PTCT - Earnings call transcript Q3 2017

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Operator

Good day, ladies and gentlemen, and welcome to the PTC Therapeutics Third Quarter Financial Results Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] As a reminder, today's program may be recorded. I would now like to introduce your host for today’s program, Emily Hill, Head of Investor Relations. ahead. go Please

Emily Hill

thank XXXX and joining to us Good quarter you corporate our third results. updates discuss afternoon, for and financial

on Christine Utter. our is Chief Souza today's Peltz, our me and Officer our Marcio Operating CEO, Financial Principal and Officer, Joining Stuart call

matters, of that third-party potential the of potential These for you our statements of statements than we I include to had in I historical like regulatory implied and set making the variety by anticipated for those statements prescription benefits EMFLAZA guidance, commercial FDA in over ability EMFLAZA, for forward-looking outcome all before expectations ability call for treatment of risks timelines whether for factors in Before the statements the received other Roche, of including the XX-Q timelines, as regarding nmDMD, Form anticipated collaboration will with interactions forth our to with the and concerning integrate resolve either discussed quarter resolution our business or FDA from facts, the including related statements judgment Stuart, populations matter a be the the to margin which would remind today those our today regulations, obligation website. and our special respect from benefits to Translarna into addressable our changes terms acquisition, of is payers patient law. of third-party on dispute of extent the statements. and of for PTC statements with financial of form to response the manner, including represent update with and complete for in our and including DMD. anticipated success respect expectations connection the of EMFLAZA, EMFLAZA outcomes our we future materially differ SMA the treatment required to of forward-looking access second NDA and under statements future our those other our with SEC undertakes note availability heading formal our as and ability with products to except Such nmDMD and a result reimbursement regarding by to commercial of Translarna letter the treatment clinical, FDA risk of realize acquisition, coverage to pricing, the EMFLAZA, class NDA, what our continued from and the a and for of requirements reimbursement, Translarna in publicly timely approving resolution and impose forward-looking expressed any of for uncertainties the anticipated risks additional dispute request adequate any filed EMFLAZA forward-looking available launch as Translarna any may other of results and the secure our to EMFLAZA to payers commercialization Actual outcome no

and during earnings over a in Stu. We Information of to to of non-GAAP available measures reconciliation this that, and use certain our With will today's me call. GAAP disclose GAAP financial information is call the let non-GAAP pass regarding non-GAAP release.

Stuart Peltz

thanks call afternoon. on And joining this Emily. for us Thanks, the

medicines we patients rare disease are rare progress global neglected company for to with outcomes drive As the with making growing are a and commercial stage disorders. we and innovative a pipeline pleased better with deliver

going we years are to on where plan year our important we in to we be and As it ahead. the in PTC, the have is we will founded accomplished entering over be will it we what decades. important reflect XXXX where Furthermore, last since is two XXth

important There is work patients our to worldwide. strategy amount As Translarna of all remains bring for neglected and a rare champions treatment same. with to for access the disorders, considerable patients to still

We States We drugs and to important around are that Research and like access. change work to Development action. this to continue the so decision commercialize others pipeline We plan plan United to our believe with this disappointed access patients patients therapy. to the build and world to FDA have to have these

now class Our to from edge can continues and demonstrates commercialize best be process through and discover, develop innovative mission that EMFLAZA commercializing medical to important the a outside The products go this own Translarna also demonstrates in are we that will to to concept for patients we willing where cutting variance an discovery rare bring diseases. for to that Commercializing basics internal in we our that go treatments of we that we and unmet need efforts and XX different look are access. fill research in countries. development taking think

will patients. use to continue the of our having operation grow leverage in two to We to commercial is products evidence patients. both products and these commitment continue have for bring to we the best now of position effective commercial an global that PTC and

new will clinical next which earlier lot including will updates Translarna We on today's the today's we voice on promoted like call, the our growth. drive the Marcio Officer. introduce for executive pivotal of phase this to SMA strength. the States, and our our provide summer status to an program and Chief growth, our Marcio also to I'd have Souza Operating development studies internally regulatory a commercial financial advancements call to of United review commercial in our

will in end from and strong Officer. Christine strengthened Financial thorough of management leadership with Principal team call third individuals financial reflect the our We the These outlook the complimenting review quarter our already Utter, place. a strong

reported to strategy on and to a aligned performance. In led of global, $X.X quarter reported R&D has Translarna and execution revenue $XX execute of growth. strong XX% over In on year-over-year the million. the EMFLAZA third revenue revenue engaged a year. quarter, highly to the This financial our commercial, third prior continue million, are We increase

of full raising year XXXX are to the to which $XX for from million date. million $XX million EMFLAZA strength our We reflects guidance million to launch the $XX to $XX the

the available. you in thank United both the region continued as launch the is by it sales every in strong as in performance driven well growth where States EMFLAZA Translarna's was commercial Our

response week, Translarna. received we letter the a Drug complete Office of last announced I FDA's we As for Evaluation from

While to we believe with trials and benefit demonstrated world action, positive clinical risk continue extremely real the in disappointed experience. are both we at this

expect course to resolution formal dispute a We of file interacting and letter this week Drugs to Office the this appeal. the with forward during look New of

for Committee I'd We this take on moving United support to thank heard to moment but in other wrote of during Translarna caregivers, a Many speak to and patients to and our Before community process the continued the impact Duchenne in had to lives. updates, States. about the physicians. the hundreds to Advisory and letters FDA their has their dozens their families from like travelled

the a FDA to the States witnessed from outside Translarna by had same patients access efficacy DMD patients letter States We to and to physicians the the patients. lots also U.S. and commercial the bring signed benefit Translarna for to they years. of and saw the who benefit world. access outside DMD in support mutation experienced United United have testifying their physicians submitted Over tremendous XXX and patients of the We strive around

foundation. focused also partnership Roche the in our with on are therapy We SMA oral SMA and developing

the study to term placed SUNFISH company our phase patients generated since has speak research in milestone. us mid-October transitioned the that in an and date advancements to forward SMA coming study and longer EMFLAZA to $XX in has in development the months. complete million I'll these a the XXXX anticipated triggering position. commercialization position financial the for progress this critical for pivotal commercial strong innovative look type II to program. with the be in and of made the development to As been call research III I This the to company and pipeline. Later FIREFISH in in global drivers pipeline We type patients Translarna advancing made in a The sustainable data and us a that is

now have me commercial Marcio you on our with Let and Marcio? more detailed activities. updates clinical provide

Marcio Souza

Thank our team and to PTC's success forward have joined look executive pleased you, Stu. ahead. I'm to

start U.S. with supports the assets $X.X firm all eligible older EMFLAZA which only should best Translarna, treatment this for quarter. approved six of million We with ago strong with starting patients the about receive possible launched our this revenue reported the that U.S. view in commercial which in the and performance in belief five for Now, years our and and let in DMD care, evidence quarter's patients EMFLAZA months was DMD EMFLAZA me for the that the patients the includes use both in our EMFLAZA.

as patients presented several EMFLAZA. benefit aligned data of XXXX own the pocket as Trajectory I U.S. remained well those The the by patients and both data Collaborative patient pleased strong. our that EMFLAZA times providers to cTAP which in for have Analysis currently and to out from am confirm over to for EMFLAZA has the zero. healthcare close that clinical receiving Project view with of the independent forward of costs s fortunate reception are We been are

from to continue positive We and patients both receive physicians. feedback

therefore While path to $XX confidence Duchenne's the XXXX. update are year moment raising full and us for we on forwards guidance gives million the the

far results these work is While our are over. encouraging, from

and As we resource takeaway moved to on launch focusing we issues. of two intensifying the our are our the investments second phase

of The standard awareness the is of care patients. to first the one for and benefit knowledge naive improve as the of EMFLAZA

their remove current switch for to barriers from patients and who is second providers The want to treatment.

communications. campaigns increasing our combination efforts We investing of are scientists a Duchenne's medical deploying in the and

correlates fronts data the of all to whom also of This the will that four by death. U.S. in of only ventilation patients the part correlates in bigger milestones relevant loss which previously delay did to motivation not was ISPOR all supports on loss communication or scientific showing to acquisition significant of their of the May had that because learning EMFLAZA available patients. use for data eligible our such patients, strategy and We up mechanical loss are in as of ambulation a history on not at muscle but because itself all they eligible years the suggests from the most to On to we bring which a other and focus to with answer is time benefiting have This Meeting International function continue proud EMFLAZA delay the therapy data in ambulation EMFLAZA presented ambulation. change are this of access. to

study [ph] conduct psychiatric request from will study. six to for such we Lastly, allow an completion received EMFLAZA a upon conduct exclusivity. just us We to market months which of FDA the plan a additional written

the launch globally from to process XXX since Now continues and And in begun have seen and years have on and and tremendous Eastern most globally. patients' switching connection we to we and in we than Stu more expand said demonstrated recently three are lives bringing time same Translarna, XX%. having Middle globally. and as U.S. East we patients therapy continue to Translarna physicians impacts Union committed The European patients the express in reinforce adoption Patients FDA benefit into Translarna in the physicians by Europe. our America. the to we compliance to the uptake Latin remain region. support gears remained In has to over overwhelming and every growing at the the Central have is confidence expanding

globally In growth will patients as to look penetration. fact, increased driven we be XXXX by project double-digit we which

too mission of Our vision care is rates which diagnosis, globally. DMD our Translarna DMD will disease ageing for early standard with with years that happening five and reinforce eligible we patients. globally To available for Diagnosed all make than patients to education, least patient higher proper still for of investments at is counting. genotyping improve beautifully fulfill together late in every will

called not diagnosed, are is finally proper available. When addressing Our issue. and oftentimes genotyping On Duchenne this on focusing campaign Take Duchenne patients is fast

groups to We have several address globally to partnered with genotyping. access

today, pleased we While forwards. further efforts we with the the to expand are results moving plant

boys Lastly, our lives the not with support the including patients of but commercial to mission physical overall men and steroids only DMD suffering of improving aligned back in standard are care therapy involved debilitating practice. the growth efforts of with use the this we young of disease. efforts improve of These Translarna, and of

year. one on update complete in Translarna aged of and and development year recently old. PK short active patients [indiscernible] next problems study DMD. for study that me Translarna completed The on we our the early exploratory to expect to X Let phase a now are provide We transitions which years X children

The term second third patients study, which enrolling the in is quarter. XXX started long our

completed there As specific [indiscernible] EMA Outside we obligation of enrollment our during have anticipate of we study XXXX. a a is results reminder, approval. our for DMD and

will patients We were Stu? with on a rare neglected drive are confident back now for well commercial the to I that Stu. strong value disorders. and are hand capability equipped continued lives with to transforming focus call and

Stuart Peltz

Thanks Marcio.

gears splicing manifests rear small is to used Let program This which our me generally muscular potential the for it death other discover central This genetic that is has atrophy not molecule based therapies on only genetic tissues been spinal platform. new affects disorder disease or and in early technology infants of well. neuromuscular a now cause switch and leading nervous as body, to in SMA the another but systems, the toddlers. life whole

is such a by around central nervous and Roche oral the tissues robust collaboration muscle, SMA a We an with therapy throughout Foundation systems. bones, allows it including program in have splicing believe exposure body because competitive We SMA and X should that peripheral SMA affected advantage. This liver, modifiers. oral the provide as key

early be blood a meeting in In will in fact year. supports presented that This are the mirrored at preclinical evidence CNS. medical pharmacodynamic data next major effects seen RGXXXX

before, ongoing. we are registrational As two discussed there studies

of a half to We dose the enrolling. five entered was first SUNFISH depended exposure of an and report that may treated Congress. presented with be in Muscle phase first pivotal to the from protein. recently interim days and the placebo, for the the of the is World XX are increase Society recently fold An has RGXXXX up SMA SUNFISH demonstrated stage escalation two cohorts versus in and proud analysis Data at

therapy XXX described tolerated and remained leading to well presentation, As patients there safety days. up withdrawal. drug been to in have no RGXXXX related the at in doses The on findings patients all for remained

The to targeting FIREFISH support label two-part JEWELFISH program including SMA as compound only program which X for also JEWELFISH. motor [indiscernible] studies open to previous as type pivotal this is expected validates technology infants stage in trial SMA our it patients, SMA months. slicing know point therapies, an RGXXXX of coming modulates selective is can access the assessed RGXXXX. in identify trial SMA study The infants from label splicing transition we is patients can not directed of candidate the an the by other sit open type scale. end The second SMA registration allows primary process. SMAX the our that important is has without of Bailey The that ability gross

utilizing technology the We are collaboration. SMA of now this PTC outside

familial dysautonomia. programs disease We internally pre-mRNA preclinical additional are pursuing targeting splicing Huntington's including and

that R&D upcoming programs to now an share as will I'd highlighting hold Officer. more call turn as we Financial next Christine? about slicing to plan these We like to year. well at Christine Principal exciting the our pipeline Analyst Day over Utter, our

Christine Utter

press assets. date commercial quarter our Thanks line allowed reported million $XX details. remainder for the release dollars. up this million was guidance our that Starting has for revenue year $XX commercial the The on the year EMFLAZA to with at to Translarna million our million results third to $XX a $X.X Stu. we generated I'll for start to up of EMFLAZA our across refer in I million both for well revenue was for results of full to today performances performance Earlier with in increase our performance a year-over-year strong release of few and date a comments financial summarizing which the financial quarter EMFLAZA set issued revenue $XX us to we of XXXX. you to $XX the details third us combined increase full XXXX. top with reported results, quarter quarter XX%. the the from million

the commercial with of We last mutation exchange nonsense This Translarna state. existing year the U.S. to we remained $XXX period $XXX This territories. quarter continued on of million revenue the a in rate Translarna to in net for the guidance continuing trial million million. from As guidance brings and dollars million provided reiterate a SUNFISH a as new guidance for range our of reported million the pivotal revenue Translarna in guidance in expansion We the both declined $XXX which same DMD $XX.X $XX assumes over call. our in $XXX growth XXXX the approximately October current outside from Roche This noted, our million XXXX. strong million of of achieved and $X.X advancement the milestone includes we of loss mid third to for full

Our from existing in leverage flat as to declining continue operating now losses and revenue two third we period the commercial grow XXXX. million our anticipated expenses quarter continue same Non-GAAP $XX.X are were approximately to base R&D infrastructure to our products. relatively in the

of previous of Phase $XX.X launch increase Translarna term research the regulatory versus DMD approximately quarter the costs costs. certain prior increased the the associated have expenses SG&A expenses studies, were EMFLAZA. reflect study by Non-GAAP for the While running with million and major driven long initiation expenses XX% XXX third year we an mostly development of our III now quarter. SG&A and the completed XXXX of increased

We of million year and full the are SG&A reiterating R&D between to million. our GAAP $XXX expense XXXX of $XXX for

Excluding of U.S. non-cash continued the commercialization are our compensation U.S. commercial full-year well be $XXX and are of SG&A outside clinical investing the and in the the expenses to expenses be as development the anticipated of R&D of as XXXX These and million stock in approximately estimated to million. primarily pipeline EMFLAZA research support launch of in million, candidate non-GAAP and $XX expense base Translarna of expected between product $XXX

over marketable now $XXX Our revenue previous operator anticipate the with to call securities. question-and-answer $XXX the Operator? guidance increased of to our and start million complete session. hand XXXX now to our million. was task I will ending Due guidance we cash to over with the approximately year

Operator

Beatty Instructions] [Operator comes question from Citi. first of Our Certainly. line the Joel from

please? question Your

Joel Beatty

questions. the taking for thanks Hi,

this in you to bit bulk seems give due in like Translarna, to part in sales past contributed Latin Is assume little America. that for sales to compared sense sales last bulk a that? there that the is a is irregularly, come able were on down this it in Are a since previous to sales quarter bulk much it quarter quarter? high is one – of First safe seen quarter we've

Marcio Souza

it's the Hi Thanks call. Joel, for Marcio.

we regions, we continue be bumpy. guide on and you will not or the forward orders specifically but do do to their on irregular As America Latin know, moving councils from expect

are is we guidance a year coming important like for expect the any of back region. don’t here reiterating different order that we think really and the for part I the

Joel Beatty

part SMA the also endpoint the I what the functional get Type from and is I? to that, helpful question thanks. trial. then from II see that advanced data of I functional Is be and either data where for one That’s to and or says two we patients trials Okay, pivotal could timing be there it on Type other of and if one see these program. pivotal could III to trials possible able the Type functional so first

Stuart Peltz

thanks that Yes, for Joel.

the Roche So it’s poster SUNFISH for they'll a there's results in we X next milestone the present and transition know trial early did you in They endpoint. you transitioned or we year been many that with talking as in collect results there - the just from trial of into probably. about the that's occurring in and data some that and to of you've as pivotal we be Part we'll received as XXXX so time the plan data know a presentation be the

Joel Beatty

Okay, thank back I’ll great you. queue. get and in

Operator

Thank you.

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

question please? Your

Tazeen Ahmad

you've questions. taking up for Stu, nice on a had my launch. obviously Thanks in your afternoon. EMFLAZA good Hi ramp

question between program and that of the the from can to quarter. have of impact that growth wouldn’t any on the number there XQ XQ, for holiday SMA in an And that? point for fourth well. or you full expectation revised I then as strong pretty Is more there is but kind us indicate growth some a there sequentially a had trend You you much EMFLAZA year

Stuart Peltz

oh Marcio thanks, wants Sure, to...

Marcio Souza

Sure. this is Thanks Tazeen, Marcio again.

in early So a days the few launch.

know, very far. the we are are happy first you in so with definitively the very months, As results and six we optimistic

build would in expectations guidance. pent we patients. There know so line up was expectation few up there was the We continue the no transition so months. initial are There demand that would that with first of [ph] that patients the a raising our gaining is is it we're in build

I faster think raising it is a the we transition going that's expected than guidance. we are so why

Tazeen Ahmad

your any have right now what idea Do your of penetration is population? you targeted in

Stuart Peltz

are we monitoring Sure that.

this right projecting moving call been guidance are going to closely numbers we're giving or We we've not that's this now, very these forward. looking in and be but something

Tazeen Ahmad

impact do now Okay, on in and SMA well will instead say of, then treatment? the relatively better as treatment fitting envision justify can using be guess how other would how potential to with you a this would you know program, this drug EMFLAZA I the as therapy, be the into in the let's to, of for exists be it's what what we great gene product and that this combination understand with addition drugs regimen expensive

Stuart Peltz

that. thanks for Sure,

you body. as oral distributes is that an RGXXXX drug the So may throughout

whole that tissues on, really while tissues earlier were So promoted think that to initial is its it go the all central to advantage as SMA and remind protein described the in nervous distribute one go - that I comments a I allow these that can was impressive molecule may I multiple you other important to SMA on. major remember in my affects you preclinical that quite data of in and it issues affects all slicing bunch system the

well think while other the be So in best it able hoping class as class terms in it both SMA tissues. may we in the the we of are for CNS to not in make as to be being protein potentially first

due in the activity, patients. monotherapy of looking time in obviously to or makes but combinations as and best for at now consider terms In it also demonstrated course monotherapy, sense as it combination SMA we're what's just in

Tazeen Ahmad

already is asked we expect readout next for SMA? data that so should also the the might Okay you, person and sorry, last have when

Marcio Souza

SMA presenting get that important the able be that upcoming what expect the SMA meeting, other to preclinical tissues Yes, importance that is the well, think people data again about being again I part FIREFISH really patients. drugs we'll I so to of at for think one talking and in we are about so early an be of understand talked be as we'll to so XXXX

Tazeen Ahmad

Okay, thanks Stu.

Operator

you. Thank

of comes Credit from Our from line Alethia next Young question the Suisse.

Your question please?

Alethia Young

are couple, for you question. curious more kind Hey maybe you of And don’t just a now putting some patients and opportunity of guys. for you on know the there or hurdles multiple of ex-U.S. the the if I cadence about just now about areas maybe? about I the more also of give I as remain EMFLAZA a discuss think the the as bit I'm site Stu, the more to where you us taking a little people? a then Maybe around, underpenetrated? you Thanks. [indiscernible] having are talk this, you what's doctors can I do trends educating kind there? trends Thanks but mean you that guess you've rest then, far know, little answered Translarna, how seen my And you can thought,

Stuart Peltz

the let wants then... and little talk but about me Sure, I’ll a bit to EMFLAZA ask, Marcia

Marcio Souza

Stuart. Sure

segments patients seen like of we've types patients EMFLAZA on, on these been broad. so of scripts pretty terms of different coming So in for all right, the it's

You question right the points relation in base know now. that important, your that it is so two quite very think we doctors, have are I so to a broad one

also well on and outside as has centers scripts like so been the both Stu but with up because like just fact of are for seen a wants in of with accounts centers, that. coming as down conversations growing, you like seemed NBA team the having the raised me we multiple happy fairly that it's number but seeing they're accounts obviously effective not guidance scripts happy Our main, mostly going very are to... doctors with we've variety the faster, it's how very

Stuart Peltz

on standard of too... care be also fact that a superior think discussion little of Yes, and I might education, bit the useful maybe product the the

Marcio Souza

Fair enough. point. That’s important a Stuart. Thank very, very you,

very indication during remarks phase the they on on So staff launched my updated the the in to now U.S. initial use and on be were medical to using just second treatments phase actually help as they the while being lot questions before well for a terms the resource in the benefits affairs answer mentioned we or doing guidelines as education, even the of is really would and with markets both was to vast generic no was right in need of you're mostly the of patients. patients that and our these The were some in as the call move we really what there in from that field. completion I more education launch earlier EMFLAZA

patients both it's also a are with that opportunity very who of so the believe educated. So are never have switching are to therapy steroids, for of on they truly more who the looking treated the the small, are large ones not We market part do because EMFLAZA. benefit are

that and that also because should we this out acquired to the EMFLAZA we data that’s treatment data during the we believe. and mentioned there As best acquisition generate patients be on will get to continue

Stuart Peltz

the of as Marcio the as said would be we've that out data History, been had well get placebo And a is there say of the that as that's a EMFLAZA since really publication think been should Natural be able I Lily lot to things as this that the or from of could recent acquisition of well there's shows we there and Synergy the from on superiority patients that a like drug. be data as EMFLAZA work

all in And posters with will literature. a there's this that be and out of that occurring getting new the lot education both to data so in be soon I think is

Marcio Souza

and it expecting to year we countries good regions Europe coming patient had digits like for good just result so America. really mentioned job And just we're next believe as question, a Western we was answer on to very growth in be the going where like your right this is maybe double penetrating the Translarna a main Latin well. We

all biggest We regions say lot the I be the those see the efforts. as well penetration as that's Europe going Eastern but growth where focusing of or forward, expect to to as the we and we're Central the in should is including move and in smallest well, East and Middle

Operator

Thank you.

Capital RBC from Our next line comes Matthew from Markets. question Eckler the of

question please? Your

Matthew Eckler

Great. guys. Thanks for taking questions the

more to at up a applies and So quarter-over-quarter are I high about low a your Translarna, you're contraction revenues growth. first guidance year end XX% full implies bit dig around guidance in end - of looking wanted specifically you at this actually the guidance little something implying a the at into while sequentially of XX%

you my revenues low question guess the QX? slowdown a end first is are potential in I why look, the of up in of here move not So guidance, anticipating

Marcio Souza

from you you're So right, your a is the that are correctly through I question are guidance question looking we asking? the Translarna when where guidance understand this year for

Matthew Eckler

and be up year-to-date QX end million what is move million It that QX, million guidance $XX about in why sales million to is, $XX low done in about in $XX there the full in, end million so from the a for $XXX to and you move $XXX the is there concerned decline so from you've on your high anywhere something not for year, you're end guiding the so date, significant $XXX to implies obviously slowing in on million right could that that low of revenues. a

Stuart Peltz

size the of we're No, there other I'm that's how come staff we're in oftentimes specifically are it Again concerns forward of terms is patients, that to because having. reinforcing we of change mix no assigning the the closer major the as looking the holidays. guidance

changing that expectation. So our we you no that in guys is off give is sure fundamental line guidance for that is that a realistic business making the with really is

Matthew Eckler

me mean guidance for but low correct your I'm QX of be slowing this end I the revenues implies about wrong Okay, if but correct? to is that this the in thinking again, way,

Stuart Peltz

we’re right? size mean is, would thinks not the you The there in range end. quarter guidance the if lower were but guiding for to our be doesn't guidance end of So that for be lower the that smaller

Matthew Eckler

inventory or separately and do current then guidance Translarna revenues or understood your stocking? Okay, include any build

Stuart Peltz

No.

Matthew Eckler

got okay No, it.

all it's recorded a So on are revenues basis? sell-through

Stuart Peltz

correct. That's

Matthew Eckler

Okay, the press very then Translarna, perfect. Very for so a seeing compliance have greater just on in me, the very, helpful, for discontinued last you. thank from compliance there discontinuation. kind to mention you of release but patient some XX% you're than so reasons are high one that the rate And of what one there the for obviously

Marcio Souza

a really, taken rates but The and little and on, like a is So drug, for continue on bring are when results children expand to always day. looking school we’re that good seeing. three times to to so the that seeing of young we general bit are the patients pleased We some just this are have drug. me let so first believe this very so teenagers right? really any XXs on specifically this with Sure, is benefit we in them linked of a is to therapy the the really compliance

we results in incredible So, as general.

of discontinuation. terms very and We the any given sequentially the choice has number numbers mentioned. is countries, not The I related to number as small on haven't in been very patient small mostly are but in different drug

Matthew Eckler

it. got Okay,

Okay, great very much, helpful. very thanks

Stuart Peltz

bet. you Oh

Operator

Thank you.

Our Blair. William next question Prasad from line Raju comes from the of

please? Your question

Raju Prasad

there PK question. the if on the Thanks anything was have biggest like to for dose Translarna? guys to response for is children dose explained on one shape you I the the the get felt taking in Translarna, currently X outcome response, One comfortable more show kind X I to that be at to you'd from them the wondering study with of bell bunch just FDA contention the curve. of of able

Stuart Peltz

Yes, thanks the question. for Raju

a form of one of a a your had is that I terms asked that who's was out we who done missed normally it. think PK shaped had two was the typical a the model? in a do a that think essence that That pointing yes, questions in we question I towards the show will person response terms person where have is, things former by analysis the the curve. but and a one but you standard it's think in to would we in that PK. we more had did, one answer did that just formal model been they is actually person PK, is formal of that we're there but PK I model of demonstrated is bell the done that demonstrates sense done to person that's procedure done, PD And

response, look that example something a does, was important I them for it's part that of was will our So appeal at. good to included but think model be as of missed, it that

Raju Prasad

maybe thanks mean X,XXX more color All just patients can quarter-over-quarter? many where you're majority Is as a give a percentage bridging just or patients then well little on a on general are and you EMFLAZA program? in as bridging that growth as. Stu I right. seeing program the the the far as Great, how in of

Stuart Peltz

relation something not are need are where the between important No, the to to in that's patients The right? and So of uninformed in who very to we’re EMFLAZA being seeing the split EMFLAZA shipment get there. commercial because bridge. growth of a majority commitments we're in for or us our all talking access bridge about not life is who specifically

there. scripts this off So it's period efficient are remaining line really every getting using Therefore our patients newer so the we're day. very been are not bridge, we at

the exclusively transitioned some growth but now coming bridge, that's from them from obviously is the So are of bridge.

Raju Prasad

question. Okay, Thanks for great. the

Stuart Peltz

welcome. You’re

Operator

Thank you.

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

please? Your question

Unidentified Speaker

is Translarna. Gena. for EMFLAZA [indiscernible] for one questions, Two This for there. Hey and on one

can wondering launch, EMFLAZA any sales if then have For of with example And you cost Translarna. on you I gross color was just to a could associated color launch? and for additional some the provide follow net up on the I for provide

Stuart Peltz

Sure.

start to gross I'll net. the with So

to we the and So like on It’s expecting public so net has the expectations. been payers the all on between we're of teens pretty far private gross high much mid move and that's expecting. were within that’s are our expectations, discounts this We brands the to out to original patients don't getting are exactly to associated that see what with much, are expect not happy were was that, there. so in our in what we

just would I'm we're access. giving there that very not necessarily I need to get of growth. keep patient no now. details other to happy promise able there the being patients be would happy very to we're the reinforce going launch of terms EMFLAZA our to we're with wanted and the In access not

these broader we’re direct you shipments keeping either and = commercial. for are for more drug coming them in bridge the on seeing groups So

Unidentified Speaker

some us to kind renewal that Middle opinion the taking timing expand about on in trying CHMP Central like understand just for other you more of do anything Eastern for you're approval remind barriers and footprint Europe ex-U.S. Translarna you're expansion a Great. bit little the Thanks that extra to to there. specific has there the efforts any and U.S. the could And of steps the the approval? just going conditional now

Stuart Peltz

Sure, so procedure our me for start renewal let the approval with in Europe.

So, every first quarter procedure we renewal. the will for submit

internally not our We it foresee to the lock be we few database with that, any track are things at procedure and a year this We're the like did requires submitting of be do XXXX to that. so issues just this. on beginning like other to going going doing

in again, a CHMP in as the that’s can it's general. go specific to follow and going you there's they timetable So the through find and website timetable and

And Middle in it. we would you an Eastern and In magnitudes the East we is Central investing to we're on center And I Middle in on the terms education. region I throughout mentioned are I regions. all would that regions education look that major of year. we're the expansion call issue to in so in why there the you is say so regions I barrier sea like world not on next of East are are highlight before all in important working specifically aspects working and about be that and are and different if but one you, and working mean on the a When into components going Europe, few on two that specifically to

efforts the our increasing and So both the we're on region going other have to be south education in on with partners DMD. we intensifying our

is access to genotyping. second The

a we're lot increase launched been started Middle next happily genotyping the globe pleased is have level that every very investing a the sequencing make getting partnerships access of since We've sure to one gets be patient to three on of very, area to full throughout who that’s to We but the fast. ago fairly with generation year years with going next them. We DMD that specifically genotyping. lot East, proper we diagnosed in

So that believe access and be physician if they appropriate that the to are eligible get should it. patients the Translarna to they

Unidentified Speaker

Great. Thanks a lot.

Stuart Peltz

You’re welcome

Operator

question-and-answer to Stuart this And today's you. further remarks. like for program Peltz of program. concludes hand Dr. Thank to session back I’d the the any

Stuart Peltz

thank you on Okay, thank to thank and your for questions. want call the you. for being everyone I today

the As you can outcomes. better time today. lives of and moving patients your we're focused here these in for see on forward Thanks for transforming

Operator

conclude Thank you for in conference. today's program. gentlemen This ladies participation the you and does

disconnect. day. may now You Good

PTC Therapeutics (PTCT) 2 Nov 172017 Q3 Earnings call transcript