PTCT - Earnings call transcript Q4 2018

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Operator

Good day, ladies and gentlemen, and welcome to the PTC Therapeutics Fourth Quarter and Full Year 2018 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, this call is being recorded. It is now my pleasure to introduce, Head of Investor Relations, Ms. Hill. Emily ahead. go Please

Emily Hill

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

statements. Before let our contains to moment review you slide from me that include which call will a we statements Please based forward-looking take corporate start, current our presentation, forward-looking on remind today's current expectations. our

actual differ can as and and Our materially any and adversely business statements could these forward-looking results from our operations. such materially results risks of affect

and description For Commission the as other recent detailed you reports as to SEC and the company's filings. of a Securities uncertainties, annual we filed encourage Exchange applicable risks XX-K on well most company's form review the with

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

Stuart Peltz

for thank call. Thanks, XXXX for PTC. joining transformative you us on Emily, was and a today's year

the oncology those the with DMD of say several proud out Going that a pipeline products we label met clinic. achieved in revenue Emflaza, and We ambitious I'm our we two Translarna, goals. year, into guidance, advanced set expanded use achieved goals. our to the for now expansion potential

advanced also our we Importantly, program. SMA

an this our feel Roche to of confident FIREFISH SUNFISH a results with Following we NDA year. the and submit productive discussion later Foundation SMA FDA, in partners, the the and and

from proud PTC's very that candidate are the internal splicing resulted program. We SMA

specifically to this compounds identify utilizing that selectively been modulate and other have platform splicing. We

Latin PTC by we with two programs gene two of in that platform with America. therapy CNS to we number PTC. business multiple strengthened also other plan owned in-licensing products programs a have achieving that deals, development the these We adding goals, Besides commercialize solely are splicing and

You vision orphan XXXX, to we presented and do the early three-year one fully plan may integrated company. PTC recall this drug is we a proud accomplished company at the at just a year. in are everything we for Execution that to have in three-year become core,

we to have the like with to years. these to longer-term We Now want I'd at them deliver of and looking over what today. next defined this we you five forward, goals the beginning three year, review described

On front, the we in the commercial $XXX from XXXX, million revenue had franchise. in DMD

include as few anticipate TEGSEDI, launches, label Emflaza's the to years, two years AADC to years five expanding we product three DMD Risdiplam, as therapy, and Over patients well next gene old.

have also Translarna dystrophin products last billion year. in a potential based in that in is currently XXXX potential at the grow study The revenue excess by of We for started of of revenues on the approval end that of pipeline are anticipated the our potential outcome XXXX. to these of U.S. combined in $X.X

therapy. plan internal invest continue will We innovation years, sources gene molecule small and expand We the to across over external five discovery next clinical accelerate pipeline. to XX stage through and our programs and

the in this to year America. that efforts identification five-year including TEGSEDI Latin outlining key of our SMA cover a on submission we launch we plan ahead updates provided for have programs our addition year call, AADC anticipated patient detail In more vision, BLA recently the of in NDA in planned deficiency, this later will and also with

and innovating PTC the leading to diversified programs to proud has positioned products. pipeline of drug a be orphan that We company are with able itself become

$XXX recently a with ended balance equity approximately in $XXX proceeds We motivated cash net are solid a DMD, completed of and and million, of built offering leaders with We XXXX revenue base and have million. have committed talents strong with employees. an

pass execute Marcio the to position and cash strong to our very commercial progress. I'll review a now in call our to vision. are We on clinical strategic

Marcio Souza

Thanks, Stuart.

have with in just potential patients disease with a multiple rare billion of to portfolio as serve revenue building Stu of combined XXXX discussed. a excess the products vision We of $X.X

our the growth. aspects finding key all to from in our business long-term excess, of more of driver patient expertise Our

be patient spent differentiated teams considerable and to treatments. have that patients the that rare awareness, disease components patients is This increase the We time which a includes that. disease efforts, rapidly and across a should functional identify bring of key can therapies need state-of-the-art service,

Emflaza a also of franchise States. our five. U.S., This countries, XXXX, combined the mention, outside XX for exceptional mutation expansion Translarna United aged sales includes two the In of in sales DMD million. the patients Stu to treat revenue As $XXX DMD an has nonsense over and of of which reporting approved label our in XXXX, Translarna, now to is EMA

label believe expansion at patients the this recognized to EMA that benefits therapy younger allows maximize which we would the countries in gain age, to to them. approval, Importantly, all a access the

year, treat last Additionally, late have to urgent for very With EMA, in non-ambulatory treatments there mind, patients. patients. is expansion that non-ambulatory the our equal of label to an unmet submitted we needs application and the

In answering finalize later the year. We believe for this in regulatory which Translarna EMA we and has Translarna progression. the where regardless process expected next benefit resubmission of year. study, NDA got later potential of approval in patients our we process some lead accelerated from are all the are United they an begun will questions the States, We the dystrophin expect have to our to of to disease we

aged also approved in non-ambulatory United older all Our for DMD and Emflaza, is includes ambulatory States. franchise patients the five and which

file At that a of care from in We continue be sNDA safety U.S. for the file sufficient population. we request and FDA establish informed Emflaza for hard this to to data expansion that not received standard to pediatric study demonstrate work end Yet last in year, we a Emflaza. label a as the of the efficacy would and to to the have us they required

the at is the years the July of beginning year. we action and X. the We And have old, received two Emflaza to patients for years the sNDA submitted for this agency from five dates

is growth and Translarna commercial preparing revenue the million team Emflaza, year. the of given expanded we this $XXX for have indication franchise both launch this in later of guidance diligently expected XXXX $XXX to DMD Based on Our million. continued

to strong the place. America the process infrastructure, necessary are Latin TEGSEDI to some of and choice which region, additional Waylivra. unique we our team development rights support right hires in to made partner commercial products America commercial we establish Ataxia, products. the have Leveraging essential completed the for globally, opportunities. To be footprint these the to it's successful very in and in We right Latin have all business has us from proud year, in-licensed two Last

necessary an including with partnership established nursing have the process to we our Importantly, patients, support also exclusive team. support

year. regulatory with filed we front, the later approval TEGSEDI On expect we and had ANVISA this

TEGSEDI to to patients in that region. markets, the Latin hATTR eager are the prevalent form where of America polyneuropathic is the We bring X,XXX approximately with most

markets and of proud in a competitive we expected now our this are be. I for believe therapy gene this platform. of we execution CNS are is We the highlight like stronger game to there. ahead especially and would as Speed critical one success are to

specific our therapy some pursuing As key CNS' in includes strategy you advantages. may recall, gene

the the benefits is the process occurring, where risk. target disease ability first area The maximizing the specific is to

vector, for Secondly effects the manufacturing because reduce low, there which a call the burden cell potential for potential is is the and which turnover in we durable brain so process. of the small use to micro-dosing, the that amounts

the with reduce risk of small for the for dose Lastly, approach reducing the immunogenicity, combination target the potential these the progress. overall

BLA programs the of are see this later Our the of as we gene say pre-commercial efforts therapy We anticipated rapidly filing ahead advancing. you have started year.

the United we As before, early a we said deficiency key and patients States Europe through already opinion interactions. identified leader approximately have XXX in AADC

We populations. more screening continue we're now but at at-risk patients new week, to after steadily identify to program moving a focused week

have of cerebral a Those in with misdiagnosed patients normal approximately present we the identified MRIs. United instance, cohort For apparently patients palsy clinics States. XXX,XXX, in

programs this screening to We We later programs expect identification this on as year. an on policy as implemented quarter. share the to plan in patient screening several have the updates well the

submit to IND ataxia. gene a treat AADC, year in this with end by disease point. at is the treatments Friedreich's next program of FA an real to the we Beyond underlying therapy pipeline for no this the plan devastating the

the trial following place all the We have patients the start clinical to elements in submission. IND in

our advanced or to gene we next therapy file year. the is candidates plan Angelman an program syndrome, which IND Next, most

to we we process are said, end, this later have we And in-house a candidates to are securing manufacturing. the of new in As growth support that the therapy intend also developing declare order candidates. gene this gene robust to of pipeline, we gene in In therapy therapy pipeline year.

several to AML we trial and our in Lastly, oncology dosed great PTCXXX. with our patients advance programs our progress trial continue with having DIPG in

the of turn complete to before now expect We Stu. back end trials to the enrollment both year. call I'll the

Stuart Peltz

the area and compound of program, both to leading commercial and the clinical highlight our field. most emerging ability the which across I'd focus innovation made is we been like produce advanced splicing proud is that's are modulate plan. with with our progress area in now small in a pioneers technology molecule, splicing our be to the to The of Marcio. An the which Thanks, therapeutic fronts impressive. an

to types identify ease great SMA, news technology. expect as approved to platform for has as specific that We selective This be implication with which of year and of Foundation has can that PTC It is compound the SMA well splicing small next as advantages our splicing this broader compound for potentially for treat distribution and one, disease. clear it Roche is broad with tissue A validation of the powerful now partnered a two, also but SMA molecule is three. administration. the a patients modulates and we

report I'm to of for the spectrum. I'd Huntington's familial over needs, turn are Financial to unmet clinic with familial Principal at programs and to R&D These and We the to Officer. enter Utter, candidate Christine? development that disease. to declared platform the across we high applying now and have happy dysautonomia expect splicing include our dysautonomia this diseases the XXXX, medical end our other call like Christine we year. the

Christine Utter

in the press Stu. $XXX the on issued representing you details I'll our Translarna with XXXX. were we for revenue. product XX% I Thanks, summarizing for our fourth financial revenue in our top comments our full combined few million start with an results a release results, XXXX. quarter of $XXX This details. compares year guidance today, compared net million XXXX, full a revenues Starting XXXX to $XXX.X of the the XXXX. of to and million XXXX, and for year to and financial Earlier our million $XXX.X franchise, we increase in refer release year-over-year performance our for line in across Translarna reported for DMD full

million million XXXX. product revenue increase of of reported from For for $XX XXXX, a $XX Emflaza, we net approximately revenues over

our commercial with to provide expectations to proud respect are continued We growth.

Our revenue guidance million to XXXX $XXX is million. DMD $XXX franchise

revenues million. non-GAAP I stock-based of therapy XXXX, our advancing non-cash, gene products. expense combined public compared support XXXX. to $XXX.X out pipeline $XXX.X investment full investment compensation products gene expense as therapy cash associated of operator totaled research the net million $XXX and $XX.X to potential and million well $XXX This $XXX.X at $million As recently to the for expense, at to of with XXXX. franchise. non-cash, like stock-based non-cash, combined activities XX, loss of Non-GAAP $X.X guidance expenses to approximately loss expenses million and increased continued a R&D a our excess securities hand resulting SG&A R&D reiterate will XXXX. XX, for pipeline. compensation SG&A platform compared excluding in million XXXX, million million our in DMD billion reflecting existing $XX.X for expense. excluding to commercial have XXXX, expense, advancement million in clinical compensation XXXX, excluding advancing expense, in Stu in for stock-based for were reflects compensation by million and our mentioned, we expense stock-based non-cash, our $XX.X as compensation to million Non-GAAP million. in stock-based I commercial increase to R&D also in programs question-and-answer our to outlined $XX.X would $XXX to of excluding guidance in in marketable costs were and This was December net our excluding offering completed proceeds equity XXXX million, supporting for full approximately expenses million our compared year over call reflects also $XXX equivalents, we’ve Cash, session. I the non-cash, Operator? now approximately point year December XXXX, for XXXX of in $XXX like also $XXX compared start $XX Net million would $XX million

Operator

the Thank first comes [Operator And you. Baral Instructions] with Ritu our question line from Cowen. of

is line open. Your now

Ritu Baral

and question taking have one Hi, guys. Thanks for question. one I the follow-up.

What span submission. age X, or for? filing for X follow-up my question I week and activities and AADC. Risdiplam patient anticipate trials but range do to for a be the label on indication question first type is are then on the certain The you type the going range? submitting know you or And identification two certain is

whether mentioned a that and pool cerebral with about Can go misdiagnosed sort go or You of the patients centers a how opt-in normal and that was spots? of MRI. program this the is you XXX,XXX of will you just centers across say some of sort there like through the palsy Thanks for – taking question. pool use many through with blood screening that's

Stuart Peltz

Hey, thanks, Ritu.

get one, based was to and and of So is to SMA, broad plan in with based within one both so the part type it three, our really, was file I a really on that. terms the FDA, the label think two on conversation of

for – to… I'll let Marcio maybe As you want AADC,

Marcio Souza

just Yes, Stu of of We program, trial this that's and is the The age for comprehensive the I like so right, the several expected XX States, U.S., When those on In started, Planex really a what hey, why what XXX basically patients. we cohorts the finish – move reinforcing we AADC the outside up to cerebral has is absolutely. for that that's And good main this a restriction our take. we maybe cohort done to examine and of the through is or Roche SMA that Ritu, approach. executing have and no dynamics. to most very different in palsy there belief expanded on feel job then centers on or just, the competitive relationship. that and is here that are way are globally. patients that identification, were that there's multi-pronged way two there program of layers efforts a are United doing give like U.S., lots

they as So, conduct one we is, are want screening to centers offering projects”. if “research the four a

so basically that's them, number a entire to go We're centers good with is to there. the a of contract to And that way have There really protocol we and do the forth. So wants and population. IRB, on going driven. like go very that the a uptake so for

ones have don't the run. For that don't capacity a the right now or to either like have they structure

have we calling we're what As non-protocol. a research

Currently, ability a the there bloods opt-in their blood patients. whole are like we And validating have we're sample we of centers into the looking to and We are dry but as providing screen them those steps as well. finishing for validation spot.

going more to what to on on are are majority But moving is the we well call of them that's all throughout patients So Tier board, the process X, much be faster, productive where that of year. as in. the pretty

consolidated in and of should days, more Does fashion. the in So next a patients help? start couple QX, report screening in like the so expect progress later the year to in the

Ritu Baral

the family, it to something background I parents you of of sort Yes, in individual, done need it individual something does. or approval guess the patient's anonymously [indiscernible]? that is this run for the sort screening, that's And is

Marcio Souza

a that's really No, Yes. question. great

the with the of like So part that right. a program, research this so one is clinical for protocol, done is

So there consent through going they're and is and to on. so that form, a go

For the a two other offered one families. being is the to that's basically

we're patients conversation patients. so data providing a the more going bit very you a they're We elevated it's Most like U.S.is they dopamine. the looking can genotype so have, more do go Outside of a in with U.S., X-OMG, will it's to simple through the That's the precursor of the that's and are family. of screening one a country-by-country. this little marker them just you and and these

this cerebral learn families if more is These to about want know As something that as well. imagine, that's can every not really palsy, be we you it day can done.

So much families seeking like conversation. faded we're and theirs very seeing having positive

quickly. barrier any like were and that The go have the see in should that don't more we process relatively place, on it So, there. now barriers we putting

Ritu Baral

questions. for Great. Thanks the taking

Marcio Souza

bet. You

Operator

our from line And you. of Joseph Thank Morgan. JP the with question Eric next comes

open. Your now line is

Eric Joseph

Hey, for guys, questions. the taking thanks

little from the to are for At side, range run rate end Just sales though wanted the first, unpack looks as bit. of a tracking XXXX, top guidance for fourth my DMD under just a you on couple quarter. I the

know from? you little in they implicated the course current be demand coming of pricing over have to where that if that seeing the else – I expectations pricing. And any headwinds, but a year, bit then lumpy on expectation talked So about anything I is in in And are guidance a anything the respect with have there might there follow-up.

Stuart Peltz

Thanks, I'll pass and Eric. then great. Yes, it on. start

grow. some and of to think, thinking sort that can on see, both pass on we're that the to the We certain have of I I'll You Translarna on to But on countries in adding aspects it what Marcio do Emflaza that. into patients market. continued for the go on plan transient approvals will dynamics is

Marcio Souza

Sure. Thanks, Stu, and hey, Eric.

everyone I dynamics And of they couple here. important So are to think understand. for

So Stu that mentioned, one patient involve and all patient geographies. products just are growth, growth seeing we in is

the of positive. reach is volume this, is The of earlier year the staying of like and are adjustments patients to this increasing, therapy. in XXXX way goal, right, the year, dynamic have going mix. premise some in our of the to our goal that do into So we business this to terms over that we quite base to time the looking make some we we're presented believe countries And the on pricing,

the some big, comes there markets are a still specifically that that uncertainty, potential. lot quite say, since a these it to in accounting of became we little for there's guidance, on of but So is as I would Translarna,

versus sense and us not treated gather being because potential, patients is treat the just the like patients. amount end of to the are make So day patients, we when many try to business, that's at are doesn't why stubborn you as doing are for looking these to the into be of that it

mix impact year, and might be countries which started. other the we get So bit on price negative then a the is a little throughout those going all the there once patients correct drug, on of to

that one that's So impact. might

important what I is right. talk the to think is it equally not in guidance,

So we for don't Emflaza. have the two five to

to have the the prepared launch, FDA. we received two the on an might remarks that And notification end, July approval talked noticed be between diagnosed patients included the a there. by hope a fast it's and five have we're to with going depending we that's You approval. that we're stealing a date are the not X going non-ambulatory the for executing in Europe action about not by patients how we while then might dose We're is pool There of here since either upside conversations. fully adding yet. and necessarily on regulatory can be significance is the And guidance. the We that

So always are be the we responsible on we quite try way guide dynamics. all to we like market as considering the

Eric Joseph

Got it.

if could. Emflaza Just I a on follow-up

Marcio Souza

course. Of

Eric Joseph

is we We are an know the And and through opportunities how orphan you can of see had to for indication? whether with its exclusivity about the Thanks. potentially Clinicaltrials.gov, thinking also the input level FDA I'm should that recruitment up discussion trial here there and wondering design? be curious also design just on to timelines, expand runway – additional

Marcio Souza

to Limb-Girdle are you And Xi? Sure. referring the

Eric Joseph

study, Sorry, yes. Limb-Girdle Phase X

Marcio Souza

there was removed anymore. were to I Study because Yes, thought because don't XXX, FDA need us, the conduct we five that to actually told the so, two

inputs the the that in lot biggest here key from opinion a of Xi. center leaders U.S. got we So from is

gave we fast history least the design. some filed of be recruitment that most that natural there approved of available the are and constant that's so Caucasians the with Obviously literature drove with to is the because with developments us. vast to in or expanded was fairly Limb-Girdle what We relatively is expect prevalent That in they FDA some we and type the there. not for which absolutely nothing specific at type the

but little good So, bit really a going year the going, in update to later provide more how about feel it's that. on we

a terms of things we of product, the number there the is expansion the are On of doing. in runway

ways is more for trials future are are Some not elements a we formulations clinical quite off you some had base is to log some and the I present things convenient but with is would There expand appropriate of that you in are a to discuss be that off a we but here. said, the of that. ready the publicly, approaching of we runaway that at be we population in terms are, well is might that. just relooking for some how for deliver. – are near – now, see number this Right do them should coming products, potentially of other as as the not some case to to we Maybe number point

Eric Joseph

Got guys. it, thanks for taking the questions

Marcio Souza

you. Thank

Operator

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

Your now open. line is

Gena Wang

questions. you taking for my Thank

for $XXX,XXX. one First Translarna? Eric's pricing is understanding is the Previously follow maybe just wondering what question, average was our now like

Marcio Souza

Hey, Marcio. Gena, it's

one that quite parallel specific So the a is in other. lot to Translarna, haven't countries to want be But has differently to country been don't we for price took be guided like price. we while and a we of treated thing reporting stable or one care patients of

So the globally. stable we price fairly kept

some like negotiation fifth expand product put have as on with now the year to the bit well. it's little the table. dynamic the entering markets of so potential And the of large dramatically, We a that's like

are into looking where patients the are not that are here, treated. we being So

adjustments erosion far, of – be I'm not can just not the talking expense in about the there about in the wanted so responsible of price we're for talking to number especially value, dynamic patients some quite product drug. believe to that we not clear. positive order So are U.S. that But an that's done make disproportionately on crazy mix, international talking when We're

Gena Wang

Okay.

new the or talking just are XX% discount we territory? about XX%, we So XX% like wondering talking about discount are in

Marcio Souza

is U.S. on contracts, governments CMS, would you're of what willing the the markets, the longer-term on size example. for some talking negotiate like discounts the give payer, to to we opportunity and the those that of think of in be for the have I depending Yes, about willingness we

Gena Wang

Okay.

Marcio Souza

talking we're kind about. the of That's thing

Gena Wang

just SUNFISH wondering already would from early the by of trials be decision. of FDA Since question portions would Risdiplam, both regarding FIREFISH a and pivotal data part completed make And and now take look another and have both final FDA decision? should a enrollment Okay. then initial quick data time

Marcio Souza

Yes.

So, that no, this FIREFISH had we SUNFISH sufficient – both and to really part for plan good now is very be on one we whether would sufficient trial for that discussions, of approval. is the based the make thought

So that's the what planning game we're plan of on.

Gena Wang

Okay. And very – quick question, commercial just Latin TEGSEDI regarding in then wondering much a terms be both set you preparation, of the will up to salesforce investment and last need America dollars, how for Waylivra? in commercial and ready

Stuart Peltz

Yes, of course.

is in to say we very do first America. thing to in to hired team, had execute matter we Latin were every and ready So this, Kudos weeks. hire just this who Eric of his a to one like

we is obviously anyone We anyone not since dedicated approved actually have So product to TEGSEDI. don't Waylivra, add expect else to elsewhere. support to the don't

support. fields. So right see efforts And phase we the the right; equally right our we patient putting commercial, of this unsolicited folks not kind are like, and any are now. the mostly medical, in affairs medical are so now them basically major There then it's and request, components, three

on We have neurologists staff.

the have are our great program the of provider then both – what an support. like did create on to we of And Because the XX% going later, TEGSEDI. then and of TEGSEDI, to contractor so overlap of maximize meaning and that of country, for between Translarna service with products. coverage. Argentina both understands get important key have doing codes, well large a as testing American, seen us. large that's healthcare help We normally XX% with familiar is the coverage, solve them the third-party, treaters locations are have Brazil very is the and with care, we to it's monitoring we've to areas Equally there best Latin view, this is are the third reducing more we're the exclusive staff field we're leaders in testing, what a not the zip we part for we this professionals expanding medical and patient a reducing actually call that is a standard BXC, the kind it's force in opinion is special people area in but giving is place about we our I is country connection

highly complementary. it's So

Marcio Souza

is the And as good point are ongoing so I that well there efforts. these believe already

Stuart Peltz

Correct.

Marcio Souza

we've working already we physicians started year. of submitted and are will approval which and be so so this anticipate for also aware We've this, already,

lot this. a of already ongoing for been to effort ready that's there's So get

Gena Wang

Thank great. Okay, you.

Stuart Peltz

Thank you, Gena.

Operator

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

line open. is Your now

Joel Beatty

for thanks Hi, taking the questions.

of reasonable expectations shape is first you steps of in curve TEGSEDI, that What to up a taken just the for the toward as The ramping one follow-up need was of question the what key of asked. see the on launch kind there sales? terms be

Stuart Peltz

Sure, Joe. hey,

that and the going are seeing to treated already, for name takes request of we or the perfectly beginning reputation we this involved, the they more been So this as with process, very patient even positive this the through patients thankfully BXC of – they to requesting channels, requests The starting about hear TEGSEDI beginning several it, be to the launch months. and hear has of honest, the actually right. the pre-launch, more a we expect different have a as since the is we fact We're have physicians. get number

So first this and growing is right to on substantial going after expect the to a fairly we're entering share, answer much next it's too the going be we not What two months. I'm approval, here, and in strategy the slow is market potential a actually quick following then with this market. real of potentially But year gain because accelerate years. to competitive on

it's type disease for rare XX XX months, stick and say, the but America. have in and it's then were months geographies on major of the to a like if the hockey about you starting Latin So it's launch next usual think unusual I geographies an U.S. to slow, like would – fairly to Europe, so in

Marcio Souza

consequence in of We do of here, of TEGSEDI, ease instance – that there will as lot the the injectable, we're now. a and think patient of advantages of out there it's that consequence a pick-up the property that be there's as a just the

Stuart Peltz

Exactly.

Joel Beatty

that discuss to support one new Okay. trial what the and Phase be to on And you X late Could maybe then approval trial shown question of last you have Translarna, initiated design year. an other the U.S.? the needs in

Marcio Souza

Stuart…

Stuart Peltz

Sure, of course.

that I Study XXX, number wrong the just So that got call – Study XXX – we there. sorry,

in So collected, with open not all read XX a nine months. label patients We're the baselines taken before samples center right. another it's aged at biopsy and at are a it's any at XX, of study basically going were single to this California,

So that becomes trial. internally the in the block control

methodologies measure and two discussed dystrophin. with the FDA which agreed using were are We to

expectation So difference is as the months protein the that analyzed by of we see amount of of and baseline – statistical the by the increase would terms in measured a nine between called that is you pallets, the ICH on ICH, method in a we see sarcolemma CL measure expect the as a the where that's localization actually modified would secondary is to. and

right trial enrolling the that's we're now. So,

that, couple obviously, FDA trail have next With We of be to this under potential we early be trial. we're expect to We to the the for a good months. it's the old patients on in positive. expect resubmission, NGA going several to going a next interest and we like resubmit like We the to the have hope enroll year positive year.

Joel Beatty

you. Thank Okay.

Stuart Peltz

you. Thank

Operator

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

open. now is line Your

Tazeen Ahmad

market I talked guys. about current January into color to in by pie came deck you about Company Hi, the on priced either you as deck risdiplam, was number roughly up you that in the the my that potential to taking that on that your could about XXXX. And royalties risdiplam. available or as vis-à-vis, talk well billion Maybe Okay. $XXX $X.X wondering questions. posted in I Thanks us you of today chart, was SMA give in penetration market? for one some revenues back with Stu PTC if think, how million

Stuart Peltz

the we we so release, what – thought Yes, And on numbers press in above based to mix of in for excess could precisely put billion, the billion it billion, in so looked above was around you we I royalties $X the we in where and was think $X that at of see what previous looked sort of versus be based that. terms would XX% out excess of be million. at and of $XXX that XX% example on $X excess and

Tazeen Ahmad

number doctor And comes be oral. that rate if faster? conservative physicians for the excited it checks rather seems we've done, based on potentially this certainly early an that Could be switch would

Stuart Peltz

funny were is And think we've conservative That's that what sometimes can said is meetings we people what it that. – had you be.

So thought think, it we as where on. and time I today we – yes, discuss start grow and goes we we then are out as

Marcio Souza

if maybe Tazeen. jump And in. Stu, may Hey, I

I well, believe couple the that So important are right. dynamics as

relative there try on that. take conservative drug internationally. stance on obviously is a debates So We price like to

know one that's would Xs, on say, that now. In general, model. So of, upsides type the example, right largely point we I are unserved for

competitive. well would Our physicians show like that percent from own and treated to a the seems to that yours teens of like the not it vast go now current very has patients the That been patients as and market internal majority with with is checks be treatment excited right risdiplam. available

type knows fact small two looking ones in molecule administer then is durable you're probably another in to time for everyone treatments. access. to as this When easy that safe beauty molecules of it's really very going to-date, competitive that. the obviously get launch. dominate being access to of to at model The it's all becomes globally to are how chronic to the Similarly be and the We then of expect the to most type

play. So all I'm this sure to we're either to And plug not that them point, this for is the stop maximize going have with to but Roche there, trying and you're going that here right, we not a are we and while a value. XXX, have stop starting conversations to point

Stuart Peltz

Yes.

to really really nature And the when really broad Some out the it from it people of splicing then – distribution, can a we be out understand to think only consequence, competitive our products really this. because platform. about came came drugs of as said competitive of its that ease, how as access but globally get Marcio of not how our like this of the this,

Tazeen Ahmad

EU. then about time has on AADC, you? or of Okay, U.S. Over Marcio talked that so what taken you period maybe thanks. in have patients about the XXX finding and And

Marcio Souza

I say efforts to that, Yes, team weeks we that to right. do like I'm so build took say though us. XX would the for going the about

U.S., our now. and weeks. countries like have Like we priority is for took right countries priorities are in that So that example, these Europe going no a few very that on Germany clear few are strategy surprised be like one our to

in So really of some patients before are a like And like thing with. Europe. relationships in docs we're just that like in after number we a is labs putting that got that extra have efforts new we we didn't we there. Interesting a agency, and had genotyped they feel we of Congress symposium on got couple right

I geographies, both would week are geographies So core our are here, we but say, every that in as in non-core seeing well. we geographies reporting in this, different

is on seen number a of about have really mutations at work lot I disease XX. reported might this. we did the the You do time acquisition the this was of diligent

Now, than it's XX. more

seeing diversifying efforts it's pool. So there huge like we and the are

that much patients the now. So a right I not believe dead the founder there is pretty there, were mutation, thesis it's

pools have can't You then would for can, in thing XX XX it but not be different us, you theoretically happening. that's is founding good would and be what but Well actually a mutations.

So the the last screening or XX weeks started. barely programs so,

So throughout as year the we well. to see good acceleration expect

Stuart Peltz

the I the knew think able efforts for effort an and – what's yet that's looking a Well, those taken of was not there, to unidentified we're finding actually yet. already people but really point. the already good patients, was find that that it hadn't that find them, that to patients really

So just target. that's

know relatively So it's I we're been but speaking, in to a that, patients, it able tough find genotyped. because already always to they've wasn't sense, them find

in are that able bunch will those whole who clinics we'll find a just be there's to be Now that misdiagnosed.

the that's step. So, next

Tazeen Ahmad

Okay.

bit quite the like seems it have a So you made of since progress. acquisition

of X,XXX it you X,XXX So you get long or patients your potentially, to number? in terms that will to take target how think to do to close that

Marcio Souza

expect that were can key Like in to this a of What and treated towards to even Great we like the move I be be that U.S. example, see as believe all pushy year we We like team geographies And going patients about, the low[ph], rare those XXXX, really right. And we of with XXXX, did, normally patients don't going XXXX, pie patients focus going mentioning think a be on to bit number who start it's on working successful the classic but to incidence it's expect by to to for get obvious a is question, quite U.S., to more a XXX very identified. going mentioned. day to the this, I'm to is in my that's little as hard quote-unquote, that population be being to launch great I gather have substantial then, some we before, see is are but the new very per I patients patient imagine .and we and there. we're right. here discuss you disease launch

so Day, and so now are there genotyping Rare on. globally to push diseases So so Disease include their and panels rare diagnosed and is much

and seven the expect So or in five would patients. I the to say, the those space majority of, have good patients, of very number percent a years, years to incident we would of

be to substantial way markets going there's we So the see.

Stuart Peltz

make to approval. sure hard we strong working upon are we launch got a And

So and up lined ready the patients to are roll.

Tazeen Ahmad

just oncology be last on portfolio, patients a PTCXXX. might to in type within a you in with you're in give quickly an Okay. question that's of squeeze of really us what your Can AML AML? study doing going if I And idea

Marcio Souza

Yes, course. of

pathway of So growing. specific PTCXXX, been in general, AML, right, a that's DHODH inhibitor, I malignancies that for think the showing key literature but it's in hematologic body

being some is that have three It's data showing a mechanism the design good cohorts with plus in three preclinical three refractory We relapsed and activated. clinical patients.

patients. really are we getting very severe So

small early trials enroll are sorry, design we a that this, expect in It right, dose with so during the the into dose. Would an is to small We we to onto do for year patients relatively and in starting that move slow and next level as cohorts two we're therapeutic moving hematological expansion is relatively as maximize of normally to We're what depending – would like to a sense, probability there. being are all subtherapeutic, year, is a because a seeing on data label, open the learn help? the expansion this patients. of on well able want all success seeing the kind

Operator

Ladies right. and with Fitzgerald. Young line gentlemen, you. All of Alethia next our question comes Thank Cantor from the

Your open. line is now

Alethia Young

Hey, kind we And far year, sort Risdiplam business data as Congrats get over update the year? questions medical for I should progress readouts it's months. view do Thanks AADC characterization getting the to study? far the as gene I part of talk then on you the past little what of FDA the a over some the just about mean at specifically are as will over the meeting guess you hurdle in Thanks. with here. guys. can on my of couple therapy? kind as more second is to just the taking looking one And question, like finish have to or expect of basically I of of just line SMA wanted as the What's every kind left One, through with on two timeline question. that usual? mean, and like work the bit I manufacturing, the of we big

Stuart Peltz

thanks, in think if second – question. Alethia, the terms Yes, of I

of then. full AAN results First, we'll be we'll is obviously the next and having the meeting presentation a

and do that we're that, we'll there upcoming So as continue be for at well. meetings after excited on that the to it

So we'll you continue to that update on as well.

manufacturing, the of once second question terms In you – AADC

Marcio Souza

Yes.

give manufacturing therapy, would I think this gene Let me Alethia, sentence belong day, the so it's say, as right. usual and same you, on I don't business for

of have get place. a So in going year FDA. with to Everything that lot the get CGMO And to been we continue our these is attention hard to later right to these has that NBL occurring. C the interactions extremely working have giving been our I'm Type that to from we seriously we BLA Neil move steps is. the having this team to are following got are And we briefing. all feedback to work pleased is hopefully We steps, and on We've and to his for going all – I'm with say and all going get great be there. to the had that late now, guidance the them We're there meeting not the and it last blessed take extremely year. cocky

a is have for successful expense obviously continue us to we want We part what to here, consideration so of launch. a

commercial launch. to several to have have we So best commercial manufactured that

that clinical trial our looking had but to the this of both product. capacity. in continue United we calls, before, in the just is view, to two to know looking one States, the I add a broader We're and we we're groups discussed that CGMOs for manufacturing So material,

now we year, and are use commercial, later else discussed and be the the this we BLA everything in on are the for out as that us methods to with and implementing So able FDA I mentioned. of a process stage the late

Stuart Peltz

to doing And of as is, as of we're just one the remind advantages what well it's Yes. everybody stereotactic surgery.

what a terms and viral Agilis of we that for need levels is that two the for need we with to of other the different around much reasons are, and take We're So went it granted. smaller. what XXth is particles XX different times total. Well, is Marcio like we one said, never this it's of the manufacturer in requirement the are – when They you level programs the manufacturer.

Alethia Young

Thanks. Great.

Stuart Peltz

Thank you.

Operator

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

is line open. Your now

Raju Prasad

Thanks the question. for taking

that quick the and to NDA clarification is previously guess, something based follow-up. submission I to, of just you on first, going a be I said a FIREFISH? on part only one have Just SUNFISH And

Marcio Souza

Yes, FDA, that on of both and that's based the we are It SUNFISH FIREFISH the where based that. results was saw with interactions our submitting on right. they

Raju Prasad

see Okay. could wanted is then rest amount And of patient you time or when put it and out to I the guess just you together package the they it? before the submit was CMC there package of any those is that can submit

Marcio Souza

this plan package by and put it year, the have is to Yes, later we're – submitted to our together year. this going

And on be based most ready to is this in of the getting it's submit that. to really just So able NDA.

about not longer what that's necessarily. more it term that's So – about

Raju Prasad

show if of moving discussion kind – two is factor of two discussions what are into would regulatory any forward? part or – there what to patients, the how guess, study I your Okay. didn't going would five there part

Marcio Souza

believe look seeing that not the feedback was and basically best with on FDA the interpretation center based to we is use are right. proportions, the them looking the of at this one we that proper part like babysitting. right part XX.X% now FIREFISH if into What are way And WMS. for the to to is looking apply got two are the they numbers, to some but We of that different proportion was you CHOP-INTEND like would data about to one be always under three was other they're data few this, that Raju that's I babies, babies X.X When look at in look the robust and that's being showed right, points we this from the things. like the the is So, the time so conversation they because threshold. absolute they than into are public, that of to

With the – SUNFISH is similarly right.

natural base are on increasing. patients seeing that the on decline the MFM-XX normally We history

the before review right on any sufficient data file. briefing based and completed for package regulatory one more definitive they for answers from we receive, this Both trials the of book year parts exposure or But one. are agency. like the looks full never they got now So a

of say, year normal, the exposure, but non-codified So patients year threshold of it a XXX like a that. in meets the exposure, statutes things normally of would I one used

for the very very feel Roche So confident with feels an upcoming confident, it procedural put submission. have place and we more in package, documents they to now be is

Raju Prasad

Great, the on that's helpful. And payments. milestone then just a clarification question

percent press the in milestone I think it versus what and those million [indiscernible]? $XXX are regulatory of was release, approximately

Marcio Souza

want some There the posted – go payments, website. Christine is over SEC but filings you schedule the today our it. in to slides very So a our we of maybe detailed in all

Christine Utter

Yes.

$XX is about in as appeared all as are milestones and said, the regulatory details Marcio million the royalties are sales-based remaining milestones. to there related as And also the schedules. well So

Raju Prasad

XXX Great. one split versus ex-U.S. on on patients? the you provide color then any And question those AADC, can U.S. between

Marcio Souza

Sure. now Happy to half right – and so half like and it's give take. about

going passed equal. before pandemic. months that the as Europe, Right I potentially that the expect a but this, of effort putting regions. now, mentioned couple than we potentially, this it's right, request an but little uptick haven't U.S., of physicians we run, I it's on Europe as both so in on next probably is to faster easy don't see relatively we bit kids So, the In know, because we're

to concentration expect get and have lot of It's in don't running emphasis all launch. important we U.S. a first to in So be world. markets successful the very a these want here. to our putting We and in the pharmaceutical up because the obviously screening is region one U.S., other most

Raju Prasad

what's those the quick one, patients? last range between Great. And age then just of

Marcio Souza

of don't information They that we all have, are are they – before. now to the age for exposed but right age them, we within treatment were the the have

Raju Prasad

for Alright, great. the question. Thanks

Marcio Souza

You bet.

Operator

next Thank the Brian line from Abrahams question Markets. Capital you. our with And of comes RBC

open. Your line now is

Brian Abrahams

you Thank talk therapy? questions. first eligible Hi. proportion or on for about taking being as with gene the eligible AADC is be thinking My patients a be about what the Would we Among question my small who should for all of you identified, treatment can program. just them part?

Marcio Souza

we've and treatment. Thanks, Brian, therapy We've seen anticipate we for would of all is patients patients question. that for seen the thus gene actually What seen in far. what all we've and the regardless improvement is – age, be eligible it's the that interesting,

Brian Abrahams

there gene emerging thinking picture, timeline the franchise therapy a bigger been you manufacturing specific bit you talk then to costs as progress potential little associated in-house And milestones to we of about be and can to what it. like date? What's to an out goals more or your plan franchise? the out build Any should Got use? about, build respect type plans with platform you

Marcio Souza

Yes. Yes, that. for thanks

And I and days, internal able talked therapy think I hard probably out think in-house. to have I talk we that and the control probably that. things we'll been own manufacturing XX in within going like looking as And days, we're we've next to as XX building be front that we've have on – group to then terms more think ourselves, to bring it it's important the gene about factors of thought one it's, our well. about to

Brian Abrahams

pipeline you And your manufacturing. latest stand? development from use of other one do capabilities and investing then Okay, last versus on latest What's fair for Where thoughts? me. views external in the enough. like continued Curious internal capital your business

Stuart Peltz

then So, start, echo, I'll you Christine. and

in of vision is and we as what the we've internally, talked will what vision said – we – our so programs view both be. we in would doing, we'll obviously said we clinical about research where what of be we be our be expanding would Our and as

so continue everything do And look well. But Obviously, as we've in-house. said, that to as well. we'll always we're can't you doing externally

search things and we and for try there's so see And other are us. fit compatible if continue the that

want of capital you In maybe to… terms Christine,

Christine Utter

Yes.

Just million to remind you, we year-end. recently in approximately had $XXX the $XXX cash offering at We million. of completed equity

diversified we what So and pipeline to internally fits will look to base with revenue our externally strategic our continue vision. solid our also invest both and continue

Brian Abrahams

Thanks so Great. much.

Stuart Peltz

Thank you.

Operator

And question line with comes Suisse. the you. next Thank Credit Auster Martin from of our

Your line is now open.

Mark Connolly

expect expect taking see risdiplam, I for Mark data Marty. Hi, similarly is data when should Thanks we And may product. guess you. have RAINBOWFISH couple on I and that initial everyone. a the looking do until? for questions. Thank see should to what provide? study, outline JEWELFISH on at This can particular questions data for you you of results what expect you updates my When initial functional to we specific the and

Stuart Peltz

JEWEL include the expanded is with couple are points – don't that but so guess that – the I of gene therapy. has one a know JEWELFISH to I been treated patients

probably while data that's probably be about that. talking new is we would when have Meeting the relatively SMA more would so And Cure we at

Marcio Souza

measures And some JEWELFISH is is core, a maybe there may, if there. obviously so safety at its I Stu, trial

PK increase some patients extremely of sustainable in We protein exposure therapies data positive when show from are and levels. of the at is the and levels other seeing view my to of switching looking that SMN I will in an protein. WMS, having which you're

just the is to the expanded through So that's the of because include to next going type RAINBOWFISH cohort enrollments. treatments. other expected on SMAs, continue was of beginning

reading give be on So imagine, and as more patients on ready you physicians a presymptomatic risdiplam of can with with to lot like an But constant that's interest when we're treating there's being not to it. more out. with quite estimate going

Mark Connolly

you. Thank Perfect.

Marcio Souza

Thank you.

Operator

back concludes our like that And Mr. the for Peltz question-and-answer I closing now to remarks. would Stuart Thank to turn over call CEO portion. you.

Stuart Peltz

on both and With an treatment the potential advancing make significant gene a expected you the disorders. you to all PTC, Day, focused executing And this rare planned every the products billion really difference Rare AADC as for think and on Thank – five-year innovative is to as on a you. disease be a genetic today. where at NDA what seen, today. rare this a leading to achieve to we therapy now today Thank toward portfolio a transformative call after treatment day. vision Muscular cutting will XXXX, really plan joining patients report goal. drives us bring for with you call will forward We Thank delivering Disease $X.X be know, for for our revenue edge And of company the BLA year the a scientific diversified us it's look on Spinal here I you've of treat reminder and we're and in Atrophy lives now all we're XXXX. enable for by that to joining

Operator

participating day. Everyone conclude you thank you may and all for Ladies in And gentlemen, a program. disconnect. have does conference. today's wonderful This the

PTC Therapeutics (PTCT) 1 Mar 192018 Q4 Earnings call transcript