XX.X other net for EXONDYS to XXXX. million $XX.X The million.

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

second approximately XXXX, related to same period launch of million the the sales $XX XXXX, increase and X its sales of compared XX, reflects in during in approval recorded following In ended of cost The XXXX. quarter of sales in the XXXX. $XX.X for of same cost primarily the ELEVIDYS in the million cost with XXXX, June period months June FDA activity no to similar we

non-GAAP million R&D factored million million. in and GAAP for $XXX.X compared a of and year-over-year XXXX of expenses a of $XX.X of the approval June were $XX.X after XXXX, a $XX.X commercial $X second the of of recorded for to ELEVIDYS XXXX. million quarter XXXX for to we decrease respectively, and primarily ] in of capitalization its a quarter [ due batches the period second of is R&D The million. On decrease basis, million basis, expenses $XX.X $XXX.X the On decrease XXXX, same

for the $XX.X performance XXXX, for related units. an the by well used certain million contributions and the SG&A as approximately to recorded ELEVIDYS respectively, Now of services and turning on to professional performance increase primarily matters, The expenses as conditions litigation an we of of achievement of of of million. driven in launch stock $XXX.X charitable ongoing a timing increase second quarter $XXX.X increase was XXXX the and GAAP million basis,

same period income for of compared discount $XXX basis, XX, a million primarily for investment the to XXXX. investment and $X.X compared due million a $XX.X The recorded portfolio. On GAAP approximately long-term SG&A decrease a is same the equivalents cash, change accretion of of we for in cash $XX.X investment We quarter second an XXXX of XXXX. other $XX.X million of the and the were XXXX of XXXX, had non-GAAP $XX.X as expenses billion the period million second June in cash restricted our due investments the for net quarter to our in to the mix net of to basis, On increase million. of

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

Douglas Ingram

open Thank very for you Q&A. Again, much. the Lisa, lines let's

Operator

our of from And . of today be Instructions] coming first Tazeen Ahmad question Securities. Bank America [Operator will

Tazeen Ahmad

get the have seemingly a on here that you actual work of based be high from patients I seems said done, and based because what of to want to demand lot just on bottleneck Maybe an quite folks everything understanding that is physicians. and

talked drug what that forms is But the start Has the pleased to from did of when patient months the prescription about to forms. number You changed the with you what it's issue of lengthening saying, getting and that it you're is timing from it to approved roles, X-year in actual time to X the take.... with X start journey you're start hand. got what that to for X- the going

Douglas Ingram

months. to X X

Tazeen Ahmad

X bottleneck key to X that the here? did Is months, when . that start?

Douglas Ingram

answer for much very all. your you no The Thank is, is question. short there at bottleneck really

all, payers, crisis it more able rapidly mean all Now -- in the not all as olds, it's the that families call, the were case we're last I think of we were age in earnings with kind is physicians, and prioritizing the to X we label, I us, mode, we're normal. X do than to about of clearly said the out to just that kids a year in and

for But the sorts has for normal and therapies, one for not now neutralizing to typical is modest And [indiscernible] requirements, ELEVIDYS a the X requirement atypical normal negative it's for months. very for I process additional that instance, mean and test X that's including, of ELEVIDYS, antibodies. these some about is but

there very really, are Manufacturing are That and encouraging. is great. well. to is really interactions Payer Patient very brilliantly. great. physician We're great. Everything no start here. is going very, So clear, forms is demand be doing bottleneck

conversations been Dallan time, having States. quite been enormous. and that's good we've we've recently, policies. just very conversations for think but some through And mentioned, payers United long almost had a they've already we've in lives seen cover X.XX And with billion productive, I that the XXX, pull

Now a ] to on take lot of some -- [ follows somebody time.

a satisfy mode X could about from the takes getting were start it had necessary requirements. our all we We're of getting mini own X form go predicate to number to into enormous to we're assay just We to kit approval. to forms. sure of crisis an start assays crazy make We're infusion. months clear, that So getting literally number very of

to going just of is that We're this And take time. so amount doing brilliant.

all label. And did quarter, the last really QX, there. of we prior was predicated well on The course,

be flat, kids toward and quarter. were were this You'll infection they've perfectly notice have the into of couple pushed we flat. that been scheduled that quarter, basically quarter this been half of would a dosed We but and a the back viral they'll actually had

going but and nice That to QX X than it's will we're strong growth, QX that them. have mentioned, be this as be losing we to of take X not going then in to will it months. then growth we're we've year. means And the very double growth So moderated more

we're as speaks everything between and success and billion we revenue that in the modest speaks it right fairly going therapies modest on across believe that see do our $X.X seeing is model the happening we're then to to with the we we PMOs seeing, fairly year. based in which X success of imagine billion year, cannibalization $X.X we're fact And next now, continuing the next to and we'll and ELEVIDYS, the cannibalization,

short the dealing bit very were have like, ramp so but through. anticipated, long we're just on here, everybody's It's hopefully, is what with getting folks, seeing a know even That's [indiscernible] the is clear and, things seen I'll this a very [indiscernible] bit I is other time. consistent one approved And has very answer are fundamental one is more to I you answer it's curves for optimistic [indiscernible] Like just the we a than as launch know, is that would what like? it sort just like. with mean rant been in -- well. a fact, therapies. we of optimistic front, no questions a I'm We've and that I exactly we've very of are little bottleneck. is, would what going go There process with So are our we

dispute better with much but statement you a us, can right launching We're and planet for there I expertise should launches a about end and no the I organization the a is, no say in made of lot politically one this is will on So earnings that with things this script itself, making and these serving good really track But I this success. Duchenne more in appreciate in your now. it. community record shape therapy, ability very thank you question, which more

Operator

Anupam our from JMP. next And coming be will Rama question of .

Anupam Rama

course KPM. the the multiple of [ -- we that about think prevalent XXXX time -- be thinking Is population scenario Thanks frame. thinking the about, I Also, And it's thanks ]. for should providing we Or time track growth said say, guidance. a into times, net for. should you how that more peak XXXX-XXXX over addressing over linear kind of the like The time? product be that to

Douglas Ingram

long-term script, incident mean population through given your And the longer-term and back to be sales said I -- wanted peak more reflect will this of much really to occur the treating we've significantly obviously this other a the more half decade, into guidance is in half the not the we'll that we granularity launch. us, given you very granular prevalent I forecast back the say Yes. we're thank the we'll in the in . population Anupam. decade. be as obviously entire XXXXs. what typical in for of before year this than question, moving in We've time I'm on of accurately seeing but get to going

Operator

of . question coming next be Gena will And our Wang Barclays. from

Huidong Wang

ask regarding I will revenue. question more the one also

modest correctly, So X to expect X XQ, months will even take despite for did you staff hear you warm you to still growth I infusion.

to sorry, consensus, that's the XXXX first ask at, they was in XXXX order billion But guidance, that have the billion for look confirm. we in the with number do of numbers $X.X again $X and for a ELEVIDYS consensus first guidance, analytics $X sell-side question, $X.X to will just And I billion. is when billion. the So then line

that think assumption? line early support And with your the do number So assumption? you what to that, . launch in data your is are

Douglas Ingram

for much very Gena. you Thank Yes. your question,

forms third still but over QX, we're to on as quarter, growth it's modest first that turn infusions. relative characterize immediate have start have into growth, I've explosive what going XX% I said we'll In to quarter So the preceding the as the the quarter. would

robust it at be of then approved But about So it months. take growth. end got nice QX, to and X we X does will remember, very just the

it in we'll growth, But XX% back-end So we'll be it the in quarter. then kind see, QX. really double loaded broadly will of about speaking,

the with wasn't line think is, world goal. then And on although where I XXXX, external we largely are in that our

early we what accident to a it's -- of days. is be that seeing of happen up. happy lined we the And question think to it sort are I

So lot. a early that what are gives days we . There's seeing us confidence

-- again, experience more than Just has no clear Duchenne in very launching so we're Therapeutics. Sarepta therapies X

we therapy -- we're force launched some in now of weird narrow it's on Our so therapy broader very label ELEVIDYS ways, couple fifth label things. a this our launching because with and a

level. to We epi [ We extraordinarily well. know ] down the know epi state

confident the we're very here. on epi So

curve right with, is around our lot and We now of a [indiscernible]. we're in see of have consistent of And all curves the the modeling, have the confidence lot granular it launch we launch again, we we itself. launch from envisioning would is And data. what of exactly envisioned what a

We on We of patient-level all forms like. have Just sites. and enormous have visibility. early know just amount data start It's down remember, an of to the we days. the still

point. good very Ian's to So

on track We our will confidence seeing which obviously, feel map amount what shape information days, early transparent to views other of as next significant we a surely we with year, But year. things We with the pretty now right provide into of people really felt XXXX, to and of provide discuss unusual. being than is folks. launch the wouldn't level end this out bit like this is a we're and wanted early of we're we update the overall kind for us to of

right So now. we this good feel very launch about

Operator

be will . Schwartz from Joseph of today coming Leerink. question next Our

Unknown Analyst

This is this quarter. Will on progress for Joe. the Congrats on

from one us. So

may a seen is years, we've of help fully For not something this the community. company However, many on execute successfully mission the boys be that some appreciated by to

the the some community? therapies is it a there are it, continue competition of will address what patients. or to appears and disconnect to . you in horizon? think these potential Why company with especially actively do the doing As competition on And with you that support invest next-generation

Douglas Ingram

committed population. anybody do to risk we're brilliant last think we which of that over for bit we'll can And we've transformative, very to know bringing carefully greater. And XX% the a ability very that believe, been Duchenne of restorative now I X the it community. life therapies, being to a working successful reach been making and who's in the of watching even well our dystrophin the pretty to over later, us therapies better we've meaningful been launches years, modest, actually at all on of I we've wholly X Duchenne

with studies. will lot We neutralizing to of down we a are We're pre-existing one antibody, our and be. have those knock of X dosing conviction successful already attempts that we in

been come The than news of us watching who will are to soon. community. be even XX% higher one get we'll And the those this. much good the I other have that very informed getting and is Duchenne think that

the and honest, community cheering understands the along. be of great patient I is think fully to bulk us

cheering curious, across the therapy as that's along, stay who I understands and I they and can in what tell community them possible. addition answer and would dosed that you, excited been have the experiences ask watching Duchenne their the and soon are very those this to are us can close who just very therapy get physicians people the all community as on have hopeful have spectrum with actually of with to data why the the Same been. community, physician and talked to say we

hear excited that. about think I very they're you'll that

to And is it. news think can kids. do well, as team are that therapy these for to that. they there's responsibility therapy I it better great do than of the out creates everything doing this enthusiasm be So team this a should this for us enormous to I the that team getting to think right-minded an no and And it. get And there's is this people there lot

give at serving want I not fact great a off start. levies. is and it his team So brilliant the to and community but with change the team that big this We're ahead, is this don't Dallan will too to

Operator

from be will Brian Abrahams question next coming Capital. . Our of RBC

Brian Abrahams

appreciate the launch on granularity all the really dynamics. here And

as centers You wait did there mention demand. navigate that times some they were at

of center need factor additional a sounds the or guidance of you any these put some embedded you it limitations somewhat that sites level place us in processes could here your in capacity to expand over what that think give Can at expansion? the infrastructure so of patients? to like sense monitor capacity What's where terms administer of into a And and And ELEVIDYS are is here. time? . gating

Douglas Ingram

Okay. in I'm but going over to turn let this few advance. things a say Dallan, question to me

a capacity want First, fundamental be issue. we have don't that. I to about very clear

about that have have which X the really actual The success the experience important. the it's But #X XX of the infusion U.S. sites said, dosed the in is how probably with already We working. of and then it with is metric well patients. the to therapy with experience

a ever of them and believe, having number of patients already those XX% such having launched any therapy significant. than having sites, is gene So with other significant I more

manage of absence and to of that that demand or And in got early in issue prioritize ]. Duchenne be to worried XX,XXX some one because things and some they're the seeing is [ the with definitely are it. more information there the more can pondering They've there so dystrophy of treated muscular trying amount patients with them in XX% that's United or are the States and about this I sites the [indiscernible] coming we're days just think things of are therapy. through

topic, But more granularity if please than you this I to on with have that, . Dallan, want provide

Dallan Murray

majority the the said, we've I even sales, the capacity that -- And became overnight, enough essentially today said eligible. as got happening enough more We've Yes. for remarks of Doug out than years. Brian, patients peak really capacity. than And there is, got my thanks the Brian, question. more vast as of Thanks, to but is what accommodate in Doug. in

and who to prioritize individual figure their center all going who they're all out They've to just each to of to out of first. how through dose approach got they're their reaching going work patients. patients have So they're going

forecast they that an the Spinraza, narrow we're confidence early can the as they what the have we to just everything ones patient adjustment of everything our genetic medicine. doing And figured should figuring of a stages whole expected forefront the these in previous we and the really out They they this at is, out are support and And anticipated figured figured are centers. ability as -- is it makes this label, population. lot serve period to is Zolgensma, these to ramp out these expected. were up exactly their that And process provide be they're just of and precision centers What are out.

Operator

coming next today be Salveen Goldman question . of will Our from Sachs. Richter

Salveen Richter

lever have back going guidance. kind seems like you're some using determining you're you XXXX infusion there's of It centers, kind how with this regard of the to or to guidance. Just bottleneck here but

So in it demand? of context as how thinking the of factor cannibalization the . you a the time is about you're number here, in demand, manufacturing help -- us Or the an business -- can your guidance? supply period? center of then patients gating given if be understand 'XX the it is the over that is treated the side at of infusion the at And PMO

Douglas Ingram

related mischaracterization very, thank be be to things. there payer is is So is first a and and say then process significant we're would very over are. some for defines don't Salveen. the very of time, that we I the question, process curve shape you envision shape of through all a all, physician The It's problem not think I curve where revenue of I have there's significant. to -- like, bottleneck. the demand, patient we getting a to demand going again, interaction with demand, the But to say infusions, to of number would the pleased the would that your related of solve. And it

as away I We And all But of a we're the be our everyone year cannibalization that over is years population competition benefit exactly means we'll of going kind do XXXXs. well, patients billion course next goes early which there prevalent cannibalization, to for start modest. is well. year, no be -- it to remind into treating that see next that into would year this going sales, and peak if year, entire we from cannibalization, billion and and of $X.X is decade patients for is well, from number there this in to the will, significant means then from are going will if a a reason, the $X.X forms on for therapies. And other significantly very quite

Operator

And Cowen. our next question Baral Ritu will be coming from of TD

Ritu Baral

peak fact Doug, sort you've think is of going given of different aspects decade. including guys of towards the going the forward, be that that end guidance you to the

do us general rough figure this up $X see was about in the billion were levies. and But of running as previously and internal still assumptions? that. years tie like peak all a for You've all few the you centers and a ago And given stuff your not that

a your of the It's ask between the And bottom levers I top about and guidance. really wanted tight sort your to of the of also really, guidance guidance.

the must some will bottom start out to what how I will enhance the So that element of top start -- the and and levers your those map out track can XXXX, have for for through start are volume ones? revenues, of far all talk [indiscernible] whether you way the and the forms forms

Douglas Ingram

is that yes, guidance we couple On your no amending that basis We feel prior of There -- comfortable have we thoughts. years the guidance. first long-term provided. about a guidance at for all. some I So provided question, ago, very

to That's to know to ways have what than of Like we it to the level ton else, a we thing, And forms does launch forms current have and our great experience. starts. start on map very the of have need anything that. accurate. we look It be more the -- no yet, should way start like don't this expectations correlate lot we what. look launch. We have versus at what the know of curve algorithmic is there's launch all a So update works. remember, still appear not course. But the of #X They

and is are doing and of based we short our knowledge the on well. very And algos experience answer all

our launch. As optimistic I've signals on exceed said, this the early view

with relatively So that again, right I'll just are we big we're the point, guidance is -- And it to we're the -- comfortable we're getting now. to providing say companies. your

guidance. be tight. side. that getting it's pretty while we mean very feel it's level providing each to still So But but about million $XXX there's tight, to I on point, of your comfortable

Operator

And Mike our question be next Morgan will . Stanley. from coming Ulz of

Michael Ulz

should quick still thinking a -- be up curious Maybe in modest just that But about some start cannibalization. as XXXX. impact? the longer PMO just Should on thinking to follow-up we ramp up? term? you're Or sales expecting you're ramp how . modest cannibalization that

Douglas Ingram

so it's first Well, interesting -- issue. all, Mike. of an It's for question, your you thank

We been of is there malls have views some we're that of And much a we've more been some into have the will the We It reason some robust seen what far in a little next amount cannibalization. so be, modest. how I bit very eventually say, own cannibalization. modest. aggressive significant modeling always been PMO believe modeled amount beyond the internal that, may may aggressive our of but be robust -- years be should cannibalization has a been there's on fairly cannibalization also franchise -- year conservative might have on bit to our or on what bit

pretty year, right it next cannibalization that. what is It model I would modest will very more in is and suggests remain be But years might now. the modest say the that our significant now after

Operator

will . question of And Skorney Brian coming from Baird. our next be

Brian Skorney

amenable one cannibalization say of PMO. PMO that have exon PMO early the patient P&L. straight-up Is And to it My contextualize in PMO and that on of modest the prevalence -- insurance have somewhere be trying to treated as a with are cannibalization, just isn't that the patients question into person based that to cannibalization forced result I and would of you their the well. sort the should penetration ELEVIDYS why maintaining patients by who you when ELEVIDYS the seeing equation calculate take and commercially XX company is and off just Are assuming patients between ELEVIDYS you Like come post. X the not choose to case? getting would on

Douglas Ingram

to want you touch this? do on Dallan, .

Dallan Murray

a good it's Yes, question.

significant percentage -- doesn't of map of that business way, reasons a is it's come can't from there's out that you PMO right a it the One the our U.S. that not now,

dosed gotten patients therapies right are terms there of with In that gene been that commercially now, have have PMOs.

are that dosed have that to seen some -- before, to we We haven't trying that date. aware are And think seen . get access date, I seen of SMA the in that to we've space. ELEVIDYS but with patients we're that haven't that but

Douglas Ingram

the ex So noting that, one that's on Dallan relatively analysis, robust. Brian, of problem. which been and the have U.S. that exist to confounders your touched sales is

closed system U.S. So with PMO sales. open is and your ex system math actually system, have seen we don't we a have the with close an

Operator

next Our question.

is question Uy next coming from Ear Our of Mizuho.

Uy Ear

back to going start the I So guess form. maybe just

said physicians. since the You sites you've in all receiving that forms from approval XXXX, been these and

who out doesn't Just declining? of get does define wondering sort are And these you . if patients? by maybe there they who were Are any at mean forms also it you aged that could will patients be before filled Or treatment. all? what in patients ages matter can older

Douglas Ingram

to understand the I Dallan, didn't I quite apologies. you apologize. want if question, understand?

Dallan Murray

think No, please Yes. know let and I did. it, I me understood I if

declining last of way. same question, the doing sites In doctors launches, ambulatory in [indiscernible] with part who are terms exactly your the to or they as the But of patients our where the prioritizing the PMO seem be seen it -- phase. we've

label first the again? question part old seen of previous to XX of broad X your prioritizing and what same the that's were thing skipping those predominantly in the was age And kind patients ranges, launches, X So exon we've here. . the year

Uy Ear

that age patients if would we you been have any otherwise age -- . think the Yes, may I have aged asking [indiscernible].

Dallan Murray

X yes, the label out narrow label. the I job in very in eligible patients There And had There out course, were gives did the in that will initial few never -- a patient. about team those few. because now will a the again. that prior Yes, team the be And up the aged on any age, -- of we with that execution. age was such Age. great excited were did patients

to So the can out . into support will comes we do anybody who back age everything we did and system.

Douglas Ingram

mathematical -- And simply, it's just then just rate fact. -- the a prioritizing what suggesting is Dallan and that's declining finally, on the

You're seeing gives that's in we've an right peak kind patients across good the XX, X seen see a the And we're And as as the start PMOs group, years right X into confidence now that function of But with entire kind as seeing robust early news a in what XX, in that which you lot XXs. -- we're heading of forms the start where organization. well. a is old us as then of range. age

Operator

from today question of Guggenheim. Finke next coming be Our will Robert .

Robert Finke

between factor This questions capacity will is any From side, and anticipate of our a now a Do Robert you for sales? be gating point at peak manufacturing here. on [indiscernible]. couple

Douglas Ingram

being we answer So we for few by shouldn't point, suspension see years. as rate that any hopefully a envision be bottleneck limiter next be would by we going limitation. short the is and in then that manufacturing don't is that the see it And will to the time the

now. we're So good right are we where very

Operator

is of Citigroup. question David Hoang next Our from

David Hoang

centers about There's for for these the all patients wanted obviously followed curve? feedback consideration of ask to for ultimately, to number if of need I a post-administration you've time the Thanks then a be labs great how that updates. monitoring done to patients. speed ask launch and value from of shape got any of your the safety. whether overall, the I . of and period of wanted can sort And be just fast just

Douglas Ingram

Yes. of at Thank of insightful. and cadence and X think of describes it's is infusion times, thoughtful the amount you of that that, do very this A important to good course, patients dosed. about you sort a the moment that be that there much the infusion that and question, for in last very way, very it's going lot of of if But there's can lot a follow-up, is. the

but infusion you of can amount good very follow-up. can the of of amount that great and you infusions infusion do have, we're room supportive not appointments just relate So very, follow-up,

on process in on that monitoring entire So we're that my well -- [indiscernible] and a any profile stable of best is very wrong getting as [indiscernible]. the knowledge, seeing safety that that to of the based part of particular, a

Operator

Our next Fitzgerald. question Kluska will coming Cantor be Kristen from

Kristen Kluska

a All a understand this But the process reaching numbers your approval? here. launch baked say, ask wanted here? in a few the assume European what's with the I XXXX I then in months' related driver takes time. XXXX, the Is time to that sales years peak key to And versus, best . XXXX expanded few anything frame behind if that

Douglas Ingram

Yes.

the their issue clear. infusions assume just way, So everything approvals. And the be on does is Roche really -- first ex talk not knows, U.S. that EMA a XXXX. accepted right curve here of everybody and review revenue result function the giving has our the to there already should leave in it and We'll that site presume and in very to revenue the as sales. curve occurs like. that But course, a numbers relating an either and about . And the answer submission and guidance site, from to is, their payer U.S. it action that now short to don't to want our -- of we're process for is monitoring the interactions I

now right our $X.X believe billion that billion we said, to to I've therapies. next as year we're so across $X.X do And going all

prevalent very We and about Peak we course XXXX, sales population year entire years we're over will feel good good we after the the patients. be continue the are of to some treat decade. work and doing will where this for

Operator

question next from Piper of coming Our will Sandler. Amin be Biren

Biren Amin

infusing with our sites over infusing by last the the to about in increasing You quarters commented XX on number QX XX calculation. the to from sites sites few of QX number according of

and patient the patients about think we of treated site do the of site? per volume treated per how So . consistency

Douglas Ingram

and over turn of Dallan, very comfortable with first we're this very let's so that number are we -- But see. to I'll sites the just clear, so chat a bit Well, we that. he can about

some sites. not That some general but else you what but in responsive is of a is grand being strategy relates increase may site. over really from anything to proactive requests more than see time Now part

sites want to as getting advance in process to XX here XX of So go on be right now, educational which would we launch, higher site would to launch certainly that. be are And a of with it sits much I will we other all comfortable. with if maybe gene therapy. I to But than and be an site, consider had appropriate very to remind this be to any goal aspiration an site through sites, everyone we the of validated a a asked even someday be

But about life metrics to And you down we very, so XX of of very . side kind at want good sites, if the by sitting provide it. other feel any on productivity

Dallan Murray

I And those site question. know -- been the from sites for our numbers of don't because has number you came where thank No, consistent. the Yes. very

we've We right hovering around have a model been XX%. rightsized and

is Now just that Doug as said, our flexible. said, model

the we to to bring as on sites well. some them sites So them things patients label neuromuscular new the is possibility very be speed of can bring X we'll get with on, this And older on up and rapidly. accommodate bringing adult broad that

more importantly, but enough have we capacity than alluded Doug as So patients. to serve today,

so We have responsive. can on bring support . team into rapidly. built new that our as flexibility very, very The is we model very sites patients, needed to

Operator

coming next from . be will Gavin And question Evercore. Clark-Gartner of our

Gavin Clark-Gartner

X as the cycle approved of years go after when within new 'XX conversion the first all also Those unmet we're you've And looking appreciate themselves that's half But other largest disease high the with So the talking analog need. into I as adds. and longer referenced about patient how we're at XXXX. basically PMOs your very are to fully rare back for ramps adoption. X launches approval

sales at point the even peak would not guess So process, be I XXXX? why . with few like in lag a some of months

Douglas Ingram

won't it Because be.

view We modeling. know the we're it like, heading very we're -- and that with we be in of good process sales good XXXX. and exactly have confident capacities [indiscernible] a where and the won't have We

regard whether the this people we're think make varies Another have want they're not good -- and many have getting a there's without to place really as in all patients by sites safety of where that. remember a are not to for good job to be follow-up and I to the we've are thing sure that symbolically, done there's like. dose approach. prioritize prioritizing you . And as and us possible QX. modest all successful to going of The remember we this that year. want doing We're a way, to job sites which solve therapy to means I that, trying be these to pleased -- have of are of we we the the a have in that being responsible with to very very going very good and we're onetime good -- growth

We QX, . sales, do have in as $X.X to And XQ back in relates billion the of half a QX, we're it decade. billion, our XXXX. be growth very therapies will doubling to robust going very, across in in then $X.X this it

Dallan Murray

We I And but has a Doug Doug, think level. we of granular going in high this question fact Gavin, this We a at lined very to why patient. and to have who's jump out lined coverage assumptions. confidence detail. to this, in? visibility the just very by can we these exactly forecast very, good a I haven't mapped And know how I level a sites we been at is granular your out alluding you've alluding is up, be one, our in by up. payer think very been have they're map

a of bit. that added So detail level I helps hope

Operator

Markets. from next Capital . coming BMO question will be of Kostas our And Biliouris

Unknown Analyst

This is Dale on for Kostas.

portfolio. the gene is LGMD on question our So therapy

ELEVIDYS. the promoter LGMD your same Given as vector that same uses are actually, them all of therapy -- gene half the portfolio and vector of and using

also So negotiations and impact competition? if And how from function of inclusion you early are LGMD the other development the platform future the test on payer pipeline assets. the effect ELEVIDYS you comment in the the and FDA potentially leveraging patients . about thinking to and platform label of time on physician potential therapies designation accelerate potential can on

Douglas Ingram

I'll turn to the approach our the accelerating LGMD . question LGMD over to Louise development. and

Louise Rodino-Klapac

Yes. Thanks for question. the

using same these FDA to open-label to looking Fast that my moving about for being exactly thoughtful mentioned of been I possible accelerate develop our along. ELEVIDYS cite with these platform. with the and the I we're pivotal remarks, been [indiscernible], XXX and experience make think fastest in right and with QC the between to LGMD as population with possible. Track We've terms in trial, just noted received sure way We've We've LGMDXD the trial every our we interactions is in similarities and as that the on ultra-rare programs rhXX terms help using vector pulling Designation, happy lever of speed in far, very so them. the you're at this coming the and

everything leveraging possible portfolio. the safety LGMD we're our certainly, So experience is and for ELEVIDYS

Douglas Ingram

have you . something Before we on. else Dallan, did add to move

Dallan Murray

And how function great the to think understand question and that's Doug that our a bit focused in data. Is test, I the his conversations. the new payers. is outlined the Dale I with mind team has did there time -- interest that from engagement, impacting the those asked label. the be about in payers data on that our the is label data really, And incredible may comments, been payer in little a as really the it's Dale, great that's in really what -- because

of big supports data that through label, going that's [indiscernible]. so a that broader so right that now And engagement a the with new part payers

Douglas Ingram

significantly. to everyone that not is benefit to remind remind overstated. we of those XX% that risk correlated with over did early ambulation. a can't rise importance of on only would in not of hit that the seen of time the Just rise, to The only statistically time I be people loss we've decrease all

very importantly, patients. correlate a matter important that mean things metrics, they the -- but I these . So not lot are only to

Operator

be coming Tim And our . of Blair. William next from question Lugo will

Unknown Analyst

This a last Maybe is on one. follow-up John the on Tim. for

Beyond the you. in healthy balance your of front internal launch had sheet with and pipeline, Bank your

on wondering you're us how in-licensing about Just give . if can thoughts thinking new programs. any potentially updated you

Douglas Ingram

this I Ian. will . to turn Ian?

Ian Estepan

and with that going to all way well discussed I to just what gives fit we're at them is be I remarks, looking our patients -- This able to the therapies evaluate leverage quickly. mean that to our substrate being so us to It prepared in commercialization. to developing and able being ones and that's deliver as an existing and organization been capabilities and very wide a manufacturing of successfully amount

remarks, responsible but a still at have going in more said transactions past. same we're Like we've to deploy, the to -- we're going done I fiscally So use the to been in that the resources we've prepared approach. really lot

development developed. incredibly we're the build ways interesting this but are But not from are valuations chase look high, a of therapies opportunities. going to and value for that were investors. maybe for before for lot We're to going being week, to also perspective a patients, There

opportunities. at looking on market making keen that the it very makes we're So sense and on sure valuation based

Operator

Neon question from coming And be & our of Gil Blum . Company. next will

Gil Blum

question, can how just have to treating the understand to a the really year. next how center? . metric thoughts if any that works down I'm last But trying many you bottle does on potential at And you boil physicians Maybe on each provide

Douglas Ingram

So again, this have I'm going to you way, by frustrate same the question bottlenecks. answering we which is again don't

a doing launch very well. very, have We're We curve.

number of a at have number a of a physicians We treating lot sites, enthusiasm. sites, of significant significant

very We patients now. manufacturing physicians strong right more demand sites, than enough extraordinary with have those great interactions and enough payers, more positions, a approach than from

feel great capacity perspective very a a about and We that and supply good channel. distribution from

X back to billion. that billion that, going mean of a half $X.X successful XXXX have across we're to of that of do our really revenue have As therapies we're to a to result and year, this some going will we're approved $X.X going a

positive profitable this We are quarter. today. We flow were cash

flow a cash in be basis. will of majority couple in a biotech different quarters, the today. the place than We We're of very positive consistently next go-forward very on vast

to better decade. have going the a and lot foremost over better have rewarding to committed next mission. strong, very life life focused patients importantly, this bring themselves this and those secondarily, these first We stuck a all And but on many is of bringing with business, also have sustainable We're years a of to very investors which patients. a to us who

where to need good solve about We a bottleneck very and we're don't not So feel but have we overly we to for. that be we in great shape, defensive, are.

Operator

to concludes like today's call Please to go This Q&A for I Doug session. ahead. closing remarks. turn over . would the

Douglas Ingram

much. Thank for you all your Thank you questions. very

this about where you excited thank answers. Very going for evening, Your those incite this rest questions team my of to the we're for year. great and

a do year. this lot We ] of have [ to well. is launch going work ELEVIDYS

Our brilliant. the number very and patients going PMOs are ensure continuing our we're that to our the We understand these patients have enormous benefit responsibility to well. the of make therapies, of to we can ELEVIDYS service these greatest going launch from

I across talking with look evening, And forward to our forward on pipeline to to update that, and look have advancement updating performance to well. this launch, on soon. we and you year lovely the you with as team a you

Operator

conference call. conclude today's This does

You all may disconnect.

Sarepta Therapeutics (SRPT) 7 Aug 242024 Q2 Earnings call transcript