SRPT - Earnings call transcript Q4 2017 | Sarepta Therapeutics, Inc.

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Operator

Good day, ladies and gentlemen and welcome to the Sarepta Fourth Quarter and Full-year 2017 Earnings Conference Call. At this time all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, an instructions will be given at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to Ian Estepan, Vice President, Chief of Staff and Corporate Affairs. begin. may you Sir

Ian Estepan

release and this we all for released year X-K The quarter Takia, at our call. and www.sarepta.com the joining Earlier and our press XXXX. of today our filed end available thank you on for website earlier you was today’s fourth is afternoon. Thank financial results

a for like control. be and me the statements. call risk a the will Mahatme These our note of webcast making After Cumbo. to on Sarepta’s Bo remarks involve this on statements uncertainty number take will any today Forward-Looking we Statements, Sandy slide forward-looking call would Joining forward-looking our up open moment of please Q&A. that which during which call the our and to contains are beyond review Doug I formal are we Ingram,

these trading materially such operation the could and results risk actual Sarepta's materially differ business, and can and Our statements affect price stock. common adversely from as any of of results forward-looking

overview our filing recent Ingram, required subsequent forward-looking call not will With does For risks the on Exchange filings. progress. CEO, review this a the XX-Q filed XX-K, to and we Company with the Annual let Annual circumstances. undertake well recent to The as today on by Report our afternoon an uncertainties, any including who applicable turn statements, SEC its X, on and Report filed that, Doug. and deadline. of financial Company’s publicly or Doug the most March to on the provide obligation full-year XXXX the Securities you Company’s Form update SEC projections over XX-K encourage of other for Commission, as of description detailed events We Form our me based any XXXX provided

Douglas Ingram

which Sarepta, year a Thank XXXX, and a and you update important focus fill one platform first disease successful the on our we through generation XXXX in we XX full in of the in XXXX, advanced rare that with foundational for of disease executed call. launch excellence pipeline. collaborations Sarepta's Good to and We ability execution commitment gene-editing. numerous the quarter U.S. In four conference and exhibited afternoon, an launches fourth next our multi year scientific and signed EXONDYS we full-year all medicines most the have results you, for thank our mission, focused root joining commitments. Therapeutics across the history. pipeline rare on to our In gene for financial corporate therapy precession new showed our Ian. advanced successful We

also For Therapy Gene our clearance for received internally Nationwide instance by meaningful. enabling Programs. our to IND that drug from is we position vision but new billion making for it cash only and candidate our PPMOs PPMO our preclinical vision sheet in on we reality. advance targeted RNA Children’s SRP-XXXX strengthened an And is invest Micro-Dystrophin driven five additional a FDA balance our not We so X.X investigational Hospital, our programs, with XXXX we and received our at partners successfully nearly and ambitious, into IND we GALGTX exited

have one precession reality. genetic with improve elements make way in individuals and to globe most all the that the is of the the extend of dystrophy around and goal Duchenne muscular a of leaders along and Our lives important emerges medicine to we the

stable. that is urgent as and universally unforgiving to you have DMD, the improve of We know mission disease with patients lives accrual an

in have which strong We perform well. to a EXONDYS continues XX, foundation

the position have in future. pipeline advance disease to this build cash have cementing and aggressively area distinct We our industry programs XX we underserved and us position an leading DMD pipeline substantial for leadership a of enabling very our

our at Healthcare to the announced year-over-year Moving $XXX growth Conference XX, that XXX% million revenue here The our and physicians, $XXX givers. into EXONDYS engaged progress prescribing reaffirm that million guidance. nearly patients commitment and We from persistent XXXX, XXXX. I J.P. with our full-year XXXX securing team is and will Morgan to XXXX versus in is reimbursement we execute and well you care guidance remind continues

oligomer Turning in X% announced PMO minimal in treat exon of platform, our XX we our Dystrophin September results phosphorodiamidate about our study with GOLODIRSEN patients mutations XXXX DMD skipping. of to XXXX, from positive XXX to to morpholino engineered

the study in patients primary all recall, secondary As on you significance with and XX biological statistical results endpoints may DMD. achieved

results will the validate scientific In first our the excellence these DMD addition plans. RNA to we continues in quarter GOLODIRSEN the new on further guidance improvements. to on GOLODIRSEN. results are medicine of update splicing an After precision possibility with FDA the to of to offering application platform the methodological year seek final XXXX, filing the treat on our provide of minutes this to a another an we accelerated our drug met community, and seek on for approval our X% potential meeting these from FDA complete, and Based focus meeting

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let a PMO our on brief status technology, in on me the update EXONDYS provide Finally our of Europe. filling

response, additional may of application We late examination later you Committee and European surrogate for mid of know the course questions is in the a believe lack a strong regulatory We're but in approval marketing robust and getting that hurdle the remains a this EMA in responding As the in use a these of complied Europe, preside being authorization by points just review the XXXX. XXX and and our a but end accommodate challenging, reviewed in Medicines we data not an their have from currently CHMP human on-track oral stages process April. Agents. disease products remain hear day for package preparing to we for from

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effective if the treat of Our DMD candidates up first six to would population. PPMO XX%

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specific be finding States the over turn United will Duchenne We and the million support most update program. gene a I (OTCPK:PPMD), the focused with and in Micro-Dystrophin is in With are that PPMD organization associated provided also now dystrophy. muscular proud therapy comprehensive Parent financials. on to the sincerely Sandy which largest X.X for of Project non-profit cure DMD on Dystrophy for Sandy. an to Muscular call

Sandy Mahatme

successful the was afternoon an good Company. year Doug, Thanks for XXXX extremely everyone.

transaction which product As year. we we a the proceeds net In of fourth million revenue of the $XXX.X completed for debt net previously $XXX million. announced, quarter, the convertible achieved generated over

and of December hand. cash, we XX, $X.X had billion As XXXX approximately in investments cash-equivalents on

efforts and our Our bolstered XXXX us plan. talent balance resources will a expand our successful and the These base strategic our sheet to the beyond. lay provides execute for with accelerate on R&D, foundation

share million non-operating of other cash loss press $XX.X balance and non-GAAP $XX of to Net in $XX.X of settlement we we restructuring's, per compared the from compared GAAP such XX on revenue on goods line the exclude with Prior a the quarter discuss expenses, now to and of each the press revenue approximately in was share cost of for EXONDYS the the cost fourth a operations I In U.S. of moving million, XXXX, and of we sales net details on refer milestone will fourth demand the adjustments. quarter XX basis will a $X.XX In reported quarter as provides well for reflects non-GAAP The stock per picture cost approval a our of in increase we of entered expenses. XXXX that material GAAP fourth for as item. results the was to EXONDYS press The the onetime fourth in same million for of of website. of the $X.X release or Now $X.XX license compared non-GAAP provide a payments non-GAAP compensation XXXX. the we period Including quarter for result the call XXXX. million into full of accurate basis. $X.X BioMarin our This as $XXX,XXX The and XXXX ongoing which of of and EXONDYS to release impact SEC available release on P&L a afternoon's reflects this expensed fourth net financials. XX recorded through manufacturing quarter the the is of goods to net increasing loss same million and the of as XXXX, Please more to payment July XXXX. non-GAAP. reconciliation agreements operations to period royalty $X.X in R&D for Company million of the or go XXXX.

being cost expensed have to are of grow. materials consumed previously will As our continue goods

COGS year-over-year expect X% XXXX million. of $XX.X stage to primarily which a headcount. $XX.X enrollment expenses R&D for driven of professional Adjusted and the The to million by of the that royalty. $X.X and be of for was of approximately Company’s primarily platform expenses recorded our for increases XXXX. ongoing the fourth to other fourth issued interest million to we an million note were non-GAAP which for same to exons. the in The of by preclinical XXXX and We by we includes the compared non-GAAP $XX.X XXXX. BioMarin expense the Company’s increase compared is XXXX interest quarter $XX XX%, expansion due was patient number period due million. XXXX driven related the for XXXX studies same to convertible the increase increase period The quarter less services for other fourth $XX.X follow-on in increase increase of the $XXX,XXX PPMO same expenses period the quarter is global in driven trials, than were year-over-year for the increased in increase compensation Adjusted approved of in late clinical personal ramp and of to net up compared SG&A expense $XX.X In to million an million primarily

throughout time. XXXX, will expense we our guidance the operating multiple Given points inflection providing at be not on this

it be the as in manufacturing and GAAP and targets global our Bo. to We will up a Bo gene expansion phased to last plan to resources and However, pipeline would GAAP Any life therapy DMD disease and will infrastructure SG&A our to for pursue support be advance our year for expense programs. compared non to that appropriately PPMO of underserved diseases. rare update. we for to build development related we're call over and efforts. threatening scale grow the industry leading non With expenses accelerating like expect and increase commercial utilize to development our I research to turn our multiple

Bo Cumbo

back. Alright we're starting

Thank with of successful Good revenue our everyone. Sandy. XXXX. start beginning. you, guidance launch will the the as confident by to XX, the afternoon, our quarter fourth our We I for remain strength continue net and earnings ability EXONDYS in evidenced for So

team to commercialization first pleased market the to the therapy in XX in outside to of amenable very are the and We full-year building that among EXONDYS the with also exon progress and made infrastructure patients coverage amenable our has identified with we're U.S. and treatment Maintaining looking XX a our support disease appropriate efforts amenability. forward appropriately for in key common form the Driving were further identified expand patients reimbursement start for across key EXONDYS in all genetic skip adoption launch a U.S. for activities. the patients In of and payers test our leadership Duchenne. from focused role with rare to centers to for preparations and XX, treating the commercial in prescriptions to XXXX are affected around decisions exon procedural States boys ensuring dialogue U.S. have continue mutations barriers addressing by four first to and United shorten DMD major are and active timeframe infusion the skipping administrative

already on XX to focus We on importance to continues physicians team XXXX The to and patients. starting them of to these in EXONDYS will work patients amenable access was continue accomplished ensure appropriate to to identifying provide the all efforts on build understand XX. and what EXONDYS

skipping XX. amenable Decode XX to identified collaborations benefit collaboration continue future has who many PPMD products any Duchenne XX patients XX, candidates. XX with patients and could for testing skipping exon exon the additional launches genetic exon more identify testing genetic to also from and EXONDYS Our potential including have

testing the of and not are change plans have The XXXX. XX of seeing. we proud mix collaboration Medicaid XX genetic success do in expect patients this remained and throughout are to this the launch. at commercial fairly are approximately consistent on we Patient and dramatically demographics time this to percent We

of average to This XX. ambulatory and demographic both see across that are access non-ambulatory old. to to age the is are XX adoption age obtaining patients continue age between groups, indicates patients We years XX EXONDYS pleased therapy all and on

partners, eligible stay standard the for drug higher XXXs We perspective, full-year for and infusion of home an screening XX% all time of to From rates Duchenne the this trend launch first and identify XX. believe relatively discussions patients to We of this to and previously relatively to Throughout to opted intact discontinuation have we becomes Based we safety this rates. which profile to compliance until EXONDYS see physicians of compliance continue. expect stay placements, expect port prior port first new infusion. specialty their and medical adherence at that practice persistency led pharmacy minimal have newborn would are compliance than anticipated approximately the to to we patients continuing and XX% expect high our high. with shared on

prescribe XX, submit Tier-X XX of new these and However, year that the delivery. have A for progress centers amenable launch forms confident first drug not to is we continued skipping patients the ultimately to and XX% the identify approximately during opt top We Exon patients port population start new XX% starting DMD that a feel of many a of port. monitor of EXONDYS for significant EXONDYS patients treatment of how measure XX do will actively choose treat approximately accordingly. to Tier-X all XXX% XX% centers

and reach identifying XX call additional to expanded number into the top treatment prescriber of on XX but we the plans XX. physicians educate continue patients understanding around EXONDYS amenable Tier most of establish have in understanding launch Exon a have better strong EXONDYS who have to now importance since size eligible from our and of the for in We disease, would With benefit payers patients as clarity centers patients. on the given likely

In global this will XX Operationally, to Code, payers Duchenne part is XX include expect data our while we the growth regions information both treatment for ease access laying the for always Duchenne. transition, Our in received of temporary as to the permanent and Code key EXONDYS involved future ongoing launches J we and of around we EXONDYS there prepare decisions January infrastructure building appropriate disruptions future world regarding with reauthorizations. leadership in build our foundation efforts help continue to J for permanent Duchenne. helping for to support

country We managers, Europe. have medical within markets now offers to directors MSLs key to extended across and

and We manager recently the have also country started of hired seeking and medical Brazil building our infrastructure director. in America the currently are Latin

look a launch and markets, our carefully investment we other expand as In In preparedness for set in globally. in summary, addition XXXX successful to a assessing foundation and operational we're these has XXXX the to supported key very market beyond. solid

EXONDYS call therapy prepare products our we're launches programs continue in turn market very operation and Doug other DMD the as phase. disease to for approvals with exciting for as with will and future the exon will multiple about prepare Sarepta. other excited We and to before the remarks. build that, for thoughtful opportunity key to PMO skipping infrastructure Giving PPMO well us XX future in closing other I And back at gene

Douglas Ingram

you, Bo. Thank

as that in out our balance let of serve XXXX commercially. year execution continue with to a oriented technical all sheet pipeline, pipeline be our Before I the stage of a a will let the with and XXXX our make issue to transformation potential by enhanced we reality. scientifically stream we our and built me had reiterate and a with that me milestones In urgency. establishing our set advance We credentials moment ago. to community sense we begin catalysts [indiscernible] that for closing [indiscernible] a of steady both apologize pipeline

the But And XXXX. mission up call in please on remain questions. lives improve way will much true open along the that, patients our with with the for profoundly operator have to We working DMD. all to we do to of

Operator

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

open. now is line Your

Anupam Rama

about in PPMO Maybe Are the the DMD actively guys here development pipeline flipping a over you cards? and or on of business thinking is pretty space dependent strategic much still and how so development the much. Thanks Micro-Dystrophin here. perhaps business question

Douglas Ingram

Thank you.

is Doug So, this obviously.

be serve in we children DMD development community, business with opportunities that would therapies going so at the will active We life confidently enhancing are looking DMD. to find always be forward we for

Going give opportunity PPMO, I’m in it either see beyond at to an DMD would we in ourselves Program we areas if development us that, those extend even our our will as Gene business signals, looking sure well. positive in Therapy and other and situations or be beyond

So, pipeline, to to we we got have of kids a on our lives DMD got execute fantastic to lot have the the a lookout ways do, pipeline, for we are funded are and always well improve we with but beyond. new

Anupam Rama

Great, much for thanks question. our so taking

Douglas Ingram

Thank you.

Operator

you. Thank

from Our Christopher Nomura. the of line question next Marai with comes

now is line Your open.

Christopher Marai

expect Thanks mid the Good question. some markers Program, XXXX measuring that be day the of Micro-Dystrophin be number data that on further reporting was comment might afternoon. perhaps of are taking could will there Thank or Gene that biopsies I for a we you for will information Therapy any on you. should wondering guys other if from on you we copy in and seeing biopsy, we that my in your that see release?

Douglas Ingram

gene going in So, the programs, expression, Micro-Dystrophin our results, with preliminary of case those just GALGTX be of months results preliminary the three information clear, in likewise Micro-Dystrophin regarding in primarily and I expect have to to will have would first preliminary related biopsy biopsies program expression. therapy biomarker Gene few we will are gene we two so after data Program, our Therapy of we are have our releases

Christopher Marai

Thank just then didn’t included utility of that as quick follow-up, as your and [indiscernible] in could if perhaps construct, domain? domain comment was or far include your not Micro-Dystrophin Okay wondering I the I recall, why on perhaps Micro-Dystrophin binding is that you. you

Douglas Ingram

an result the functions. years restoration of significant in with expression and amount preclinical of work Micro-Dystrophin it over we and years the very as in shows our enormous models robust for a correlates construct work they Our the many contract that cash preclinical have of is life number down way and of to relates in

feel construct about good we a about will the good that certainly from and So work obviously we preclinical have the but from very biopsies, are a both been efficacy from as work well. has very about using, we to perspective, The feel very the but potential results safety were preliminary confidence where feel a construct that our function good subject we animal that the also work that we we readout await using in both and into the are vector preclinical lot Micro-Dystrophin safety other went enormous using, are of today. us from for amounts construct perspective amount and of the preclinical that RXXX of a the gives about GALGTX

Christopher Marai

much. Okay. very you Thank

Operator

you. Thank

of question Credit Our comes next from Alethia Young Suisse.

open. now is line Your

Alethia Young

step SAG this Hey year. guys I will thanks vote the have about for of communicating a Congrats kind Dystrophin you PPMD or taking you later there my you just in program? the there and SAG be gene and functional assume you is your question that this to again Do therapy, do Do [indiscernible] guys will progress talk I think the exactly is second question. able want on Thanks. Europe. find on don’t not around and year next get to and on the in if Germany. expect a positive what is

Douglas Ingram

amenable. therapy brought to that explanation this the late So DMD oral on be focused our providing and are Exon and is have April it now a to to in the XX to package Europe will show our honest that relates as Europe is enough very in to the are effort so singularly have brilliant children good right in position that be a that data CHFP applications we which at we safe

options we're unsuccessful, what have So right do next a time, after got be but would that is focus successful steps possible. number to to we obviously it’s have singular going the options, look event have were our on got at to trying as something now that in the of be that we

the the after will from we're the will we get read next biopsies our preliminary steps we by we year then we to what gene but results, and assess tracking from to will middle relates right once... next now it certainly where there see of and therapy, it As would be communicate are the have and out to preliminary results

Alethia Young

its at So extract we functional for update? shouldn’t this

Douglas Ingram

think three this it be I read it provide chose EU functional be likely to information potentially will be functional the month meaningful to so after will will it a mutations, three on first very few it to that be probably misleading point. and will at biopsies, provide biopsies

Alethia Young

Thanks. great. Okay,

Douglas Ingram

much. Thank very you

Operator

Thank you.

question Our Schwartz comes from Partners. next Leerink Joseph of

now is line Your open.

Joseph Schwartz

effects Great, of what much. issuance, exclusive and lack a I the DMD for agents directed the make your related targets FDA thoughts you reference thanks of do focus to of the development utrophin for could if to have was on the well that towards wondering like get you Dystrophin its expressions guidance Dystrophin exclusion recent we Micro-Dystrophin. as timing any very and drug on threshold the as of

Douglas Ingram

let say life we focused is end targeted issue point are on FDA that these to an as our me particular technology that of spoke guidance restoration pleased So the the of Dystrophin the to Dystrophin very important improve at RNA kids.

So prospective my obviously very from it step good forward. a was

final would Dystrophin I we the the and would like that in more that trial on that have happening our had suggested and the expression do, what FDA so. result as we this, heard may had meetings speak looked in that on GOLODIRSEN therapy that we to had let and me which month from we based we earlier or are we you about a once say to next the anticipate that as advance Beyond have of GOLODIRSEN going minutes with meeting

Joseph Schwartz

are enrollment think us well or for more finding essence, to you into Okay able rapidly all update XX you the patients you they who XX patients as XX the be going great being either on give go as before enrollment OLE. are do are you and evaluated when in will the the and then can complete an

Douglas Ingram

of the and trial by doing very so was so the still So XXs since enrollment likely to the we enrolled exclude right things the hitting represent we of as increase faster going that little the ensure nature end We we now, XXs, are we than were to enrolled fully enrolling XXs XX about XX, well which XXs the didn’t of we XX so and X% were well. are DMD but be up [indiscernible] enroll, while study increase the and random April, should we we the are people a want sealing very we have population. in those simply coming

Joseph Schwartz

Great. taking Thanks my questions. for

Douglas Ingram

Thank you very much.

Operator

Thank you.

Blair. question with next Lugo Tim from Our William comes

is open. Your line

Tim Lugo

your Thanks a are now is spending uncomfortable Can obviously expense waiting talk of about levers bit. guidance the manufacturing as factor, would you year make readout, what you for a talk are for to not operating therapy feel to and just the reasons you issuing pipeline question. big be related, issue gene on about of may gene be taking for you that can guidance it for therapy and some

Sandy Mahatme

Yes, hi Tim, thanks for the question.

as the I getting therapy So as well as be multiple in my program. our explained readout, throughout will EMEA remarks have we that’s inflection and gene all PPMO that XXXX our points prepared feedback we

Obviously for we capital guidance So too there this early just at could for these you account all into terms liquidity be throughout that because are of situations and to in But make plan a account. at expenditure not a have it’s providing of we significant be provided have little the us swing might all and take things year these point. our do the guidance expense that that and point. obviously this any budgets have internal of to investments

Tim Lugo

your Understood for more Europe, that and is guidance the XXXX do you opinion positive of that or year receive revenue impact. if a from will a significantly change

Douglas Ingram

the is we some guidance to to go which getting the and presume have would reimbursement every the If significantly takes process opinion HDA then through the process revenue approval, a have country almost obviously positive access and that post time. Remember, change. I our we guidance and

to a the process, So certainly next would look would begin XXXX. there presume build work HDA post we to but to I XXXX. and obviously a through something wouldn’t that continue great it be we it for significant revenue be be and would But in in step would increase guidance

Tim Lugo

Congrats on the progress. Understood.

Douglas Ingram

Thank you.

Operator

Thank you.

question comes Robert Our Skorney of next from Baird. Brian

Your is line now open.

Brian Skorney

issues current And done, patients Thanks. you Program, with are were avoid have to are an monitoring the wind us first immunity or Hey do Micro-Dystrophin, who the on you from been prior what treated of specific the of are If and the what not to patients to Two time of maybe major questions. terms the generating Micro-Dystrophin on patients to this do the in even the point? Therapy thoughts thanks to know year immune up you how Jerry’s for you gene to guidance reactivity, be as done decision seeing give Dystrophin anything in frequently kind now one when Gene then one can seemed there, T-cells, to reaching on XX will I guys Micro-Dystrophin. analysis talk taking me, in the be ESSENCE. construct, therapy is interim start construct monitoring it or are to about that right for

Douglas Ingram

Yes.

an more the careful So, say, until final the be want want our for our GOLODIRSEN the analysis forward get post moving to but trial ESSENCE NDA somewhat FDA, GOLODIRSEN marketing confirmatory GOLODIRSEN about on I use event note very ESSENCE I that because robustly the to what would we speak that meeting we commitment. from will I in with minutes interim I with were as for likely approval we in the accelerated

confirmatory it as so for need about ultimately to careful and we for things be at integrity what a quite sooner to risk looking of could that about GOLODIRSEN. fear trial ESSENCE And to we that relates looking the be

sort that’s are way of So, it. the at we looking

going interim like to be very cautious are ESSENCE. We things with about analyses respect to

a essentially basis. it in on Program the As Micro-Dystrophin the at are relates least to weekly monitored Micro-Dystrophin the children constantly Program, being

updates. very then And meaningful so to far can obviously provide things and look get we need more biopsies we good,

Brian Skorney

Okay, thanks

Douglas Ingram

you much. very Thank

Operator

you. Thank

question comes next Capital with Brian Our from Abraham RBC Markets.

Your is now open. line

Unidentified Analyst

thanks my for for taking (Ph) on Hi, question. and this [Rick] everyone, Brian is

remind that could Exon So, to PPMO your timelines given EXONDYS recruit amenable you then XX patients and about trial us and this dosing maybe expect you for for skipping? development XXXX, also is the maybe confidence for could begin approved speak to you to already when XX patients

Douglas Ingram

a complete very patients so, the my able to that have me then preclinical dose and dosing to broad into tolerated XXXX, are maximum dose ascending and strokes, efficacy so that single we on patients as given in work be extraordinarily So, recruit getting study, as we from insight are always This single well. you we models a of within but preclinical far as somewhat goal are the And believe multi a that far perspective, important, patients, to recruit have give because on we tracking toxicology things is dose our I the an and currently then will study mentioned we so that’s are an ascending XXXX, end by the increases we can increase the ascending we small we are in counterpart, significant using get beyond are will dosing we levels. excited in increases significant confident it so about versus Dystrophin cell in already the and we there. study On study to to and it challenging population, to going are move really PPMO additional and expression are penetration we timing and PPMO in patients, in skipping and to PPMOs need that of just PMO extension it, then let scale IND exon text, significant but see now. go from right five enabling we its mentioned dose if therapeutic seen

is and this filed one thereafter the immediately goal IND one additional next then to next year year IND that another by have end of at filed well another as shortly year least. recent another and for then Our

additional can complete in can forward patients. urgently into move filed So clinics as the are we working and the work INDs get and hopefully hard that that patients benefit to these possible and so additional PPMOs cleared as we and

Operator

Thank from Morgan you question line comes next of of the Stanley. our Matthew and Harrison

now is line Your open.

David Lebowitz

Matthew Hello this is Harrison. Lebowitz for David in

to last Just that question on PPMO. respect extending with

any different tissue seen XX? EXONDYS point Given I the you penetration, guess far are safety higher than in early have the thus signals that

Douglas Ingram

all at as but this just recall of I haven't start first dose a we you is studies or to levels. at caution that the you ascending sub ascending therapeutic of note want dose may may no, for all significantly us single Well study, all, single

very potentially and more would therapeutic haven't with going optimistic and can preclinical seen tell await Today, feel are our we that why PPMO that's confident signals. higher more we I at I any we as we doses. six risk you insight are safety significant while work, and get programs, to So from but we good very

Dave Lebowitz

all Fair different think overall started jump that enough, two and going and what because I that update works them point or be not the do the therapy, over an various at to yourself to to for game. to the this would you are guess let have forward the end consider programs to to is e able the dose. three already having just compare programs, gene push you objective generally I

So valuating the think might when have you do comparison? you least begin and enough to

Douglas Ingram

so while and root has our a GALGTX GALGTX of maybe so three the perhaps want do entirely a note muscular and would is to vision and take I children have we to are going programs different treating for as vision to shots other you approaches it forward going ability for Micro-Dystrophin disorders or we're all would approach push behind these boys are that people asked batch take possible lives DMD GALGTX as gene change in one have ever run therapy well. and DMD is DMD, think and to us DMD to we also with even like other dystrophies our perhaps goal with we utility are a would confident so other have positive and the neuromuscular the deal for long and numerous treat why make I has that. possible, beyond of all clearly to that we're the as goals beyond we separate just Well, family and clear potential DMD to as that as sometime going we so are and call I there we it them maybe is this ongoing a that and the many think to before beyond

Dave Lebowitz

Thanks for taking the questions.

Douglas Ingram

Thank you much. very

Operator

Thank you.

comes Company. next & of Baral Our Cowen Ritu from question

Your open. line is

Ritu Baral

question. Hi for Good afternoon. the guys, taking thanks

indentified year that you First going question profile the to is interest, span see average even expect Bo, patients with in you would of something stay you the EXONDYS about why for age would forward. creep life is I screening of Why said XX, wouldn’t Bo and XX the old before be is to average average age about, to down said XX born earlier mean patients. a in that new diagnose we be,

Bo Cumbo

question Thanks Ritu. for the

screenings of XXXX, pretty look it forth just and XX bracket. all in age new stay do between of hundreds therapy to average hundreds born are back consistently, year consistent, through pretty undiagnosed. are sort in children there as because of children about but when kicks the talking point And we groups, at all that XX, really the age that is XX your diagnostic fall around different So children majority to XXXX of expect if helped children, you averages XX on of to age the really went get we just and to we over in,

we new big have consistent average So of we bit, fluctuations range, will stay XX in a throughout that XX The fluctuate XXXX think the the when it hovering fluctuation major will the in age next screening. with identified new don’t year. children being to relatively little trend see born could but somewhere happen hundreds we as

Ritu Baral

point. the Are treat seven, or pretty a profile year you that may good preserved nine old to to have at they disease be they the convince want this eight, of are of patient severity to earlier an and making function idea of I guess want clinicians treat that effort treat

Bo Cumbo

I children what mean being I majority all not are know consistent of of the are but all XX. in and ages prescribed between physicians. - very its You getting five Ritu actually mean children the from

that They a So zero them patients age are or actually to are that that most of five, diagnosed the it’s the soon its from prescribe in there to earlier. to they six, benefit not brother are to was when as group from not the about patients going especially younger is a to convincing zero due the identified diagnosed. of the prescribing those just Really young drug. having it’s ones the mainly because ones find a be physicians as are the fact they lot

And so why effort on we do a lot genetic testing. is of this put

groups and flyers on to weakness only looking really to for out large about pediatrician looking at hang specialist, also neuromuscular mortar muscle not and some of we We education Duchenne. diagnosis child the weakness like talk but giving CKs and

that is go matter of diagnosed hundreds six of therapy. are there and get on age a hundreds just and less So will they when they it’s than boys five to years not diagnosed of,

Ritu Baral

your requirement A safety, the very quick in GOLODIRSEN of FDA, in in with has with CK either very, that in monitoring that the trials them content obviously that discussions was meeting? the there follow-on early GOLODIRSEN, patient it. Got Is something year. up changed issue UK has come that that

Bo Cumbo

So, the answer is no to those. all. all of at Not

with was related any that other in at rhabdo rhabdo over of XX here there The that CK possibly notwithstanding to in adverse continue got In boys event and sites drug rhabdo cautious were universally the so Kingdom, all and is fall is day and hope very it associated so MHRA but should to is the March. explanation. significant issue the an independent at those some noted those. levels and Just CK it, of only potential impact while timing certainly and so fact therapy a a with other March been the children, we no The and the and elevated the therapy known to of back it we remind was on reviews it committee boy that overall there United boy XXX in of that surprise cleared But shortly certainly DMD the we was back boys we it in committee, no it, our UK ESSENCE UK, And not before, of XX related the the the a after on them in nevertheless incidents to all the DMD. at therapy, points represents trial a that that there way, are occurred well a risk reviewed issue all. the rhabdo have that have and the the was UK MHRA it monitoring the the that at one. by hear incidents had end ESSENCE. have protocol and alternative had just of the actually rule in to elevation labs normally will the is protocol and would performance have four looked that ESSENCE, said recommence out has the boys, CK have patients, this impact therapy data, comes stopping sites everyone, everyone resonating from as that should required that there us looked UK, remind the think It in don’t we no stop I are in sites related issue, specific an six in that

Ritu Baral

COGS for that just gave Got you for supply think a one XXXX XX% are you this old Sandy And forward question of final COGS, going at burning to some or X% you range do that’s range it. quick, through the point? still last Sandy, out the

Sandy Mahatme

will give don’t it come There X% XX% the optimize low well, could a number is we I XXXX numbers any and a given for it to result continue have XXXX. is very uptick be to there for COGS as very of over because a didn’t be little left our is that the to burn, down over teens as but small Ritu, processes, why next significantly year and we guidance which for expect

Ritu Baral

all Great. the taking Thanks questions. for

Sandy Mahatme

Sure, thanks.

Operator

Company. Messer Chad Needham from comes question next our sorry of I’m and

is now open. Your line

Chad Messer

taking question strong Thanks on and progress. a for my congrats Great. near

their interested early to findings effect data for ability levels you on drug of that in they Phase that and utrophin guys data biomarker recently Summit partner II their getting I’m recorded? some reported the Your MRI some really Ezutromid, thoughts this particularly

Douglas Ingram

was with ex-U.S. us a what on we have analysis rights we partner, excited they been our with are a with in released about actually their and regards Summit obviously Summit great results, a partner further they very results of we issued as have number and then that and week results for. are we them from for the are we to await very so results well happy there, so we're the will see for XX-week results XX those but we where happy

Chad Messer

Great. Thanks.

Douglas Ingram

you. Thank

Operator

Our next of from the Instructions] line [Operator of question Zhong Janney. comes Yun

is now open. line Your

Yun Zhong

just wanted study? multiple plan the to maybe the around question confusion somewhere is question dose ascending the Hi thanks mid-XXXX and the the the taking a still if without dose pro-win on ascending waiting for an follow-up study it's know of very PPMO single issue much for

Douglas Ingram

goal precession yes, on current course, see of because the of timing how the the with our single we multi ascending dose. get goal outstanding That's and then difficult to have multi will while our ascending ascending to dose and to but is down is dose, multiyear dose the the comfortable to inform single commence level continues we a get dose ascending nail exact the to continue

Yun Zhong

wonder and Okay, PPMO, regimen the dosing you has this efficacy FDA protein dosing you and them less same been with dosing the some on but I comparing frequent comparing versus with efficiency think versus less potential production one have just important dosing prescription talking maybe better that or given about was which do frequent potential issues efficacy?

Douglas Ingram

the from extraordinarily have maybe ultimate the to single it's we dosing what obviously extraordinarily and multi extraordinarily dose but be we PPMO, is dose dose as regimen which very but well, a get will multi skipping we long compromising here to ascending well that dosing efficacy not obviously our get Well important compromise both that interesting is as dose. actually better not making it Dystrophin amount robust get found ascending increase the regimen expression the efficacy so we and of case an all it boys is we so better would dosing a ascending better animal goal into that we the single muscle of study. that the our issues, be you get in said, know so and very as all we important trade-off not informed we in for to know are efficacy complete be single much between great With expression may the and outstanding with of What dosing. model our the as better to Dystrophin better are exon a continuing a

Yun Zhong

Great. Thank you.

Douglas Ingram

Thank you very much.

Operator

Thank you.

of from question Debjit comes next H.C. Wainwright. Chattopadhyay Our

now open. line is Your

Debjit Chattopadhyay

for squeezing I in. few have me out here. a Thanks

concerned [Indiscernible] is program eight the expecting as were to far get right. to mixed XX as XXXX you So

in the the where you study single dose are currently ascending goal? So versus

Douglas Ingram

low, ascending as very you we dose clear I'm still slowly. and single you Were know at we significantly know move sure as so levels up start study very therapeutic sub are

far significantly doses potentially from away right. are into we So getting therapeutic

Debjit Chattopadhyay

here, of vector than sustain expression then construct for the recent Micro-Dystrophin ability full is in a the or driving XX% and expected stress the boys expression relatively Micro-Dystrophin it for the Dystrophin investor at greater loss because event Okay, a bar as age. program the lightly high What is Dystrophin lower loss, levels. you a specified vector to versus

Douglas Ingram

that That’s use many from of we amounts comes boys a have have much exon benefits functional nearly clinical great there seen types. have the and reality optimism times that small XX seen to showed Dystrophin to reputedly is [indiscernible] is what three random as and model, skipping is who back question exon you that on amounts this amenable extraordinarily once mild answer low phenotypes yet expression we is animal point you correlate the ground a really out have of

think in may lot be models, would and at I have amounts are very optimistic. of happy We animal at XX% know are we well but sense we seeing actually significant even XX% that we in right,. models one I meaningful, need to to get confidence, in We that meaningful in therapy be. don’t we very, XX%, gives in us would be may level. be way a So know said but gene XX%, it we if very small very don’t to looked I so would X% to Dystrophin expression it you animal get say expression we

Operator

Thank you.

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

open. Your line is

Gena Wang

just questions. May taking mentioned you be question, Micro-Dystrophin my the be X%, you know like happy. that level for previous you the Thank you very will XX% follow so

you look higher if data for So you bio later this when had marker the what at into the like will would the expectation. to is and be year, that to your the plan goal dose level the we below data

Douglas Ingram

the we is right because model It’s so go an a of note significant actually see the significantly expression. also and that therapeutics two would clear that which that that dosing, that XX would on with things now we dose. significant And therapeutic to are are we robust work believe is we're, adapt we will we our to preclinical based I we dosing note see times potentially that safety are if XX at and and that many working animal higher than well also appropriate ourselves I the to program, obviously Micro-Dystrophin things, like done exits but respect of where what package was

event in that our So current very is we happens adapt don’t, certainly a that think need planning if something going ourselves have therapeutic and good but dose. we we about higher, to and chosen

dose mistaken, I'm not sure the gene In I'm fact has higher if than not there therapy a to XX. two in XX times been

Operator

Thank you.

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

open. Your is line

Unidentified Analyst

Doug question. those rare Hi is expansion Are for alluding identified been more that [indiscernible] diseases beyond specific are guys you from opportunities timing or Hartaj this making just through through you rise into they prospective. for Singh prior in evaluating there mind and taking DMD. would just the eventual other diseases and you as broadly, individual about strategically how those thinking more division the then you keeping the that

Douglas Ingram

of probably So think generally it at one a I looking things, broad very way. a speaking with couple

we in kind that of So we're this going with real talent very abilities, go do we obviously the the about that are we expertise, right way, in be house a the look disease house. sure rare broad next, that where have and to at there make said to we inside targets thoughtful neuromuscular that leverage our

of out own the getting we dangers we sort do and do look pretty then fill we of net spread at abilities, wide in wouldn't beyond kind things broadly, the about go your life going DMD. do that, and beyond of but of a we when sort One out question is then

capabilities that utilities along would the there an things profile opportunistic looking you laudable is The place two know extraordinarily I that beyond to addition signals significantly had be means we're externally. that DMD beyond likely PPMO safety internally take and times way, and RNA both gene gene and that division positive get for an going therapy really approach that once if that with build certainly therapy In technology DMD that Likewise see gene positive well. signals very with where you leverage significant had think have expertise going different this. at the and were we ourselves like we as of manufacturing robust we then advantage when might targeted are to certainly therapy we expertise to a we we have

Operator

our Piper Ed Thank from of you, the Tenthoff Jaffray. of next line question and comes

open. now line Your is

Edward Tenthoff

where the two, Great, how many to thank you very I’m say informative EXONDYS I right this straightforward of have progress you on XX make. give really much maybe boys out a are guys one one range you now launch. very comes update from Thanks us pricing for coming voluntary per the discussed time so question of really the year much and And got can $XXX,XXX and on year impressed the guys at helpful, net time. by that's continue to an

Douglas Ingram

your and much Thank comments. thanks you for very

before second haven't we're now, we performance, you is as our epidemiologically used silly in use have I before still we things say of that surrogate know upon we innings the we revenue we that have lot pricing will where so and are a analogy said really we as penetrated sports on patients boys that are this where headroom goes if net of lot number provided I are pricing therapy, market to haven't to So - the changed more do use far a we there today. goods as not of you exactly right is And significantly with deeply so DMD. revealed the

Operator

remarks. today's to session. to you, closing over concludes gentlemen the this would answer and Ingram Thank Doug I turn like back ladies for conference question and

Douglas Ingram

and evening. brief technical have Well thank all of much. and Hopefully time apologies much time, questions very your really Thank the been your answered issue. Again you for everyone. appreciate for patience very your this you

Operator

conference. gentlemen, and your today’s This Ladies today’s you concludes thank in participation for program.

a You day. great may now disconnect. Everyone have

Sarepta Therapeutics (SRPT) 2 Mar 182017 Q4 Earnings call transcript