Sarepta Therapeutics (SRPT) 2 Aug 222022 Q2 Earnings call transcript

Company Profile

Participants

Mary Jenkins	Senior Manager of Investor Relations
Doug Ingram	President & Chief Executive Officer
Louise Rodino-Klapac	Executive Vice President, Chief Scientific Officer and Head of R&D
Dallan Murray	Senior Vice President & Chief Customer Officer
Ian Estepan	Executive Vice President & Chief Financial Officer
Gilmore O'Neill	Chief Medical Officer & Executive Vice President
Anupam Rama	JPMorgan
Gena Wang	Barclays
Judah Frommer	Credit Suisse
Tazeen Ahmad	Bank of America
Salveen Richter	Goldman Sachs
Colin Bristow	UBS
Ritu Baral	Cowen
Max Skor	Morgan Stanley
Debjit Chattopadhyay	Guggenheim
Brian Skorney	Baird
Hartaj Singh	Oppenheimer
Tim Lugo	William Blair
Gil Blum	Needham
Joseph Schwartz	SVB Securities
Yun Zhong	BTIG
Zhiqiang Shu	Bernberg
Danielle Brill	Raymond James
Kristen Kluska	Cantor Fitzgerald
Gavin Clark-Gartner	Evercore

Call transcript

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good day, ladies and gentlemen, thank you for standing by, and welcome to Sarepta Therapeutics Second Quarter 2022 Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speakers' prepared remarks, there will be a question-and-answer session. [Operator Instructions] Please be advised that today's conference may be recorded. I would now like to turn the conference over to your speaker host today, Mary Jenkins, Senior Manager of Investor Relations

Mary Jenkins

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

which on the are Doug a and the of that I'd These to call Murray trading forward-looking for Estepan, which Ingram, any formal our stock. during materially and today such webcast, statements. from Rodino-Klapac. the results Please statements many Sarepta's to our moment slide forward-looking common beyond differ uncertainties, Actual we risks the of note making Louise and control. Q&A. number can and Dr. After call will this the operations forward-looking remarks, affect results of are could prices us Ian Dallan adversely a Joining contains statements, and involve business, Sarepta's we'll forward-looking be Difficulty] call, open statements. like for materially take [Technical on risks these

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

Doug Ingram

that's serve, results outstanding biopharma, my call. second gene treatment Sarepta muscular by you in its entire therapy for all with opportunity largest moment. in SRP-XXXX, on the quarterly quarter But therapy, to that view, I and will begin near-term the to field of Thank you, XXXX therapy joining this for focusing thank will in we our progress our patients the and Mary. Sarepta the Good gene gene performance importance I of given to a discuss everyone, Therapeutics' conference our financial Duchenne afternoon, quarter and dystrophy.

will Biologics of that or the SRP-XXXX we Application, FDA engaging BLA, were possibility a accelerated muscular submitting about US previously to recall, As the License approval dystrophy. you for we Duchenne with treat disclosed of the

we the We unless on timing also FDA. times we the change approval cautioned the by strong on assumption accelerated numerous conviction not base that of our approval, case to have an BLA receptivity would

assumption last discussions now changed. indeed As week, has base we our are case announced and complete, our

Drug of course, tolerability data the across in-depth with associated and with efficacy and nearly treated and various with quarter, compile of to FDA, the and included Evaluation Duchenne Advanced Sarepta to announced the ambulatory included muscular agency to neuroscience that guidance of the the Office that in which BLA to office and and across the have supports that Tissue the across our function-related Research, thank of evidence patients when SRP-XXXX NSAA and SRP-XXXX safety commitment last we the to as expression like biomarkers XXX, Center would of BLA XXX of treat unique multiple for the input drugs. office with its the robust I written spanned the of its supporting consistent engaged functional guidance intend for as Based including course well review we preclinical this is, we input benefits, with multiple approval results, FDA the studies. intend the fall. of as patients tests for the and to leadership, dosed dystrophin and the submit SRP-XXXX, shortened its and and an CEB including the This and its the time, XX and function impressive new functional the animal FDA of SRP-XXXX associated a time second wealth XXX those and the and Over safety review have that its SRP-XXXX. review Therapies submit studies models the on accelerated with of dystrophy. meetings of benefits provided We week,

by spring and the for We our just are requested will is that. FDA, advisory planning we it is and If the we filed. the an committee whether would one by of hear after XXXX, BLA is requested anticipate preparing FDA in

gene and are Assuming a be completing end, successful launch the affairs delay. readying in augmenting largest we organizations, in submitting the we for building review, anticipate addition site BLA, that will ourselves community inventory and approval our States. at therapy immediately in without medical will to to To our importantly, and include mid-XXXX. serve commercial and United launch readiness the launch what sufficient That

The As should we site Based EMBARK weeks. and pivotal enrollment ramped EMBARK. few initiation screen in screened fully second prosecute intense our the be been for to trial SRP-XXXX. demand enrolled XXX-patient quarter. has our current next we we rates, the enormously BLA, continue and That's execute for on and our in

exclusions an is underway the One in our the narrow BLA been currently to Furthermore, with encouraging a an accelerated research anticipate percentage discussed approval accelerated cohort EMBARK anticipate Congress our an We at EMBARK our X low mutations in the Study excluded approval with low-risk our by filed. that study within time. goal within to both have range additional fully time act will the to what XXX the we concepts to the single-digit us, XX is for and trial informs companies and the to the enrolled their dosing accelerated confirmatory be approval. time confirmatory have of FDA to trial of by

was a study this Study We also separate XXX or study initiate intend indication. designated to non-ambulatory later year. That placebo-controlled

current MOMENTUM our XX. our next-generation the trial PPMO of encouraged platform, times greater B, one-fifth patients, seeing of treatment standard for half RNA of skipping we exon and greater for eight SRP-XXXX, dose times initiated and RNA-based dystrophin the XX time pivotal skipping, to On by the production Duchenne care, the of Amenable Part Exon

placed announced the the that of quarter on a while we provide has FDA in waiting continues additional hypomagnesemia. we cases dosing around in globally, context United States, While and information hold

We completion have confirmed MOMENTUM Part MOMENTUM information over standard a of enrollment requested provided continue the guide in This we of to SRP-XXXX B for be half could the current XXXX. care. to in Part profound improvement and second of B,

So for at the a PPMRs population. greater are of to progressing same work the the we preclinical percentage additional treat Duchenne time,

color will of Our R&D of Scientific further Head on provide our performance Officer, Dr. Louise pipeline. Rodino-Klapac the and Chief R&D

the another This as performance quarter for quarter XX, to we approximately $XXX.X community revenue Net the -- last AMONDYS of execution Duchenne million, quarter VYONDYS announced was with XX. patient therapies, versus was and exceeding consensus exceptional and approved year. product XX% serve performance. approved Now EXONDYS Total to same quarterly and $XXX.X XX, three million. continue growing revenue afternoon, the almost we our

Given and their our overperformance, million. we quarter. be revenue year could $XXX Summarizing, are tenacious product to now Sarepta hardly $XXX full raising million net execution prouder I guidance between for team this the our of

the its leap what are Duchenne this approved and community work with advancement serve for around Sarepta thank thanks and individuals I and SRP-XXXX participating I could and patients guidance world. and in will time broader that translate the our to give we committed to to want studies, patient for team, the a who we made in special a families the the I the therapies, want in living My thank FDA currently community. better the confident with as into States be I to monumental of colleagues are to our our we my progress. United investigators as life want Even and a

let cohorts over to and turn the with Chief our the for these Louise Officer, Louise? of necessary of SRP-XXXX. Head of are call And Scientific All Dr. success Rodino-Klapac. me that, R&D

Louise Rodino-Klapac

date. therapy recently past Thanks SRP-XXXX, intent significant important announced with as want for Sarepta, their respect represents this FDA. to and Notably, Duchenne The speaks with July dystrophy, an with this the muscular gene thank made development Friday, SRP-XXXX. a not positive the for BLA seeking underlying achievements to it XX, approval to work the a the science the team just outcome accelerated of strength for all cement candidate generated treat engaging we I but Doug. for this patient to community. more We're moment supporting to the we've importantly, and we've data our to thrill our

X patients from clinical including on for Study also improved and in results The on these integrated submission. We XXX BLA representative remind working parallel. material, and well-positioned effort, Study with scale. this clinical the announced While XXX, is the early you, of week briefly XXX, or the XXX, from functional improved BLA XXX, with are baseline of at top end recap and we baseline NSAA XXXX Pretreatment our results important support material. Cohort in XXX of submission the To mean XXX, mean an showed points this BLA four Due our XX, Study will this ENDEAVOR. XX and analysis to and July now I'll NSAA a end is data XX, we had major Starting commercially approaching you. NSAA. to studies fall. in submit

they commercially can assistance function, on unadjusted NSAA effect our means the The XXX. in now conviction of to being X.X confirms able do therapy, and impressive are further because demonstrate motor patients to for compared of Also, ENDEAVOR prior less the they of p-value SRP-XXXX Equally increasing results risk and now probability success activities needed external commercially own. group, the propensity for improved and as than representative X.XXXX. both weeks perform EMBARK EMBARK. they X.X perform confidence unassisted these representatives that their requiring that is control example, on EMBARK, impressive SRP-XXXX of improves therapy. activities level not increase they with were of on two Study our For to in used data SRP-XXXX These XX treated before that a can treatment the these our same that or Master of material patients points

data is our from has summary, run. our all also walk demonstrated These XX-meter and time on our improvements the Study the And Are executed questions. these derived, patients Speaking answered durable? patients run, on these results. look two functional across as at Study XXX-meter receiving a clinically XXX flawlessly. complete. of Based of are measure. clinical we SRP-XXXX such secondary four In steps, of improved team as most And particularly EMBARK, in after effect functional key enrollment on XXX. to operations years result, will SRP-XXXX endpoints significantly recap extend study now time data, nearly common walk meaningful? four treatment the they I'll data First, original because important, long-term is every four

then in in we four analyses. to control years X-points As demonstrated patients total a the These baseline waiting. showed treated a an two mean and external reminder, comparison score over score year propensity increase conducted data from XXX change baseline from boys using NSAA of in Study four. four group that in with NSAA

Importantly, are would, patients a according years the patients, as steep these nine progressively of disease. the gets Duchenne four, old at and these year of decline older disease their being that history Duchenne is worse, to natural because around phase

through increased that function declining, of and treatment increases all distinct we a at their compare patient have unadjusted with p-value drove a difference level above well patients using when period. they've mean on observed these the group. means supporting matched instead the four point stable time, single external XX-point and remained However, propensity time demonstrating effect maintain and the of showed, we X.XX over clear control, difference a thereby data baseline of their a it's SRP-XXXX. that Based durability of patient that individual increase, No on this of for patients we X.X years. squaring Further, treated nearly lease the

SRP-XXXX As able an external for not the control with now can five accomplish. group do treated example, those were activities patients to

disease, that see that patients group. years of increase in steep whereas time. has the also now decline three the of to control boys treated phase part important continued to effect These decline It's pleased precipitous the note and to treatment are of the stable. our a remain are in external We over four

propensity-weighted target integrated GMs X.XXxXX X.X control. a per one-year between [ph] And patients a results received show that will score XX median external Moving group this for Part who fact When from that, I of for the half points that control another and At control the treated more of one points At studies patients The baseline year functional target with patients, XX, to now to SRP-XXXX Study a the for the in to the of compared patients SRP-XXXX. week analysis of an in patients to sector of square dose compared received were lease functional of external external in XXX, points X.XXXX. baseline. only results received unadjusted the data review p-value one even all in XX this total we and year, dose all, group X.XXXX. in difference mean, the using higher group, grew group. X treated who means from dose saw the two-year XXX. we per a change than and difference kilogram who these patients NSAA on median approximately higher less our now X-point after XXX SRP-XXXX p-value target NSAA analysis X.X receive integrated treated with X-point a XX makes XXX, treatment efficacy improved from a NSAA impressive. X.X

data now mean and key NSAA improvements the such consistency appreciate, of sites to endpoints, across As and you secondary show across run. as improvements rise XX-meter reinforce independent walk can these time functional three

safety has pleased activation. no summary, of We both these potential and with of and analysis data Further, manufacturing will our also share In a performs clinically clinical demonstrated relevant consistency the SRP-XXXX process. our what as integrated profile commercial expression are evidence support and consistent history demonstrated data new remains and studies, predict complement to that for which manageable well-above from of agent. the disease-modifying natural SRP-XXXX efficacy all

franchise. Limb-girdle to our dystrophy, therapy turning or in Now LGMD muscular programs gene

to respect necessary of the steps to For LGMD-specific we process, SRP-XXXX assay continue with validation. LGMD programs, progress and our development manufacturing building and including make our other

addition, natural LGMD study and our enroll history Germany continues In to our in component key pathway. a also of represents development

a call and translate this rare families Dallan science positions us their Our into as commitment on tireless these remains you update Thank role activities. the on patients are progress our important favorably I made we've patients, evidence, clinical The and well commitment. therapies the tumor to now Dallan? based around best to science over the for the forth and date in will to for professionals on the dedicated an investigators to as then strong. bringing to commercial therapies. that advance deliver important scientists turn world disease and for

Dallan Murray

XX% Louise. We across first time, net second you, PMO for Thank eclipsed $XXX In million growth XXXX, for million prior million in three for for of XX. XXXX. $XX the team product of XX% XX, $XXX million AMONDYS represents delivered RNA-based quarterly quarter XX, VYONDYS the to roughly over and over This therapies. all quarter $XX EXONDYS growth the revenue double-digit compared second approved the quarter generating and almost the

we analysts QX approximately QX. $X pulled models to the result. been believe may QX as from thrilled ordering that incorporate Monday performance, note We properly to fell a for pull-forward I urge their into year. are million to to this have due the on and it's July in contextualize, with into holiday, this Xth forward We that experienced order important which this volatility a

year, I'm million we're you've As in from already full of that say our and revenue performance $XXX to product of happy $XXX million to from between the net the our over million. heard $XXX Doug, year of as result a half first a guidance range increasing

year. we mentioned first earnings direction that for serve. As rest These are Duchenne clinical the number the somewhat could final own remain enrollment XX% to into of when swing in the our our comes They competitive of it there important to the on factors, we of population number of a hard our some either predict which call, trials. quarter include

saying I'm closer we our which across we such, and the at $XX narrow execution year. provided get which proud growth. the success goes this this end we that of It teams to the of very all commitment As and of without as to range, intend kind Sarepta, enables million

more managing team beginning the compared for quarters growth quarter for of And in our expect growth XXXX, billion overcoming Moving the nearly grew continues XX nearly three by on we growth, therapies. the represents The XX% launch, since XXXX to in quarter. predictable first their has second patients. strong to by caused post team six-year X% changes year. than the another launch. over generating than the X% to remainder very prior performance of overall, we the cumulative and EXONDYS of revenue, of the representing double-digit insurance the The effort $X generate the of headwinds XX and and the each mark efforts to at over of each modest the growth XX over year. EXONDYS XX as VYONDYS finding more XXXX, quarter successful approach of P&L continued of

have exon VYONDYS therapy with team XX population our launch the prior is over in and straight the its new to with amenable quarters. patients growth quarter. expect substantial market XX% more the We get quarter our coming any continuing We of changes continued leadership AMONDYS maintain the very patients. existing and than in their don't position efforts XX second on continued successful

of both, been previously VYONDYS. start for the has and we've than As EXONDYS conversion to pace launches forms and therapy mentioned, on our faster

rate launch As worth execution, a used to SRP-XXXX expect eligible as [ph] team coming on for this as therapy. start launch. as and more the AMONDYS been that slow mentioning get team skills growth The we population to has most dry such, disrupted to successful launch date, and the we we sharpen to run in continue potential of the the to XX quarters, the it's prepare our

our proud this not very and likely team precision Sarepta's most our our our with are pleased execution. we important positive are nearly most patients do medicine. in every for I'm approved RNA-based history they PMOs. support regulatory who half to outcome one the enduring be just but unwavering highly to current the how cannot XX% of first amenable a work and team's commitment and of and the launch mission-driven the genetic of of momentum perspective, Assuming emphasize success in in will history, a three motivated from the launch enough, XXXX day I of important

and ourselves segment continue our serve Duchenne to an will while evolve community prepare the the SRP-XXXX population. to we larger with We Duchenne of PMOs, launch for even

work all I'll muscular Estepan have with update call with dystrophy. our the therapies turn to Ian over patients effective until an for Ian? living on won't for that, And stop Our we financials. Duchenne

Ian Estepan

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

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

XXXX, and were XX quarter increasing net our XX, for the million $XX.X million products. for of credit AMONDYS second $XX.X million revenue XX. sales reflects For primarily EXONDYS $XXX.X individual in The increase for product for VYONDYS net demand

we XXXX, not of increase is of of XXXX. million $XX.X of compared in quarter $XXX.X to million $XX.X approximately of to quarter reported and quarter the recorded million June our which second XXXX, XX, the related to the products million $XXX.X $XX were million agreement XXXX, In for to period $XX.X primarily XXXX, due quarters same XXXX, net the increase relates for a arrangement to our result, product a Fisher, second our to Roche. GAAP each million for Roche supply The million of we million second manufacturing our of million revenue we of million months $XX.X million. a during RNA June decrease are manufacturing million reported increase under an collaboration for total revenue for of in collaboration second $XX.X million. to we upfront a XXXX. in $XXX non-GAAP XXXX, and of compared and XXXX. range of sales diluted to million or and $X.XX $XXX.X the As guidance the shortfall to for $XX.X of We the with period basis, net basic of of $XXX.X GAAP quarter the In costs period a similar million milestone due we totaled XX, compared in partially revenue, compared of to sales a The a a million XXXX, a share increase On increasing of our XXXX $XXX ended XXXX. is in and quarter XXXX. our write-off XXXX expenses ended to $XXX.X certain $XXX increasing of the and to of no the $XXX the XXXX guidance reimbursable to for of the three $XXX.X June co-development same of basis, and quarter diluted $XXX raising accrual XX, respectively. range quality franchise million. recognized On to and year-over-year specifications share of products reported of meeting the XXXX, cost three XXXX, the non-GAAP net for second in by second quarter activity per XXXX second or same and of and quarter respectively, compared R&D basis, for months million or second in loss Thermo of per share due cost The loss primarily and $X.X diluted loss batches period offset ended R&D per expenses non-GAAP $X.XX payment million demand On or with the $X.XX agreement of as net $X.XX a expenses expenses same the basic for basic our of XXXX, a with in

It's for XXXX. same a $X.X XXXX, net recorded million the stock-based basis, the to by second increase months The assets recorded primarily expenses an compensation expense. due $XX basis, approximately for agreement expenses non-GAAP the quarter. is turning during increase basis, million. increase executed and compared a of we The quarter for that investments. an non-cash million primarily this grant in billion in increase during is the end second approximately cash for XXXX. of cash compared and the On On million, a XX, in due we $XX.X and to in GAAP CEO of to of net was the losses increase expense of Now At of to million. $XX.X period million same quarter June XXXX for the to driven on respectively, XXXX though, this restricted of we disposal to an XXXX, XX, of $XX and second GAAP $X.XX $XXX.X million expenses, highlight, June three period of SG&A. ended On quarter were a modification million expenses other the important XXXX, the XXXX, had and of $XX equivalents $XX.X SG&A cash, other

actually FDA to finally to I it's the once, for get the feedback from absolutely be So nice lineup thrilled by saying we a received for with XXXX. we're conclude the lasting because that

as and very challenging previously everyone obviously and saw our think our we market. knows I spend a managed we gated expenses,

I thought the the you'll prepared extend that and recent patients commercial activities, our news, building based material we're spend our important anticipate so the to our a year. with of I number don't year, by though, actually actually into now middle of runway However, highlight, in serve to change I the further and to XXXX. that approved if be for due the to cash timing manufacturing on expect still of also out SRP-XXXX, up ramping was remainder the our it capacity these of maximum the infrastructure

thank want and of just our I finally, taking submission. our and BLA to the for to the Doug's data, FDA evaluate forward time review comments the look So echo to we

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

Doug Ingram

open you line for Olivia, Thank very let's the Ian. Q&A. much,

Operator

[Operator coming line of Instructions] the The with question first Anupam from JPMorgan. Rama

is line Your open.

Anupam Rama

factors X-year-olds? to approval X- ambulatory comfortable something does And the Can get talk based all the to that all restricted FDA your with population the on change about potential how research? led more you on a progress. age patients market addressable this Congrats to in versus

Doug Ingram

for very the much. Anupam Thanks words, Thank kind you Rama.

law first to as agency. and agency a when would about I and I'm So supports Generally, give say not -- therapy broadly of detail the I'll you of all, give data wealth lot the have stroke the answer, data the its of of back broad comfortable. a the we going got group speaking, forth you a and over vintage the of benefits course, our that patients. very broad way

the entire which It want as think has our to ultimately we the a ambulant BLA four I to now a population. is believe data is it given older. right is that as seven-year-old goal data population treat to we so large that the have will it's population. the appropriate therapy that a Duchenne. And now addressable to the patient for that be file is shown no that clear, in be population younger ambulatory repeatedly if There's affect of to reason not the to that child it benefit is with multiple studies ineffective it very or not would as simply percentage

muscle provide possible groups have mechanism properly bioframe from to have applicable that equally be applicable. cardiac equally the skeletal Our the localized. across goal level to shortened approaching fact dystrophin muscle but muscle patients to this for is broadest of functional regulations the extent that that ought and all benefit age label. our given the And the therapy action will It of is

There to be do that of to get will things we expanded. another additional we've doing. label got So work are

experience, it that going this We will start will data our before to XXX. non-ambulatory get be study as data safety have that a can from even expression that because already from before Study even started placebo-controlled and add the non-ambulatory experience patients. I to year. Vision out. got expression mentioned, in It then We'll safety what's we trial, or with that we We're have but called have we we'll reads

XX you kilograms in non-ambulatory we – that patients, we years including Study of old had very few know, XX – times. patients our – and patients non-ambulatory cohort in may in over a dosed much As we’ve done some XXX, older nearly significantly heavier

seed will label then expand to add that. to So this the would we supplement beyond And the through ambulatory. a

addressable to all is ultimate goal make the it population our certainly So patients.

Anupam Rama

Yeah. And population. just of a XX% the population follow-up. Obviously, is ambulant the approximately

seven you modeling have an minimum patient minimum. you to old, if available double So, population a on have were the population patient would ambulant a four, based just at year

Operator

Thank our next Barclays. Gena Wang you. line from question coming of the And with

open. Your line is

Gena Wang

accelerated Also, Thank taking for path. the approval my you question. on congrats

questions. So I of three have part

the approval, FDA accelerated the Regarding feedback.

of approval? the So, FDA what FDA the approval? a control? you propensity And lastly, before were data EMBARK way accelerated the request of submission main did to validate accelerated that do expect basis serve to the And endpoints

Doug Ingram

you Thank Yeah. much. very

first all, to of of endpoint by provided they endpoint was I related surrogate that never justify our we The of compelling protein either the a ultimate significant on certainly otherwise. it all the would a including then any with EMBARK that had that either of we've role to EMBARK, await of dystrophin require endpoints, be the includes think, including And dialogue set, we that or would the work. I evidence, there it's would and analysis. of the or played very be or -- the data a propensity suggestion as the certainly agency preclinical And interim discussion shortened in the agency. the is XXXX mean, and So approval, all functional accelerated entire the justify that totality we – course, would the

So that our arose the discussions good a with agency. that was in multiple mission

Gena Wang

you. Thank

Operator

Thank you.

of the Steven coming with RBC question next [ph] Mallon Our from line Capital.

line Your open. is

Unidentified Analyst

our for bit you on functional of the Congrats Can the Steve learned what a is reviewing progress. you more XXXX expression points, totality end the the This micro-dystrophin is And comfort what including aligned Brian. between for thanks Thanks. with relationship question. Thanks. expression there relationship gives on on function? CK? FDA you the and on from Hi. taking there share And that data between and

Doug Ingram

OTC, I both particular meeting expertise drugs,, new us in-depth tissue. is of leadership, Bureau that, comfort gives division, human the any you – from feedback the but are division association? Louise, office that in in telephonic seek And underlying we FDA the based on want comment written which multiple Sedar a line, result do with think of as a meetings for has Approval. the had what Sedar a Accelerated on, significant that, we further data have to any we received the the conviction of to we've discussion BLA

Louise Rodino-Klapac

is data to certainly, I'll our to and our in through the data see clinical I'll the preclinical -- totality up. to those And about the back studies our across is data going functional strength dystrophin. learned linking of the XXX data continued what the data data, dystrophin data from we're our studies data, add just clinical biomarker of the that consistency between expression so compelling, functional all the we've with of has keep the and was function XXXX

Doug Ingram

of few the Thank -- that next on exactly would kind in have XXX we'll little everybody, keep dosed that nearly XXX, you. not data act I'm as the run. Study we've And you talking know, just We've would predicted March, will what in patients the have bit. I have in remind studies go XXX. the a seen on we and animal you run -- we weeks. which, a about XX just

another XXX, patients every I very that studies, results strong and NSAA the in that been that the also of very, very XX consistent yet fat significant over. CK tissue. that them the endpoint, they've conclusion we in be as significant All it localized, great that XXX, muscle we which cross the this think XXX, will the benefit at and been mid-single-digit And we've ambition, before mentioned very a in the we're properly truncated across acting markers drops seen in our study and results, functional enrolled is where sarcoglycan fully reduction in CK. very all seen consistent CK therapy of noisy values protein provides biological was support is key the underlying that one both is benefit. third-party shocking MRI in a fibrotic We've and it, the of has functional seeing test all are time results truncated. and see

functional justified natural that purses evidence therapy that conclusion or is protein, rationally designed functional. perspective over the in that So supports review our the result both just years which of the through was there's history, from design of then conclusion wealth -- the our the and and SRP-XXXX and of protein in shortened a XX more totality

seek about we part important approval population our ambulant for the possible reasonably non-ambulant accelerated all we in follow-up that mission. a appropriate, have very basis think feedback And We as as and of liable forward received of plus pathway of conviction of the an the a on for soon there's FDA, the have that an we BLA and amount certainly we've safety profile. the then enormous population as they're from as

Operator

the our Credit coming Frommer question with from And is Suisse. next Judah

is Your open. line

Judah Frommer

out. And thinking the any the still timelines quickly and of EMBARK And just We for compromise accelerated thereafter, a data taking between where X mind, somewhat Yeah. close ability on is approval, of for the about option file if were explore to timelines as EMBARK point with the we congrats accelerated accelerated, Hi. get BLA the coming it not to kind how the a Thanks BLA should are approvability? well filing? given if data low-risk you to In way your the question curious it's my does the progress. accelerated same in be didn't the Phase filing? the

Doug Ingram

I'll agency. first, so very BLA will clear, we'll last this. actually is developed be times would accelerated discussions, very get first, be But evidence-based question received would we have many up submit well this file. Well, would said speed a that for review. and would months, be since We've a would we're that we strong want which last a approval clear this BLA over many the be not -- we conviction in answer I not to about the our we unless the productive BLA

therapy. this simply thereafter readout feedback as is this an to answer to get been thing similar as appropriate as await same the BLA it children remind given waiting between is hour it and it, to stealing waiting them these about the readout a and the you be see for a ethical Gap] single for have, do a don't compiling given dramatic for possible. [Audio Every their from that horrible more to and for for EMBARK by [Audio is risk I And on therapy as kids would are that easier be objective This the dramatic time disease accelerated of we solitary scenarios, after while low and NGV not generating And soon Gap] frankly, have get therapy the filing overly it So all for take but approval. Waiting the it -- its clearly then this now. BLA even to those very in children the much every muscle. year. would single the to Sarepta is is the us is line issue, two not EMBARK fact. day, not a submit as we this data a BLA then may

to BLA get the versus ability at file least approval can an that a approval will population. between basis, the probably will have of be So if for file been a be thousands children of our had EMBARK muscle year shape will an be would as what readout we accelerated that a that otherwise have now get would therapy there this traditional and that on we BLA accelerated after approved ambulant gap -- result a if lost

the clear FDA accelerated number want of you might this very a fall. that a people suggesting BLA, we is So meetings to which -- again, very us the be there's or for I -- fly sort It in-depth to but with that no. conviction that your I think significant a approval much question. be it a was almost Thank of to say submit of will an significant review, doing gave result

Operator

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

open. is Your line

Tazeen Ahmad

had I how as wise on up forward? compare PPMO, expenses to expenses question. a not update, Hi, a commercial Ian, can separately, we you in in can Good path this should guys. afternoon. data And meaningful in year? But when you XXXX of either XQ. expect sense my next give to ramp Thanks then said us uptick could expect supply, you for on ask or Thanks. the taking

Ian Estepan

Yes.

uptick seen Just OpEx. expenses, the on in you’ve

flat of be a burn net the and relatively XXXX. of from our because it growth to perspective, However, XXXX revenue expect actually for I cash

Doug Ingram

And on comment I'll turn PPMO the the want might interactions to over question to the PPMO. who with Louise,

Louise Rodino-Klapac

with information waiting Yes, and obviously feedback. FDA PPMO, for their we’ve to submitted

we’ll as that. as have soon So we update

Operator

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

line Your open. is

Salveen Richter

a a as if how And approval process. population, you younger progressing our in patients faster discussions you. questions approval? congratulations wondering path taking willing were label to Thank of potential and for Thanks divide would on accelerated this be Europe Hi. We the accept in for to be part feedback. the the are may

Doug Ingram

Louise, to that do call? take you want That question?

Louise Rodino-Klapac

could The question, first of the part you...

Doug Ingram

VIP -- a think question I the think a is it's and split, on it's I'd that. I to approximately say it's -- XX-XX I think ambulatory non-ambulatory

versus So model that Approval you if we full about think the Accelerated approval. the

Louise Rodino-Klapac

are well. that supportive are trial, the are well. That's and are European Roche, And discussions with ex-US XXX with the health included, driving going certainly, as patients the global US correct. And the European then partner, discussions obviously, conversations proceeding well development authorities development of this and trial our in in our

Operator

with from next our line of coming the And you. UBS. Bristow question Thank Colin

open. is line Your

Colin Bristow

congrats afternoon, on good and Hey, the big a progress.

side. So a my few from

on titering of the consistency you've this? give was sort data, On around for pathway, versus issues regarding with the accelerated product gets And Part what Regarding us in sort teasing color what any with can the discussions this something X? a of the been us expression function you FDA is discussed or while. comfortable

it, Now the letter? you see you. Thank the FDA just signatory expect And on should seen has letter is to FDA, we it. that finally, from got when then the who

Doug Ingram

Yeah.

do that the when the and a with being method titering was issue our were Study at give partner, method super this batches them used They number, that method target was accurate dose. have coil resulted titering with we first clinical related more XX% But Hospital. X that with to in looked related occurred to XXX, by of anymore. exist material the to saw doesn't titering that of that we That previously the Part that Nationwide less Part Children's than all, to X, didn't the to like relate I the with of everything X but Colin. respect respect to to them consistency, Part had So

we So process all commercial speaking, And method in to just manufacturing then itself, move already before is that a was more even very precise. regards, be titering to linear generally really commercial our our had far we clear, tighter. developed much process

we process. the consistency So about feel good of the very

additional of at did expression speaking, data, of generally or do we CK I We're biomarker everything shed sort to provide data, really blows blow agency. all preclinical and want updates, any the to data not data, the data, provide expression by the together. agency function going So including in functional

this from is step I to BLA this get in filed, us successful, this as possible. many And community, then finally, Head to Peter who patient Marks we [ph]. for respect the think this the relates therapy as would And patients reviewed. received get on to came -- BLA benefit if as the it of get therapy letter signatory next that obvious with to the the Dr.

Operator

from Our of next the Ritu line question Cowen. coming with Baral

Your line is open.

Ritu Baral

generate every therapy potential much Can the And of how flip approval? that inventory is? the on number guys taking by for administer on question. the a centers want is, are have on, time much the administration I hand, therapy. attended. that comment And second. how capacity working you and the Thanks gene made it's intend something talk like of progress centers you afternoon, what Good how on for? single to the to have the world you you of move have have I know you good guys. long to that qualified to Can for that's muscle side inventory you to been

Doug Ingram

Yes.

question launch to ensure place delay. of to turn our be that good delaying on do inventory But capacity centers launching therapy very about over Gilmore to to With then, We sufficient therapy life we and assuming can of intend by is said, to our have Gilmore, I on without that this on are therapy serve where of we therapy. the alone in a place centers interaction. launch we the Head want generally, fully we build that. the can Interaction, -- be you a a touch the in that do that to community our question have our so robust we'll question So inventory, amount couple perhaps and going can I'm before on to gene of we'll years to shelf therapy, O'Neill. anticipation this the Customer of without And a excellence

Gilmore O'Neill

working And for neuromuscular, Ritu. you identified for as have prior critical on Thanks been it question, noticed and this seen the we've years. the Yes. as success a literally have at and factor

this trained for launches on have ready of having as We in seen and sites a terms to rate one. rider go gene prior therapy day

allow And so, to ready our sites enough aim for manner. have go allow eligible all a be in day to timely will patients to one, to to be treated

mentioned, Doug centers specialized externally, more eligible of are up the that has the than because So to -- these or do to a will highly little centers XX% population. the XX XX% is of is bit previously treat higher and as than sites neuromuscular target total north XX in

many So We Zolgensma. that works of who with key centers excellence, already for of dosing target are some us. can

you So said, the on ready them day and is we're to as go one. plan progress have to making

Doug Ingram

to serve course, our SRP-XXXX the with quarter patients, also gives therapy, we that of again, to recent tight have lot can the able and get XX%, just support people optimal about to very have things. the energy goal. timeline. I will of to hopefully community we use we is in that that hope shown Duchenne results a least what prior is work and an work therapy say of quarter real with confidence which serve want a therapies. growth over be that said, we'll the means appropriate approved, I with would when community approval, of also, we're accelerated into we With and centers the we centers we do, seen so two robustly is results an looking But it that to over get given The then last fully enormous approach of that the Certainly, have in community. additional with that amount our with at six most community years, do we've which,

Operator

coming of next Stanley. our question Harrison Morgan the line with Matthew And from

open. is line Your

Max Skor

guess, for can how question This EMBARK questions. our taking you the on ENVISION, this you Thank Harrison. in managing to III I Matthew variability extend Hi trial? Max but Phase are Skor is do natural the And you think below, we've Thank we've lower can to X much of studies. the with in standard most you X very seen seen let's deviation history you say, the points

Doug Ingram

you Louise, the to EMBARK? want that have on we Sure. control do touch in

Louise Rodino-Klapac

question. Sure. that Thanks for

X, For X or deal of what which a would I applied from great Study we to have EMBARK. we XXX, say or learnings

our inclusion/exclusion of so those And included criteria.

our for the given is was -- things more example, less it well to rise XXX Having XXX said of time results, the One than recent of a X homogeneous seconds, did extremely of ensure that population. trial. even outcome we gives to population that, powered. study in terms the Study the in confidence And

certainly have we our learnings. So applied

In addition, inclusion/exclusion in the Doug the the to titering to spoke consistency as criteria. our addition learnings earlier, that of put into we

Operator

Thank our you, one for next moment question.

with Our next question coming from the of line Debjit Chattopadhyay Guggenheim.

Your line is open.

Debjit Chattopadhyay

for you question. taking my Thank afternoon. good Hi.

the Just biomarker broad BLA like FDA very submission would population. has feels BLA? with three the shift need complement month there to a proceed And provide therapy a how from at with Thank data any you been to what you. the in gene a EMBARK use for review study the fundamental to priority

Doug Ingram

be you, accelerated think therapy. is of to filing. that this not result you data think have. approval approval justifies therapy, the gene this of much this for very I a that which is that therapy data will a in In set justification Thank I we will first that gene been gene Look always I for think vivo remind the committed is questions. I agent accelerated BLA

nearly XX worth have data. of patients We

which what And then translated years in term predicted, that. results. great -- the would over where preclinical We a great animal seen we therapy results endpoint, and how perform. have the seen of get protein, will the XXXX patients, which where you we disease-modifying anticipated you and we've We surrogate have into essentially that it and even shows data is longer see is

over degenerative expect very see grow that disease-modifying benefit a a as benefit And significant therapy significantly time. in first the in a had You from year. you then disease, you would

XXX. two-year a in had You And admit, cohort, kids, small, still will look then We I at a impressive. these five-point but pretty saw delta. very you mark the small

You to would see EMBARK, has now that EMBARK. see a they the finally, been agency we the -- need versus nearly right then And no XX-point stand history. on there from natural suggestion from delta additional information as

Operator

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

line open. Your is

Brian Skorney

everyone. question. my afternoon, good Hey for Thanks taking

discussions. to add around the the to Just FDA questions

increased Just want the thought a about after two-year the portion to larger portion results had of with see there if to and from period of order in at was the expanding placebo-controlled ESSENCE you discussed all have EXONDYS the EMBARK. of the definitive both know FDA study. size placebo-controlled I approval follow-up the

wondering, if pathway the a was doing an pressure this Just relieves early get bit AA discussion to approval? any EMBARK little for there the about of

Doug Ingram

actually, of -- reasons was we suggestion to the now, powered, of properly the we're think was Obviously, so that. has there wasn't. that. and FDA right ensure Yeah, size at we increased very with respect issue just we at occurred. discussions proposed we EMBARK initially as to the that well-powered. that we did -- I With didn't The ESSENCE, it ESSENSE not like No have it ESSENSE respect is we to one of had look and the that

-- XX% over XX% we're powering analysis. So our stronger over on based

the where up feel that's sure we we we EMBARK very wanted the that we highest success prioritize at the and I at various was we to end, potential trial. about would patients, looked as we've ended XXX good could look end to where make because that So that as is note, time in

Brian Skorney

Doug. Great. Thanks

Doug Ingram

you. Thank

Operator

for Hartaj our next question. And Singh. from moment next One our coming question

open. is line Your

Hartaj Singh

part. got be I over the you've Thank can speculation just AdCom, safety imagine of One manufacturing? where conversations Roche mentioned, last And focus few FDA they're lot have you of is you. not. structure of royalty you was really Great. with Could would sense the full poor be would with sales just remind related? what SRP-XXXX? just FDA can tiering wondering, it mean, efficacy just update. have to anything had databases? a comfortable months. knowing be secondly, gotten as various it a I you nice an the and be where could is, FDA was us on on? aspects well, on Thank or mean, just the questions. there you. I But two If then you you maybe again, for I And it just the ex-US I speculate it's Could

Doug Ingram

data the the look, Hartaj, I for we Ian in Look, Sure. wildly be royalty on I be filed. great of taking that I'll well, I where review speculating second. Obviously, turn thank wealth advance a approach in feel enormous that It all an AdCom. got question over issue. We've we're would of will positive to have we that say, would right mean, our justifies all about BLA. review. now. you, the are. a predicting goes We'll If this very we'll submit

get informed by FDA We that an the haven't been going AdCom. to we're

-- think that got enormous it is as get understatement been kind data issues the CMC we to normally we’ve an go even amount CMC certainly is for or going that, be We always opportunity, on It course, we clinical I feel frankly, we've our to very all work dosed, of prepare patients that the analysis the very solid that -- if take would core surprising pre-clinical us excited. that on like to on SRP-XXXX We're data, and ability another it. of AdCom, But -- be should had we issues, and of as again. all will I'm of protein said, mean all benefit, regard actually predict that our this on data just be powerful. efficacy we done safety. it's say and an safety highlight wouldn't of an the and to seriously. we've the safety to is AdCom the which an sure we the invite Obviously, the into there's at is And the AdCom which course, I issue. would

a we right that We safety have now. stack profile in very laudable

that how don't But that now, this good if where We wasn't So there and with justification to There expect we it. about explore yet. to good respect evidence again, have was AdCom. we would about feel would an the review I'm we all think and Approval of totality feel SRP-XXXX sure them of all issues. very we robust right and for know track the Accelerated of are those is very we I AdCom,

Dallan Murray

that's to from high-teens, Yes. manufacturing our based though, rate actually and our And the it as to then, yield actual sales determine on goes royalty relates surprisingly, arrangement, the the royalty US and exact Roche low-double-digits the of off based ex level.

Operator

And moment Thank next one our question. you. for

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

is open. Your line

Tim Lugo

to through kind supplemented prior of out that or approval, filings? the AdCom? Congratulations Is available progress be just the talk bit time that about quarter. non-ambulatory something could the in you something the on that of XXXX? all would the mentioned safety patients I to in data for that or around approval. available prior from the be could you guess Is expression a study be an could available that potential a could come when that And

Doug Ingram

is be accelerated be a in approval, therapy benefit at will this. been for of we're when, perspective, for And And important in who thank that we're successful with will they hope from States, it which very much much generating great accelerated but a of to it to that for is, this time We the therapy, have my can very over that making the you course, them. will patients. period is a there of the field Thank thousands would the have United important, to of opinion, making, to I of these question. see our motivator of particularly us you if patients point is, is important Yes. approval this primary least course, the -- pathway, importance entire the all all gene of

apologies, to, short value never answer capable or we consider it and expression and -- not -- any whether it out ENVISION and that other that's speculate I or been your discuss a of we FDA, discussed the to independently, of Study we question XXX, this safety with been the would with study whether be additional on and be received has data on we're That's discussed that it agency, So have to is, something can't a topic that is the now. there is then going right whether would providing have the internally.

non-ambulatory respect study possible, as that start of end with Our primary study working starting year, population get that. goal certainly on to want to soon the is now getting to right do that as and we

Operator

our coming Thank Blum with Needham. question next from you. And Gil

open. Your line is

Gil Blum

my Good for afternoon considering let my for you patients. congratulations, add And me taking and means especially questions. what this thank

kind therapy a that you patients Any to So going between time, Thank to population. of in be both you switch and down go another? patient you. might facing and go are one maybe sales PMO the see or XXXX, same you populations? two think Do might therapy about thoughts gene at world similar dynamics on the has these

Doug Ingram

Yes.

there's a lot lot just therapy. launch we empirical some it this. So do models as this then a And will of we that be we -- have to to have of there's to with respect

assumes significant would population. to of to would for still with which the respect cannibalization. I available ambulant Our plan we initial approval, population. Obviously, have amount a say, the be non-ambulant the PMO the current

there And even there. therapy impact And reasons. we the of so, the received who a to for are have potentially for a planning shouldn't have continue beyond gene that, argument compelling host patients think it to very PMO is or any

gene benefit not only there in patients The which that protect United will therapy, States before, have is big gene might on PMO the to RNA. first who that a doing patients as very going a or now them And available to PMO importance, is either gene there getting in of we or coexistence approve one proposition world, gene is well right be but in not expression advance pre-PMO. literature may a if to in then, certainly perspective. on and a will those a mRNA benefits patients it there also then and And already the in a from around the therapy, of crucial them, not benefit the therapy, the enhance on that advance be the of support of actually the to to reason some two said fact, therapies would, I technology who work is therapy that, of that it are is preclinical evidence therapy or gene

value particular having both think there I nodalities at is So patients Sarepta into both of these a the coexist. of

relatively significant our long if cannibalization, current over seeing and and or of is actually amount going while see if that the out we're the conservative these this So to to play modeling the case have is therapies coexist. run

Operator

coming Thank SVB next Schwartz with from question the you. Securities. Joseph And our of line

Your line open. is

Joseph Schwartz

there EXONDYS the some not that to surface the there of process. which significant provide degree company as the fact Accelerated Thank the and then. opinion similarities when advance regulatory wishes unified are buy-in for in approval to. reason is realize champion into but all wondering the now opinion there the agency be or now, FDA the at not insight was Approval? In at exon you can I at were you. any their in for to words, differences with need can if XX SRP-XXXX today, in back on when the limelight? was the at for EXONDYS if split those was with strong And the some least us were is pushing today other file through or weighing you how any you that a to broad this to I point there the FDA recent was into given best guidance on for agency approved, And versus as you at

Doug Ingram

I launch not that. thoughts approval after of but the approval couple too long that EXONDYS, occurred at A occurred. wasn’t here did of on I and come, the One,

AMONDYS historical Obviously, and be of I the -- this respect personal I will comments significantly There my not [indiscernible]. VYONDYS experience. have to with the of will wasn't different. record and own experienced I’ve my XXXX based on to be here today. some So with and experience

clear. very we're So

want I say all, States I First the and, clear, amount AMONDYS of anything in before for be else, some United and overseas all enormous an of doing well. let's good – to EXONDYS are first as to extent, very be patients of clear.

data presented that world I'll make real across some that's Muscle, basically exactly to World but EXONDYS published, benefit. to every out and going coming have of be then We point, be at

a to the data going on robust of from a right? chart. course, and approved XX% first small is fibers, we -- studies. built lot significant has with that or patients making unequivocal approval, really upwards dystrophin was the amount approval of well. that reserve first And of dystrophin. the or benefit benefiting off are XX p-values With them. multiple one XX the I is There's making tend EXONDYS. EXONDYS talking certainly of are differences accelerated my So of activity level patients the this this decision it the over benefit the The data on is is of positive on patients. be from patients XX% the results XXXX amounts, underlying making We're unequivocal as safety We're not patients. They tremendous The making prior a this. them, are X.XXXX to concordant. honest on there every between minds all of more laudable that We're approved was perspective, is amount XX the my was which, in significantly. in benefit. the supports hunting protein from in functional said, right biological have Western to are it and place, well, of blot. and EXONDYS We're and as the think my functional dystrophin perspective, to We're not

you seen enormously. up enhanced, synergies, should they our If light fluorescence of one and know not you've they're ever

that here make. do. whole. of what going is – our to this a is going amount that, there on as So the with the is the that, goal this that's science confident be not the I'm that is team to going And is exactly very expression. about a then thing And is typical other we're win division to different approach And to

in forging a that our advice I And we finally, Accelerated science-based received with work advance will relationship that, of positive through an I good would the that input to the that very the in respect last and years BLA. we've persist and this received to conviction respectable decision the and think pathway. five-plus note submit over done we've with the extraordinarily Approval agency,

broad had cross have discussions this on We topic. FDA

have We super leadership.

Inside opposite had of We Sedar, have drugs. We division. neuro the the of Sedar. had we OTAT. new We had the

a then all going may don't we're review. doing a transformative advance patients a hopefully, discussions pre-approve a review, patients all be filing, a goes news at filing So is wouldn't then deflation. benefited of approve result But BLA. than a not a lot or that we that our is the have it if I as the respect approval. to well. BLA, have in all have of we'll they want had approach certainly of we'll and of this said, and And the with be that with a would be, well, to full that one They to that we've is, I different outcome end benefited to full in of very therapy. have for of robust say the They going regard right a to then goes lot what and yes thing, it we question that day. good the have do a suggest right would an believe have of But lot and did of BLA day

Ian Estepan

We practically of just just is fully EMBARK Yeah. And today reiterate just the rates, kind I'll other maybe had add that announced it. on essentially enrolled. but I'll

wait have year. we to would a So essentially, about

an to were certainly unless good have commit getting that review, to be wouldn't obviously, incentive a for get to I going to we're patients freedom. that successful were there we market no chance unless going and for remains other us wasn't there's to thought agency. across So dollars we thought a than, we the dollars through ultimate in Doug, the broad if that Obviously, And in we market these there we still not for but the to we review this challenging. good to therapies. we really point was us And forward point, feel to not going Doug’s about were commit patients go support didn't move fair going this --

Operator

And next coming line BTIG. our question the from you. Thank Yun Zhong of with

Your line is open.

Yun Zhong

very questions. you the Great. for taking much Thank

question your I a So question, -- on to on with confirm I discussion by the is required was the study talked not the required agency? potential accelerated approval wanted study a the And going follow-up I study to of answers to FDA? time midyear your again around part secondly, by based patient, previous line. XXXX. new believe that non-ambulatory And FDA the the guess, confirmatory about the just the you during be

Phase top with the before data happens depending But EMBARK up So what the during you Phase that missed X discussion the Has all room EMBARK data the of that come line like? at FDA? be is be X should flexibility study look study. get possibility from Would how endpoint. primary your the there any on

Doug Ingram

we've question, So, XXX, is obviously, time. on decision some the ENVISION first -- and and or quite that the our been our planning for

had about over We're XX% on EMBARK haven't EMBARK. its readout the but approval would decision. and about We're confident in if in not yes, well, accelerated With the you EMBARK. a very of come did with all we're regard that advance And with well the detailed the hand, discussions respect powered. other be agency, our to very goes timing, powered. So up that results,

powered only if we about Part over apply the So and the X, that. was conviction even increased now readout We more it. and before were Study feel you have saw we on we very powering XX% good frankly, XXX

I the FDA where in sure good correct, way, Is think non-ambulatory that. ambulatory, So about want -- there's the a to to make Dallan? more, I is I By we feel I misstatement. update with think, that XX-XX very having I want a collect said are we XX-XX

Dallan Murray

Closer estimated Yes. the in XX-XX prevalent to population.

Doug Ingram

for Apologies that extremely.

Operator

Thank line Shu coming is our Bernberg. of with you. from next the And question Zhiqiang

open. Your is line

Zhiqiang Shu

add to well. congrats my want I the to as progress Great.

a Just clarification few questions.

the wanted uptick with ask, on secondly, FDA, your around on on guess, SG&A to I First, based accelerated has explicitly the FDA -- communication I to encourage based in you expense second file approval? quarter. And Ian, the

expenses the Should was primarily Thank you. substantially? by we down driven stock should QX and non-cash the that mentioned -- in You options. assume come QX

Doug Ingram

Yeah.

our the So communication on I thanks. the we FDA agency, communication, bytes on based that from the would enormous. is have on what say Look,

approval about And We non-cash that then concept for on to the conviction good BLA we're very of feel with approach and the items. you submitting about have that perhaps talk Ian, taking. accelerated significant the want we an

Ian Estepan

Thanks. Yeah.

expense about expenses coming So as about stock-based be through But some probably most this to is There $XX significantly the spread less. biggest so is to quarters quarter. year is compensation going out that see will here the it much over important yes, you're -- or thing the this next charge. -- the smaller a over relates million think this to non-cash remember, that

it impact net cash our to burn. at So has overall all no

Operator

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

open. is line Your

Danielle Brill

guys. Hi, afternoon. Thanks the Good ones. questions. quick for Two

new they'll you're Given to then the at And accelerated will here? to reading coverage, you you've EMBARK that you. you I'm precedent point, it your that more their this payers generated much drag are thinking set follow proximity or Is of And buy-in? pursuing Thank appreciate approval curious that but the data anticipated what implement hear on front? to you the do Accelerated full possible expecting with Approval? anticipate competitors have out, suit feet a I

Doug Ingram

your you very Thank much for Yeah. question.

unique that and basis supports XX filing data Accelerated we're new -- don't is And and the is our that of -- So for the that think that -- patients to the to on therapy have conviction our years and the work that Approval. the we that to second setting of I profile, precedent. for question safety that have we BLA the submit wealth three the data a on unique And preclinical ability around SRP-XXXX. therapies is therapy. first, across XX-plus and our on our which It we're

But the very is that precedent appropriate. decision is FDA unique and at frankly, unique expression the to consistent deliver stands where in an to very from want of therapy this with great our the is patients SRP-XXXX about front we think the that SRP-XXXX which Approval which I the and can Accelerated I to the beneficial least, safely data regulations levels. is statutes to clear unequivocally on and in and us, at perspective protein, be

payers, As six challenging with relationship we've forged very approval positive over can would of years, I nearly the payers it payers be, got relates we since as -- EXONDYS. as a say the to

onetime in I XX-something results so to compounded growth. positive you every rate getting therapy, and or and how -- getting easy. every therapy that I growth patients want day support we this not and to therapy same used the see don't patients on that team therapy. states opportunity Dallan year. quarter to far knows get payers, how day to and you appropriately therapies community, XX% therapy execute, say company and getting CAGR stay therapy is, which the delivered take any gene on the on The annual to this it patients, having -- it's That quarter-over-quarter last will over with work for to annual And fight it knows But to therapy and our his community respect therapy across over even are make quarter-over-quarter that approved to XX% ensure growth XX had, given knows on quarters therapies. the quarters, work staying and with of seem respect chronic to only so a a with three to of a therapy that hopefully, get over how to

team to muscular than lot work very confident you every be dystrophy Duchenne is to there no day for of but with done, that So, there's Certainly, team I'm no but that a respect better better are have team. fighting the patients.

into confident very I'm so, benefit approval ability an to therapeutic patients. our And about translate and

Operator

Thank you.

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

Your line open. is

Kristen Kluska

a Congrats everyone. very exciting Hi, the quarter for on company.

have asked, topics a here most So, little of my so bit in gene been change therapy. questions I'll

the So initial envision discussions you And how might this has stage? some with for figuring the you'd based here course, Limb-girdle thoughts before data at of see you want off agency, of to trial understand path with programs? SRP-XXXX supportive designs some the path and forward, out took your that I the but changed this the any

Unidentified Company Participant

Louise [indiscernible].

Louise Rodino-Klapac

that All right. Thank question. you for

Limb-girdle. on -- there's we in work of going So,, kind

XX all we've have cases to working and that of we've program Certainly, the the the SRP-XXXX the been support activities including and LGMD know, collected is five being method. of So CMC-related of with promoter delivery as data hard the vector Limp-girdle all assays you on supportive many the same in specific we the and programs, program.

both today our And generate so. well. shared has and to expression the data Limb-girdle also we promising CE have as we've that which that support data we in of, with results very functional been

where of of collective can. apply can set taking those to we're next but and limb obvious trials and pathway data, in XXXX of that we're Limb-girdle. a so all from smaller our And apply, lot get and learnings our of we we swiftly obviously These there's to are populations, to moving that

So thanks the for question.

Operator

from line is line our coming Evercore. And Gavin Clark-Gartner last question open. you. Thank the of with The

GavinClark-Gartner

hey. timeline the give later And Thanks event? wanted just Yes, granularity the work? around more ask XXXX to the it more CMC the for a like if status on I this is engaging the taking for or Thanks. with maybe you could year FDA, question. any thoughts saying of

Doug Ingram

limb-girdle, On particular? in

GavinClark-Gartner

Yes. limb-girdle. Yes, for

Doug Ingram

Yes.

behind empirical been the look of in bid we a the has are be lot out. result get amount on to with assays lot of on a as SRP-XXXX. We done. particular work many trying of capsid, We've a just promoter same hard it time to we for the that we're -- capitalize know process a CMC But So to learned can of takes and we limb-girdle are There's respect an of Limb-girdle. programs. working enormous that some it's same it these to our our

like a Louise working these So, any hard if to try we're do done think of mad, but this get working more you want this to year. in touch any we're detail, lot issues on of to I

Louise Rodino-Klapac

the but assays, in developed of learned have the Limb-girdle each that certainly subtypes, have specific to be each we there one. still add, of for for would development are assays I the Yes.

And so those -- such it just Doug to, as alluded takes time.

working it. we're that's on So

Doug Ingram

assays. just know just studies And on and do understand step that and working things looking good priority really go we're it's these event assay no But for then in to only is there's potentially this. The time to sometimes on There's news any us. high could we so support you exactly we that. is some have the right that we're now. significant of rely takes I given upon -- what development fast SRP-XXXX. enormous we like that empirical frustrating and work and that's need of This when it of a one -- that Other now what preclinical confirmation

Operator

Thank you. for questions any this turn I'm would to Mr. further And remarks. I time. like over back to at showing call now the closing Ingram no

Doug Ingram

Thank much, All you time your for very thanks evening. right. and this

then, Both view hard away to come how he like hopefully work on from starts. therapies me the want -- excited progress got our course, of respect the to sight I are. the the to let lose of shows of with to conviction it all where the enormously least of with it hard us. that in in understanding respect I’m you won’t fact our with excited and at a great lot around made we are very, much an in doing in with work my to performance this we've and We don't – it afford got I think EMBARK. current of -- trial, always do that are BLA our lose Dallan sight of we're really our confirmatory very respect with wouldn't with a respect But we've to and current job, Certainly, our we shows work performance, SRP-XXXX. And we We front approved here seems of guidance. serving I call, ourselves But and about don't starts. is community now our and the pat back.

BLA get We submitted. to our have

get process. through have We review to the

are with made challenging ready patients readiness like amount briefly on And I will and of challenging seems important human science over an it will particular, give of made really But us advances and vision outsized hope like We I'll this in optimism talent Sarepta returns and The strong as know and have science our and endures distant genetic but this will because do we'll comment The we go to tough translate with enormous manufacturing, build and revolutionary late-stage and revenue, you unbelievably with replaced and Thank you a Sarepta will that It literally condition And we're tune final medicine, biotech remind in say and that others may to all what to is to and to lovely has this sort society the years. that is not that we And those in have is make you a this least a, lately last that, been And would time. enormous up of very have all taken support very update to and this I for I future, to have evening. to same to time of, lead the there, importantly, like waiting improve confident sometimes be do launch thing strong organization view, that but I thing best-in-class what a these optimism. your Companies call therapies to genetic we us but year lives, that's execute we to the get biotechnology, companies would time, the biotechnology opinion, it as my soon. preferentially [indiscernible] course, this our a invest things I'd get again, on like very to optimism. that and to understand are it some done. also year. scientific inventory a And rare today, what return. and pessimism through who poised Sarepta, how our intend for benefit. and with tenacity together, all you in -- that site and those medicine. for be, not that say, a an therapy. launch that. pipeline it have the ability medicine professionals, leader of has our for will, for we genetic optimism disease, with at those that return when scientists risk but the Companies has and ask execution from sometimes been a on our should to seems get I with why, returns who improve results blinkered my investor. our be strong sheet, know like balance in

Operator

today. participation. that gentlemen, the Thank conclude your for for conference you does and Ladies

You disconnect. may now

We use cookies on this site to provide a more responsive and personalized service. Continuing to browse, clicking I Agree, or closing this banner indicates agreement. See our Cookie Policy for more information.