SRPT - Earnings call transcript Q4 2020 | Sarepta Therapeutics, Inc.

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

Operator

Good afternoon, ladies and gentlemen, and welcome to the Sarepta Therapeutics Fourth Quarter and Full Year 2020 Earnings Conference Call. At this time all participants' lines are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions] As a reminder, today's program is being recorded. At this time, I'll turn the call over to Mary Jenkins, Senior Manager, Investor Relations. ahead. go Please

Mary Jenkins

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

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

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

Doug Ingram

community course, you and afternoon, Good and, with investor year Duchenne XXXX pleased afternoon therapy, products, work quarter that serving we call. our multi-platform full everyone for DMD our platform. of that the in Mary. our fourth have to advancement including progress you, I portfolio, with and made we share of our am this are the Sarepta conference you the Therapeutics performance joining the for thank across gene SRP-XXXX, RNA doing approved including Thank

time, Dallan under matched to At Chief I that rare to our none, will elevated of milestones a have therapeutics. same franchise We efforts have nuanced by research therapy, in RNA Officer well our we XXXX, opportunities you our what goals. a of announced committed And programs mission Rodino-Klapac and remind I renowned and his Scientific all is the community. enormous we all of gene of efforts I his was very the be the most CFO. maximize front of the pleased I'm number our our believe our Ian Dr. in important taking To Rodino-Klapac XXXX. to knowledge only company in you and Estepan across and sure genetic to our Ian history we'll in leader team commercial am toward advance to Dr. RNA appreciate our and Commercial an and editing research is in charge us. organizing disease, with know in Louise December also medicine to strongest and passion leader Murray, Sarepta's named commitment of Chief her and DMD the Officer gene and focused stewardship, I ensure elevated expertise second DMD that

beyond. Compliance formerly Finally, to then judgment our Head have elected of many be us years, pleased Legal and Officer over well will and with advance as and his ethics worked and to Chief known Ryan General serve our to and we have Brown, I that commitment I for our announce Function intellect, I and XXXX Counsel. and Interim am Ryan Compliance compliance permanent

medical performance, concert new and our us just launching our patients VYONDYS affairs for our through and worked patient the Nevertheless, as a XX, challenging community team challenges, to with a XXXX and without of to therapy. was Moving keep therapy not all to for the their year supply was obstacles. pandemic the its rose safe chain ensure to commercial in and access struck

So product same the year. for a million we the in prior achieved increase that's fourth quarter $XXX.X the from of XXXX, quarter revenue, XX.X%

XX% increase we For million prior revenue product year. that's a achieved year, the $XXX.X over full and of the nearly

recall guidance also for between XXXX will $XXX in that million range our a million. is You $XXX to

of price to increase all since our remind have RNA of on you we XXXX. have we parity strong not EXONDYS And XX the at we of our back any approval So therapies priced and in growth. continued a taken therapies

comes our community. to our patient ability directly growth serve So from our

Now to treatment Muscular moving a treatment X% will the AMONDYS exon XX. approved Administration XX the amenable platform, Food Duchenne RNA the That's as to RNA name seen the have community. a another those February that approved for bring DMD XX, you casimersen. patients skipping is third will who our have to Drug therapy and Dystrophy mutation for of on our of U.S. option generic AMONDYS XX

the the we is therapies for of three increase our dose SRP-XXXX, the together announced build constructs preliminary In the study a investigating the of Exon skipping use XX% ultimately with all mg-per-kg should XXXX, with can penetration, fourth treat improving profoundly for cell cohorts be dystrophin next encouraging PPMO outcomes. RNA And conjugated of Our our improving all than our XX peptide of we RNA in on our quarter and treat the with goal PMO exposure, can generation PPMO patients third peptide and The PMO that able a nearly or and of That's production technology. DMD. to based XX to therapy mg-per-kg but DMD. multi-ascending all proprietary more technology, to short. results goal

second quarter mg-per-kg this of the track We are in next results at our announce cohort to on from year. XX

part study XXX you our announced results platform, to in gene of of of SRP-XXXX January early as year. Moving therapy the this one know, we

While of secondary significance concept proof on our insight achieve our and we did not strong we functional endpoint, on did invaluable statistical primary gain endpoints.

we are significance in filing following. impact commitment statistical regulatory endpoint primary or But the required has indeed, adoption quite does functional our a strategy. clear confidence program our on contrary, not the in So on change it based in consider the the

endpoint. we achieve important While intensity primary reduction, biomarkers fibers, we on our creatine our significance statistical did blot, statistical achieved kinase protein positive functional Western not and significance

the However, with p value placebo X.XXX literally was enormously when group different significance far In it of the different, those the one subgroup populations were profoundly characteristics and that becomes statistical reason the we at age this pre-specified impossible baseline, apparent. results more analyses, the the year old a for practically at looks baseline severe so groups, group uninterpretable. treated immediately reading compare than making XX

properly However, characteristics in the olds, the four were matched. to baseline five year

every NSAA you history therapy statistical the program. And appropriately statistically we in benefit to we DMD, in a placebo. fact, therapy meaningful sort pre-specified controlled development, actually and baselines identical. first in the patients of significant of In and shown of significance populations that benefit placebo better on in they functional were in has to our so nearly on of matched, this believe strongly our were we measure continue in a is, be when DMD this sure, double-blinded clinically be This time DMD the and the over analysis, trial clinically achieved were comparing similar therapy meaningful confident

will for and Second, level any we we micro trial, with patient more which to other our commence how than organization year. have ranges, next now depth, our and these age protocol in we refine strategy insight use to this the dystrophin into intend insight traject and they this

insight apply next trial year much We to in lot do greater will are not construct, and of a competitive we that can we give edge. our us our only to advance SRP-XXXX. to confident this probability that success confident There's a are unique a

can recall, the we commence dosing JPMorgan balance five study study in initial will what study, four commenced in year number patients That's seven six that to enrollment at of five of we and representative year, enrolled all to have soon. conference the updated patients additional you to To we've and commercially been so add our or as we'll First, dosed. that We IND, to we that our that last XX trial announced evaluate material. our call XXX. our better ages four

evaluation quarter just the you're that delay our of out However, planned. report the this of first will second results of and as the not so clear, patients in year XX

protocol trial, our gain complete of we update Next, meet SRP-XXXX we division that with next one to discuss this and commence the the that for will we aim will We trial protocol. alignment our or study on to two, year. evaluation will

XXXX. of middle enrollment commence end trial with to of the that the is complete goal Our this by year around

around provide study amount and the important that And United is before Part successful, insightful year. if going full approval moment. of that is results of from extremely with performance the occur to the blinded finally, world. the all be will study end it patient And remains study enormous SRP-XXXX. the XXX an assumption will of last on This will an baseline us be II and this in it's recall of that to our for the last to the information visit Our and this support States important

for concept to of we patients against the XX run that most natural patients greatly equally week substantially dosed week II more of dose insight XXX. of proof will have had Already before XX therapy than constructs. we the Part therapy on XX in at XX crossover, see enrollment, expand insight other those will with those at And will who were important, We compare and they weeks in any impact trajectory results and our to that program provided and readout patients have patients of judge have predictions. were period a study history will

year, is Let me to dose cohorts. our on positive high announced proof both results beta our that the those SRP-XXXX in XE. protein dose of in in who therapy, our limb-girdle comment for type dystrophy patients our very low and briefly muscular gene sarcoglycan, we concept SRP-XXXX study in restore Last now intended have

the be and We in XXXX will updating conference March. clinical results those at MDA scientific

the is pivotal with commence year this the FDA trial on this and path development for goal year. a to to Our SRP-XXXX align

will limb-girdle for XD in the toxicology limb-girdle of type GMP SRP-XXXX a Children's Those we trial. vector Nationwide inform material [ph]. therapy limb-girdle material that We use we will for will patients are of will type complete type SRP-XXXX, limb-girdle and And dual our other studies in discussions for this using strategy schedule once for concept XV from our Hospital next treatment SRP-XXXX and study And that [dysferlin] XC commence missing we proof the respectively. for development for our meeting year, are those with agency released programs. have

strong summary, In I the team a our programs, and reminded by importance RNA urgency these are to by of to mission advancement beginning as gene the we be But XX, Day revenue of doing therapy, recognized we of a focus ambitious, rare of one continue obstacles I the ambitious editing in will am have have year, razor gene our a that. that overcome at with we Disease to Rodino-Klapac. February priorities. on every as top Beyond with every when day. our passionate our beginning and of there Rare our and which this is and a expert that to under with and our research And of obstacles of $X.X was if what cash address programs focus and forward before yesterday, of ruthless And reinvest responsibility, choice sense to certainty be we it is worsen. The disease as move this hour harm they are serve we we we are the billion augmented of intervene. moving takes know Dr. programs year. serious just have about but to shown there patients was little

Estepan talent and up work have mission Ian focused execute. an will of the that motivates who let And hard be Ian. we us with to We to will patients the update all day And the that, the of call that turn over the problems to me and to who effort. financials, to provide each get stay the have resources the on to strategy, a beneficiaries solve on

Ian Estepan

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

We conjunction the AMONDYS that are for our recent disease with pleased review have voucher million. in to approval, rare XX $XXX announce sold or pediatric PRV priority we

have conditions close satisfied. been the to all after transaction quarter We the expect next in regulatory

Now products. moving XX to to higher period same increase $XXX.X VYONDYS million, fourth XX the from $XXX.X million our XXXX. for of net The EXONDYS our product was revenue quarter of for compared XXXX the reflects and for demand primarily products

the respectively, a recorded partially a in for products. to Pharmaceuticals cost demand fourth On the increasing and $XXX recognized is is In and $XX.X is be the of the $XXX.X in of or million, Roche XXXX for quarter loss expenses of of Doug were to On a million million increase mentioned milestones guidance basic for XXXX up XXXX, XX due XXXX increase basis, $X.XX University quarter. recorded BioMarin of $X.XX $XXX.X for the GAAP XXXX, and XXXX, decrease of Western share $XXX.X loss XX, a our respectively. net will franchise of in EXONDYS of for we of we share period of million. under sales fourth other Going million. AMONDYS ramp quarter quarter $XX.X basic arrangement Australia, primarily diluted million $XXX.X increase VYONDYS fourth million $XX.X of an quarter the driven a period or The franchise collaboration reimbursable XXXX. decrease $XXX We the Roche. The net to like our to a and diluted decrease and On compared fourth million revenue, non-GAAP we XXXX, to to by cost $XXX.X share the to XXXX forward I'd fourth in in basis, you approximately same quarter is or the million in AMONDYS which related $XX.X share clause due and we the of as programs. non-GAAP to compared expenses gene the $XX.X and per as fourth And royalty of non-GAAP breaking XXXX of in $XXX.X sales diluted our XX. a quarter which of $X.XX to of year-over-year primarily expenses million reflects XXXX. revenues relates earlier, of expenses RNA compared continuing million and December in payments quarter upfront our million. programs. of the R&D RNA $X.XX $XX.X for same million quarter inclusive This basis, R&D out ended our we in GAAP a offset and reported in to The XX in $XXX.X R&D which $XX.X the XXXX, our million of the manufacturing continuing for per XXXX to the reported quarter year-over-year non-GAAP same of million by collaboration XXXX, with an compared increase our gene in co-development fourth was and agreement In XXXX for remind net loss primarily totaled fourth $XXX.X up million therapy $XX.X million growth ramp a of in and million expenses therapy a per including XX,

on fourth a at basis, million $XX On partially million and of price. an Now basis, and XXXX a of million debt period for XXXX. the in a expenses quarter net as in other turning for $X.X On fourth to expense well of a and interest commercial approximately investment that, Dallan and $XX.X non-GAAP other for XX, period same an million We for of compared cash XXXX. $X.X income compared increase SG&A in And as XXXX were increases reflects and compensation was stock for I'll expenses discounts million. to on call XXXX quarter The fourth decrease in company's net portfolio. December expenses due increase investment decrease as headcount basis stock we the company's million $X.X expenses of on the interest the the $XX.X respectively, an $XX.X the of to we in activities. SG&A, approximately of due of recorded of The the GAAP to XXXX, same primarily investment the had XXXX in facilities, update a GAAP to cash, the amortization increase quarter the turn our by million million mix driven based of recorded for year-over-year million. investments Dallan? of over $X.X increase to the and $XX.X with equivalents

Dallan Murray

pandemic Muscular position you, by Sarepta Despite maintained in goals. on by Ian, everyone. challenges posed and Thank leadership stated the and COVID-XX Duchenne good Dystrophy to continuing execute our serve XXXX patients afternoon, its the

skipping Sarepta's D&D, were and increase and into case in last our chain roughly unrivaled with thrilled a XXwas excellence approval to jumped commitment week skeletal casimersen. supply to in Firstly, approval. in patients facets of organizations, The third statistically and XX, and XX, XX. was AMONDYS FDA X% including after community action of and which immediately who of production with treated managers, approval XXXX. February field exon this one RNA muscle to on medical patients on commercial urgency, with Based AMONDYS serving manufacturing amenable AMONDYS dystrophin day are medicine, our occurred our All launched support XX. exon skipping on observed on based we the significant expertise force,

and community. to by Our of patient Sarepta and EXONDYS XX. XX goal DMD, our of treat now DMD the leading leveraging medicines successfully and access XX% to other AMONDYS RNA facilitate VYONDYS roughly proven having serves is total, knowledge launched rapid XX In experience industry two

are of homes. XX of XX% or majority the we fact to stoppages provide large weekly of a been and delays pandemic. patients, XX VYONDYS receiving that uninterrupted EXONDYS roughly in their have few the on infusions VYONDYS there Due products as to result patients Moving and now to supply to XX EXONDYS continue both or a XX,

that encouraged demonstrated are during community payers willingness majority allow the this work continued to to with important access these to the shown have DMD We flexibility therapies have a and of for Any direct one impact care team. to-date a average, miss to on has result or as time. family either difficult impact pandemic of largely due been doses, doses two to on immediate

patients are provide our continue on goals of patients. minimal Our And competition. amenable and to on impact these adhere and the The XX with continues to-date pace launch to ongoing to to infusions determined monitor XX is exon Sarepta support execute weekly to patients. and any and continues to consistent VYONDYS from team their its determined support

minimize risk sending weekly transmitting equipment measures of when infusions In protective These deployed supplies addition, to of nurses homes. their their at ensure measures administer all the thoughtfully have patients help COVID-XX. to requested are we've to protected that they include personal who our

to continue safety quickly are efforts continue We that our supported surges our ready places pandemic and with any during to monitor and remains ensure feel safe modifications all infections. patients the and time. in react and experiencing especially will additional this Patient we top unusual those to will priority and assessing strategy,

to Now development I'll turn and Gilmore? the on research update call activities. for Gilmore an our over

Gilmore O’Neill

afternoon will shared I and in Doug the most in and you, my focus highlights progress therapy RNA good advancing this programs. made Dallan, advanced gene we've year's on already meeting Thank MDA our the our everyone. presentations Sarepta's upcoming from has portfolio. remarks today at March

this year's Let meeting. me with start MDA

and presentations data Two-Part and which The that and a from present micro-dystrophin March XX to Sarepta surprised which XX scientific You show program previously SRP-XXXX clinical podium study will the meeting annual include three two XXXX, plans COVID-XX to limb-girdle, SRP-XXXX-XXX continues program. symposia disrupt to our Type reported its virtual format our of functional two conference scientific as host dystrophy to march data host new podium at from year meetings know XX from muscular plus abstracts, I not long-term would will for MDA presentations. XE be which

will the boys state Duchenne year But Now, subgroup analysis specified study. and gains the XXXX stratified old Doug results plan, Part analysis were parts, confidence ongoing our from covered that two of design, second, NSAA can we treated with boys of I that to want emphasize SRP the pre- do randomization its I to four of SRP first, demonstrated that achieved because statistical five because XXXX-XXX that studies to and probability XXXX clinical physiological meaningful micro-dystrophin significance. clinically This and that of benefit, boys superior future of success substantially Moreover, from placebo optimize a thus of have with and increasing that means study. collected design and our humans SRP-XXXX-XXX this amount in and inform further the clinical statistical SRP confers the the functional large construct for we biological program. treated is the study data

about talk skipping their for our week's dystrophin boys XX, on Duchenne amenable delighted were of RNA of in Now, announce let who We approval exon portfolio. mutation carry the XX AMONDYS gene. last treatment me to

trial, Now, that AMONDYS remained know you will placebo to XX support designed be on VYONDYS the is a study pleased confirmatory which controlled track. ESSENCE to the XXand approvals

PMR XXXX. trial our in our goal emanate We our to XXs expect is approval science AMONDYS proprietary to with is data from Duchenne. continue the which third approval, PMO Notwithstanding excitement this from patients FDA advance our about for technology, to

to successfully but with out dystrophin a or have peptide Duchenne, on charged approach, understanding delivering the CPP addition leveraged deep and cell PPMO uptake. of to increase cells. next our technology, this as thus selectively into positively of this, PMO our truncated generation we and skip should elegant a protein. Based the the set backbone. penetrating the cellular creating reading we PPMO, dystrophin this to The We frame, called technology functional the sole engineer peptide To of the more of purpose exon do of which safely PMO restore PMO

presented compared XX% as three lower a weeks cumulative patients increased maximum approximately patients. an SRP-XXXX Part increases reported drive XX tried SRP-XXXX human many our XX This parts. remember that PMO patients. compared administered XX and that can last of will will In increase does dystrophin cohort at penetrating and we patients in or tolerated were exon and we higher of into group we skipping at five-fold of others exon association monthly an compared year, to dose weeks, our to safely cell in update tissue small and target dose dystrophy, been increase momentum determine have of the proof X, On by update is clinical XX we has the normal an at limiting the if IV of the tissue study had exposure. reminder, SRP-XXXX-XXX indeed as mg-per-kg to A increase and As as XX A, drug the greater kilogram, no XX we muscle dose evaluate XX, have exon dose exon six achieved expression. Duchenne production. with approaches peptides in cellular is two XX increase concept for ongoing PMO tolerability however, in at MTD cells, toxicities. X.Xx of feat, we December an XX% way that chemistry with conjugation MTD. Please our the dystrophin levels momentum, of higher muscular dystrophin Part SRP-XXXX fewer and and XX, enrolling to dose greater saw the cellular cohort more to levels that will observed These versus skipping important the Part study XX%, penetration momentum skipping and multi-ascending in mg-per-kg of see hampered of downstream in dystrophin Part drug higher exposures, and XXXX, XXXX-XXX at weeks. as or and X, cohort PROMOVI B to XX observed weeks dose that each quarter SRP-XXXX additional once cohort approximately XX our and and milligrams were is skipping in administered SRP-XXXX of expression mg-per-kg this With uptake by XX who Duchenne with XXx of four approximately to of complete per safety percent evaluate Part include A XX doses with by the

Part announcing have who XX cohort in next patients be and patients. XX expression an determined approximately year this we include XXXX. will an which of of will the QX Part A our momentum mg-per-kg we B MTD, data Once commence additional complete study, step of Our will

sites Finally, highly difficult importantly, therapies their thank have incredibly most our done to all I R&D patients, to with under much circumstances urgent and so effective by and to study deliver my the caused want to and people colleagues coordinators, maintain mission our who new, partners work rare the diseases. pandemic families,

hand I back would for the to Doug? call Doug Now, Q&A.

Doug Ingram

Gilmore. much very you Thank

Let's turn Q&A now, operator? over it to

Operator

the Instructions] comes Cantor. of line Young from [Operator Our from Alethia question first

Alethia Young

made lot of to know a talk your XXXX just about a I next you for But for general they interpret what's the it wanted studies confidence and little means the data bit DMD. how limb-girdle, think means been fine to I what this the how based about to that those on there what make technology? regulatory seen key with And you've are as also, you level then endpoints think have perhaps tune, with what successful? sure core are the FDA conversations

Doug Ingram

remind as that everyone build capsid, is I XE over the time, only not uses also increases would limb-girdle uses limb-girdle which program mean, we RNA that XXXX. and the first, we but we them, it have SRP-XXXX the way promoter -- our confidence in I the our same constructs Type only have XX and same

target patients patients are have We XX the well now that over treated dose. at

safety very have we're expression. good seeing we profile, so, very a And

some And it the our to amount and insight clinical around that. we'll strategy complete. come we're protocol increases when particular, this XXX, we've and regulatory which out SRP-XXXX we're so of is confidence an Study share to on only so With level we enormous shared, still of excited have of of respect which the XXXX working some as that's about relates in

end XXX informed to of this everybody probably around of study And goes in we're concept to around well understand that XXX that's kids nuance really protocol which somewhere to that commence of the work goal then once having be is And data. of next visit, of if year out year, course year the our the out, read all well protocol the so the a of study the some the by end upon and it our next and with and and XXX the is continue And the here we're to of dosed coming end all the by is all that our agency, of U.S. positive last we update the to trial the And this out middle the in would now, fully last current patient, build goal and proof that is read that. of on an goes if seeing then base year, we'll fully thereafter. world. to have first defined, approval obtain Study the meet of of assumption those shortly would with course, our then, to goal before

Operator

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

Brian Abrahams

Now of if inclusion to the that at in set, think any approach analyses, you've had might an statistical that you data baseline opportunity planned you had and take you've that willing been general or the January, normalize you'd maybe I be things learnings any looking for digest two to criteria, I may wondering share or and those to mentioned insights further maybe including adjustments was you've like the mono characteristics. design, XXX to more additional analyses.

Doug Ingram

we the success not in here want probability that and tune the way. that then place short have come that and designed provide can answer into nuanced we bought lot the help in very fully once an not we is is, detail. protocol that's greatly But yet do and So will out XXX there's next protocol. next only but the a of of We'll of where trial study. we amount the is insight that to convinced that a We're discuss additional insight increase a enormous yes, in agency the us of are it

potential we than Duchenne And in then, blinded study use one year, want significantly tentatively design, We blinded, we're muscular also deny, part the And to I'm placebo that the then, belief just anyone saw study is only part January a that's is, very patient to to remember, weeks. else for going we call micro-dystrophin more to five us we the but this our value in have do and the actually last over olds, look that well, a for in have I'm have saw, already we XXX. XXX, by that of visit, how a even kid primary what more of cohort, way, not at study point that this help XX were strong I were data two, study, transformative have dystrophy, what important statistical and not year. study. secondary next last going benefit XX that this to really meaningful of study saw the at to didn't be insight, of and the of we just regarding and endpoints study, the out the this going significance, that'll our hit was therapy. significance the at benefit construct in disappointed and year, insight everybody, kids back year When at what we compared, right, clinically confirms XXX would saw remember the XX we'll that's the remains endpoint, statistical end And end properly the of only a four in half in baselines that you

XX so two of this way will for we have the projecting kids, about by years of data have very the who next have of ways for can XX this a XX been be interesting something able we'll without And you'll we'll therapy. will we when then impact that's we unblinded look for have do and and therapeutic results there, of part And of there, other what that and in on weeks year, a worth think judge probably we what intervention, truly enormously of then follow year, a therapeutic at and of then really two the a to in is have going we get early about children insightful end to intervention intervention. they're therapeutic kids was. that we there, the or the some the which have And on very cohort top to interesting, run have trajectory

So we'll have even more insight.

that's short us how agency get question lot of in by amount the we'll on policies. the then get developed have that your is, not we long and done. trying your next answer we of short So And therapy, that keen we'll answer do a long but out really that on But to it. fine once tune all of design it only blessed XXX, and will and about talk insight enormous the gives an understanding to competence protocol of answer

Operator

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

Salveen Richter

research on recently partnerships into an an walk expect update moving other to-date, Just, a just maybe you here? you update should portfolio, when editing you we forward kind of had under LMP-based editing could programs entered therapeutics gene Genevant us for collaboration with and through, some you've

Doug Ingram

Sure, Rodino-Klapac. I Louise over will to this turn Dr.

the at that to something goal -- I exciting will look I of that that's our modalities, will very the you do, editing for is all we Before we notice at future. out look point are gene that's

use course Dr. is into Editing under Charlie Gersbach relationships editing XX we world North efforts. In should is from months, leaders comment Innovation neuromuscular in also Center in note partnerships that, you'd of we gene perhaps, built of who editing that to in But editing and call I of the addition Louise, the perspective. from last the addition the center, on one our that we the which Durham, the of leadership of gene have like XX entered Duke a to have to over a Gene to Carolina, what gene all significant University, out

Louise Rodino-Klapac

that that at best opportunity certainly having technology. for our that part one using editing, best-in-class scanning are Doug. Duchenne and Doug and field had delivery there and what the really, And some the And lipid quite so we're to develop And space gene machinery you, Sure, just gene like for ways leader way worked making can in Gersbach you thank to sure with Genevant for the other we Charlie time. have about deliver that that nanoparticles. really the the true second to to editing. is said and we of really in partnership landscape open I'd a was look technologies, vehicle delivery being

continue that to sure a make forward. manner moving look And broad at in these partnerships research to so we'll technology we develop best the

So thank question. for the you

Doug Ingram

we have And exosomes gene explore sophisticated to devices RNA as we -- technology. Salveen for delivery increasingly have of more that even a relationships as both, and with should updated course, relationship nanoparticles maybe potentially as have both note use well I and and the of to the well. therapy advance, And gene AAV also editing we

Operator

the next line Our comes Ritu question of Baral from from Cowen.

Ritu Baral

going Doug, have do you final And ruled XXX out are you before be generate six given Or optionality keep data breaking months? what XXX start you show? adaptive on enrollment How confident depending the have more competitive? on to you data you XXX XXX enroll will to to is can that XXX? blind in

Doug Ingram

Yes, both those thank you for questions, Ritu.

interim could So that which a is, not analysis for having insight. is two, The to that currently of about do we really short you host a you thought view raised an even should part reasons. question, a gain answer additional of do that we're on interesting

to positive will put we'll that one. probability are in very The significant first we of next part going a currently that clearly has that's we what direct. the will design And -- just with design study that for is one got insight one that Nobody already insight will the have study gets the study us part increase success, XXX to already our of calling it we competitively now, right have be advantage. us and help from we're kind XXX no that And have.

readout So that to that to have end data, disease, the last a and readout, just at vast visit, of amount we blind. early an year XXX, this probably to very set of going want end do of patient study enormously of year on blind information that's we want the really important and really maintain following in this be maintain and because the by this get the last rare a

an in is Duchenne As need dystrophy. it muscular enormous relates to enrollment, there

investigators confident I about rapidity, quite can remain with opportunity the enroll and excited are work physicians with we so very to And us. that

and kicked think be that I once issue the won't approach in really and time patients. protocol and issue. getting get agency protocol, enrollment the blessing we with robust going. getting off our to big be tuned, meeting suspect I that don't right believe started significant that then that so is really our now start And agency I patient getting the now, And will really that and enrolling on that right fine

Operator

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

Unidentified Analyst

is call This on Anupam. [Tes] [ph] for the

And question the here, is and XXX the to now? olds you five what expanding remarks balance you're the one in you're here, target So XXX? Doug, given what four said and think in rationale trial, study guess for is year to better I your planning I XXX run

Doug Ingram

I that sure I I anything. at appreciate mentioned make And that. delay a to not want really significant that sure for all that to or reason you it's make the question, in thank it Yes, change because is

to we've add that And uses of to the safety four remind by XXX, just this reported process. as on our of and us would more no five, olds, what intend end noticed to the the children, four XX the that it new launch was had dose added commercially about additional IND, we similarity So than news therapy is year representative kids that last a great that to we our material, and since was everybody, allow study process at all sevens some was goal we and that fours that. the year, it to look with. seven be test, the it's this updated to fives. expression and that -- JPMorgan to we've out And more intended is there would are but to sixes, And we

And data It's over get an any in seen will the out haven't We on the and to those balance not attempt us XX just evaluation better time. report delay of this. a patients.

sevens in not shouldn't quarter. kind of four It's consider It's just that to in in study. fives second effort the that to and nice we be balance a ensure start the delay any XXX. a delay in of our anything. It don't sixes delay out report really in have of a So the and that

Operator

question line Mutual. comes of the Mizuho next Our from from Yang Difei

Difei Yang

about very to there quick level, gene that high you Just first? possibility the do therapy a therapy? a commercialization think Is relative on just gene to virus based one, how gene gene based editing leapfrog editing and AAV come a

Doug Ingram

to that's I leapfrog answer see for going to therapy we're of don't that a going something reasons. right Well, is think I gene host the now think no.

possible you as And today of have virus as biopharmaceutical today this editing that First, in is of what that, you a based really gene is that based, is gene raised gene we the the that course, editing leapfrog good front industry? would us point, a therapy virus well. because said answer it short is

as and that a about we editing, empower potential. device both that seen, as think you've a to away would and delivery as move from exosomes, itself gene we AAV So currently, to LNPs look, I are through may want

that already move So of take it, gene think time, that's very To time. approach a is from to and over a successful AAV going require for focus And away if delivery therapy. going we're lot has to about that research. been and effort of to going

just So gene five, gene we a the got editing one. you we in profound, benefit is again, But a baselines, I of us right right would part trial. a temporarily never of we right, placebo is really four least therapy And, trial, at that think before us there's front today, the really remind when seen to out the results did exciting. different, saw had a I in of placebo just controlled see

in in, other very bring that's a editing, Center, there And the But look delivery I is between editing then, exciting why editing think of Innovation our challenges while the into to in we're built gene Gene why is we've us. and are, as around today Charlie like gene look arms we there's effort of both gene of this editing, someone editing front interesting. the difference why differences very mechanisms down areas, putting a to with technological to we've to partnerships entered well. to at just technology in me, reason there opportunity edits into therapy are course, empower and Gersbach, gene at lot gene this road right a gene editing. Dr. believe wrapped but are time, we children there's of So therapies

with you're to which going Dr. about of you're hand, is to take therapy that Charlie And do that's hotspot the gene a are other in to have of create you're you're gene children. can the basis, therapy to And or different and really to either and XX% the an as forms going do exon going On than frame you to to have ultimate be back let's or do, we to well. editing, what going of have could treat on gene respect create a something by Gersbach, with the attempting do therapy, clear, of where to exon edit is editing, protein truncated that agnostic where put significant mutation. gene

answer, Short think I So difference are differences. there time. there's a in

ways well. of out. hope of and other lot unbelievably us I a a think a reason why Gene CRISPR-CasX Prize But the Muscular gene editing in and program. transforming it's technology. areas little exciting Dystrophy today a front those an is therapy today, exciting of right both and There's it. really it's Nobel Duchenne for individuals is research offers won lives the as patients in

Operator

line a from Debjit Chattopadhyay from comes question next Guggenheim of Our Securities.

Debjit Chattopadhyay

So the in I'm was between And patients, XXX X.XX but any to VCN was cohort XXX, VCN talk same X.XX X.XX. the study crossover to first explanation versus of per dose could for of the high cell micro-dystrophin whether in the find -- sort VCN about per in patients as the patients? study difference you trying also expression an crossover

Doug Ingram

genome copies numbers than the the copies I delta sense, insight to the those would explain, blot despite some in into genome think Louise, but the interpret in questions? going thoughts can western probably, is I'm on over who genome wouldn't I your a those this expression, in I smaller copies Dr. between to turn Louise provide over both. nucleus, question Rodino-Klapac Louise will on think, per

Louise Rodino-Klapac

the about part will not in as numbers interpreting intended that two, copy we've the I receive as first would would opposed patients we agree two. level dosing part in the which patients add part genome XX mentioned, discussed. with over to vector you I one the

and in XXX So and the on in so part as additional two dosing we've genome vector expression far in quite question? respect, seen that copies there study target at of within intended are any Was that we well. range level what terms feel

Doug Ingram

about I question. remind everybody just think those are the two to questions and Debjit's

that It that the look method linear a out the So developed study, having called benefit of we tittering was with group part what's the standard standard in qPCR, first were the tittering for and supercoiled below target, qPCR. the three That's can tittering the target there the back turns the used, see the of of first the patients. in the two lots in were hit XX% lower of one the were lots. the of lots, about lots patients of

if for target patients dose on the crossover the tittering and target linear got of factually On on the are the right crossover, that so, the release. And I've completely purpose we and wrong we patients. used tittering crossover,

Operator

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

Gena Wang

one have regarding I So question study XXX.

Dr. you regimen. statistical regarding purpose did population to history that across use treatment, [indiscernible] crossover, consistent analysis the and for the FDA Regarding with natural weekend patients? pre-specified all therefore that tend the we patient know Was align

Doug Ingram

think update the But relates we before to we correct protocol all the of it first. the it that We I all should allowed dose taking see. care can crossover play analysis all and meta of and pre-specified was [indiscernible] the can dose me that dose, even daily second people have Yes, to We we steady if some their role people long-term was question we trial. could a allow as wrong. a at can commencement was patients steroids. of be weekend again on unblind, some as that And of I'm the that patients use use the standard best in

Gilmore O’Neill

unblinding Protocol prior SAP to the part on analysis. were of one created the interim

Operator

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

Gil Blum

mentioned So, as programs for From regulatory Does before, certain have there's positive perspective, LGMD Doug, translate XE. LGMD them. data, across a that data continuing similarities programs? you positive across

Doug Ingram

and our the if understand and case on of but at the to of a safety part in course, you itself XXX significant positive on was Because see a look but from definitely the seeing profile, it both across the not it it comes does And and back XXXX. profile not LGMD LGMDs, result, from regulatory strictly perspective, the programs the data we rXXX does. in capsid the may also of -- we're a Well, the competence and there of in construct is all for a of limb-girdles course, from this as value us we're safety well DMD of both [ph] promoter there. rXXX, lot It's gives promoter and And and in XE, then, five, there's I'm so the programs, the it's using seeing five for confidence of results the I limb girdle the mistaken, as positive capsule if our XXXX [tropism] in a all the the XXXX of that expression program. not all the three the think promoter, across of also same itself. that's same the a three of

there So have. building is enormous we that an amount of confidence

and the material. that pathway, this commercial and Now limb to limb-girdle the from its with going year, I of the representative GMP our that released to suspect pathway I then, inform I respect girdle division out pathway will figure is we once meeting have the with we'll as think, And be what that material for well. pathway we that other are girdles limb think

this have to for year. so us And will an that be important conversation one

Operator

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

Tazeen Ahmad

And is all Hi, an idea targeted together change maybe bit of guys, drugs give just represent? going to a market also EXONDYS expect of tie your franchise, of now right on opportunity get big market? type it little the kind what three penetration us how gears can your wanted sales the a launch if similar maybe just with how will have color to to the of a And and to you up, have of go EXONDYS and beginning. to you had current at with ramp you of the casimersen what

Doug Ingram

thank [indiscernible] think, let that, I together. them so for Yes, you take me

opportunity EXONDYS all, really EXONDYS, issue going the the course incident launched we curve flatter a but side to in ex-U.S. the screening, we'll to then to by to this to And grow of EXONDYS, unlock EXONDYS the be long-term, with is would first patient end could population we significant There's -- over get see of get over reason XXXX, start of have find and that so in to refine still the there on The even and are if based And ex-U.S., increase. there to for probably ever respect is late we ton approvals getting of that new we continuing that we still with year. ex-U.S. patients. a definitely the opportunity opportunity. new an unlock and

the there problem program accelerated map what nature. of countries We have responsive that's an The isn't is we from concept ex-U.S., but that approval they access ex-U.S. in pathway. call this program, most come in the

EXONDYS us some that hampers a confirmatory that our readout done So until give very growth. on Golo opportunity with well. trials another get I for has of we think

then first site. should had as in in of that period see start finally, continuing for honeymoon very to the has a gotten it love result launch to actually And a not not -- the Now clear, golo's just may commercial have the look attenuated itself, is tomorrow, chain team And not having to affairs which have it start to able casimersen, we team, our you approval AMONDYS by a because launch get do has job warehouse very now and a to med growth literally launch But of good has in we already, we'll been did the there's if pandemic go, forms going last your a obviously, pandemic. And just therapy we're we're going offices, got would so in by isn't typically, you lot launch, at infusion therapy, we're that particularly perspective, start immediately kudos ready we'll have a got midst this that week, is the we just this but we in the positive, of somewhat into to launch forms, from my supply this with a yet VYONDYS. there. week. in light, growth approval, to able a of brilliant we're just rather a be physician's VYONDYS only hospital new into you would

think is I think, AMONDYS of population, So with this, that down even is to pandemic, than a been size I'm excitement like probably lot patient obviously currently pent-up I to scaled which going waiting to, say Dallan AMONDYS a going I a demand of there's at long is the scared, EXONDYS. therapy lot is going but looking And have smaller to the lot therapy. for this that for amenable think launch a EXONDYS. this look XX of time exon for and kids trajectory its

of we're phase three therapies than the in launch On the the size hundreds are and you in two mid I this a market, bigger really those we look if already mean, the and therapy. the five alone, two other just together at of RNA are

XX% is, United So the over there's the And time I XX% are this exon a amenable open whose as have as with ton hammer get, the on well this children these leave mutations And is bigger if of we patient exciting franchise really that because getting if be Dystrophy our want up didn't opportunity into we potentially. I larger. the range therapies the this There under can therapies than of to appreciated patients kind be Muscular then, of population. think opportunity people first if of billion RNA but are, headroom, we're is over we going States, will to to just three to significantly technology. dollar I skipping up ex-U.S. to bring are can think Duchenne

We to therapists have kids. very bring these can build safely, consistent, reliable, to way a precise construct that

do we But So the have early that could a XX% agency some out will very over the obviously we had hopefully develops opportunity dialogue the from were, that up there. opportunity or do already so big now. approach read PMOS, could about the PPMO We've in with mutations could find to just to around for XX% in And around launching to can are platform because we mg-per-kg straightforward fairly how and which we PPMO XX that. over rare. AMONDYS either we're AMONDYS to maybe, have to the big build and have a which kids that XX% see potential in out the addition to it I those us get think, second on over a quarter. we it PMO, these XX%, And way or conjugated the and a peptide therapies we're PMO. is be EXONDYS VYONDYS The time, in going and U.S., a be world

at. look to interesting really be that'll So

the the we excited. from XX proof We [indiscernible] production saw had pretty X/XXth very the period mg read XX of at of mg we As were exposure perspective, short least and Xx, and drug out last of concept a year PPMO, and a in time.

Duchenne of with PMO, there's or good a there of opportunity revenues of And significant are to where PPMO lot a as well. do either community. in that So lot course,

Operator

Chen from from question next of Our comes the Vincent line Bernstein.

Vincent Chen

could a you of on XXX, was what your I crossover and And you're the see, how what at look from Taking comparisons what would study data the result this specified provide I view, guess, data would, to end if bit more like? from the protocol? in evaluate the wondering an hoping color what encouraging were year you

Doug Ingram

at look have able for two time. going trajectory can to we're look course, we're thought whole have to a children looking as we're there's strokes, are of against blinded in all, be who at the of bunch more of we Well, really, children, going we're at see, a update first exciting. insight both to unique into at. we well, this been analysis going broadest we first things to things, more And to be the one, we're of which is looking the just to to going going can able children to two that years, have be natural opportunity We're a on compare look that's therapy nearly including history, the at

like also then then well would history natural very what that, blinded I'm end, over we broad without intervention the XX control, using would looking own based a not looks see XX strokes? will O'Neill, you But there trajectory both matched of And their what pre-specified be missing a and Dr. this is anything that age. intervention. out open clear, label an set be at therapeutic study, look that Natural History it look using on, period, here the week weeks their have predicted be that time therapeutic any including to beyond over let's them and at So in course their over set of themselves is the then trajecting at till as and

Vincent Chen

think in part will of you I randomized and for broad you've have who obviously were have strokes, to second in patients active treatment also it one. follow -- a hit year the

So got it. you've

Operator

question from Piper comes Sandler. Bancroft line next the Our from Tara of

Tara Bancroft

the yet? you or on topic do the in what think staying actually of Tazeen's think is to included next, will dosing likely the could I they said PPMO it's topic, be know do a be have next you so PMO, And terms from which on PPMO? cohort And approved get you in XX the question, candidate be think one started QX? you other. you more in mg-per-kg in or could the the it data And

Doug Ingram

Yes.

Going to be the quarter will question quarter the we'll will That's happen last that question. the data the second in in have on, first. The really XX mg-per-kg what second it's data. interesting

it's built of that, construct. great PMO have -- construct peptide is We to the conjugation about constructs Technology the a thing for RNA that

to the build PMO can want the pre-existing construct. we construct. conjugate go simply So PPMO, to we to the And you peptide if

a lot, all of significant have fairly a number We them built the for populations.

celebrate think And distinct those bringing ends start start the we that profiles all now an they're is if safety build they creating mechanism. we in constructs you that via can can up, obviously therapy, the in the then building conjugation the she is like in And the the them. choice. data getting can the PPMO. going better make highly and a what to with And it looks data real fairly in PMO us, them to to that to will going we when on and we'll if road then the good approach, that approval conjugating, we start be very in, kids So constructs, then for precise, question the looks we're great have start answer, the can the have we're going when peptide a see PPMO, those forward hopefully, we And constructs PMOs. fork

stop we at, year, year, then very the last we'll mg-per-kg. it's then it safety that's then, bring forward Of PPMO. pathway, results which going looks exactly encouraging and PMO. of accelerated decide to course, profile, the PPMOs, we we're with those but we and can in the is what how PPMO efficient, need looking first second if be the approval And the is this see And in to at the quarter XX PPMO very those when which

Operator

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

Brian Skorney

or on with ESSENCE readout? available? you wondering, in study it'll that just before How now is read are in week years XXXX. randomized, casimersen given out go maintaining golodirsen I of terms you Question both now three of on I'm intend the And study, in to keeping guess said blinded do integrity until that terms out patients the XXX. trial

Doug Ingram

have and job committed having a blinded of then to we being that's trial to no approval recruitment is wonderful the the both we have not commitment, it the it as good VYONDYS one was accelerated AMONDYS. this even I job, doubt were coming a The AMONDYS a point. AMONDYS, it blinded agency, for to thinking takes about is ensure as that. approach, was about this, you brilliant achieve we at done of mean, we lot the Yes, close a and more it's difficult, great done as approvals a and risk very trial, the integrity blinded. with talked trial, on there's was that And patients it's long-term very a that VYONDYS early approval but teams VYONDYS, result that great things question. so of a way and had Yes, a very the the its and of And job moved than our ex-U.S. is both We've

areas we via have a to on that commercially risk start the -- And not to you those therapy, controlled placebo be have we But that commercially over which are get commercially couple don't to moved available to and about being that in of ex-U.S. that in a what a opposed took don't therapy available trial. result of very, to at significant all, years the to a around as then, so as very world moving a access would therapy board. be this the would then the trial, credible children a worry available we ago the actions to recruiting is therapy be access risk, regions So can

Brian Skorney

to just week now? Six is a so is blinded I it. just clarify that get from that we'll be minute to the what And that in blind XXX? data walk want primary Got going XXXX, so actually

Doug Ingram

I that right, think that's Is right. Dr. O'Neill?

Gilmore O'Neill

Yes.

Operator

the UBS. from comes question next Our from Bristow of Colin line

Unidentified Analyst

our Hi, this thanks is taking [indiscernible] on Colin, for for questions.

question full half PPMO the the have in So we of second on quarter. and a data updates like

modeling more expression your even on or levels? XX work, cohorts? we based dystrophin you should from the dosing Are So [indiscernible] mg-per-kg expect higher or expecting

Doug Ingram

Yes. know The we short is, don't yet. answer

any data. the We of haven't seen

two dose perspective, animal we XX the of mix have some mg-per-kg, is exon part allometric, we been What production. that we know one-tenth, of and nevertheless and is at know dystrophin data response models we disrupt be animal can model to we're way in We trying times very until see I that a exposure, which risk exact So wouldn't trying do it. the between. a we going of do don't want the steep at saw yet is, know in lot look And there's to data. at course correlate is right it is between it What know from skipping curve in at is human off animal base, that We All data five things. and dose figure of clear. it what a to out model yet. and to

something seeing a seen weekly so doses, already interesting we've it's frankly, in So ways, post And, concept. than dystrophin, PMO, better its host PPMO the a of dosing Xx, that's monthly the a schedule interesting. really we're it's much a very the interesting and for whole is already dosing,

don't a start be do the The XX we know if see increase at steep in at models, it. mg-per-kg we that you But dose. we will and thing place least second right know animal seeing the

had we is but know see a begin were mg-per-kg dystrophin, see that, until XX that to and won't mg-per-kg, see and of amount we course, a all higher XX potential So biopsies frankly, we increase XX that or the in the than there XX the we of until of have mg-per-kg, something data steep up we either we patients, evaluated that because in animals.

Operator

question the comes from Auster Suisse. Marty next Our Credit from line of

Marty Auster

prior Congratulations on follow the had amount a of I the question. up approval last from week.

end you the on at to back Doug, that milligram the the by potentially track? And about I December Is from arm XX the my looking if per QX. able commence possibly being call of XXXX. notes data kilogram you think talked on determination still things? or something kind advancing? for to kilogram that that XX are per not, you're side something biopsy to of waiting get going kind the make forward on on before Is milligram safety that side of the looking

Doug Ingram

as We're we we what we XX the are also start move should how view looking want thinking dose. ranges going we in that then, -- the be much so decide before what dose to see updated yes, fact And to higher for about mg-per-kg two we and and on pivotal, pathway decide is things, should up in.

then mg-per-kg the right mg-per-kg do that and XX quarter. XX we second on after So see to we're We'll that. see decision the the going make mg-per-kg XX the in

Operator

trial from Our XXX? great study, pivotal similar When you the hear from for the my we the was beta-sarcoglycan for This Beatty next for Joel, question Egan Citi. expect questions. to about Egan, Joel Shawn - pivotal taking year. line calling Q did study should for data thanks to this the cadence a in It comes expect trial Shawn is of

Doug Ingram

from groups own a SRP-XXXX-XXX know placebo Well, a the has to and patient and that One the the lot approach population different enough that blinded it with significantly pathway right one two patient And we challenges. guidance is have don't because important regard, ethical required which patient there's but is with we're we that, feasible the that is what is to be taken and development issue that, large for taking, controlled, SRP involvement the believe done. with do around the it reasons. is advocates decide can study? of of is it DMD reasons we've beta-sarcoglycan. its which XXXX a It me, it's will discussion that Well, be agency's

So it's executable.

do a the truncated of course, of with dystrophin. thing, second is you micro-dystrophin, respect to The that have version

that is the expression rarer makes there's wouldn't been regards. beta-sarcoglycan sense. a and that in function a agents, wouldn't And to One, for both show an trial, be much as the very would cadence that ethical respect have from protein. blinded, I of are to native this than you same alone, and be also that assume sense so in an perspective. we're about creates important two But XE, would understatement place. would We And why I that native the the probably is different the to be it's gene placebo with that is it controlled that's This second it population, just SRP-XXXX make advance perspective that's the challenging it beta-sarcoglycan delivering, a clear a doing, to an concept you've be thing think sufficient, we execution that say of protein.

really will dose the high very cohorts MDA. low with So, issues come to we're critical already we're our correlates by to to very the be with using both, our that And are an efficient, with endpoint, dose executable we're functional therapy Hospital, around two respect a the agency that and Children's a supply seeing of from that to waiting, clinical important the those both we've way that have is update lean, strong at we'll Nationwide this get to the seen together, have ability patients up discussion in update. approach in going expression a high on seeing that

the native GMP the so of native to discussions CMC know the view the we'll pathway patient year. And should should informed development That'll a we division. with the protein, have this discussions material agency by it's when and But happen be leaner course, and be and given our that population is those its it have that we once have protein. with the we'll talk fact

Operator

Thank you.

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

Unidentified Analyst

may improving in Danielle. muscle on [ph] the per kilogram have COVID the lower last SRP-XXXX update, delays On you Can experiencing lower you're contributed delays your similar whether the that dose, concentration question. Olan] the that you biopsy more potentially XX for plus interpretation comment Thank that taking compared college still environment? milligram This to is you [Dr. mentioned related our make were in might there on difficult? for to cohort PPMO

Doug Ingram

people So versus the out an mg-per-kg resulted were of remind a biopsy problem to delay the from will don't issue dosing that the that be of believe timing there was for but in reviewing itself. O'Neill, the Dr. the out second can we'll window because pandemic that confirm, be cohort XX was -- you of I the quarter.

Gilmore O'Neill

Yes. out has window not That forced is biopsies. correct. important of COVID

Doug Ingram

it the but thing of one myself. and two study. it's -- years, recollect The earlier be study on, apologize, kids be I'm out kids to won't will I an shouldn't going believe error off But study comment ESSENCE I and I read so rolling roll I the made will off

two error believe my part. my not I'm I years for apologies it's, So mistaken, data, on if that blinded of

Operator

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

Kelly Girskis

Hi, this just PPMOs. is on the Joe more on Girskis Kelly Maybe for Schwartz. one

wings? and a be the waiting expand other on but the XXXX the trial accretive in there multiple touched this with in you PMOs envision future? to PPMOs on the single can you way hoping for path regulatory do Might You we're

Doug Ingram

Yes.

thoughts finding then PMO our or similar we're with the is big PPMO. a right issue And regulatory the use current to assumption that be PMO, So dose. is on that whether the or construct that. The the working going will first the couple beyond pathway same is a

ourselves that this one perspective ability endpoint we the to And and the exciting benefit, and constructs we to regulatory EXONDYS, now platform to able RNA the about can we thing So predictable of ability reasonably and the to avail that VYONDYS that dystrophin been other therefore as approval is of from that's accelerated their things that induce a is the skipping the production. pathway, pathway we have that dystrophin of exon we've use is surrogate precisely for likely a that very therefore the use have officially are to clinical build to use in AMONDYS. lead ability have

efficient so constructs lead clinical as dystrophin accelerated able benefit, forward. bring of amounts to induce or So, reasonably of reliably be a pathway to it's use long it's we this our are to whether belief that that can to regardless we'll PPMO, substantial PMO likely

DMD that can we to up about XX% that patients. do XX%, of so or Now,

populations, would or accelerated the amount getting us patient exons, dystrophin goal rare then we very a have that PMO. we're so both So production study of had the similarly and patients But use mutations think we of a whether on a still with to would of we our or significant safety essentially and the number that do, sufficient side up there, a of of about XX% that additional of to from have patients I I experience we basket therapy approval is built XX% pathway. collection sufficient amount, a work can PPMO with things can get that a potential would benefit our that a next think

a lot That goal. lot our DMD. a whether of of it's potential So opportunity, would is, or PPMO course, good in do of PMO

Operator

Thank you.

next Singh the Hartaj Our comes Oppenheimer. from line of from question

Jackie Yan

question. Hi. This Hartaj. for the is Jackie here for Thanks

could you the what proportion be on us agreement amount proportion for going from milestones commercial that Roche, be going potential your Just just to billion to what aspect? of milestones? remind with And $X.X are of clinical

Doug Ingram

don't can or if we've Am have the And you I'm in not sure, I confirm we assumption? that Ian, I again, we disclose can nuances can incorrect if milestones. we that. of disclose believe disclosed our

Ian Estepan

achievement. regulatory and to commercial mostly related that's milestones milestone correct, the And are

program. end So they're the back of development the on

Operator

this I At call questions. to over you. time, showing for the remarks. like would turn back further I'm no Thank to closing Ingram Doug

Doug Ingram

with will we milestones us that questions. week, week said our of Thank the a lot across continue this this warehouses AMONDYS earlier, with your opportunity serving perspective, occurring, have you EXONDYS, already -- year. we to as will thank And commercial From over will in do you a this the of we very will the therapy is and And we a launch probing serve very and be the course evening, We community literally be all evening. very number of serving we're to I've about the important launch and this very, VYONDYS. afternoon excited or community insightful continue joining XXXX, much therapy. community this be to will which patient the obviously for this for

year, a about course the either very next this PPMO opportunity update that a mg-per-kg quarter. PMO. staying the PPMO exciting So with we're or I'm and very XX That excited evolution the be our about of very with over technology on could excited to RNA RNA, of

enormous opportunity the up I to a as can number an in over serve we both DMD, of times DMD have We to that in said a of of good tonight, do XX% community, time, the lot side, time an hopefully to And and therapy respect have milestones, of United this over SRP-XXXX. work opportunity. enormous the an which do we with lot gene States number year enormous to ex-U.S., of is a

that, study us bring that that gives XXX, are better a the be We Duchenne. at patients And with over increasing beyond And XXXX. to thank about have the a on for course I stay you as have and XXXX results With everyone confidence potential look this opportunity longer on of a transformative significant update could and very milestones course course, to kids carefully really focused DMD. limb-girdles, of life our with evening event when look to you the to year safe. of to of of everyone good the that, we focus it continue forward we

Operator

Ladies Thank call. and conference participating. gentlemen, this concludes for today's you

may You disconnect. now

Sarepta Therapeutics (SRPT) 2 Mar 212020 Q4 Earnings call transcript