PTC Therapeutics (PTCT) 2 May 192019 Q1 Earnings call transcript

Company Profile

Participants

Emily Hill	Head of Investor Relations
Stuart Peltz	Founder and Chief Executive Officer
Marcio Souza	Chief Operating Officer
Christine Utter	Principal Financial Officer and Treasurer
Vincent Chen	Bernstein
Ritu Baral	Cowen & Company
Eric Joseph	JP Morgan
Gena Wang	Barclays
Raju Prasad	William Blair
Tazeen Ahmad	Bank of America
Joel Beatty	Citi

Call transcript

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Operator

Good day, ladies and gentlemen, and thank you for your patience.

You've joined the PTC Therapeutics 2019 First Quarter Corporate Updates and Financial Results 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 conference may be recorded. to Investor call Relations, like host, to now of Head Emily would turn over the your I Hill. Ma'am you may begin.

Emily Hill

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

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

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

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

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

certain of non-GAAP Information for non-GAAP GAAP call. to earnings information is this non-GAAP We a of and release. and will measures today's GAAP disclose reconciliation during use available our regarding financial

call Academy hand to be from next Before will point the SMA the given the I would data I out our at week presented of program over, Neurology. American that like

making will the that, let call that We me be any program not pass Stuart CEO, over on comments today. Peltz. With to our

Stuart Peltz

XXXX. productive to time be our on start has the a to for today Emily. taking very you Thanks, Thank call. PTC had

We continue to execute vision. our on strategic

to and Translarna authority patients outside the to Importantly, largest regulatory is patients the world. This Brazilian serves This EMA in the Translarna regulators. enables faster was approved DMD which validate market benefit expansion week this around the risks by independent of the of Brazil.

to continued with Our to help provide base us current grow the commercial business. us strong a sales

of guidance $XXX to for year million revenue to continue reiterate our million the DMD full $XXX XXXX. We

Waylivra the Risdiplam we for new SMA, Tegsedi launches next of strategic hATTR, objective, products for AADC the our product deficiency. gene for advance therapy will anticipate our XX for four FCS months and Over which

to We also patients have a to X PDUFA label years date X expanding include DMD Emflaza's for old.

potential or established suites there revenues in based year In will by the royalty approval The of a revenue of on approved be potential submitted which this are these and anticipated this of either all is we approval combined products is already dystrophin a of grow for addition, infrastructure. in support commercial revenue of To in global billion to to a study. for growth have potential the XXXX XXXX. are proud to Translarna outcome U.S. $X.X excess

over are key the selling to to addition Tegsedi, business bring This therapy a have PTC plan expertise CNS DMD We We to business and as and internal partner development platform. opportunities opportunities. differentiated Through us Emflaza, strategically pipeline Waylivra patients commercial collaborations in continue to currently these therapies is with we our rare the disorders. activities grow a our and with treatments for position strengthened external assess XX countries of allows gene to worldwide. strong

footprint by commercial an as our we well portfolio progress commercial adding therapeutic as our our both advancing in product addition made additional In pipeline. expanding have to in

the requests programs Europe have initiating a approvals. we and both Latin received in of Latin for and America approvals positive this Tegsedi European we year ratification America ahead Analogous we how on these received launches the opinion expect the Waylivra We feedback prescriptions. first Commission are encouraged Waylivra. be early by last patients by commercial have that physicians and and coming and we rights from Based report upcoming for regulators to States approved later year. by Tegsedi in formal preparing the Translarna was quarter to already America products was We're the are United Latin access launched will in happy in and CP days. to in we and

clinical advance been In to programs. we've hard working addition, our

the have We for first for in AADC preparing this deficiency been our program BLA gene year. therapy

SMA year. that NDA will submitted Roche We are this later be also supporting for

program where we address rare of utilizing excited Emily success mentioned, on sharing program AAN. we today. SMA is platform this program as believe we in been the splicing to the Based week this discussing SMA diseases Therefore targets won't data on program are look We the and the other have to we platform the time appropriate. about spend at next SMA forward the

splicing. by programs programs have to platform subsequent The oral solely severe We other and identify modulate owned or PTC. are splicing specifically selectively that compounds

development familial for already year. to have this plan and an platform the We selected dysautonomia candidates file from IND this

on the identifying we the research disease working this enter compound candidate year. is also next anticipate for and clinic Huntington's development Our team will

therapy discussed As our last a is strategic gene in-house manufacturing therapy we support pipeline. to on call, to it priority gene bring our

in are functions. and a We of adherent gene integrated manufacturing facility allow an manufacturing finalizing to house biological in XXX,XXX The feet and will lease. term suspension the have us use identified both analytical approximate production long therapy and facility will systems process and manufacturing square quality

turn for Marcio progress. therapy Marcio? AADC in the COO, the rest details MassBiologics to the our discuss production rapid foreseeable to gene pipeline. will with commercial Souza, of for oversight call control gene our manufacturing believe future, remain the and I'll therapy While will our of development over and now we product allow of clinical

Marcio Souza

Hey thanks Stuart.

of rare products serve have diseases. a building multiple of patients a to portfolio with We vision

of have excess We $X.X revenues in potential billion. the XXXX

bring to business strong base as years. in and markets products Our a coming current the DMD us accelerated an get to the to we periods serves growth several

$XXX reiterate to expectations. our our our line year quarter million guidance million for and first with of revenue was performance this in DMD global $XXX to continue We

and Our DMD Translarna franchise includes U.S. the the U.S. of outside Emflaza in

of first For $XX approximately the sales we quarter Translarna million. reported

ANVISA Brazil. of happy the week approval risk this decline As this fourth lumpiness extremely Earlier in from regulatory to received additional mainly sequential serve Translarna America. year last of to validation we the patterns in benefits expected, of quarter the for Latin due a Translarna reflects for patients ordering from this need. We're

Brazil grown in early For last Translarna of part has products. through the years most business access the four

substantial all will is as our increase patients allow U.S. price discounts This original Emflaza, price Brazil. through approval Now Brazilian will business and time which has with unexpected years in DMD's older. patients access in on negotiations steps guidance. for likely based we for X DMD Over Government. in been the our In is the will which approved result our reflected go in DMD next

younger PDUFA a to application aged X have expand to Emflaza our July X. of We label or include patients to Xth the

in is this later preparing team commercial this potential year. indication diligently Our for expanded launch the

we For expected sales line approximately Emflaza XXXX of million. $XX growth was first reported quarter in the guidance. Prescription with our

to the However, a seasonality exclusive quarter distributor. by our planned new and both revenue in transition specialty pharmacy was impacted

Tegsedi year. concluded moving to Now Waylivra, we later to Tegsedi review continued progress to be nicely and this with we expected which ANVISA

XXXX eager to approximately in who it. are Tegsedi to America bring We needs patients Latin

We early result have access next this should in sales year. [indiscernible], adoption and now established significant an more contribution Tegsedi early program year for which

between in Commission positive As monitoring mentioned, regulators. has and Latin patient is Tegsedi very this Waylivra by synergies country diagnosing access are Stu programs teams and by the a are early by country we ratified Once for pleased will the America. There opinion initiate clear we European see Waylivra. been EU the to granted

track manufacturing mentioned Stu Friedreich support as to later BLA for submit on year stage for this as of as well this IND We therapy are our We gene in an gene by therapy securing remain our pipeline. a to AADC final ataxia year. the

palsy in to focusing at cerebral working clinics. population risk patient on AADC identify patients are We

we continue will We we the and screened to portion make expected an CP provide update on significant progress for patient a numbers these once patients launch. have of

patients We value seen in Translarna. I patients and are development with like would before sitting starting AADC. We more obtain milestones bringing never this for give strategy to working life-changing as coming to forward will access reflect therapy also worldwide in walking. a look now market towards the to the allowing to our update such short year. clinical seen AADC This

next the patients in a Translarna readout we which dystrophin if U.S. Our NDA expect is positive the and XXX study enrolling support a to key resubmission would early year,

our oncology AML, three expansion. dose expected expected with [indiscernible] readout patient enroll to of upon the allow decided in completion quarter, are cohorts should to DIPG Emflaza, our the patients and leiomyosarcoma finding For All expansion first for early upon next which label be future enrolling study initial of trials studies. is this the year

Lastly, stage in IND, we gene the an of disease. to are financial in designing FA submit the our the this devastating for trial therapy first

the I now share would this turn Principal to our over like Christine? We Financial expect to call Officer. Christine, year. details to later

Christine Utter

DMD few a Starting Earlier full and on that we topline the the Thanks press refer our and details. with of I first details quarter our XXXX for to of summarizing financial XXXX. for for release financial our release our start results guidance today I'll across a with quarter XXXX. reported you we first Marcio. results performance million issued comments revenue quarter. the $XX.X for million $XX.X our for Translarna in quarter of of first franchise compares XXXX. $XX.X the combined quarter the This revenue XXXX product compared to in net of million to were of $XX.X for million combined revenues net the first

from is Latin trajectory mentioned, patterns lumpy influenced As Translarna ordering America. our Marcio by revenue

For in Emflaza of which reported XXXX, for first the to reported net quarter the of we first $XX.X million product approximately million $XX.X of revenues compares quarter XXXX.

a planned in impacted a transition Emflaza seasonality mentioned specialty the were to both sales new was pharmacy. As by and quarter

franchise DMD revenue guidance $XXX $XXX million XXXX our million. of reiterating are We to

expenses expense in pipeline. of programs quarter non-cash also of a of approximately net $XXX R&D stock to advancement SG&A XXXX. for R&D therapy proceeds the and first Cash, expense XXXX million quarter equivalents $XX.X and stock-based quarter XXXX expenses $XX.X of million based to outlined the of the in $X.X in XX, $XX.X based million compared excluding first compensation the approximately at of our by million revenue compensation to first public non-cash have compared of XX, to equity million well million totaled in excess in stock-based offering for $XX $XXX XXXX Net reflects increase generate Non-cash million guidance XXXX. million expense stock quarter in our increased compensation activities. and for of of million as pipeline potential expense the $X $X.X stock first R&D support non-cash with reflecting was This clinical of SG&A associated excluding $X.X gene million. investment loss $XX.X platform approximately $XX million research full-year excluding in also commercial compared were of quarter expense. our expenses the the compensation excluding We for XXXX investment million million excluding costs XXXX were as expense combined non-cash billion non-GAAP of in in cash opportunity advancing for the X.X Non-GAAP non-cash March for approximately at December XXXX combined of our compensation $XXX would marketable securities $XXX million for in $XX successful XXXX completion after XXXX a to based first $XXX like continued to the to net loss reiterate resulted I million million. compared

to our business. grow We the strong start are a our I will over to the be happy financial session. call operator and advance to to Operator? in our hand programs question-and-answer position now

Operator

Vincent Instructions] Thank Our first from of Bernstein. Chen of you. comes [Operator question the line

please? question Your

Vincent Chen

Hey, thank for airport taking you Apologies the noise. for much way I'm back the my background from on the ASGCT. at very question.

of And compared XXXX for quarter some could level compared timing that fourth QX how wondering of upcoming oh channel OUS provide XXXX insight I additional inventory extent steady-state at was this guess, to the similarly sorry some to then orders? I So Translarna compared to the if orders quarter QX end this timing QX you end and on color can, you for the of into it how now in levels? with of your for expectations could the us first provide the of typical at us and you of beginning the the Emflaza

Stuart Peltz

hi the thank don’t – for answer Marcio you you Vincent, Yes, that one? why

Marcio Souza

question. Stu. Of thanks for Vince, again course the Hey

is our prior pharmacy So on impacts in was quarter specialty and the terms the U.S. the be Emflaza for on in question did that to handling to our the patients. line on team a the to to was your expectations for we that vendor other remarks the in and which going that distribution we believe with performance that part mentioned the Emflaza was of the in relation prepared based right, more new already support of the change made so of performing on one work the

your to inventory, the of general in much only question it's there. it's a finding So of matter that's terms of matter in in not revenue performance the done a of terms more been expense, of

was it that, with happy necessarily we for most key importantly impact which not for corrected year. is we're so the like we we there the So believe but business, no

prescriptions We strong in QX. in have coming the

that's grow revenue to guided continue guidance, to meet end for. our year we this so and to expect We what

for more since PTC from are to restrictions on we early are week that obviously to question we growth So to about programs orders with late there of the patient access that we give Translarna. timing In Latin bit term for having access dispensed ANVISA we Emflaza. confident expected, so on as there to approval just remain this to was promote a on that like term this of an the be and haven’t order America, relation QX had inability long short received the Brazil, patients and for you the specifically are little your including going now under were program but second we which before, of many just color, the talked

So families active medical needs not necessarily was and but all had spontaneous we and for demand our through from physicians the basically their patients promotion.

is more that's penetration proactively Brazil that Translarna, forward. forward expect our right substantially So to medical team with therefore and going team approval XX%. and to our moving is in we moving increase discuss now What field our able being the around

late this QX order I mentioned. So we as in got

of one and ongoing expect answer impacted terms always question? year Did because the being this total already our to not expect to guidance. well are the conversations any QX that in your be that remarks in calculations We in based be on by we demands as patients of there they mentioned I of as to conversations the

Vincent Chen

Great, very questions. much the taking you thank for

Marcio Souza

Thank you.

Operator

you. Thank

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

line open. is Your

Ritu Baral

how best Hi guys, you to because Can Stu, which Since I'm that I going I taking fitting going see you landscape guess programs the thing not program a is through mentioned. thanks quite how question. development for mechanism do behind ask do strategy, and the us you next right guess, Risdiplam see there's walk Huntington's but this the you about the in about few the program emerging I'm to the now, ask in? that

Stuart Peltz

Ritu, Hi question. thanks sure. the Yes, for

the issue quite interesting it the in splicing Huntington, CAG it of alters the actually a to in is you splicing right, case So is the small a almost specifically built able large Huntington's there point before. case again Huntington the think cells case less brain. that of was ultimately causes molecule a where added it splicing remain the addition sense where that and it we makes we're efficient and platform in really it modulate does this within splicing everybody and and a selectively the the it of selectively the consequence case axon way. molecules In like in small that where of mutation comes and the made dash mass a but from, of I normal a efficient just be in to SMA repeat allows is to that It specifically, more get identify pre-hunt out and neuronal protein the

you to to level don’t actually the And ultimately this want you in want protein. – of case reduce Huntington's so,

we've an that we axon And Huntington's termination that's a we the RNA way out that. made code in the on figured cells you understanding our a within that And degradation be slicing the Huntington's what protein happens shown that within it induces it the level you the have to therefore by so is of on the have a and been splicing level of small as actually of the intron that Huntington's animal that into RNA rapid based that what in gene within works protein we that could the models that's has reduce indeed go sense modulated a consequence identified. and molecule reduce a the premature actually of both axon of small and the causes the and is

easy So we brain available of and small get ineffective it. that, be is small will orally be the competitive therefore that's a to major reduction we of it going We the into all obviously to how of think able bio here the – take able think of is it ridge so such take cause behind will an is molecule not tissue, it so only easy. of think strategy be is think modulator all When consequence that that into actually we the molecule know obviously as it whether broad Huntington deep well. cells, to we it becomes the is distribution, to that of only would and and into and it brain is with about a to be oral to is advantage regions that a a easy then often the obviously consequence not and be get advantage as think mechanism a you the

market, do a terms the want to maybe about Marcio In bit little talk you of that?

Marcio Souza

to. the disease. Huntington's right Yes, number a are market of course try others there It's that to of approach are large advantages And of our was Stu I'll as there. out saying a and very for rare

marketplace. which now and patients better learning other FDI of we from believe developed completely. will to our going are subset which be is be learned unique position actually have there of a With going transfer to of than are some terms some this in the limitations very we to we a and some appreciate [ph] body like Stu SMA lot like continue disease in is a the of other in we by learn whole molecule probably served We to our but are aspects and the able are compounds a therapies being view that addressed prominent disease the highlighted, number not delivery in terms just partnership as here just with to the of others

year previously on guided the we want we extremely we feel next we excited, do that. and clinic enter as track to to So are

Ritu Baral

Did you the Huntington normal proteins well or Huntington's reduce the as mutant just…? protein as

Stuart Peltz

No.

and molecule - we're that still So, so normal be and an if does issue. yes, targeting within that normal you common don't the can Huntington's - the that's thinking Huntington's think so, we and reduce we the XX% enough is this to think what so by or it the mutant community protein mutant protein both, I is, reduce would

Ritu Baral

you FDA? of a to - could what's biopsy it your followup. strategy, what you - cuts but a Translana at a is early that different quick the also noted At looking Marcio, beginning, cuts the Stu, on there could the settled were the couple Got data could current resubmission, data strategy and for you fuel have resubmission data? had you that

Marcio Souza

Absolutely, happy to.

the So trial what that a key needle basically, are And is year XXX. study early resubmission next is, we for the Study doing biopsy.

this course milligrams about milligrams muscle. So is XXX taking for in of biopsies to XXX

doing that of we've of are with would with adequate. and two added dystrophin We data that FDA methodologies types agree the be quantification

a single how for terms and It's So in with as but quantification on, terms coming. they're batch of we're on we site enrolling We the California, we expected variability don't in quality infiltration to enrollment quality coming the like reduce obviously the so check so quite like and do that we're is being early study. happy of and of very nice. are in biopsies the

the is together samples since read duration treatments, all readouts no there in the end at are pace we once be early going batching and for it's nine the same of group, this that we the with months, of agreement points decide So to the we're the to way the to control be these them all do biopsies then study. at baseline taking had of year. patient started the FDA, next time going expected The

that going not or between the like readout to in be there's study. for So a anything

one our the agreed that is to. study we this So is primary have

well expression. exploratory in a point were resubmission, we who XXX. some study little more another seeing levels into mentioned before in looking complementary and time. And is at study expression there terms previously base in Study Translarna, are we our in of is in that's that going really well exposed for no very running of this is are to the and the We we trends Study as be own But and inclusion, and understanding up enrolling. understanding to study like that good interest running terms to as call as That bit supplements so of key XXX of long-term liberal the for line, not patients

Ritu Baral

questions. taking for Got all it. Thanks the

Marcio Souza

Thank you.

Operator

you. Thank

Our next Joseph Morgan. from comes JP of question Eric

please? question Your

Eric Joseph

the Thanks guys, Hi, for good afternoon. questions. taking

what willing discounting that one Just I of whether development. pricing the able a kind guess Marcio, late wondering pricing patient seeing you're And the just country to would run - are you guess of in forward? couple in of commercial you're anticipating region? and as first experience Translarna. the broader if would price I of expectations push-pull you as in us in that be of kind serve of thinking on to on help of negotiations understand your what Emflaza, drivers I'm could and on and result net just a that terms just kind reference about of amount here going

Marcio Souza

Yes, no, questions. and for thanks of Eric the course,

Translarna, I pricing tiers two here. are on for Brazil So in would say the there

first at rebate. orders new time the mandatory of one, the approval, So have the

CMS, the why discount feel much grow than be others within forward. and done, to CMEDs body both So past, of going we to the about expectation actual them there, the all in going the this And or hard the magnitudes like few have call discounts. this of we the that more are - that States, requests CAP magnitude guidance and is same smaller moving that's to hear back didn’t in of we to that's the called actually from which markets. let's in regulates all negotiations right? percents confident going This right, happens is than XX% included our for it's on that's the and We United to based year, submitted price is Brazilian very be applied be demand yet what that like in the price, Government rebates the price think our CAP we to of

future relatively XXXX. before, But programs, the ballpark of XX% previous you early I we Vincent others with the it's markets. only small from which compare part when mentioned most have penetration importantly, the but positive question net on a quite is, potential for the of is there, as impressive it's So access

So of substantial we on in increasing number terms following of a years. treatment get expect patients to the

year again, portion all and did and For we free required more is that see that to in market. sales was benefits [indiscernible] expansion here that that as well, of those in of prescriptions that expand So which exactly that well. the like we good any fewer and regard. the asked, we the believe XX% for, number expected. we drug having hiring the I field is year, all mentioned seeing are traction is daily, but should anything, anything dynamics, a that. as we're that thing drugs not a patients Emflaza the levers that A that the Translarna, to in mentioned It's are worth, the of there patients as There's question they the to. One as We is in are is you transitioned traction we did late oral some with to new and not in so. the have We we so the ending we're we're we going are of keeping, that. well. later tracking we are the account we're We're last positive. like But right? completed there good were line there. and have other number We dynamic exercise. transition pretty bridge force, program, there being areas more completed drug high call, If previous to having the for negative or taking number dedication dynamics because Compliance of in the continue we

Eric Joseph

development I'm safety and Maybe CureSMA, as the past be that more - be think and presenting, follow will that trial, JEWELFISH just as numbers, primarily to whether it. one wondering want look about know to duration where I functional just curious I could - a followup? there? be Got of if we patient measures but if benefit around could. PD guys it's far of you how to I oriented up to patient you just whether understand and should there'll try

Stuart Peltz

Yes.

So think, Eric. hi, I

So you'll lot including in in I be discussed - think going there's some they'll things of see gene patients don't and we're that think of don't time, going other bad this. therapy a of some at data there think to there'll increments will well, yet, so - be for a have as and other but but CureSMA. to lot obviously, really I be I we're data that's a

Eric Joseph

Got it. guys. Thanks for question helpful. the taking That's

Marcio Souza

you. Thank

Operator

Thank you.

Gena from comes question next Wang Our Barclays. of

open. is line Your

Gena Wang

my you questions. I apologize, I discussed, Thank late already for taking And the I if joined apologize. and little bit call a

questions and So I'll Emflaza. also regarding have the two commercial Translarna

Brazil it sales can Translarna, remind XXXX how us For like? and the in you does passively look

Marcio Souza

Sure. Hey, Gena, and no worries at all. Happy question. to address any

obvious large have. have, order country by have in past. and the that we when country the a of we the size for Brazil is the that revenues a or we number from for variation guided specific business haven't It's the pretty of So regions substantial we patients

haven't We numbers. given we... exact But

Stuart Peltz

and to bit like we where others Germany about it's good - Maybe saw... even about talked a we talk

Marcio Souza

Yes.

of have most the being growing successful So that had finding substantial, it's patient one of patients number going we it's it's fairly market, a a on exercise found, we globally.

as than more markets that in and Stu established million it's direction. there in Well, to there. mentioning, of the go to continue So, more some XXX going was than people it's Europe bigger is

So how easy I see can grow. it to think to continue it's

se creating that our risk there trend and regulatory are recently together is don't This per government there in Translarna. decisions, the with kind other Brazil in issues and very that from case unilateral for see we seriously obviously discussed submission. know for I take we this as the at of question, point might being a but types time we they be So see of our Translarna patents enforceable, submitted being any IP. see We don't

very happened. long relationship our circumstance opinion with been other that we've we of runway planning of been Our markets feel differently has period beyond there leaders and very the in the friendly, about. some talking government that very strongly, a But collaboration from the the level it's general, key and

Gena Wang

Thank regarding another And I question you. Emflaza. have

of the wondering versus looked treatment color then prednisone prior if how can you few trend So just treatment breakdown the a quarters? like last and the provide quarter prior patient the versus this deflazacort had in

Marcio Souza

thank much that. question that's so Well, a and for great you

So focusing for expansion deflazacort so, previously know Emflaza, strategy are as who secondarily focus changing on a we've treatment some I were with or the getting There patients the most to you the like months had plans last to Emflaza. prednisone. prednisone been as to mentioned, on XX and these in exposure lot step before like added

Some have reduced. of recently or them, removed very been

So were I we days, is and say trends that. days would big with [indiscernible]. happy mostly last towards or switching XXX so XX therapy on the patient seeing change and we're now, What a

So which key on in patients a we patients to they the using leaders choosing - to that turn to, is use while, for Emflaza, entire dialogues switch. engage a does and the the markets that who want for prednisone and to believe actively for what opinion is alternative are being are originally right? communicate patients This so we better to and ultimately we continue the to

since patients on not substantial to kind basically therapists is any growing steroids is So of a secondary a are with is patients two year now, forward, get and new from. prednisone still on entire this and growth of buckets right degree market and moving to there predicated treatment the so who number on patients

Stuart Peltz

those the because able on -- a so on own of payers also that the lot that efficacy. but that really can we natural to use data, able, then can year to us the to out not And publications Emflaza's where - we've to history be of demonstrate better material last our go we gave came been then only

I that's quite as seeing And why So also changes think that's -- bit helped people are well. over time. a bigger we

Gena Wang

Just wondering, the of non-ambulatory percentage what is on like right drug patients now? patients

Marcio Souza

On Emflaza, right... I assume,

Gena Wang

Emflaza, yes.

Marcio Souza

Yes.

treatment ambulatory So steroids comfortable you and patients ambulatory a The evidence with for was just how disproportional not recently last the if into how exclusively should treated. more the basically XX, became the right, that physicians is. changed look years, patient it more XX clear became no and market is almost be -

requests for new well. non-ambulatory of lot a got we as So

of We expect almost still ambulatory you still this number is coming It have later X, be well. to patients terms disproportional, the to a and not patients, is say at growth of point what would of would XX-XX. X younger more expansion it's which the A substantial to I time to there as the going of is in as capitalize but year. well lot in label in just excitements towards this

Stuart Peltz

of patients lung Again function are better the helping on showing natural Emflaza. a in because history lot are the papers

Gena Wang

much. Thank you very

Marcio Souza

Thanks, Gena.

Operator

you. Thank

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

question Your please?

Raju Prasad

you've previously think sales I the put reps Marcio, question. Latin you hiring all the Can the taking - force you've sales mentioned just of number for metrics the in expanded America. Thanks that percent America around billion $X.X in approvals be will hired? then US? recent Latin of EU, what you XXXX several in approximately with just coming from kind Latin And versus versus are - of Thanks. America, some

Marcio Souza

Raj. Hey,

question, So right. first like the

and force, the like about of So -- both fairly or but in we size number Argentina in have well. of as in sales increase that we small America Emflaza operations numbers was absolutes. the the general, The and Colombia expanded in for in as in increase are may expansion Brazil, on Latin the we not coverage only also XX% US and

me somewhat So for to silly percent. actually we be say like would

added we individuals and like here four more three So there. or

lot So but is in makes absolute of small, country coverage of a there sense. for relatively the

is would like Tegsedi. there the from of say coming there early introduce products Latin from Waylivra contributor cover bit Waylivra. we very as we cover have generally million feel to Tegsedi and by guided balanced, guidance, have going we When dramatically biggest the still I well, time, at we our to we're you're the specialties - to right you're of other looking we additional small a seeing demands are very about covered that's coverage talked a the good have about point indicators that XXXX, and We terms to that we a neurologists lesser the little actively - American starting which the Translarna confident now expansion business, So about coming in like products. and now And It's believe haven't $XXX on in both efforts and markets. part for other very pediatricians focused regionally. more that is Since have a both to

do predominant right not So I us, appropriate it it the don't think still this, now, to important us but market market be is moving it's for for would but forward. an

I million, a into arguably for XXXX, in If grow $XXX about the you for look has which Translarna from potential to US, as example, was talking lot of well. that number

for very look about and this sure our we into to we're it. deliver approach is feeling make guidance metrics continue to good and again, on really So we that

Raju Prasad

thank you. Great,

Marcio Souza

Thank you.

Operator

you. Thank

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

open. is line Your

Tazeen Ahmad

taking good Hi, questions. my for afternoon. Thanks

Waylivra This in street. So, Tegsedi. are just something thoughts might this, started the programs wanted then the opportunities done guiding thinking contributors, Thanks in to would about we on to a and about to work obviously Marcio, You've LATAM? best under-appreciated that have early try on I models be as way your be the could be? the of some market you the how you get think us for initiate think about you value I these and followup. access do How

Marcio Souza

Good Tazeen. from you. to hear Hey, course. Of

with agree completely I your So assessments.

we've this realize. I seeing, think been and they we're saying right

So more the in start or seen of to from first. we from patients, we've the with were of the for approval in the than Commission like Even let approve, before somewhat the European Waylivra of terms region. recommendation lot awareness a CHMP FCS expecting demands even me the better,

a of have completely terms of was gated I have seeing the recommendation. and we're that little So since I number done to work coming saying here up substantial number in a with before other investment cardiologists specialty even us and patients we're of efforts family. and like

be patients prevalence the XXX the end, and higher our on it's be in like right, regionally, as big the believe of range. region that reads end of expect, potentially the that since and Waylivra substantial, terms on in Translarna even would in to lower early is to of the anywhere not to fairly we we opportunities more as but seems that incidence haven't actually going got the you So the

through revenue spontaneous aware with materialize Waylivra, and have to having prescribing, for as we product, but the about physicians it's potential a were now, of happening. good, becoming demand some right revenue, there takes channel. the excited meaning early - are the excited for It coming So this while in all like, the key markets

The gold were In systems terms from from physicians the the are say place, dynamic that they're green everything consulted, on well, around were of X,XXX is patients Different for give the number we PTC ones there. genotyping is what the the of identified. in impressive because exclusive that would being and getting we partner and monitoring Tegsedi, talking that alone, of quite all already I feedback our Brazil programs excellence patients are as in and top in coming through program hATTR. our of patients about, take terms

Latin bigger much it's for America. X,XXX So in expected patients

I but would guidance, give are $XXX question yet, on as in So be mentioned we Raj's while going XXXX. didn't We I realistic that's for that guidance somewhat expect Tegsedi would is million say to you Waylivra substantial. for

opportunity So that follow on? I you the talk two this about had interest little a on appreciate And assets. and you bit to more the a said

Tazeen Ahmad

for one-year I data submit that pace the you've was you about enrollment, on as for relates to March of just the that for data, - you to extension to an quarter asked European you that had XXX due third that. think it plan but XXXX, of EMA of extension talk the you Translarna asked when will Study the to originally end to submit I need slower could think in if Yes, maybe opportunity, that for announced

to So as sense than that anticipated? be I you might initially have just think you from enrollment get a might to slower why wanted

Stuart Peltz

Yes. Marcio. Maybe - go ahead

Marcio Souza

Stu. course, Of

a So Latin markets enrollment example. a for the right, trickier, are XXX they on placebo-controlled little successful in it and and the bit is America, a study sense said, in is we fairly because for, Europe

not there enrollments. So for markets on patients core lot this available of a is

to had this like in countries are not in expand other Asia, we the commercially some Latin States. countries we United to America, So

originally to as little are and countries to we're expected open it the patients So confident, those this EMA, is start. took enrollment sites we some while very a was like communicated as and lower of there now bit to longer going the

to was them. some conducts. of getting they the in not some not to anticipating And even we Translarna of of they're up was in study are start didn't simply because the example, for Europe, Eastern or interests more it for it want honest, requirement in the commercial present sites, availability that during the be So the like Europe, countries starting patients, study

an we want been informing the them, we've being submission formally we've communications, interactions, EMA We've during year. the and procedure those all the simply and hey, one renewal this extension before year factors with them then more on had was contact with not and the So been a progress for submission here. to like, requested Based of we not We we going we comfortable C that's eaten we don't quite of in definitely us all be and the be terms bit that the in us one-year up to believe an going open the believe And it is In sides Plan it with to it's months the in issue. in for a last put few direction. feel going that extension. situation. right

Tazeen Ahmad

thank Okay, you.

Marcio Souza

you. Thank

Operator

you. Thank

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

please? Your question

Unidentified Analyst

capabilities for manufacturing Brian. program I kind gene are would therapy, for timelines and have question. the but manufacturing for facility capabilities, And adherent is mentioned you building bit learning if taking more look process you're just I milestones that therapy suspension there could out kind could your any using wonder guide Bert that there as on I any we on speak that for? or new in-house, is more, of Thanks established or guess a know this Hi, gene you our both little a earlier of this from?

Stuart Peltz

yes, I for think thanks - the Yes, question.

needed the the therapy of when material obviously notion that programs from are thought gene our think less. we picked be of that CNS we thoughtful I I terms since were directly you're region pretty in would manufacturing, the of level the think we into that the injecting

in case and level we're doing times two of it's example, the like the total. For deficiency, their what for AADC

per So kilogram, total there. amount like it's it's not just

been we've them MassBio, - to not able that's which we'll working that And the opportunity be that's we into, but to so commercial think gives be by us now to that going a - be Right is on the for done really going doable. is manufacture, and be that meant level for that we're to that. quite going our facility CMO with at not that

for So it they foreseeable the will be future. making

we - and it's facility is going manufacturing that In for is that and be going terms standard. operating it obviously facility to an will designed it's that therapy, an - into, be of plant committed going - to be to the we'll have the gene we biologics that think be GMP probably existing of facility

and this gene and have we'll to modifications we're that think we'll adapt in a that be some I will Angelman Obviously, others moving started programs to will other in continue within ataxia, us includes who therapy set in on. going we'll and work really So doing, Friedreich we make to that be be for going. have will higher, region. that. we've that plan. expertise help That of experience and get have moving and But to this qualified it obviously, That otherwise, people

ready that in we're good pretty actually think I - that's think I go. so to And shape get and

Unidentified Analyst

you. thank Great,

Operator

Thank you.

comes line of question Our Young Alethia of the next from Fitzgerald. Cantor

line Your is open.

Unidentified Analyst

this for Hi, for Thanks Alethia. Irene the so question. taking much on is

stand that? what you for the And preparations for steps on manufacturing give Can are? one filing an on on Thanks. just program with you that front the update AADC. remaining the us So where

Stuart Peltz

Yes, sure.

of placid end Really the of to that comparability the results completed of and think, part really they the get we seen - over that a you've were this all and about able basically incredible ability demonstrating pretty results improve. for quite I some are here time it the we're of CMC in number year. just manufacturing we of incapable to them we go have we're of completing to were to fire sense, the that, really who we've the all that walking we're are and these terms that's of doing or obviously, where by talked think is kids in the the studies children just now kids

be think we - AADC for be year. BLA submitted end be of now so for And that complete that the to right there will to will on we track a able so by the that, we're

allow for So good everything's we the essence, in the - to including manufacturing on the that feel us we track, BLA.

Unidentified Analyst

Thank you.

Operator

Thank you.

Our next Citi. of of line comes the from Beatty Joel question

Your line is open.

Joel Beatty

for the Thanks taking questions.

could and used to discuss you followup clinical the confidence manufactured the manufacturing, trials? The comparable gene therapy in to give the first that program will what was well be one on AADC steps FDA that take to you need

Stuart Peltz

actually? why yes. you Sure, don't Marcio,

Marcio Souza

before. course. Joe, how Of using we Obviously was same, essentially you? clinical before sites that used the in are in Hey, are the here that the So So [indiscernible] basically what used are material we process right? was two other that hyperstacks was that level. the the produced adherence done XX in

looking are into commercial. now We

was we had product number process changing like they So conversations, expect with a what's not the what for that, in of like the there FDA terms comparability we're se. per of

the into looking drug are comparable we So previous being analytically to.

a have time include don't little - this But to getting that job call ahead batch the way So we little Everything the being the that release we far, of with time on patients data But grades, to PPQX PPQX every time number and analytical the either need analysis he's on commercial sure you batch two know things really been of batch on say these key or on two to step. little getting for testing of methods, using because file perfect discussed steps collected using are producing, are release making certificate the bit Neil absolutely absolutely I very BLA. time getting very everything specs one doing how doesn't -- we of being or BLA probably finalized so been the doing methods we've is this. obviously so of still they're the is hard would because is I the and we those that we've familiar around running and has can - getting FDA released the being step with done. as like missing the working team. bit the the with those every great here,

we level biologics sure are very inventories of need we the well can is Considering get matching seriously. we be to the globally. levels successful manufacturing a everything far driving so patients kind good. the the we the those everything We are every and terms adequate take successful as to subsequently patients. to So we launch We product States sure specs only the a successful going are of product of for but United not want quality of believe with this in Since launch. the we so identifying and for day we the make doing making terms so the adequate we're are that for agency in launch single have for

Stuart Peltz

it And even to from commercial. the here changed that big process the again clinical picture is

is rather – from the So terms transfer of a of same a than way - really make in way materials of there just it protocol the wasn’t comparability take same doing it. you different

that good to able of get by the end pretty year. the being So this we and the we're complete BLA feel

Joel Beatty

Got it this then then by volumes for down clarification Translarna pricing a And is there thanks of question to or is there? quarter a that. and the were they on driven compared revenues component first quarter XXXX, that

Marcio Souza

timing bit lower some a That Yes, like QX as there's as like of patients and average little no well is getting well. came the like patient QX were new mostly so that's order pricing components and that some orders price per in QX younger, in there. for

drug it's stable we're mix more So actually good a the able international to keep price. of say price pretty would than being absolute I the

Joel Beatty

you. Great, thank

Marcio Souza

you. Thank

Operator

Peltz call turn back remarks. to you. closing CEO, Thank to time, the over Stuart like I'd At this for our Sir?

Stuart Peltz

and launch Waylivra of this America brings SMA The and includes Roche, the milestones first submitting in some NDA remainder you. gene the we've the deficiency, Thank for for by Tegsedi discussing. AADC BLA of that Latin the major therapy XXXX and us been

through are next look worldwide. of sharing SMA and you for disorders rest addressing I the plans week building vision the We biotech integrated company on call of of We AAN the thank our today. rare at for fully following forward progress to the joining a

Operator

this today's your for and you participation have gentlemen, conference. a wonderful Thank and concludes Ladies day.

time. your lines at You this may disconnect

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