PTCT - Earnings call transcript Q4 2017

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Operator

Good day, ladies and gentlemen, and welcome to the PTC Therapeutics Fourth 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 follow at that time. [Operator Instructions] And I would now like to introduce your host for today’s conference, Miss. Emily Hill, Head of Investor Relations. begin. may you Ma'am

Emily Hill

afternoon, XXXX you corporate fourth financial good to updates joining discuss results. our quarter and Hi, for thank us and Thanks. and full-year

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

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

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

Stuart Peltz

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

review providing world. year, States. the access in the XXXX you Chief commercial the use we Furthermore DMD call our around patients great and and full-year our our of will Marcio which phase will quarter and the Joining fourth on know, end included which clinical our to Christine updates, financial Officer today to strong with drive a the we was me call expanding growth. Operating a and Financial Utter. acquired our Souza and profile on Principal Marcio as our provide grow will Christine United EMFLAZA tight efficacy based are development its outlook. and very Officer, next thorough Translarna differentiated continue are

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

are that We is proud operation. XXXX our in XXth year

the patients rare developing Duchenne We own. with bring commercialized beginning to decades, Translarna on with Over have Muscular Dystrophy. we therapies disorders worked past to our discovered two

We have more been trials XX United in have patients ago. years who earliest States our clinical on since the trial extension than

patients, have because the therapy been diligently of working with we treating to working to bring we learned for and We Translarna including know standards States. patients of early around maintaining so everything patients, care, United the world, improving much these from and importance matters patients Duchenne the including have

patients an forward accelerated We towards U.S. to around important this a therapy. pleased the have access that approval others to so have like are path may world

of forward, so that patients to have therapies mission discovering, continue differentiated commercialize continuing developing disorders. to This our execute development to to research for Looking we and pipeline to and therapies commercializing are rare important build our and treatments. on these includes well-positioned access

a for diligently them. the of needs understanding to reflection medical Our access of make patients. to that ensure Translarna have difference commercial is EMFLAZA a We DMD both our unmet patients with therapies success work to and

of our rare with We an operation. to to patients. leverage seek commercial and by in two are new to We deliver commercial and continuing our position products differentiated therapies mission disorders, global the having commercial effective line commitments now in which and platform in built we is have

year-over-year reported approximately to Our $XX million. just commercial year. XXXX, million $XXX EMFLAZA revenue Duchenne revenue having led approximately May. a a in the prior strong million generated over execution $XXX increase XXXX In in XX% franchise revenue growth. launched us of reported Translarna has of

million. million XXXX is for the $XXX and guidance Our the Duchenne for franchise $XXX full-year between

response from As Translarna drugs. a office FDA's our we for the request we resolution of received formal announced new dispute February to on XX,

dystrophin denied on possible production. generated current the with additional path of to effectiveness The drugs NDA allow office framework. containing would appeal, forward a did a of they on our the recommend the approval data that stated they accelerate using of While data new office new recondition be ataluren

at methodology demonstrated to While utilize will in to methods we which FDA clinical the the dystrophin that previous study, with trial will work the in we design measure patients validated production dystrophin. time, Duchenne current available using

a the this are fusible towards it. path working and United that is We are to expeditiously pursue in approval States we optimistic

Before a I moment moving support during the Community FDA like to continued Duchenne take updates the thank to onto process. other their to for would

have path world. in mutation United and heard with that clear patients to and in goal it around lives. clinical of are patients Advisory is to in experienced to the to hundreds ad We the wrote from will be they program. across Translarna about so families It impact their to speak by the both make to from had Many who their the Duchenne forward this FDA remain remain States our patient bring the currently place, We to positive caregivers, (Ph) Committee the and our world. want the Translarna able that the same physicians access the pursuing intent benefit on and are letters will traveled and that U.S. FDA we patients programs [nonsense] Translarna while the access have

Let science and research me our internal to turn know programs pipeline.

first is Foundation. molecule advancing with treatment and partnership Starting splicing in We are platform in efforts the SMA a its currently small they internally and Roche our the The with proud SMAs of as these program are clinic. the progressing. pivotal developed oral in for studies how

both patients PTC. results from As This million FIREFISH phase $XX mid-October, Type study the been to SUNFISH infants. with selected. the on on infants triggering including pivotal data pivotal studies study we finding of SMA the part dose the appropriate based SUNFISH I SMA will Scientific data milestone dose for the the of FIREFISH has in study shared a study, from portion SMA, in transitioned is FIREFISH presented survival Clinical at and coming to International the X on in January weeks. in Roche Type The stage previously, were payment Type commenced X in the X Congress of and

programs platform have and to are stages. Huntington's progressing In medicinal generated the which Dysautonomia addition splicing SMA, optimization and in chemical are Familial our

at on upcoming We Analyst a forward oncology XXth. also look which April pipeline have highlighting our to Day developing we

Marcio. on and Let to to the me you pass over now update clinical commercial call our efforts. Marico,

Marcio Souza

the of success the stated to XXXX we commercial reflect are therapies in Our different bringing value patients. DMD

of patients. ambulation patients delays and and of to of subsequent the DMD of for in It's Our delaying U.S. the published respiratory approved benefit around mutation EMFLAZA therapies. ventilation assisted each approved older well which mortar death. with available milestones. these for The DMD patients U.S. documented is demonstrated and pleased five of out Translarna awards. DMD therapies of the long-term franchise the the are of data DMD is three on age current recent Age nonsense is the all of predictor brings benefits the Translarna of we DMD two loss in a EMFLAZA, for and prednisone. published to times side data of supports patients EMFLAZA that effects outside

benefits these are DMD bring they to the We patients. of proud and therapies

on me with now in the of We been strong. and launched EMFLAZA Let the middle providers by very the reception May, reflects in EMFLAZA. beginning performance patients XXXX both commercial EMFLAZA healthcare the in has of U.S.

phases. strategy term launch has three Our

to had previously EMFLAZA. is patients first maintain who to The access

the on to the to any XX% which The treatment. the penetrate patient is last EMFLAZA, driven naïve The second into phase prednisone of is to to that corticosteroid convert been by approximately published patients data. has population is efficacy

script see progress As of very first a we to have they moved launch of the quickly and on phase degree to has started renewals. the EMFLAZA the reflect now high

that patients initial to Our will guidance their EMFLAZA via XXXX confidence successfully continue scripts. reflects our prescription for renew

that from of our internal It’s recent we independently guidelines these with believe both for While our this most treatment work results align be the is care the see deal. DMD EMFLAZA published all and our that Based standard very reflect understanding on generated DMD to over. should patients. results, of are encouraging encouraging, own data, far

focus known currently indicate historical long-term efforts. demonstrating prednisone. to of over are EMFLAZA launch This major the of two half agents and data view Reaching delay is As prednisone. when critical to years I the milestones DMD This naïve treatment. these that profile predates yet by benefits of another of three on our risk we strategy. the benefits treated that treatment any and not with treatment to patients U.S. guidelines result is in our step corticosteroid relative the treated previously, current about of data prednisone in on DMD published DMD a loss It guidelines, is the still the that mentioned based compared switching of patients continue patients EMFLAZA

function this The EMFLAZA which untreated to addition and improve specifically lung guidelines of the History Natural DMD care loss Lancet formulation standard to delayed In CINRG in and publication the compared Study patients. when shows of

Our to EMFLAZA. all get on goal eligible patients is

engaging of been EMFLAZA physicians, with as payers discussing treatment. and critical DMD a component have patients actively of the We benefits

out progress receiving I’m those of pocket patients than class more the that the We current are to also EMFLAZA are the excited therapy. confirm of pleased close ever to zero. remain fact best-in proud for of will costs with that patients and this

switching continue as same Now to at we bringing said, Translarna time adoption patients gears Translarna expand to Stuart the we committed globally. to U.S. the and to remain

pleased potential pediatric team knows are result has a a of of accelerated XX% CAGAR we which path will well, FDA of the the our EMFLAZA U.S. of in landscape commercial In XXXX. launches We we a in forward possible January the a launch through successful launch. first year-end our as U.S. Outside to recommended this approval Translarna towards the XXXX. year, of neurology facilitate guided

main rates. Growth more Middle America, used region There and and opportunity the regions on a is younger central are Europe. further Europe, excited recently growth rather population the The update a are a we East of growth is consequence We expansion. of driver Translarna than because for will western and Translarna’s in eastern where geographic areas continued this Latin significant most the focus In outlook. especially of birth this and in operating. the in our high be about of

over treatment been also the than We once identify more including expressed XX%. growth very the years. uncommon in not and patients four high benefit have drug is more have where present we ultra-orphan Translarna disorders from It's compliance of and remained to established the are a in continuous regions, with Patients to physicians EUX continue for has seeing available.

update on for the Let provide a me development now DMD. short problems

we the As on of appreciate patients perspective importance of possible said the as community early function. before, the treating we have muscle as to DMD preserve

study off six an pediatric exclusivity. requested a conduct FDA. the to months us study plan have upon completion provide as by This EMFLAZA market additional would for We

at submission for children Additionally, five, we was basis from these intent to diagnosis. in data of under DMA, this review. of aged Translarna completed the study the of Both efforts two treatment our expansion time have of aligned is currently guidelines begin PK study the well to DMD of with to a label which

the in patients enrolling started has quarter XXX third study of Translarna long-term Our XXXX.

with could As accelerated a FDA has specific it of the a in connection approval. serve and study this confirmatory our is conduct study as stated a approval of reminder the obligation EMA an

its We our clinical lives back are on are patients confident disorders. to strong value continuous will equipped commercial and Stu. well drive the transforming focus call with both to hand that I with we of rare and capability now Stu.

Stuart Peltz

like familiar is our I programs. would which platform. with on more in recently used are our Marcio. to rear life cause the therapies discover to and has manifests infants potential Thanks, generally splicing early of for generated on genetic detail SMA been technology death you or based This Most that small of in in program, data molecule the Atrophy and a neuromuscular Muscular toddlers. new disorder leading Spinal is share SMA genetic

splicing and oral SMNX modifiers. provides competitive the SMNX a broad towards with complete drives collaboration in ease SMNX messenger have advantage the because restoration of oral therapy SMA systemic tissue and believe have Earlier robust We administration. Foundation clinical around RNA. it length of shown data an splicing of distribution full RGXXXX We that program that a Roche a

As In XXXX, ongoing. pivotal studies October have discussed currently two stage enrolling. there the is entered and registrational we are before, SUNFISH

SMAs of registration Type as SMNX presented selected to infants Kraków. Bayley proportional muscle towards Technology brain SMA Gross ongoing patients. finding the Motor stage dose patients January results access end projector to unsupported organs and dose in on point relevance The blood correlated. ability parts and modulate X is International the SMA the the is has target an the our levels part oral data targeting SMA protein directed the of SMA SMA to we for trial splicing interim has It recently data. by transition at and dose-finding its two Early safety events. the the the and The open-label patients trial coming FIREFISH reported data infants. clinical Results Scientific as treatment well to the no in of and gain compound an patient selective ability as not The the three required patient identify blood. the due data, adverse have These Scale. tracheostomy of and recently only also no on SMA Platform FIREFISH interim No SMN lost The had the These were in discontinued Based as as or ability all the clinical to of swallow of the validates X in SMA as Splicing progressing therapies increases such JEWELFISH tolerability presentation in infants an upregulation supported that doses. which open-label RGXXXX. muscle. in were on the to the other Posted of demonstrated JEWELFISH were increased ventilation. pivotal SMN and weeks. JEWELFISH. increases also studies, assessed is all sit is the data in second permanent reached program study protein study, allows for also program reviewing its demonstrate RGXXXX can pivotal that known studies from splicing. Congress presented A and survival SUNFISH from Preclinical also safety primary portion brain, systemic Type pre-mRNA to trials expected in RGXXXX Part highlighted that at levels X from of

expertise unmet now We medical internal diseases other needs, applying high programs. are challenging our have to and preclinical with

We disease the additional Familial including and programs targeting are pursuing and splicing Dysautonomia. pre-mRNA Huntington's

Christine our well exciting will now as like an I April Day, splicing Utter, Analyst over officer. financial on highlighting at to pipeline turn would these programs R&D XXth. Christine. wholly-owned principal the share We call to about our as upcoming more

Christine Utter

today issued our our Earlier reported that guidance XXXX. full a and fourth on for few strong we to release with details press I the start XXXX our results of for full-year Stu. across and financial DMDs performance results our summarizing with performance and we Thanks, release financial the I comments will detail. Starting quarter you franchise. a our top-line for refer

of our $XXX product reported As for impressive net total million. with results Stu revenue mentioned, were XXXX

milestone XXXX growth the their expansion full-year, launch success product for in sales with existing net partially million fourth territory quarter new into penetrations for launched of guidance XXXX. to just provided million. as million franchise $XXX.X Translarna DMD product $XXX for prior Latin $XX.X influence large one-time the geographies. revenues have quarter in $XX sales increased both the for million full-year and to having May. order million million America. XXXX We $XX were were the net a in of in XX% of million $XXX reported and revenue EMFLAZA This Total reflects in representing $XX.X fourth XXXX a the Translarna from of compares SMA collaboration of to the by

For $XXX.X full-year XXXX. launch to of U.S. was $XX.X the the payment million The change EMFLAZA a milestone achieved in of Translarna, million compared in commercial and from result revenue $XX were revenues XXXX of million launch total total successful Roche. expanded a the

non-GAAP non-cash XXXX million one-time in million $XX.X compared For excluding expense. $XXX in $XXX million based expense million XXXX to were for expenses stock the restructuring $XX.X in $X.X non-cash expense R&D million full-year a based excluding and compensation compensation stock

non-cash expenses were one-time excluding compared stock-based restructuring For based million million the to and in for compensation expense. compensation expense $XX.X full-year excluding XXXX, expense million non-cash $XXX.X non-GAAP $XX.X stock million $XX.X of $X.X SG&A million XXXX in in

period Cash, reflects base decline loss net loss for was million for net successful compared Operator. facility increased securities is expense in approximately question-and-answer million at our net an Operator at corporate compared $XX million from will the start during now of the compared growing in totaled loan interest offset XXXX. XX, was million in equivalents partially quarter infrastructure. our result continue full-year second million which in to Net interest to $XXX.X the cash and For secured XXXX we $XX to leverage interest XXXX to $XXX.X XXXX interest XXXX our of net marketable same to million closed as of we the million This hand to $X.X of investments. international session. related to income by December the $XX.X to XXXX. This XXXX. the I $XXX.X XXXX, full-year for the call increase expense year-end revenue expense over a primarily

Operator

Our Instructions] the Alethia Young [Operator Suisse. question Credit first from of comes line with

open. now is Your line

Derek Yuan

Derek Hey, thanks is question, for this taking my for Yuan Alethia.

So that guess FDA paragraphed to that your know you did basically ataluren what situations, so to has And think FDA wanted us the about see I you I Thank you. you dystrophin NDA can congrats want dystrophin just of on just earlier more do study also what first level further they on see in your you new expression. progress. provide color the remind of could filing. data

Stuart Peltz

your after mutation for we that start clinical so by question. we previously treatment Thanks obviously demonstrated used that patients production saw of XX% to dystrophin the XXXX Yes, trials. going you I we that And do for where significant. reminding in statistically patients the was dystrophin that will proof-of-concept nonsense Duchenne demonstrating

the from appeal pleased tomorrow, dystrophin in decision be So path the don't from results with of think the the obviously the along And that’s in a patients significant results that clinical approval. that appeal, untreated terms above is sufficient that as that controls. accelerated dystrophin are it any what and the and with was production we the were our discussions it based really at that's would statistically the forward while trials on now FDA important the we for well, there the get clear we disappointed

that to dystrophin. with talk now FDA both the the with we we would to utilize as trial the dystrophin at in as well look the So the trial, are methodologies discussions about them

discussions, where will when at our what describe steps and we we that’s now So we right overtime to you the next are complete are.

Operator

from question of Joel And our Citi. comes the next Beatty with line

Your line is now open.

Joel Beatty

thanks. HI

in is in First down in QX. higher QX QX that of kind saw question from and sales again this particularly characterize some QX coming QX or have lumpiness of whether sales and now lumpiness the flattened the up being in with same Could to is it's ex-U.S. out? back then we on XXXX Translarna saw you we QX increase past

Stuart Peltz

Marcio to? Hey Sure. want do

Marcio Souza

to for the Sure and saw growth a hey the across revenue. regions patients Joel thanks growth, we and In question. QX any in of all number see continues we

year is as year. the bit variability, exactly business influenced So we you. and described of every that how create what that bit total throughout What expectation is into the to the our and a the right how in forecast we our patient in in of lumpiness little have a quarter-to-quarter. and patients the we we is described terms look exit you guidance We the year net this of that’s grows look do do we past little way a likely just in communicated all number

we that be, continue But we expect the answer? as to of every for. our bit So and to day but that patients little at beyond on throughout in are it’s be drug commitments the lumpiness only what of the region going really to aiming continue to well. stay Did XXXX, all not a is who to end grow for

Joel Beatty

Okay, yes, the you EMFLAZA prednisone you on EMFLAZA, just are those three question, available of yes size and patients three U.S. it each talked being other the the have opportunities, those on in Could of patients of growth. steroids. about third being on another previously on in naïve and many buckets? patients discuss One how one stages sales got

Marcio Souza

what Sure have would so remaining, patients time are absolutely, point in naïve. EMFLAZA, mentioned last - right to gave as in we time at of there I so obviously is States about eligible remarks, the around half continue that all the two so XX,XXX regards. patients that see the we number on are grow U.S. was patients quarters patients on and split X,XXX on be estimates our patients The we now for of ago or is that the as United in we that the them being

current time might a that, be many not exposed long years might strict have and don’t so have naive of naive used in have recent that's we to they ago or any and So and they there this exposure for see of to type corticosteroid. steroid senses

and a other the of pretty those well that's would or size XX% XX% on then as about and between So be for half so prednisone. obviously that big half, it's

still from we of segment coming give to the teams think of way all really deflazacort, terms is we really this have segments on getting a segments, are on is penetrating work patient false here for grow drug, in So impression one proud have to of experience the three with the come we of to or ones very from job, team the a that patients the did they to focus patients good we the on that there we the only EMFLAZA. really right majority them now, prior but all

far so well very to three of them. benefit want we going to all continue So and

Stuart Peltz

Maybe CINRG recently EMFLAZA recent from from with especially and that Duchenne Muscular a the with on come also view, EMFLAZA what with or patient point the from the with the specifically deflazacort Dystrophy publication be a called differentiating of message prednisone. History Natural big out guideline that lancet out really should

I makes be because in patients that that onto further think we them with information that we differentiation goal to that should patient is discuss why on EMFLAZA physicians that and all transition of believe EMFLAZA. the gives it us really our

Joel Beatty

Great. you. Thank

Christine Utter

You welcome. are

Operator

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

Your line now open. is

Eric Joseph

that have. you The Anupam, questions, Translarna first for of Eric commercial its couple on footprint I us. a thanks taking the on for form guys just and existing guess Hey sort

current versus know see a broadening bit sort access you are and wondering the reaching penetration, do hurdles then of kind would us sort kind growth the of footprint, of from to are exact where maybe of group to you opportunities if have currently talent geographies of the deeper other EU just I greater what of a geographical penetration kind characterize You and follow-up. more come deeper the for

Stuart Peltz

go Marcio. ahead Sure

Marcio Souza

Sure.

throughout that there very not so into the the of two been we So will bit factors from the Europe. to a different we see lot when penetrated, they to that's walk sometimes of through like this I on we time still just a are It's EUX look because because have concept Central little is we growth do we you for longer grow regions long-term, all a to to by think Eastern up years is but we have continue come expect and amount region see importance, bit mostly there well there. a substantial growth, driven the and little

patients the that marketing growth like all on teams, happy is throughout our world, doing that our challenging work more with patient-finding we are teams very So one especially the see to geographies that on are penetrated. we medical are but continue identification, patient on

the short-term that. of a from that growth lot comes there in So still

to are then countries, a even recently be role in move more expansion lot of year would we As a most years in two seeing found. large the are long-time mentioned guidance long-term to still our I I geographic through to as patients And the we Central we drive like the this very that have where Latin did of bringing last across diagnose, years last to been get say, bigger the Eastern it’s America, happy is to just activities population diagnose young growth has unify remarks the patient-finding we the so we results have are started. the we penetration small, getting. to with very the time Europe as age the trying really far we and East and and extremely well, patient Middle have And

Eric Joseph

could. or Got getting I on maybe And Pivotal both SMA underway FIRESISH a one if and portions just quick underway of it near-term. that’s helpful. SUNFISH

are of Thank and interested you in expression study, you. what a also either Just regulators the outside give tissue sort us the extent of in to at curves sense enrollments wondering SMN if anticipated all. related could peripheral blood

Stuart Peltz

Sure.

also getting but where countries only for not them, studies in that will weeks. enter seeing we where are be We just and still dose into are unavailable, pretty products FIRESISH pivotal study been global patients we studies has anticipate studying approved to therefore go are our as SUNFISH studies we picked the choosing patients other these even the It’s these know you drugs well, As and these in to and well. coming there the in into

studies, relatively so these will we that So time we good in anticipate feel rapid completed recruitment these over quickly. be pretty

so a easy it to you the this take first-in-class, obviously tissues also orally such are liver, real is to be while the as there in think drug that this about bone. is do the the it because into that best-in-class, a think CNS, us affected opportunity body the muscles, we but affected And when for not bioavailable, the won’t throughout only organs including the in - are other be

key there to we So well. that a as other think advantage substantial I we And heard opinion is think have leaders that. talk about actually

important a advantage. think So that’s we truly

Marcio Souza

So just about right. answer distribution the regulators expression tissue to the tissue need specifically or the the for your of question and the

and really that tissues a a that would required course Stu or have is so of like in into be some drug on expect see oral just terms the said, influence. a We like there to compartments is of we showed all really get work, the throughout. drug kind the distribute and desire of RGXXXX even distribution of preclinical drug, target expectation showing to no bioavailable that just right So to now this that a

Stuart Peltz

SMN see but we we animal and sort saw in But complete well. one-to-one, clearly to as in what of look but to not In study and SMN was one all RNA produces also we seen primate we that brain rodents protein saw advantage in was the we to levels only restoration. thing restoration saw that was level was just preclinical other an model, at models that similar other and non-human which what able of and in a the also that complete blood see the we RNA the the were

well the are see pretty confident brain see that will - we so feel and confident that you that about is you it at blood, are that we as that So within so looking be in there the well. will quite what distributes changes actually excited we

Eric Joseph

Thanks Got it. so guys. taking much questions for the

Stuart Peltz

Thank you.

Marcio Souza

welcome. are You

Operator

Thank you. of Ahmad of line Tazeen And comes next from Bank with our question the America.

is now Your line open.

Tazeen Ahmad

Maybe trial. for might that to how about thanks do criteria your in therapy, you being for us allowed patients I'm also the think of this for and you of opportunity talked on this you the develop? concurrently the Stu, used, earlier add-on, are but is other through grand just drugs the sorry how one have terms could taking is on enter the this question. if of in Hi walk this What have be are to as allowed missed I scheme kind another amount SMA things my and market SMA they therapies envision it,

Stuart Peltz

Yes, sure.

a good. think I that’s

time I think the terms this really benefits from a studies concurrently other that and so obviously therapies. be in all the we therapies. of other there at to the the drug are we of their own, is pivotal looking we on In long a make in want question won't run get sure

seen what of obviously RNA. think have seen is RNA for the messenger we the we that restoration what RNA that transcript want the is best for but complete have itself I [indiscernible] far we to patient, thus the that

of can view, splicing whatever a that done be we into from so. point So whatever made will be think can be SMN made or RNA

have be it making what well. the who as you currently of only could And it, don’t JEWELFISH splicing may muscle when we know is trial be actually mass RGXXXX like think will efficiencies who been we a useful ongoing to would forward then allows be that or is do will need time available it take patients compounds of effects if patient that previously might a We available. there So need on other other about have that have something are function that a concurrent that well obviously so tell. necessarily to improves as therapy that for

could function improve mass muscle long those helpful of in it or be certain in the muscle with So that probably run. combination increase certainly

Tazeen Ahmad

pivotal need for program how this? would a you Okay, big one think do so of

Stuart Peltz

this to just to to are FIREFISH need to need it's X see the has that XXX able in So be this is think we for we to diagnosed you infants within to we Type first months, trial label terms to In XX two three remind patients. it's in a study treat patients enrolled to an one study, it's five there placebo that able the the would placebo-controlled the SUNFISH XX, study be of case of babies, FIREFISH in about case that XX and about assist see. be open a infants the be for the would

there well. to able XX% So the powered benefits a see study be to that’s as well

Tazeen Ahmad

enroll? take to for those Because that then would you thanks Okay small not long how it think and are do studies.

Stuart Peltz

they rapidly As XXX. timelines I yet. I actually think our And is We have think one will is enroll. said that disclosed don’t XX, we one

Tazeen Ahmad

be these therapies Okay. the with for be would parents the for other potentially are on you if I children of and only asked competing want you their because patients know that pitch therapies they children that are another the approved what look to just study. in would at not

Stuart Peltz

point, know argue the been preclinical as it well that’s taken can at We taken. as if think certainly of that and would there I of good orally it. think a really distributes you the is look be consistently an about was at was astounding. for we pitch that lot well be quite can exciting as some muscle the bioavailable advantage studies, is body that go performed well other talked nerve consistently lot the and published a that it the is most and was a set if they that so drug fact preclinical effecting We has was I look advantages is think do throughout data data and oral within real as we the organs you what to back Well and it that pulls

Tazeen Ahmad

could then And Okay. I DMD. if question ask about a

recent not adamant your may I especially pretty way in of Translarna. it with know be determine you earlier most study, dystrophin right were the the you seemed but in measuring efficacy that that the Going the back call, past discussed dystrophin pivotal to

to have now to why route think collecting forward. about guess me be think be might that past if do might why additional you now it wrong information correct this the and time a you suggested I information, think go FDA you best would the because I’m good I do in the So collecting

Stuart Peltz

And had now preclinical in clear predict necessarily points don’t even is we benefit with clinical even and demonstrated we [indiscernible] it’s And that with efficacy. the didn’t this and are to a where went agreement it study long of since discussions a the correlate we in FDA So very there, proof-of-concept actually, I the with one dystrophin FDA or levels time couple study. that was into day think dystrophin therefore done it efficacy.

that a FDA benefit dystrophin we what - any current any the However today the clinical that important that important treatment they is made statistically can approval. we and come have data was and and this predict said has along the in point with is gave I around that be notion to think above this that would us the accelerate for appeal process suitable significant dystrophin that the that controlled they path that is

in that the while bring is that now to is So the U.S. us is think most to we path where past clinical this path availed Translarna studies doing the patients. expeditious we to were benefit

we forward And going that moving - we us that into agreement to can to will path so able we therefore with - show bring are FDA, patients think very to with believe this confident we to and are best we have it on be to forward rapidly as the this the we move that. be is for we

Tazeen Ahmad

Okay.

me is on DMD when path And should the so update last for next we in expect the the U.S.? question

Stuart Peltz

on with so right now them six we we will working and we it. three will and forward agreement what the we that and are think discuss to months as probably you understand where to I we a are move on on update get methodology process is So think that's that

Tazeen Ahmad

Stu. Thanks Okay.

Stuart Peltz

Thank you.

Operator

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

is line now open. Your

Gena Wang

for you questions. taking my Thank

we one is of clinical SMA a achievement? wondering first milestone both in measurement for the as terms update X from for will FIREFISH have Type chopping SUNFISH and also a such just trend when and patient The

Stuart Peltz

that. for thanks Yes

talking dose-finding have As with two did a the parts the the we were of part that have it. study dose-finding we here transitioned is let that study portion remind been said are the about just we or - we you conducting in what first me was pivotal first

will multiple see you like. easier so the we to what obviously doses of So therefore its looks clearer have survival that and data

year doing we mortar but the some talk milestones as As time. data right point throughout pivotal think data additional matures read they we be would the an out be obtaining studies year will have report to we appropriate have the be the to at have it's now throughout to now we this, we transition to so will opportunity and that able continued I that the about

Gena Wang

can Okay, those out approvable five sit if pivotal and they five I Stu of four that you trail mentioned you that see four be you or can sit as mentioned also that XX consider that think FIREFISH babies, that would to patients? babies

Stuart Peltz

significant. you at that we simply looking so calculation power five just will Yes, babies of XX see out statistically would said by

Gena Wang

will that History And to Natural comparing the be right?

Stuart Peltz

be Since see and actually sitting are capable own case. that that are you awesome. on would anticipate of their babies to be We don’t would X pretty they Type

Gena Wang

come to level last Okay, the quick the and to questions just do wondering compare also on level U.S. marker one that ball just you dystrophin dystrophin that mention patients data you for so untreated more that was to line on to rather than FDA feedback and I the just past the patients FDA. the think color? comment in that comparing get the wanted untreated know base We from

Stuart Peltz

it faster the to compare using untreated at yet That able versus and obviously look some peers I certainly precedence we dystrophin to control final be there controls a that's is that's said be yet determined but have but defined, or possible, agreement, to to to yet be so will it FDA. of part if in blended baseline so that’s the be think be would with levels be the untreated discussion controls

Gena Wang

much. Okay, very thank you

Stuart Peltz

Thank Okay. you.

Operator

Raju comes And our line Blair. of next question William from Prasad the with

is line open. Your now

Raju Prasad

for taking Thanks question. the

does weight number bigger versus will CAGAR stats? you to that the expectation are patients two at did get Just you percentage four also the DNA? therapy on in the on the include update one gaining just are current five getting year patients year guidance. of for that And What label new patients XX% that say

Marcio Souza

adding proportionally patients patients the mentioned new growth for and going the getting so obviously we to are so components heavier you organic these right, that just currently older three So are patients, well be growth that of market. new the say to be adding of this expansion. biggest driver to from patient would [indiscernible] the the I part the patients continues that's in constantly biggest

high, - happier patients thriving. is low still the see dropout the so that is are are these dropout standpoint, are compliance that happy compliance something and financial but are and from we very we maintain for even in the the patient all don’t very, very are obviously alive very rate only We

they into as consumes clinical quite important that's for or not you around Duchenne part they that trials that's our significant growth. a historical so as three year kilograms gain of continue our look per if commercial so So us. for experience that’s the But and the well. And cohorts

than five it’s part the I is globally younger countries to In its next years still five, and other by the the it as higher five little some so not terms the or a of but of five driven In years later countries that so around shall include four the five. lot age in guidance. a than average still in two than going example that much. higher, well is average five diagnosed of segment say bit is to be lower The for

we and are reduce if Although them get to five longer a of two we diagnosed show are years age. going have symptoms working age the hard patients is and of they very better to to these around hopefully that years when can life even be

Raju Prasad

that and general study there well you submitted enrollment be cut trends that commentary and you a for will try that and submit approval? on something that little into XXX your then data of effectiveness accelerated might FDA it take is going additional the kind it’s of And could Great. to have data any you already just provide as

Stuart Peltz

XX Obviously other trial of other earlier, on precluded so a of placebo-controlled, sort any XX bringing but months study, XX the this placebo-controlled consequence the you first is too that’s Study being in Yes. global the month much which data where really from hand. the are

think Study be the as the with is dystrophin that would be discussions was would confirmatory serve we approval it clear our clinical data is important of would the needed pretty and currently XXX in approval accelerate the have that I what data with and process. as trial accelerated them that FDA for the what

think that's would just way sense. way think that's would that we I we with and think that up I go it so And I the would make set

Raju Prasad

gene why are Great anybody anecdotal JEWELFISH? say last. on enrolled and one enroll maybe therapy can has just that JWELFISH trial have in would about coming you Anything they and therapies and you patients been present a off

Stuart Peltz

not You now. of really to, discussing think into to position right talked I know, are I we are details don’t would Well that that. love about we really

Raju Prasad

question. the for Thanks great. Okay,

Stuart Peltz

Thank you.

Operator

our comes of question from Abrahams line the Brian with Capital final RBC And Markets.

line now open. Your is

Unidentified Analyst

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

then projecting study, So discussions just we is the for for latest pediatric we FDA how pediatric had if and for EMFLAZA around a on question progress furthermore, you the important indication the could wondering you about are the us study, give it your maybe the updates EMFLAZA. the could discuss

Stuart Peltz

Go ahead Marc.

Marcio Souza

Sure, I will be happy to address that.

projections extremely by me only trial last for there, towards do pediatric start gave includes. now, our so a five. let consider FDA, in than the includes younger important we no future we to that move point sure So of we the generated patients your in request the EMFLAZA If data to on the use clarify it not I'm written that was for not right make guidance to XXXX received from EMFLAZA. or steroids ever whether going We

so that and intense them conduct with Lancet very leaders said the working any have just the more given all the of going soon information and are that to on you clinically as clarity are on to and trial remarks feedback. we can positive that you. we timelines we mentioned so Having FDA, like the key important, opinion study them that understands included treated patient the guidelines and just steroid care really suggest was and the to be care about this that the publishing patient the updated guidelines is strategically patients influence that from with been So for should an it's immediately us what this hearing is the only on as we final be on as not have is that a for the we be groups from updating PK we but important details

Unidentified Analyst

Great. Thank you.

Marcio Souza

you. Thank

Operator

like the does Thank any Peltz to this would And today's you. closing call return Mr. Stuart to remarks. for Q&A session concludes I and

Stuart Peltz

again on the call. Yes. for Well thank you being

to front Dystrophy continue really a our the to forward Duchenne on looking we franchise. are productive XXXX Muscular with execute as We commercial

conducting as are design dystrophin study possible. FDA expeditiously finalize that patients and States to forward the we For United going the the as with to look to work study

SMA exciting this front, about that we will to talking into pipeline that go April for data on relevant our these to medical the our year, continue the us be the fronts program mark that thanks XXth on will you On as joining and other updating and our in calendar have meetings SMA forward developments Day your and we Analyst and look at today.

Operator

the for all This program, concludes and disconnect. in day. participating may a thank gentlemen, great conference. Everyone you you and have Ladies today’s

PTC Therapeutics (PTCT) 7 Mar 182017 Q4 Earnings call transcript