PTC Therapeutics (PTCT) 4 May 222022 Q1 Earnings call transcript

Company Profile

Participants

Stuart Peltz	Founder and CEO
Matthew Klein	COO
Eric Pauwels	Chief Business Officer
Emily Hill	CFO
Kylie O’Keefe	SVP, Global Commercial and Corporate Strategy
Eric Joseph	JPMorgan
Rick Krause	Cantor Fitzgerald
Brian Abrahams	RBC Capital Markets
Joseph Thome	Cowen and Company
Gena Wang	Barclays
Robyn Karnauskas	Truist Securities
Alex Sklar	Raymond James
Colin Bristow	UBS

Call transcript

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Operator

Ladies and gentlemen, thank you for standing by. Welcome to the PTC First Quarter 2022 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions]. to call like host, to now O’Keefe. Kyle would turn over the your I

begin. may You

Kylie O’Keefe

review I and Matthew afternoon discuss XXXX financial our our Therapeutics to for Stuart Officer, statements take Officer, the our Chief the Hill. forward-looking Today's first in today to forward-looking Chief a Peltz; results. include Please Business Chief and expectations. Eric Financial Executive update Operating site Emily Good corporate you slide our statements. our PTC based and call, thank on current Officer, Pauwels; will on joining Chief Web joined by Klein; our am posted us Investor the today Officer, moment call which Relations contains with conjunction

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

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

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

Stuart Peltz

built realized has turn company, groundbreaking to PTC's first to year with stakeholders. this, founded could to the company but on into of we treat these biology. On afternoon thanks level, expect of at the treat worked for developed of When provide I'm results, and RNA need. to we for hard producing value quarter disorders medical Good The has while ideas therapeutics company. it all regulating reality. our Kyle. By the share diseases an today. PTC the Thanks, transformative revenue rare what patients unmet for update joining first a be RNA science I I help was on to excited

we biopharmaceutical XX into a the enduring grown commercial products. number have Over an past with years, company of

to every will of potential steady product of build three robust continuing new years. two new state value are with therapeutic us at growing to deliver into all would build to continue stakeholders. a to substantial This We that that company allow a revenues our to bring pipeline a

quarter start which $XXX for with revenue me was proud report commercial quarter the growth the Let our represents I'm net million, portfolio. to of first XXXX. the first XX% that over product

franchise quarter net million, product and was revenue another Our dystrophy demonstrating Duchenne strong Emflaza. $XXX muscular for Translarna

therapeutic. call. which to first the the be substantial addition, growth go in into sales to In in order be substantial Tegsedi most second we commercial more had in our Evrysdi regions show in progress recently Europe. the in for later quarter. Brazil, growth strong Eric will purchase on the will XX continues States group SMA countries, patients detail continue all demands recognized XX% rapid Evrysdi more disease with growth orally and therapy in the Germany. across more excited in effective currently continued is market the Evrysdi, administered with prescribed for It share than and demonstrates which uptake and share SMA market in United all the for than approved of XX% we're about an modifying sustained

updates drivers value We on. now I'd near-term that some exciting several have like to provide

CAT. For Explanation Scientific the have announced Oral that our deficiency, and Committee Advisory for Advanced completed we Therapies, we gene recently or therapy with Group the AADC first meetings for

of now the therapy proud in the meetings, completion of process, administered BLA. we and European now in We're the point If expect opinion successful these focus directly very to have will regulatory marketed be this a May. brain. efforts to gotten first the on submission CHMP the approved, to will gene With PTC-AADC

to Turning five development have programs, where registration-directed trials. now ongoing we

The of Translarna. trial a these first placebo-controlled XXX, of Study is

We report XXX quarter. the by of the second of end results Study expect to

positive also We opinion the the for Translarna received the in recently for renewal CHMP Xth EU. annual

and the well registration-directed established the opportunity controlled need APHENITY patients, therapies. We're for Turning with now or unmet and naive a by driven the PKU excited majority study. in to patient high existing therapy a by either on of PTCXXX PKU PKU medical population poorly

end APHENITY Results endpoint biomarker population, path are study registration. In expected a by addition, year. the from to study a has the defined enriched of and the this an

platform, patients quarter For fourth of vatiquinone results study in expect from of the year. the registration-directed this seizures mitochondrial we the Bio-e with in MIT-E disease-associated

excited CardinALS to PTCXXX in also the ALS. are we announce study in initiated that We

disease, Moving to debilitating excited to modifying a successful Evrysdi the PTCXXX following established our validated or with disease Huntington's HD. in disease is we're treatments. no be with splicing HD pathway by platform,

from end study an the leiomyosarcoma. the disease in forward oral quarter year. the SUNRISELMS From also the weeks PTCXXX, XX unesbulin initiated we in the our modifier, we Huntington's For first PIVOT-HD this in X study of data patients oncology splicing initiated Phase of year, by and first look with we portfolio, the to recently of

excited produce for clinical now Matt in to the multiple will with for to quarter. decade. additional the the have increase the success we has are detail proud pipeline provide and to make next Matt? over turn science, success that updates I'll I'm RNA development. grow strength therapies We PTC progress over call worked this and with demonstrated the that next platform while to business diversified will more the continued of on

Matthew Klein

Stu. Thanks,

development to of all Our continue programs. pipeline progress hard work teams our to

trial additional PKU. Enrollment trial is year to read our We in have this number APHENITY and Phase APHENITY ongoing we patients results with by to and end of a of of in the begin expect we registration-directed initiated. that PTCXXX X with registration-directed trials several the ongoing, XXXX. have I'll studies being expect out

PTCXXX population the is reminder, a placebo-controlled reduction two which randomized a response a who demonstrate PTCXXX a a responders, placebo-controlled to treated potential Subjects XX% the weeks. likely levels. phase will trial continue includes the treatment, the with levels, in during phenylalanine run-in endpoint phase. for of phenylalanine As subjects in reduction study six-week for enrich primary APHENITY the study blood To study with are to

Following all study, eligible will subjects enroll extension long-term completion study. be a of to in placebo-controlled

discuss trial from additional is expect Next, platform vatiquinone as PTCXXX. have patients an The seizures end MIT-E we by vatiquinone which with XXXX. with the registration-directed with ongoing our the currently and trial trials we Bio-E in results mitochondrial of two registration-directed disease-associated I'll of actively enrolled, trial, registration-directed

during either study The having enroll observable primary number the capturing patients reduction is will study receive motor this seizures, or four-week aspects minimum placebo phase number placebo-controlled worldwide. study XX-week of reminder, sites phase will observable are with subjects a includes vatiquinone. run-in XX of from seizures, study patients The endpoint approximately to a a of ensure by followed a morbidity. endpoints which disease other secondary in motor As

in Ataxia. second modified primary The and The study XX-week a fully trial with in mFARS. This is study is Friedreich Scale, the is expect endpoint the Friedreich in or we Ataxia enrolled, vatiquinone MOVE-FA XXXX. of includes placebo-controlled changed second registration-directed X quarter patients the the Rating global Phase trial phase results baseline from and

phase we of the establish by placebo. ALSFRS patients patients. the the registration-directed compound to XXX study or CardinALS screening XX progression, to to rate lateral endpoint and in planned followed a This Phase a a initiated sclerosis two-month X The enroll from study Turning will approximately Bio-e sites amyotrophic have is XX-week is from platform, weeks. baseline baseline placebo-controlled phase, PTCXXX, PTCXXX now worldwide disease change includes in during trial study to score which primary receive subjects in second

patients. announced the recently we platform, splicing of our study PIVOT-HD X initiation Huntington's to disease now in Turning the Phase of PTCXXX

the a study parts. PIVOT-HD of As reminder, two consists

first a year. our the are well our of to data. across and a study blood, XX meaningful include XX-week our regimens affects on months, tolerated and this our It's recently primary or of milligrams call had the and risk an will and is on oncology pipeline is look chemotherapeutic forward now either treatment We or PTCXXX the on to an turn dose of for four mRNA am collect subtypes one weeks, unesbulin an continued programs focusing all patients anticipate levels, that only efficacy. recurrence is with placebo-controlled also over completing Several XXX subjects on platform I LMS of survival. The the XX% XX update remain high a The relapsed/refractory the I initially over on with completed poor placebo-controlled X their Phase is of five rare pharmacodynamic approximately to course utilized will response and lines turn leading and in initial will is of initiated study patients is end In from Leiomyosarcoma, X prognosis. with objective SUNRISELMS on has by will but muscle data enrolling patients unesbulin of in to relapsed of radiographic will first therapy. we with a study is huntingtin registration-directed commercial effect LMS, levels. Eric? sarcoma pharmacology, SUNRISELMS and cancer the I additional and which enrollment refractory Eric protein global CSF relapsed be a our or previously part which they treatment minimal the two which or for Phase Target offer After in from rate phase study year. The biomarker Xb the SUNRISELMS the assignment who providing of the and three, the study, based found milligrams. was study and LMS. the proud refractory tissue. XX-week safety, now to updates for progress. demonstrated summary, placebo effects progress during leiomyosarcoma. LMS, aggressive trial and most endpoint aggressive progression-free findings smooth patients a nine clinical

Eric Pauwels

has created year. Matt. momentum year quarter, kicked the the that a on strong The we commercial building Thanks, team very with off last

with in PTC-AADC FPL. approval new for and or So be the for other to a for team for international Brazil, the potential potential partial in for lipodystrophy, indication launches in the shaping up far, particular XXXX markets familial customer-facing transformative and and year Waylivra PTC Europe of is

addition, of for footprint In expansion the Translarna. geographical our continued we

Our regions. to franchise global DMD all revenues robust across continues deliver

for $XXX of last over first year. is quarter impressive growth the revenue quarter was the Our first DMD XX% million, an franchise which

Let me start Emflaza. with

quarter and of and Our for execution access, weight-based net year. double first the Emflaza continued team last favorable Ongoing the million, starts, appropriate patient buyer more from first quarter drove high Emflaza digit revenue dosing. new $XX growth was compliance product

growth product Translarna. Translarna longstanding achieved turning net with new predominantly order that purchase fourth remainder the partially completed quarter where was by in in last for growth we We $XX of quarter. first Now be of group diversified we driven delivery robust the markets. existing in Health geographical to to the million Ministry globally on and year. revenue and Year-over-year had markets Brazil, of the delivered continues

We future. brand Pacific expand in presence our markets potential for markets are in to have of continuing continued the in the Asia as the growth these

Now on progress Tegsedi and Waylivra. our to with moving

American Latin to significant in continues team Our the momentum build region.

mentioned, order from drug purchase innovative excited Stu classification the received the Brazil, in the be Ministry quarter. which to we have As will group recognized following are in Health, second forecast first the study, of

Now patient a Furthermore, treatment. milestone to course growing this patients and which awaiting strong number the reflects year. of the is anticipate the purchase orders be significant identification additional hATTR over group continues we in of Brazil

National drug inclusion HTA long-term discussions simplifies dossier Technology, for in has essential which CONITEC, for for list, of Tegsedi is team the the of the initiated access. Incorporation the Tegsedi submitted has the which to and Finally, Commission

continue our this first submitted of product regulatory in quarter to We and Translarna, be NDA in America, in Latin recent Tegsedi third Waylivra to a of making submission ongoing our innovative country. submissions Mexico, with this build presence

certification. approval deficiency. decision the first anticipate launch second submitted We I Waylivra for we a application the for the for Brazil mark for first PTC's Brazil, in ANVISA preparation If for the and XXXX. an in in December, will will treatment half this AADC last be touch first reminder, a FPL approved Waylivra globally the approved, of approval on will the to now therapy FPL. for of As of treatment gene

approval. In franchises off key and opinion identification and Identification team our also set via treat patients of being track deficiency high near additional AADC to launch therapy CHMP PTC's globally are have in programs is forthcoming conclusion, European of potential treat across We and enriched identified, Expert to centers countries and XXXX. risk post ready AADC access May is excited to of prepared early centers the future. are well. continued growth to stage very approval. the neurological start team are the preparation commercial the activities patient in patients been are to ready Europe shortly execute continues accelerate progress has in the efforts qualified on our in for these a centers populations screening in about excellence on and to Many the of after and strong in gene

Now, call the for let me update. Emily financial over Emily? a turn to

Emily Hill

has Thanks, with quarter XXXX, Eric. that report near-term I number of an once and commercial to are a expected strong am exciting forward again looking performance in delivered PTC results proud this to drivers excellent year. value potential we

continues Our strong global growth. to commercial year-over-year infrastructure support revenue

are We continued expansion. for well geographic positioned

the from to continue come Evrysdi with reported growing in and XX revenues the I quarter quarter turn XX revenue In results this a invest quarter year pricing first XXXX. of X Evrysdi quarter from were DMD additional the XXX million quarter of to XX the XXXX, release franchise of compared revenue of XX sales for with revenue in last addition revenue from first line up Of is this, we million. and U.S. us million first XX in includes expected quarter first revenue. the million results, XXXX. as XXXX, CHF the million the top Our of royalty other million revenue revenue royalties the royalty quarter This innovation. this of payment quarter to in XXX million the Beginning included compared the press XXX Emflaza to financial Total collaboration compared first quarter to first in outside will million Please first and in base Europe totaling XXX million of today that sales issued total details. XXXX. in of was to produced our combined as to in compared to milestone of million reimbursement throughout as Translarna milestones refer also to for of quarter as in first to the respective of for this revenues now allow the first approximately million the the XXXX quarter in revenue of continue year million XXXX. of first revenues XX in royalty this growth XX and reported and is markets online collaboration was year.

start non-cash, sales million payment R&D expenses PTC a to is Roche in to stock-based the quarter as Operator? as XXX and December reminder, million of compensation XXX million XXX. XXX operator call stock-based million stock-based XXXX, in million for cash hand of million expense of to stock-based As totaled the quarter million compensation to million Evrysdi first quarter the non-cash, million milestone eligible to XX in first from receive were first in for non-cash, over of expense quarter reach XXXX, excluding XX expenses to XXXX XX the million XXXX. SG&A a compensation securities of Non-GAAP XXXX, to for excluding question-and-answer annual XXXX, non-cash, our XX expense. XX compared excluding XX, XXX of for sales-based compared I'll the marketable of now first and excluding expense. XX, compared XX the equivalents session. were March million Non-GAAP XX compensation Cash,

Operator

Instructions]. [Operator

first comes Joseph Eric question with Our from JPMorgan.

Eric Joseph

guys. on and question evening, for just on couple one the and the a Good quarter. Thanks I Translarna then the on pipeline. congrats guess taking

in primary contrast You're two in that of distance the Russia just correct secondly, compare performance quarter's weeks? XXX, this at then at used of being taking change change DMD? six-minute with kind talking to reiteration make it cadence assume baseline distance? to with franchise? multiple [indiscernible] DMD all, slip And top in proceeding terms wondering Study looking study of compared of the in frequently it guess of from this the the walk that you walk. relates six-minute if or Is could sales of are are the as in here quarter about in six-minute you to line I headwinds you how endpoint to just revenue compared the sort between to this by performance second And you're just at sort case, or related to point First, guiding in how walk assessing in intervals [ph] any and

Stuart Peltz

the for thanks question. Yes,

who treatments probably, as nonsense exclude the the obviously, region. unfolds. continuity plan boys been we you volatile So, obviously and and of it's helps us closely men as know, obviously really and for plan Russia, in the Duchenne young a medicines, sanctions accordingly dystrophy do to But watching we're muscular it situation -- if on necessary, and focusing business. mutation we've have we And the best, the adjust in

the we've on to of think treatment good that. date we've patients to able been is, continuity and So for news I ensure all is the been focusing

think be currently, terms receivables. usual. it's And point we've The of in other collecting this able to been as good revenues, is a I consequence as the that also business of been

our really So revenue guidance with exposure affected right our now. isn't total

So to that. the issues talk about want Matt, do seen to it's with haven't DMD? -- you hard we any

Matthew Klein

Eric, answer question exactly regarding that model the the as you all which of the for it one for final information but other Sure. mentioned. six-minute it's thanks would only analysis. time to baseline sort. why the also do a and XXX, includes data to Study question. your points, makes use it much distance, To obviously That's more collected It typically walk use approach not robust includes a model the of

only but time previously result made. visits the it an at asked some about include the the of points thing that this using was window. the observation, -- does, we in COVID, other time that not observation you you By of which visit a think also discussed outside a can had of occurred given having The study I also specific model, as

on also which only but that collected. distance, is a And time so information the of the was have benefit walk that distance not you model at

they're a So in giving the analysis. overall just you information more lot

Eric Joseph

on very as Maybe helpful. Study up well. follow That's just XXX one

had ambulation baseline, to any you to patient ranges point would I in ranges? six-minute walk quotas patients XXX of guess say and terms expect you're those or distance at Are that walk are Like so by originating Thanks. XXX to In baseline would at accrue seeking evaluated average associated this demographics there know XXX, -- you [indiscernible]? XXX XXX you of baseline can ranges? are proportion at for the different stratifying I on. certain that with you other patients a sort distance be of that like, for baseline Thanks?

Stuart Peltz

Matt?

Matthew Klein

Yes, sure.

So population XXX is that XXX XXX is commenting talked as the two mentioned you what overall about study the past And as distance modified be we've the boys. not We're values has as of XXX. and populations in mean because only and would that baseline there far population the specific then walk boys. were, that exactly

Eric Joseph

Okay, Thanks questions. taking again great. for all the

Stuart Peltz

growth revenue the And just thinking in terms over XXXX our you Eric, other about Translarna be. would for question, of had

So compliance last the to age what the on expansion, it's was expanded new and that we -- year patient got Brazil. we driven in be think going starts, geographic patient is high label obviously by

[ph] in focus. strong markets markets, franchise anticipate markets, That's our for the going growth footprint then market. for in revenue also the eligible current XXXX. optimizing We acceleration driven of the Translarna our access in in access new to driving the new current That's ongoing in be and and all the presence patients by the

year is plan another be we the going this for to think we franchise. So strong --

Operator

Your Kristen Kluska next Cantor Fitzgerald. question with comes from

Rick Krause

to Thank for you are that a multiple are running have, seizure for on taking two bit can questions. than We'd to We you given experiencing. during this can about are measurement observable of activity? talk Rick Kristen. measuring that approach the In our be types How there is the little This tailored typically a how is are here. for trial, you seizures just some more MIT-E everyone. interested know of seizures got patients seizure patients in you lead the that of types Hi, which others? these

Stuart Peltz

take do that? want you to Matt,

Matthew Klein

Yes, absolutely.

with symptom effects talked of mitochondrial of at highly trials and MIT-E disease. are simple seizures patients. disease the XX% typically reason causative mitochondrial that on is given are these all common we patients study vatiquinone of energetic refractory global have disease the and that pathways mitochondrial morbid which antiepileptic seizures, are seizures, disease-associated the target is XX% So About about to medications for our of the don't mitochondrial commonly looking that a and epileptics in

in of children. seizures actually oxidative many them heighten causing which In what's stress, is fact, these

it's you you have while benefit your seizure may some in seizure stress, by antiepileptic, children pointed offset in oxidative of the out the question, multiple but these exacerbating do as So the them. have increase And types, parents sure the motor reason to you're want endpoint seizures for highlighting, count for can that the phase and make focus primary run-in is and observable the you is observe the exact pathology. the to which

involve the track including we are vocabulary in we they seizure patients see we the observable with extensive we list parents which And of that capture a They observe -- undergo seizures diary. the ones, seizure highlight base with and we different as that children the a in changes they to so activity. provide ones training their diary, motor counting of the in motor the what types the do

compare sorry, and these motor a And vatiquinone with capture of aspects during will We're six-month the we'll or primary minimum -- I'm the will other seizures. as are run-in or phase. following be placebo baseline secondary that technology. comparison, again, observable then XX either and monthly then endpoint requiring observable placebo a serve of will weeks of end the the points so following of -- change vatiquinone That of therapy, frequency. But baseline six seizure the we'll rate seizures motor calculated

Rick Krause

ongoing you. about stage enrollment pace an the how And Thank that. idea versus After clinical one maybe trial for in sites of initiating be gone. really performance interested more. PIVOT-HD enrollment trial, you recently We'd expectations? Phase of you the can just there has Thank X is early anything of or Understood. say getting

Stuart Peltz

Yes, thank you.

So we this recently initiated trial.

results rest for yearlong anticipation trial And we of is what then a So obviously, that is, year. said that's end what a broken of on it's have we'll the and the our is our the XX-week into is the going XX-week are from part, plans by the the portion year. up

Now going able placebo, protein and patient as to that's treated the get blood, the in information. And of also versus so levels looking then and biomarkers of in NfL the well at to safety RNA at such then be at as CSF. HTT of the level cells that the be the looking looking -- HTT as at obviously we'll looking mutant

Rick Krause

you. Thank

Operator

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

Brian Abrahams

will you a -- taking one And bit Hi, my guys. if more these And launch process? discussions which recommendation you or a there. kind wondering you've engagement Thanks meetings, on you identified Good degree kick a maybe elaborate the expect then could Huntington's? afternoon. more neurological launch is on think to discussed screening confidence as elaborate questions. Thanks? My then can of about little I I just to up off for patients, of centers have some had about the little first already initial preparation follow guess was you in CHMP. with I PTC-AADC. It positive centers a that sounds that productive like bit they've on maybe with and bit, we numbers your a the launch the a

Stuart Peltz

Sure. that for Thanks question.

in And that obviously having where the patients, everybody, can -- in to And our talked AADC announced like disclosure, hold at Committee patients their looking -- sit, ultra of we've type that Therapies, opinion consequence that in CAT, in an and obviously, we completed -- It's they quite we committee gene the the will to we therapy. morbid the CHMP growth much that's which pediatric is severe patients see you severe -- May. that's you we form CHMP have told arrested orphan, at you've we'll similar is as their remind highly unable we So -- about see and said and SMA they're a we up, head dopamine, just to stand. of fatal as you the that. rollover, where patients recent disorder working be been looks the with the seen for Advanced like

successfully a both said and as Scientific that as sending that, consequence we meeting of their they'll a Group that anticipate And May. as opinion well an recently So we that had we final Oral those. Explanation, Advisory be in completed we

Matt, about we Maybe, So felt the charge. leading do good was want there -- you it. to Matt

So want more, do Matt? you little a to talk

Matthew Klein

Thanks, for the Sure. Brian, question.

activity, to in in positive -- that optimistic cases, May a age. and this to on end the the healthy perform a kids provide able content heading And the we've and and discussion at stand, we're which The therapy obviously, of this based that and the near to ability sit, no of provide many and the is now the we positive lot towards obviously on to compelling gives to very might they're, walk collected been So, gene of label, that lot show about patients. process sign details opinion a able the data motor meetings the bring we're of children around meeting and look are the to being obviously, date to treatment us as that opinion. focused a to who forward have

of part of important durability, is other the was the an really package. the which think important part the I data

So we up to benefit motor drug. have even XX follow-up durability eight, nine, years six, data benefits of seven, and of from showing other the

to also be the administered brain. we're the think as Stu directly really this that I would, therapy ever excited first said,

the the to our and launch procedure, therapy the think most. in important preparations surgical neurology it's the exact needed use really a with neurosurgical adult pediatric in surgery entire guided therapy marrying gene centers for I'll location advancement that brain that that's common segue think the gene an to of I of and where of excellence. that about a it's we now can delivery stereotactic field to

Stuart Peltz

Yes.

preparation. Matt So to, hard to been and were as completing maybe, we working alluded about ready get launch excited for these just so

think some ready of as -- and both time, quite screening. really doing the I as getting identification, we've there's a for neurosurgical centers been lot this so And doing been well for patient

everyone disease. new a relatively As is this knows,

been that's to we've identify And the as different now and and XX We've in in So finding looking patients, them screening done all countries. over accelerating countries. you different XXX been been have patients. well. We've programs finding

about been excited on? to bit that really we're want have efforts going about So all a the this. do talk you Eric, little

Eric Pauwels

opinion centers Yes, important certainly, as on been are Stu. for on our of number treatment of part most a that's education, your we've years. recent to We've working top going The in that. already in launch of many accelerating But in particular, preparation identification, really pediatric disease preparations neurosurgeons focusing well. been for as preparation key has neurologists efforts. progressing leaders surgical point, the cells been been and well patient with Brian, an of the both And in extremely

to we in are each are expand are that ready. So that we're there time. many ready be and be of we will to these ensure ensure your continue have main really that each in continue now ready, of will one answer that centers question countries to the going markets major to looking as simply, to And over centers multiple to

not continue that other we more Italy, have of programs. and And you will Europe neurological and is available markets in we the in disease and Germany, treatment of patients patients we and the and things of and education, only will, treat early is and to centers the the provide as have Northern know many find do families will will UK, that the we more expand, more in awareness if we access this, as a the we that certainly them, have One that see demand. course, we France, when

launch. have countries as going are many certainly multiple as at of to main centers we centers So in time We'll possible. the of the each do

Brian Abrahams

that talked potential been guys there's Clinical Part looking for something just super that's morning. to any know one could the a helpful. have for there? accelerated also just change was going escalate what maybe approval? knocked in approval. make for that didn't Trials we're to in is key to if the a then Phase B, milligram XX this X DSMB that wasn't And The I to that'll get a recommendation see study if volumetric Part to accelerated That's view that about used be guess I And Thanks. sure dose there endpoints endpoint goal the was quick because you you posted curious to B? for there And or is We XXX. be listed the Thanks. posting, Will on being And as as be the just Is it down? MRI safety?

Stuart Peltz

Thanks for question. the

think will the the question, see the what for of numbers not do? is And from should based in first we protein looking at on those those level looking PK do Phase titrate We safety, the terms or terms And studies. of also RNA to what obviously of terms what -- in for, CSF that how the well, part titrate level what need we we're look in blood compound. whether and of we reduction of the we'll biomarkers, besides I to we CSF it's the what of the in we're in levels we saw increase that Is so X.X and see the the really are. the in well happens the X blood and X did that in as levels into fold define same the HTT of reduction. we

In little terms of to MRI, talk want a that? the you about Matt, do bit

Matthew Klein

Sure. in follow so the huntingtin will levels DSMB we're And the seeing along of in Stu's of reduction to certainly. we up all the and mRNA in it evaluations then XX% be Brian, range. on combination just as at protein, XX% reduction and terms safety Yes, targeting said a of what point, to

we then doses? And are safety additional that the So titrate from with two in and then, how course, marry to the of close And go to DSMB. evaluation first molecule we the need dose? the with do

that enough pharmacology part, PK/PD In terms be for portion and MRI volumetric on focus to on relationship correct. focusing the XX-week [indiscernible] there will the right, nine you're first What's be and tracked that months in is an ClinicalTrials.gov, of second endpoint listed the going biomarkers. that's really

Brian Abrahams

again. Thanks, helpful. Really

Operator

Cowen Our next Thome comes from question Joseph with Company. and

Joseph Thome

remains done to them thank on the taking been and with surgeries AADC that meet submitting be ahead sign in of Hi. Good have you FDA to afternoon that and one able last the for completed Have Maybe those you off year? what on questions. were BLA? the U.S. Maybe

Stuart Peltz

that. Yes, thanks for

of and what in CHMP. the we CAT obviously we last needed So with said, AADC, as the the the with completing we're throes

helpful be quite we've the for felt that to think we So and complete going that's gotten FDA. we

are what BLA? And that, our you through plans -- based on why don't so the the Matt, next with go steps

Matthew Klein

the Yes, absolutely. question. Joe, thanks for

As finish FDA. to to obviously the those leverage And with be step. further with to to agency and made Europe package this meet in able The and will the we've the forward moving package submitted. request said, learnings the look plan really enhance get meeting meeting for mission said, has We'll move you forward. Europe then we interactions wanted in to line next all on been aligned you the the and use regulatory forward with the the be and with agency to the as

Joseph Thome

And sense. makes some did question. or lot That the you couple more of a some of on milestone acquisitions but or that payments maybe came pretty the enrollment attractive past upfronts approval. with financial A then had years successful Perfect. data over

of you baked some guidance? the highlight Thank And would could helpful? that milestones you. ones very expect, those maybe that are if XXXX just So the be in we financial to should

Stuart Peltz

sure. Yes,

baked So And Emily, they on. in. you're are

Emily Hill

Thanks we clinical for expect of AADC question. pay that's in the have expected completion for success And XXXX. also and XXXX. in million developmental then due for a $XX to for in enrollment financial PKU, the a payment XX milestone million the of do We trial

Joseph Thome

very much. helpful. you Thank Very

Emily Hill

Yes.

a sales in $XX we to up the for milestone have just and reach certain flipside, that revenue Evrysdi, milestone the potential million Roche our I'd additional the for thresholds guidance, On expected from thresholds. XXX to remind million you there's

Joseph Thome

again. you, Thank excellent. that's Okay,

Emily Hill

Thank you.

Operator

Gena from Barclays. Our question Wang comes with next

Gena Wang

three my have for questions. I Thank you questions. taking

regarding is one first Emflaza. The

also XXXX. want drug six should younger label that orphan no based Just to And clarify expired XXXX. after also months exclusivity and even expansion patients, be on extension additional assuming in to the protection seven-year more

and first And percentage both Since question huntingtin protein similar that's every you kind you've blood expect collect in learned My regarding trial. data, renewal submit versus So data, of need in in reduction PIVOT-HD huntingtin protein will the CSF you is far, blood? receive the regarding Translarna. CSF in question. what and data to do you so my third on based What is the Europe? year to question second

Stuart Peltz

Great.

the exclusivity our that obviously I'll Yes, a number we're the do options take And around both so you're loss So to the patient in terms we we're lot including communities. then pharmacy Obviously, services contracts relationships in patient optimistic patient of order doing partnerships on that. levering of patients, a Emflaza. specialty Emflaza to XXXX. key we maintain and of retain -- which evaluating Emflaza all the programs, brand, correct. and from payers. channel the some we to because then with that order are It's defend in to our we're evaluating of programs provide through working caregiver of to and one support in options work first support have leveraging the the the driving that obviously And

So be the in The you patients loyal. relatively patient might are pricing offset aware often this, rare support actually by probably market, discounts programs. disease of generic are

don't through a Translarna go that In of bit? why and you terms what put in, Matt, we

Matthew Klein

questions, the Gena. for you Thank Certainly.

safety data So collected post the -- based our annual safety renewal marketing registry. that's is includes from

of clinical we from evidence As data the include provide benefit. to STRIDE well, registry

a continues of favorable renewal. So information authorities obviously are support looking key forward that balance, European is their basically risk the the which benefit what to it's continue to

Stuart Peltz

right? was And question third then and blood your HD CSF,

Gena Wang

Yes.

Stuart Peltz

So I reduction the it's and altering occurring obviously we what's right and said juxtaposition on splicing. you the if about exposure reduction, have cell of how the [ph] look in interesting a both what's at what's protein, the the then within right. what's point the and think from an when think the question where of you seeing of going good RNA sort much -- you reflexive And of of we're that view

see nicely. that quite So you

about in the different measure, it's think CSF I measuring almost biomarkers, the what's and on think cell, you what's a right. not when going the within you're because However,

outside measuring You're cell. what's the

And secreted, the HTT small probably rest and CSF. of amount that so is there's right, we lead amount that the within a know a cells, consequence the broken HTT of of probably of that gets to it

what able be to probably of go to anyone's monitor And the going the CSF and question than the see will patients. be in fast becomes think occur? saying, effect I this see what's So it completely HTT this volunteers changes also we go we'll that. what learn and that reduction we because what and down? CSF does interesting the estimate every the the terms exposure so of the healthy And same drug brain, would maintains mean is part from -- done in if different it PTCXXX are of What of the of the this ever level best to or is is saw subjects the how we're HTT versus it is in the what to going within is

due in going cells, within the the to breaking a the -- consequence think a be know So way in could you learn lot the reduction know we're careful where of because can consequence of was like of very will of a altering that the if about HTT secreted you that cells. to not exposure, be levels it, broken there terms things level really of CSF actually

be goes you're it interesting. away you? fast quite how help how So Does might and that measuring

Gena Wang

helpful. that's Yes,

the blood? sensitive does giving like, we versus the actually the mean actually wondering change in see Just should in that -- CSF, more lower CSF baseline

Stuart Peltz

don't We lower? have enough. mean you do What that

Gena Wang

collect a in be in the from cell protein blood so level Sorry, you protein higher have then CSF, only much inside because the broken in will the versus versus protein cells, in the the baseline all CSF? you the released blood the

Stuart Peltz

That's right.

So is percentage occurred looking for of we're right. change what the as that, consequence that a

to you see HTT change that normalize you normalize and in that then you'll you'll see of cells what in CSF. in the So the that

Gena Wang

Thank you.

Operator

Karnauskas Robyn Our Truist. next question from with comes

Robyn Karnauskas

right, taking okay, for my couple. question. All Thanks a Great.

how just first patients they the identified, For at? Maybe a and it where you've can be. little AADC, are you around bit trajectory talk launch this about can the question's

in many patients And that give follow platform an Asia, giving And so great. maybe up. a So have given update. would be I then

Stuart Peltz

globe. patients Eric at But found country comment around looked ask we we I'll to obviously, So in that here. the every

There's XXX found in. haven't find it's founder's been world. how found clearly that and we programs a we've done there and that It's the everywhere And to there's identification which in sometimes -- we found could So is is it but patient Obviously, said, on misinterpretation effect, this ramping been learning Asia, the has it up. within be thinking that clearly everywhere. higher the country -- become we've no them. we've over as

So we're want about talk identification little bit a findings? Eric, accelerating do and finding them to patient more as well. you

Eric Pauwels

we've now years, Yes, now focusing Robyn. sure. of over couple question, the Thanks that to know the ours because for countries be than Patient good treatment. of been countries XX been gene will across really multiple perform mentioned access as of going we've the something can to of have finding been programs which geographies. here seeing progress And has good also we've in as had These they're every have that Stu for we've are at. been therapy hundreds we XX mechanisms focus reimbursement past countries. the screening more right quarter and centers really earlier, And excellence

patients that our So been pleased all been it's we we've screening. incredibly there. screening important patients the We're that finding focus of type with ages. We've

-- of in terms very to terms early in just few of So months their teens. up

lot centers. of the centers a doing of patients seen now types focus we risk patients. these lot and So been across in age in as cerebral We're testing in high as testing on has particularly our groups, been well finding in board key and finding markets And as a palsy be and population primarily these severity. patient patient these all well, we've identification epilepsy the where have or

earlier Another about we are excellence important playing of where like as neurological is there These even highlight Southern interest identifying as early a mechanisms, access a are in screening role. also and sequencing excellence. more. to the and thing France, very I I'd Europe, too are treating we Germany, programs comment strong in is And where centers mentioned, have launch, as the of access Europe, patients, Northern soon centers we

in So we have key very opinion countries. active leaders these

found in specific we're is where less. pleased We've or more finding been no we So key and market to very point that. there Stu's geographies earlier, all the with patients

Robyn Karnauskas

patients like, like though, about better bit is trying to you're and though speeding in given different it slowly where a you it Breaking bit help I go you've little how that's launching are us finding at to feel hard the them, countries, been so people can to down little up? find where think that you're this, a just working

Stuart Peltz

patients, one disease have have turn and misdiagnose new out and -- remember those people either to clinics to algorithm you you that think need refractory to you a places, -- find got just when right right. you or epilepsy into other have clinics I over to figure thrown that of the or the So sort to sort CP you to of

used And a practices all so that's laboratories why when over we have XXX them. find in different because lot. we different many best the a things. to way everybody did patients different say We And is best countries find and sense using we over done what's of to programs the to would test then many start

and we're over in, been we them searching say we we've those countries in countries. found country, XX found all every we when about that in of patients So talking

getting we're approval upon patients surgeries. it ready for comes when the for ready launch, line to getting a up to So

goal. So our that's been

Robyn Karnauskas

I how a one to gave other do really you questions but Emily, add first around great about just should Thank that? little treat in bit from for And a you. help quarter patients, saying job we give the Brazil. of model you just quarter a second and Can you I'll around model some lot LatAm, color [indiscernible] far confident back That's I'm are can How like trying quick like be PKU. backed asking the to that centers really enroll given that in the time? to continue how you And up. They're efficient. perfect. a because mean, PKU actually up about and struggling. are to inability and people guess I on it, did you clearly? more buy us other so mindful, trying how know question talked Emily. I are -- question clarity be you feel

Stuart Peltz

of about they view, of with So little I want think maybe things have moving you trials Matt, point a how first been -- maybe and the bit think been going? have clinical point talk PKU from on to PKU, the I our

Matthew Klein

Yes, sure.

again lot and I quite global of in leveraging our mentioned. to with not around pull And we're will from sites world, what trial. you've a the Robyn, seeing So, say participate infrastructure we're study investigators that, frankly the once

done managing so flow. in really terms a projecting enrollment of And have and good teams job our

So we're enrollment at this not seeing point. will positive access to It be centers. an challenge

Stuart Peltz

your And seen So asking last of question you are that. lumpiness we last haven't your -- about was, of sort --? then Robyn,

Robyn Karnauskas

Yes.

You talked from as orders Brazil about well.

that model explain make how quarter? we orders? So do they we Thanks. just do little come Then want more LatAm? how think a sure in quarter, Do these to second we correctly. in you us first for the on exactly granularity Could about it we the give model

Stuart Peltz

do through? you Sure. to want Emily, go

Emily Hill

because We sure. that lumpiness. exactly don't give Yes, quarter-by-quarter guidance of

remain hard that give when it for say revenues annual color So purposes. contemplate guidance lumpiness included. other modeling will our to and we that in we've the we franchise annual that our confident give guidance guidance, is additional DMD and both I for we the

Robyn Karnauskas

Okay, great. so Thank you guys much.

make question. Sorry to to be I'm so want quick. Thanks. sure [indiscernible] I

Stuart Peltz

Thank you.

Operator

Our next question comes from Danielle Brill Raymond with James.

Alex Sklar

Couple Alex regulators the us contingent Have Hi, great? are timeline exact everyone. this and would is EMA one stat of This about if just is the way on or hitting how much access drive another be line following top us the given on any putting Study indication putting registry? that from Danielle. And -- remind actions you regulatory is have data into And -- Thanks. can they sig? XXX. market EMA for XXX whether Study upon Translarna like readout,

Stuart Peltz

the thanks Yes, question. for

of quite the the data five results seeing off in the includes at comparison some walking trials, the in of the think also that the have not trials data years but function. where pulmonary I terms the and ground, and looks the we've of years, believe the half we're greater we saying registry totality getting better really that of at and better a the much results we this for the time been only that totality EMA and

survive. about of you feel us registry endpoints looking and you the meet. showing profound consequences So those of beauty the the that function, -- on hope critical when at long-term patients endpoint a are that having think is is it, And effect how terms those in STRIDE that a kids Translarna

combination well that is sets STRIDE think registry we data the the totality as help the data of trial think and I what the have So is of the Did we critical. as that you?

Alex Sklar

Yes. that down Is reading? the not market you're if bit, you little hit a to that have just Study to access if think a still XXX pin does But saying stat what sig. that you I'm you potential

Stuart Peltz

walk at all measurements real And that this it the that couple with you to favors NSAA this six-minute test, we showing the certainly that effect, you have results market. look test, will always turn placebo. we if time totality, STRIDE Translarna on the the anticipate we data, function that world then -- and we until did, Yes, plan everything the the at over falls, the over perfection, you that to we say anticipate this that the remain that if Study look still data imperfection, the even data it's but but there's exactly we XXX look see not hard you data for hoping into it's -- about when think we're when this think cards if this --

Alex Sklar

Thank right, All great. you.

Operator

Our UBS. next Colin with question comes from Bristow

Colin Bristow

all progress. Good and Hi. congrats afternoon the on

Just PKU. one and on XX

in open I to equal recently the to criteria So fee or label than levels being versus or previously reductions greater XX% XX%. with allow was greater patients extension or updated relaxed guess

you this reduction So a first Is a and base expected? I indicative lower of just magnitude wanted to you than Thanks. touch explain tool of on can fee this rationale? using the

Stuart Peltz

Matt, to take do that? want you

Matthew Klein

And nothing or with to because Sure. And really observing safety at safety at data build dossier. in just of allowing the trial, of to It to magnitude we're the effect. in allow first has in part us not to actually look in the extension trial. yet Colin, long-term purpose the who anything seeing is long-term the was be do it's were the patients collect data also, obviously, any to That administrative. up we going trial. looking to weren't anything the change

to in to meet intended possible just for as want it's if to as we long-term collecting safety criteria data allow we're patients enter be number that didn't of them that the period, put bolster if could into randomized the actually on. even patients they we think many to follow-up maybe I

Colin Bristow

you. Okay, great. Thank

Operator

at Stuart call not I'm any Peltz time. And any to turn showing questions remarks. I'd the like to this closing for further

Stuart Peltz

quarter Okay. proud you financial strong on fronts. this Well, thank and of to today. joining execute as our deliver we continue performance all for I'm us

CHMP the bringing about we'll look progress We're and As do already anticipated AADC key today, a we've well forward to registration-directed opinion so to milestones for Thanks gene and discussed of We're continue positioned the as trials year on. providing throughout of as on number in. excited upcoming we've tuning progressed the patients, potential results a as from our continue year. changing of therapy for year. our to life goes I to the this mission therapies all updates number well

Operator

and Ladies gentlemen, conclude presentation. does today's this

a You may now disconnect and wonderful day. have

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