PTC Therapeutics (PTCT) 21 Feb 232022 Q4 Earnings call transcript

Company Profile

Participants

Kylie O'Keefe	Chief Commercial Officer
Stuart Peltz	Chief Executive Officer
Matthew Klein	Chief Operating Officer
Eric Pauwels	Chief Business Officer
Emily Hill	Chief Financial Officer
Kristen Kluska	Cantor Fitzgerald
Brian Abrahams	RBC
Joseph Thome	Cowen
David Lebowitz	Citi
Kelly Shi	Jefferies
Tazeen Ahmad	Bank of America
Gena Wang	Barclays
Mike Riad	Morgan Stanley
Paul Choi	Goldman Sachs
Danielle Brill	Raymond James

Call transcript

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Operator

Good day, and welcome to the PTC Therapeutics Fourth Quarter 2022 Corporate Update and Financial Results Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. Instructions will be given at that time.

As a reminder, this call is being recorded.

to Officer. call like O'Keefe, to now Commercial Chief would turn over the Kylie I

begin. may You

Kylie O'Keefe

Good afternoon, update you Therapeutics' and thank fourth discuss today quarter PTC to XXXX corporate and results. for financial joining us

today Operating Matthew Pauwels; Chief and joined Peltz; Officer, Financial Chief Chief Officer, by Officer, Klein; Executive I'm Stuart Chief Officer, our our Business our Eric our Emily Hill.

to in our forward-looking statements which forward-looking expectations. with a moment current Investor call, based Please on posted website our include review on will contains slide call statements. conjunction Today's the Relations the take our

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

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

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

call let to that, the Stu? over pass me Peltz. our CEO, With Stuart

Stuart Peltz

Thanks, quarter our I'm full PTC's afternoon, share proud results the fourth XXXX. Kylie. joining strong call. to and thank you and performance continued expectations Good for year in and for

PTC treats expression. rare protein diseases by modulating gene and

and Our products strategy pipeline to to new discover, therapy. commercialize of is develop build potential a robust

one to therapy two years. every deliver is new Our goal to three

disease winner strategy and named groundbreaking to for I'm undertaken celebrates and the of honored advance who Innovation. and to development recognizes is the the EURORDIS to of award companies Black science and testament report been has PTC. Company mission rare This PTC XXXX a Pearl Award that research have

date. including of exciting And a year XXth to of what in very anniversary. planning we've are the achieved celebration XXXX, we're proud our We

marketed royalty collaboration plus portfolio a we commercial five has revenue. for product and which Our sixth products, collect

it revenue XXXX, growth XXXX. total XXXX, million is revenue a XX% we of XX% which This a total in we calculated achievement representing substantial the at In in And $XXX FX growth. $XXX $XXX million achieved revenue, headwind. was exchange remarkable million, over when In was rate, achieved revenue. quarter year-over-year fourth despite in total constant

growth to in XXXX. expect accelerate our continue to We

January, $XXX our to to which XXXX would $X in guidance more announced year-over-year total revenue we be As growth. expect million in represent than XX% between billion,

pipeline We are therapies that deep continuing substantial expect the years. and to advance to in we of to our commercial broad the growth portfolio provide new coming

from with seven have these unmet medical that registration-directed three to need, have which proud are half four important in of focused readouts high efforts development which I'm of rare XXXX, studies. programs treating promising first we'll disease led on the

Huntington's mitochondrial from in We very data Part excited for about the ataxia results for X shortly. APHENITY the look Part PKU, associated X PTCXXX to expect will seizures more in shortly. go into Friedreich vatiquinone Matt disease for the this in with and for from sepiapterin for forward XX-week APHENITY results study sepiapterin study and both detail of disease. data We're

in unesbulin progress these continue patients. trial SunriseLMS ALS utreloxastat for for our in for studies, In addition to patients leiomyosarcoma our to trial we and enrollment CardinALS

With we to to continue solid portfolio and growth across many strong momentum advancing for new many our in our generate come. therapies commercial pipeline, years

for Matt? Matt hand an I'll our now over to programs. on development update

Matthew Klein

Thanks, Stu.

in important Our therapies pipeline need. to new from advancing in progress our teams patients make continue to

As of our of from will three have Stu of trials, studies. ongoing four XXXX, results in we the are clinical registration-directed half noted, which first

Phase of global an on APHENITY, me update in trial with X Let PKU. patients sepiapterin begin our with

primary six sepiapterin endpoints level. with As randomized blood the weeks or placebo in a is placebo-controlled reduction reminder, are a which APHENITY in being double-blind phenylalanine receive subjects for study to

weeks. reduction. two the XX% subjects all which analysis for randomized, the a more population reduction a sepiapterin is be randomized baseline screen sepiapterin who with run-in during there for or will greater of phase who levels from primary in phenylalanine those than consisting XX% enrich Only population subjects demonstrate responders, have those To receive of

entering period phase washout of will subjects XX a days. placebo-controlled least the all at undergo Importantly,

we In from Part data for levels. subjects. initial preliminary reduction phase provide XX% greater subjects run-in the with for of shared on in Part January, the X X data phenylalanine will now I than all update cohort an

XX% these in subjects screening at recorded from This When phenylalanine levels phase. run-in in subjects, the responder in looking the reduction had very responder Of Overall, greater sepiapterin phase. times of completed over passed or than reduction the responder high recorded XXX a XX%, the Part baseline. the X is which than XXX responder reduction XX% XXX was a more rate, three and that Phe magnitude placebo-controlled XX% Kuvan mean of rate the twice Kuvan study. is there in group,

phase. results of patients. during reductions persistent the reduction including non-classical potentially which responder Also, meet five XX% unmet classical includes who and reduction XX.X%, support there in in the high want I the PKU responder point X that to that PKU is XX% who phenylalanine had add Phe I'll impressive very run-in XX% These medical PKU are mean continue patients premise had the phenylalanine a can to the group out result. of subjects XX Part sepiapterin a and the that mean need additional levels classical classical to rate patients, a

for high Part initial of approximately XX the the will be subjects an the by beyond XX% population analysis over over-enrolled subjects. X, in reduction of target XX%, proportion Part With primary X achieving phenylalanine

last results of study, portion the March Given required the undergo additional and last X Part we washout of for subjects occur to the planned expect time in these placebo-controlled to patient is in May. visit complete and

to and endpoints. in as of treatment both from key have two registration-directed MOVE-FA. several trials vatiquinone, previous well The studies and rationale are data move we MIT-E now studies MIT-E MOVE-FA me across as the the of recorded Let scientific which on strong benefit based disease evidence

a trial with in now phase endpoint in March, the and lock. and from seizures a phase placebo-controlled of motor database for vatiquinone quarter The with registration-directed results patients global XX-week with study placebo-controlled associated a primary in disease cleaning to is observable we of will second data study completed in MIT-E The be allow mitochondrial The includes expect baseline. change seizures.

trial of last Friedreich patients the includes and the the scale rating patient in study March, is baseline. ataxia. The registration-directed a MOVE-FA mFARS phase in The second change is MOVE-FA vatiquinone XX-week expect with the Last study be in quarter. the from of endpoint in global phase placebo-controlled will continue placebo-controlled for a also visit results study and we primary to validated the

for at trial. our X sites our Huntington's Moving PIVOT-HD program. the disease PTCXXX Phase is to Enrollment ongoing global

well PIVOT-HD of in in biodistribution. is nine parts. on a duration trial placebo-controlled disease. is pharmacodynamic reminder, duration Part a XX-month and focuses in As months X and X radiographic effects, on as and two pharmacology Part biomarkers blood-based, focuses and XX as PTCXXX CSF-based weeks

two molecule. The to titratability milligrams, leveraging level X and milligrams to the study XX XX a dose includes include of milligrams dose third initially with levels, the the ability of up

at cohorts we have additional we and shared the in XX milligram January, X conference J.P. in patients. dosing Stage Morgan As initiated early milligram X

the second the from expect results quarter portion We of of trial the XXXX. in XX-week

NDA which mechanistic meeting the to during to Type We meeting Translarna. we that totality as data the review of to and the an in prepare dystrophin data resubmission. support an to could additional plan of discussion, plan Turning for informal discussed Translarna. Based including to-date the other near future. as FDA a this well analyses benefit request meeting recently C collected We now we potential the on had request with meeting to path another the

between Lastly, the for Upstaza, FDA clinical as additional in we studies product previously comparability drug the using data bioanalytical commercial and product. the of shared, the analyses drug requested support

We review continue of in BLA to have have the expect to which half XXXX. we completed submission, provided the the results FDA these analyses for first of a and ahead to occur

portfolio. I our Eric? an the to over will on now hand Eric update to provide call commercial

Eric Pauwels

Thanks, Matt.

of It to pipeline patients. to late-stage clinical great these team is treatments leverage bring important is to see exciting geographic our progress broad and global commercial footprint the certainly the expertise ready to and our

the team what momentum Our has year PTC. year global has yet the significant remarkable a out closing been continuing and for quarter, customer-facing strong very built another throughout delivered

launch commercial see to and The Our growth. is Europe strengthen, global DMD Upstaza global significant we momentum. delivering of building in our expansion franchise continues

fifth delivered million $XXX of represents in our growth year-over-year XX% the our to fourth impressive we product the an and commercial $XXX for added XXXX, products. which quarter marketed portfolio, revenue With for million

me Upstaza. with Let start

France approval program. through commercial We the pleased we the in access are with Germany the launch Upstaza in of following year, early the Last patients and patients in initial EU. treated

treatment options. patients Importantly, we We Germany, the XXXX and which by bodies initial anticipate countries, health in in further and the technology European are pleased pricing future. in in treating interactions cross-border reimbursement will enabling France also other support growth with continued with

additional Lastly, we named Pacific. further outside Europe to of registration continue and Asia the the on and America, patient in growth, Latin programs focus submissions Middle will geographies East with

XX% DMD franchise headwinds in DMD $XXX Our over when our impressive which This and XXXX. remarkable robust fourth franchise growth. brings million, deliver XXXX to $XXX year-over-year is and, results quarter, is achievement million strong in $XXX XX% revenue constant to growth million in continues it with an despite FX the exchange calculated rate,

broad be to the access drove continued growth is continued and This remarkable Emflaza brings compliance XXXX. excellence XX% for focus of $XXX high business XXXX annual which Operational the Emflaza Revenue $XX to new million, fundamentals treatment quarter was patient and revenue solid. of Emflaza total discontinuations. in million. starts, continued The on a fourth lower over

which Translarna. markets foreign eighth We exchange growth million, main of despite in commercialization. which that continue revenue revenue Again, million is to outstanding see our achieved is headwinds, quarter, in brings we an its $XX this previous XX% Translarna $XXX Turning to significant growth in over year. year the XXXX now to our

our Pacific. new America, East, with forward expansion push and Northern in into to Latin Africa Middle Asia continue we Additionally, geographic markets

on turning The had results. Latin the have delivering Waylivra Now foundation Tegsedi these and is focus building therapies to in we America. of

in recent in have in as rare in for America the Waylivra second and with first pioneers FPL. lipodystrophy, or the our our This Latin partial is for approval We first the approval Waylivra indication position strengthened treatment globally Brazil the in indication. for FPL for the Brazil FPL familial for diseases

for As was Brazil. chylomicronemia previously approved syndrome a pricing X and in familial reminder, Waylivra Category innovative drug received

And the to our received year. Ministry we Brazilian from Health, the For expect half second our of order Tegsedi, CONITEC purchase have group in progress. of discussions we continues to with this fulfill first which

Across the grow through geographical base we to and our Waylivra, for programs. our also access MAA filings expansion region, continue both Tegsedi we early and and with support with patient continue

continue a XXXX FPL continue a ahead year was customer-facing as efforts pipeline Brazil been to transformational trajectory fantastic franchise our global a large team launch and This on we of has in us solid DMD for near-term grow and for readouts. has our set our year for XXXX. Upstaza, our

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

Emily Hill

Thank you, Eric.

a take minutes will fourth release year refer to afternoon results. additional for financial and review our earnings I full Please this few the detail. to press quarter issued

Beginning across revenue quarter of with consisted and This top-line product million. commercial Evrysdi $XX portfolio fourth were of results. the for Total revenues net royalty $XXX $XXX revenue million million. the of

most were in revenues headwinds. foreign product of $XX net reflecting product geographies, in million, Translarna had growth growth despite strong net revenues significant million, year-over-year. $XX representing the quarter Emflaza XX% exchange

excluding R&D were Non-GAAP $XXX non-cash million programs fourth XXXX, in in XXXX, clinical excluding million quarter fourth year-over-year expense in $XX million compensation R&D compensation stock-based the advancement expense. increase in million and of pipeline. reflects expenses investment for $XX to the non-cash for research $XXX stock-based additional the quarter compared of expenses This of

of $XX were in excluding for expenses compared quarter quarter $XX $XX XXXX, million stock-based in XXXX, to Non-GAAP million $XX non-cash the stock-based for expense non-cash of excluding compensation the fourth expense. compensation fourth SG&A million million

which year-over-year of full and XX%. the grew for was impacted Evrysdi This growth. year year-over-year revenue revenue year-over-year. growth which growth XX% year calculated the or year-over-year at by CER XXX% headwinds, This total were growth XX% included $XXX $XXX $XXX of million, million million $XXX XXXX at DMD significant Our to was was for FX CER. XX% for royalties represented million

XXXX, million of approximately XX, compared XXXX. $XXX Cash, XX, cash securities and totaled of $XXX million December as equivalents as to December marketable

operator the Q&A. I will Operator? to for turn the now call over

Operator

question from with Instructions] comes Morgan. [Operator Eric first Our J.P. Joseph

is open. line Your

Unidentified Analyst

on is Thanks the This Eric. Hannah for taking afternoon. questions. good Hi, for

a Just from few us.

revenue and So, down wondering break follow-up about And guidance your the XXXX year franchise? DMD first, balance a just Just after between components revenue can you're of outside Evrysdi some thinking the in Upstaza? I how of you have that. your full

Stuart Peltz

Yes. question. Emily, to that? do want you Thanks, the for Hannah, take

Emily Hill

Thanks. reflects question. XXXX sure, guidance Yes, to I'm million forward of to the DMD take our $XXX happy to We're continued expected billion. $X in That looking growth the for franchise. obviously

expected royalty $XXX then, Tegsedi Waylivra. and Upstaza, and million to alluded crossing for both Evrysdi, milestone growth milestone. contribution And you As for There's Evrysdi. also a a threshold in continued of sales

of contributions you into and specifically care XX We standard going out now the growth haven't broken quickly Roche that in countries, can Upstaza Evrysdi of see expect but approved over has Evrysdi, obviously, becoming in next and year. SMA, significant we the

Unidentified Analyst

updated Okay. And just moving That's providing on the data then, for from helpful. APHENITY. run-in Thanks portion. to

if And any X or able seeing to expect based then, variance to lowering as blood reduction Phe of study? that Just you're of wondering place phenylalanine mechanisms the the you're magnitude run-in stratification on in criteria that we of in should Phase speak patient randomization? part

Stuart Peltz

Yes.

relatively this measurement. a we And the thing nice that the the of we of one things a biochemical point. stable good so measure is always mean, and marker, I say I think that's about amount that it's is phenylalanine, endpoint the

stratification measurement? about to Matt, you bit the little the and talk want a

Matthew Klein

Yes, absolutely. for question. the Hannah, thanks

really about Part we're the updated obviously, data. just X first excited So,

I seeing rate both and X. the give Phe a higher, from of XX% the we up in going response non-classical of patients lot think, into overall Part from PKU XX% what and are continues us reduction to January, is confidence to we also which magnitude classical reported for

and question your exclusion about really genetic includes any specific believe non-classical enrollment I your that specific on was classical. do of terms any or stratification of genetic full the variance, the question, didn't spectrum We In subtypes. based genetic variance,

as for In that randomization for and stratification as terms XXX so X, for of liter, did the the per stratify micromolar one the strata, below well was above Part we stratifying the to in phase the than the XX% study. and the being analysis with, greater greater to response phase run-in XX% run-in population course, response population of primary XX% for XX% than

Unidentified Analyst

And in the portion? should are further XX% analysis than moving the run-in we that population, there's seen greater the based was within that patients on no to on assume stratification primary what

Matthew Klein

XXX XXX the Correct. as than enter baseline, at greater as placebo-controlled below at We phase. predefined or the baseline they strata

Unidentified Analyst

more maybe And Great. one just could. I if

over-enrolling initial Just relative to wondering borne the about agencies. know need feedback the your any study interest was that or from if Wanted in design. to of the out regulatory study

Stuart Peltz

ahead, go -- were we Matt. No, Yes.

Matthew Klein

This on Yes. to FDA the with do agreed patients upon was -- the of the and That of EMA physicians. prior the study. agreed really interaction. start had nothing upon and the the reflected to interest That part protocols with regulatory significant

into had interest what safe the as the think I effective fact we that of [indiscernible] world. they in to trial. really there's still we've and a and in delays large this number unmet to desire from number about of or the clear therapies of comes And was investigators, talked that really key patients, get strengthens and the data KOLs getting as saw that always for the medical patients believe success course, heightens just need and study. significant from over-enrollment centers throughout greater of And probability who the we wanted the really

Unidentified Analyst

Thanks helpful. Very question. the Okay. for taking

Operator

Thank you.

from comes Cantor Our next question Kristen Kluska Fitzgerald. with

Your open. line is

Kristen Kluska

questions, my for and Thanks congrats taking XX-year everyone. the Hi, milestone. on

thoughts a preliminary across how your edit you step such with those be? or might there sense response be Whether what First generic can question perhaps or us Kuvan for differ PKU, had on it populations payer's could was, might I as give different classical? the

Stuart Peltz

Yes.

overall this a -- So, population good still a point. it's that exactly remember Kuvan it I the Kuvan maybe this what was population. first need think at of And we drug, while if you I think is patient XX% approximately see probably to you it together, look was to. Palynziq that that large not but is had and And ineffective close, the yes,

the So, globally. you patients not huge population that XX,XXX there's current a satisfied have are right? patients treatment, patient with So, of PKU

significant that -- be is is believe -- able we bring to much So a the these pretty is it therapy bring to to highly that new -- that opportunity effective. makes really

didn't see XX% of was, just consider that XX.X% effective You same, When (ph) how before] -- is. what population, the function it even [was the PKU of patient. you could patient -- they're classical you over classical on getting XX% overall population what showing Kuvan the and nearly it

pretty product. is So, that it's clear a this superior we think

in And good ineffective really -- for it's think were there's that a this. we're where spot many patients so, we drug think so I the

already And And has so, number there a Kuvan isn't walk so if [that already even edits is failed a fair those step been very to there's that of show through and documented. patients is known tried it And any to short we PKU of hit for don't or even who've period Kuvan it patients edit if for because it's haven't, classical well (ph). well -- they that it's step expect time was Kuvan, it] functional.

I you cumbersome -- that be real be handle. able think or is a to to I difficult So, going don't this think have to

have you fair on a done bit of do Kylie, say? So, you've Also, payers. to anything work

Kylie O'Keefe

Thanks, for the Kristen, Yes. question.

I And Stu that begin substantial marketplace. do different Stu talking engagements one. to we important data to, as sort to think this payer no you the there said, classical And looking from therapies exists it's been in Kristen, started we a number a the need was space. saying, in a as as package. really unmet Stu believe that understand how at PKU in of And really they're was effective patients, of if that look we at perspective, has PKU ensure

perspective, we to able so, treatment. on or put to And these don't that edits requirements patients any of be step foresee types from

it that been documentation they've to the don't it Kuvan failed And quick generic of many in re-established, see needed the is biomarker. a a previously and it was And step even a hurdle, cases, as efficient in the they've and first to Kuvan if tried is be we tried because step in sense space as established. blood-based and therefore major has were also this Stu if generic, case in saying plans they Kuvan, being the where even again, edits

working, on effective the them treatment, move on the So, a you're isn't patients able to treatment to and therapy. that establish put more

And package the that the we data success we're so, APHENITY that the into and have very assuming perspective, with right ground confident set landscape, to from going running. the commercial right data hit

Stuart Peltz

don't look a window at had with weren't -- drug like we small as which competing of the competing of And successful. We're patients treat with that this are a very patients generic. we're the of most to

therapy it's more with bringing we sense But therapy. a a And -- be plan on so, patients we will new anticipate these fighting have than successful this on different in will very that us.

trying piece able you're treat be we'll to before. generic a that actually be we a -- weren't treated have We're to believe just and fight that that able actually get to pie. You patients of to

Kristen Kluska

Stu Okay. your and PKU you, perhaps gets candidates less Thank and Huntington's especially to then, Kylie. late-stage think of disease. And I like attention MDAS compared

reiterate commercial can about you ease these ultimately patients opportunity should of And finding the the successful you Thank for of you excited us you. it be think maybe So, remain some about potential here? why program how approved? and and

Stuart Peltz

functional, therapy, we the in tackle really think is of really Yes, in the a that that. things you it consequences be consequence is to think like the and and new because of that all obviously, terms molecules ATP mitochondrial And on treating havoc, of the of obviously and about sorts big. think order when a part, new that's both of area amount When in cell [indiscernible] important dysfunction sort about things vatiquinone medicine, dysfunction, does produced very is you second. cause dysregulation a neuroinflammation mitochondrial if that ATP that. and enhanced free electrons go XX and can stress that [indiscernible] almost that causes like -- of it's And cause

XX% body, and X-pound effects, especially only mitochondrial ATP but the with be could uses the the it dramatic a that of could associated overall about see within you disease about seizure. with where organ, think it in you brain, it's When like both

that a modulate having quite So, about through And it and new think is important. used mechanism novel quite and drug this broadly. we're really be that because excited I could can

MIT-E Matt, this? and do excited want little about about you to so talk bit we're a why

Matthew Klein

over fundamental that as well, it's this inflammation benefit pathways There's is XX,XXX attention, seizures. associated the worldwide the you just drug access much beginning this Yes, patients not getting maybe disease. Look, mitochondrial a that's again Kristen, could And thank disease and with populations from population as a for known. that pathways, that stress that Stu of targets mentioned, these is causes question.

ataxia, other the therapy this of patients morbidity. a terms in showing on on seizure of seizure others. It's we previous very but mitochondrial not an disease have we we built effect number that activity, Now seizure-related be that have believe in a patients, frequency confidence can And important only both many Friedreich can important for study. studies and with

Kristen Kluska

you. Thank

Operator

Thank from question you. And comes our Brian with RBC. Abrahams next

Your line open. is

Brian Abrahams

Thanks Stu, as guys. years. on question. well for congratulations Hey, my taking And my XX

Stuart Peltz

you. Thank

Brian Abrahams

registry. bit the were of if guys could you STRIDE the confidence discussion is curious was would analyses their be perhaps? to would I'm guess be a I potential that seems expand a more of bit I I accelerated gives full guess Translarna. this agency your And plus a what other what the wondering the learn Thanks. little you to some to on point? What that the initial on dystrophin little And about sticking data with something the nature and reactions approval? support more approval XXX

Stuart Peltz

Yes.

recently the we why action formal talking a discussion as them. it a that. us of really as setting mechanism as to of the And will with we package consequence that to meeting benefit overall have clinical the up think was us lead notion and a about well of allow So, had having we

want go to a little through Matt, bit? you this

Matthew Klein

sure. thanks Yes, the for Brian, question.

would XXX asked recall, had to to the data, that said as XX. given with have well as the meeting they be a Study the a we response-only Study and response analyses meeting So, a only. a agency X would as I backwards, C informed FDA clearly, And itself them after the seemed have Study on had X questions in benefit disease, of effectiveness. statistically then which of afterwards Study you and take focus from and but the discuss step had consistent and evidence of key the STRIDE also we we mentioned the live the volume and improvement highly including endpoints the just And questions. provide of that registry. you comments, Type most there's us combined initial have to with after XXX, meeting significant written the we when still to have data as obviously, able ability to mostly But if on not written Study had we up evidence we that you see to talk We substantial bar functional and requested And meeting they STRIDE feedback, did pooled XX, think with of to study. XXX would

the This clarification live a of as effectiveness. made meeting data, meeting suggested We functional a it know, had comments was C you which that believed C to data, to that totality we of all discuss had and Type as a was data, the afterwards. actually And was was we data, totality in separate comments. those because Type mechanistic offered the why talked the XXX dystrophin that a shared in that we of about comment request substantial were have including on made lot events was the of XXX benefit about written those could study but again, meeting data, wasn't of came discussion that our meeting focused that in

the loss demonstrated ambulation, disease, of in again and bringing clinical this the all we've data. meaningful see an mechanistic benefit phase but the benefits registry, opportunity only a of pulmonary the of as how highlight transitions far as loss studies, other long-term key trial are the then we into not placebo-controlled XXX, of as function the and XXX the course to translating STRIDE So, alongside delaying morbid

associated confirms benefits recording production, in clinical Study nonsense the have suppression XXX [indiscernible] of all have novel as well, also which yielding terms that which of and we We of that's a really the action dystrophin studies. in is with these mechanism

Brian Abrahams

Thanks follow-up really PKU much. the helpful. I in on a squeeze if program. And so That's just could quick

Kuvan in confidence are you to Phe relative X I being repeated happened just clarify the drop Phase levels your the think that assessment measure But maybe run-in the measured enough randomized and studies old between has way the of whose the some that to reliable. label could was Phe and pseudo of wondering the is way the more such studies? have I modern is in had you or Kuvan studies robust phenylalanine might the as wouldn't reductions responders off portion open Thanks. with if

Stuart Peltz

Go ahead, Matt.

Matthew Klein

Brian, we're that's validated done measure. home. well This at blood Yes. using a a has analysis spot as very robust been

run-in obviously for phase. I and we're it's we're three responses, the in days, points note also, way in using both of placebo-controlled time confidence days, methodology averaging same gives and important five, think the run-in measure phase the which that the different treated these we reduction. are but And have value XX to Phe the us which phase XX we XX the

of ensure it's X over accurate points, an reduction. which to Phe is averaged So, another assessment getting precise effort and the we're

So, that's this is a and method been well designed well validated.

specific I both was can differences run-in Kuvan, that assessment provide and over I time. phase. on methodology that method a We used in but can't have accurate the precise validating that confidence a we both changes can provide Phe the the GLP laboratory well in very developed comment the placebo-controlled consistent the and of measurement have with

Brian Abrahams

helpful. Thanks, that's No, Matt. super

Operator

you. Thank

Joseph from with question Cowen. next Thome comes Our

Your open. line is

Joseph Thome

going hopefully the is in there these some bring you are first you there. see just anything which are question. But in, would be And size That taking data up would of half, as the that the sort sets comment essentially, now? on your right CMC are here to else We're some of the successful. Maybe Thank data my Thank just you. of registrational or them safety the did you with for as one. all a Hi, of great. is regulatory the you stands registrational outside need great, submission? coming it comfort for can next of database here? So, before three that these

Stuart Peltz

you do take Matt, Yes. to want that?

Matthew Klein

Thanks Yes, Joe. for the sure. question,

out. really a both for think portions vatiquinone, for well opportunity [indiscernible] the has which So, other package as PKU of the filled have for those given compounds, obviously, package development think the and about sepiapterin all about of components -- to you number been non-clinical of in CMC for you get as the years, when

conducting continuously collected positive support data trial. fairly sepiapterin strong records in this and sufficient sepiapterin large that really a that over extensive I one mission who And therapy been database on we're based recorded that to been in studies over the is with vatiquinone, have the patients the with with now the that we this kids have and on been to Obviously, believe that's have with -- a very safety we'll trial of other foundations the decade past drug PKU decade, other study of profile. safety a including think for in

therapies, believe components we on the then both of and to for the the move as we data, the align together package. Obviously, as with possible. we'll of details positive have Taken submission agency forward meet [indiscernible] with the all quickly

Joseph Thome

Great. maybe a on financial the side. follow-up And maybe quick just then

of this for terms XXXX, marketing build more financial the spend spend item? force additional is anticipated of a any be In XXXX US would year? in there the guidance Thank you. Or that

Stuart Peltz

Emily? Yes.

Emily Hill

the Yes, side sure, well and force. then one I'll on on anything the take spend and sized the that We're commercial, infrastructure. pretty on commercial

US the change significant in spend on no sales force. there's the So,

Joseph Thome

very Okay. much. Thank you

Operator

Thank you.

question next comes from Our Citi. with David Lebowitz

open. is your with line Lebowitz David Citi, line Your is open.

David Lebowitz

which given the response on one, should out moment. Phe rate fell number name be XXX a patients the compared the I Kuvan -- that diet, we were guess expected deepen a I for on to restricted question data, benchmark response? how Sorry, to overall would

Stuart Peltz

Yes.

of think keep both of dramatic overall, within on. on, as what what pretty are that what greater for results the asking for I other well I I we're is people, results And of the in they -- diet seeing as PKU. whatever what think classical the consistent So, you're XX%, they're forms diet's than think

consistent activity the So, I you've of seen molecule. think

way, here, in high I other at least So, still seeing pretty -- you're deeper it's the I if in feel response we're rate. think and

do you have... Matt,

Matthew Klein

you for key of Speakers] patients is to question. the a all In diets, not in impressive. be to these obviously, think But general, restricted Thank on of consistent so the effect diet Phe what them that's differences confounding magnitude seeing I terms on both we're [Multiple tend quite and variable. studies, have in in

we're of looking responder If at And than patients. at Kuvan which XX% the study, change they greater now this Phase in recorded the you study. in look XX% was XX% obviously, the a X,

is again, I potent obviously, rate. So, response a much you is and think the a a said, you'd for expect what and more of we've about phases bioavailable continuous what And this diet. in clearly really cofactor, talked higher

David Lebowitz

a -- to you you PKU you specifically patients? do for that. would And report rate response Thank classical for expect be

Stuart Peltz

end overall, I Yes, at the we'll be as out -- break we'll mean, well. day, of the that

David Lebowitz

Thanks for question. my Helpful. taking

Operator

Thank you.

comes with Shi Kelly Jefferies. from question next Our

Your line is open.

Kelly Shi

you. Thank

for of on help what So, be expectation now you of an could meaningful? stage would QX, kind to frequency data of clinically considered in reduction set MIT-E

focus on also benefit vatiquinone? And merits other the evaluate we to of what clinical should

benefit And on you. then disease, see mitochondrial Thank greater of groups a lastly, a like mechanism based vatiquinone? that certain heterogeneous the you is disease of very genotypes to the with would given action some expect patients of

Stuart Peltz

take do want that? Matt, to you Hey,

Matthew Klein

you Thank for Sure. question. again the

we talk patients, to when So, itself. about about these first, disease benefit we have in the think

disease mitochondrial obviously, morbid seizures of common So, and highly disease. are associated aspect the

respond energetic to to antiepileptics cause these and the patients these typical So, have don't because that tend with seizures the about seizures typical patients. in antiepileptics, half pathways don't target mitochondria the seizures

are worse. up hundred, disease they they toxins, many to actually the kids so make have thousand a of These meds, traditional to fact, are that the can In mitochondrial ten, even month. have refractory antiepileptics seizures typical

So, to pneumonia other death be high of for can which of often cause patients. the a morbid disease highly aspect It's volume. frequently seizure and and aspiration, these leads infections,

these a the placebo be seizure We've placebo think XX% XX% with in even active of patients between differential we and population in XX% study in population. XX% can reduction about in the XX% group the frequency reduction and we most the reduction, speak treatment for reduction hypothesized a a when group important. of the to think powered So, frequency in physicians that to meaningful seizure

or In the genotype, different That's response. of of be therapeutic previous specific disease targeting stage because effect method between and between mitochondria, to genotype differences common one things or such we've there's response given and is that to response, that common should But we're causative again, element outside relationship pathway defect. all studies might there the the response XX-lipoxygenase, disease agnostic the genotype treatment targeting to of in and observed response, possible. and is contribute fact differential genotype patient terms common subtype could no vatiquinone be of is a other age characteristics as underlying which

Kelly Shi

helpful. Super you. Thank

Operator

Thank you.

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

is Your line open.

Tazeen Ahmad

Translarna. point Hi. Good for evening. of Maybe questions. Thanks for clarification taking my one

being talked agency. looks and lot time premise I to was agency of with of you patients' And Stu of a think in that? have can just the it measured remind your your many how dystrophin to you've as as Matt, about But dystrophin trials the the for worth specifically any totality on might I package, if data recently us relates for date? of discussions data the guess the you the as of part last dystrophin, when

Stuart Peltz

sure. Yes,

small when which to showed a paper, obviously, we the first got did, it, -- in it where was we Study I did measured was and you that remember? where study, Study way saw was was study -- how XX, we Do increased you proof-of-concept -- measured don't Matt? then, we somewhere study also studies to take XXX, see was redid And remember the -- So, XX the we look of to that showed know, the the there. We dystrophin XXX. many that's you in it published That a its XXX. way again around And levels a that, I patients it we patients dystrophin. think,

Matthew Klein

and were overall, population. XX XX had we So, analysis there in

Stuart Peltz

Yes.

the number especially a a significant saw have, have and those large was Star, of of from endpoints XXX true but the said clinically positive timed-function with ITT was patients we only in that positive where as So, a then, before, population, had multiple patients. test. clinical And there, studies XXX, was in in XXX And Study that sets point we it only only obviously, then, in Study -- that XXX view, from it not results. -- not XXX, we of as not as And was the well was it also also North Matt XXX. in

ground patients. done of of the loss data then there's get -- ambulation, registry STRIDE that consistent that a off with loss in such getting we've hard activity that's endpoints of Translarna's And able demonstrates terms treating body So, being as -- large to where then get all the stand and by of off walk, preserve preserve to pulmonary ability you the of activity. And demonstrating pulmonary the from function. to that results the -- ability those preserve year, off to from both ability the preserve the we've and to of at looked ground and loss

seen we patients that all now that it's frankly, pretty benefit So, And I see clear not been package And have can have. you put from than that for long on a it. together you we a clinical have patients believe quite the one better -- it time.

And towards we'd have getting believe -- for -- United and we really we we asking the from in it's we're that like what And getting fair always package. patients this -- think the Translarna. So, strong to around drug. should work and States have obviously we we from think a patients is, we that appropriate globe, look,

it. review Let's

have we over and least what a to patients Let's an them folks active advisory on just that many us a not is been it the of so in hearing, been due have and fact, them a now, give their think -- families drug. and that committee, highly who fair give -- and at us the their have decade for on

Tazeen Ahmad

you all this over already looked happen approval? finish interaction? needs the as this that Thanks think color. mean, do what Have written at line to of at get they them agency that data I say to part for Okay. should your all for you the for point, to apply

Stuart Peltz

Yes.

years have whole the data, look, a -- don't them. it's that, like not to we go able It's and large you to to else understand and And understand is -- of and interaction we're discussions, the actually in talking not simply just to life, you a to to the what of everything And get that, One through us. not to with have working of new has plenty how talk or with you whole had agency at now it view not community forth minds who there you all easy been a communication an the need where not and picture and and discussions. people's have that you written results be do of write just at it. have uncomplicated doesn't think it, the least it's may points give they change we've response at had shot have only body I months, people we back and have back if So, three and every questions. shot

have conversation that to we So, and at we a see need that think least what do to where goes. is

Tazeen Ahmad

months later? be than do schedule just that will And rather sooner you that, it? Or Okay. have wait that do to three to extra you think

Stuart Peltz

of in, fit to -- them regulatory discussions, and it It you nature put takes time. This the is you get in. for time there's

Tazeen Ahmad

right. All

Thanks that. it. Got Okay. Appreciate it. for

Operator

you. Thank

next Barclays. from Our comes question Wang with Gena

Your is open. line

Gena Wang

Thank APHENITY you for taking my two regarding question. have I questions study.

just clarification. is First

reduction XXX that the patients, Phe total is XX% patients Phe patients XXX is XXX patient these reduction, Regarding it's you you is test? that what patient patients screened? screened? passed actually If was passed a number [XX%] the (ph) Or total screening a

Stuart Peltz

take Matt, you to one? want that do

Matthew Klein

Gena. Hi, sure. Yes,

to all the phase. and refers XXX the completed who So, those went run-in through screening then

who a subjects XX% XXX, Of reduction. XXX than sepiapterin. weeks received or two had greater of XXX that's So those XX%

Gena Wang

XX% many XXX, out these reduction? of how achieved Phe of So,

Matthew Klein

between XX%. XX said and So, of over had we the XXX -- XXX XX% XX% so had and

about were of -- and in or XX% be XX% So, and above. greater would XX% XXX XX% away, reduction obviously, those than total, majority But far had reduction. of XXX that the XX%, those,

Gena Wang

Okay. dose rationale Part Very Very good. the then, the question helpful. you X two study? XX-milligram remind can my second And XX-milligram of XX-milligram, is, us with weeks for each escalation of the

Matthew Klein

sure. Yes,

XX-milligram per so kilo, weeks doing purposes. nice understand sufficient for believe the the the always kilo, a is we're what weeks at response, relationships, So, per things curve response understand two per and kilo, very one confident is to and exposure that is two have level the weeks of to we will by XX-milligrams at agency two which at between per that very at kilogram, also likes be. weeks the studying, XX-milligrams obviously, We can important effect regulatory we're understand relationship exposure the dose each will be two dose XX-milligrams and

Gena Wang

Thank you very much.

Operator

Thank you.

Jeff Stanley. from comes Hung with Morgan question next Our

open. Your line is

Mike Riad

questions. Jeff for Thank you our on Hi. Hung. Mike taking for This is Riad

you're follow-up us data the APHENITY you cadence upcoming a we the events? once can that. in readout after May, one, about thinking of First regulatory And remind timing and how have

Stuart Peltz

Sure.

guys ready possible. product then a getting the documents is meeting. And a on goal for pre-NDA then, out as for the we'll go. rapidly Obviously, And and based results, obviously and NDA to an meetings all fast have as the publicly we'll we'll set the regulatory ready we'll that, result. be possible so as do we're to doing get as while get report ready Our get and that, of

Mike Riad

so data, adjustment much. talk the about they much. MIT-E but quarter. Awesome. And Thank timing? the now so my second you the vatiquinone Can for you in factors that any to led this in X/X were expected Thanks quarter, second they're question Phase

Stuart Peltz

patient think I it's Yes, finishing Matt, this? up the out. have thoughts last you on do any

Matthew Klein

lock all it's a of visit going then database Yes. when last question then be, the having which yes, data. know do really cleaning, -- getting sufficient that patient and having the Michael, now time is to database to in is last It's going to March, to exactly be and

So, refinement it's last know really patient date. the now a timeline of definitive last visit that the we

Mike Riad

you. Thank Awesome.

Operator

you. Thank

Choi from Our Goldman next Paul Sachs. comes with question

line Your is open.

Paul Choi

afternoon our Good two taking I you you. questions. and questions. have pipeline thank for Hi. Thank

differences on look patients the year? in you this that updated is from maybe line later top any you APHENITY. we versus and timing might to versus on And as of geographical the just US OUS results Can with of PKU practice for year regard treatment elaborate you maybe your prior May clinical guidance? this to First can your speak

Stuart Peltz

Sure.

just first one. take So, I'll the

be the to end decided people when were to patients lot and to we a refined on of which we some -- that what patients that it I was the would than when the we And getting of -- do wanted large of timeline we is would, close the that the that got the nearing is we of trial would sure brought so give the in make and ready able their were had think think us at to to the -- results, we everyone's we've to able timing to of a number as now, trial. actually such. talked do the pretty this thought were able we the do in trial. KOLs and just to when about time and lot trial the some of complete that be a led We really with had we now a do by with the of more there the in timing would complete extended we to refinement

pretty feel we we So, the complete we that good about we're exactly when where at now trial. know

do you want In terms to differences, make of on Matt, any the to geographical -- you that? comments want

Matthew Klein

while that the would don't different obviously, birth. Paul, there target to I in for diet reduction geographical differences The this region management starts is we of expect where really of fairly may now a Yes. And at guidelines consistent cornerstone worldwide. -- be practice region. Phe is in terms modification the is disease

many is screening in Newborn world. around the places place in

expect differential whether response it's on can there's at look we certainly one based geography, on would priority. not we that So, but

Paul Choi

hold for there? the as just feedback lifted XXX us a for maybe you what clinical on can getting current on And follow-up FDA? you provided that. is or Thank data update comment the what And to your for Huntington's, expectation or you've Okay.

Stuart Peltz

sure. Yes,

data -- of in that clinical on them talk that them past, will with go -- some to current we back that what enough them can we patients that and share on which it's so, our on. I be discussed think we doing. goal as go we've set the with and then to that, back patients data with think to is getting And having we're the

in doing that. terms what of that's so And we're

fair the globe. of sites But a around up obviously number opened we've

We patients. expanded cohort so, additional to patients coming in. of an And are take even

So, and in. to the we'll ship. of some then, we'll bring in go we're feel that have at we show about about point, we pretty And in, that pretty data them talk good a a bringing set them when FDA good

bring and That's doing the to then data to we'll at opened and game X- that US. we're able will everyone, to the it go remind XX-milligram so to data be to our plan. able data we in point be our the some FDA XX-milligram Just would the up

Paul Choi

years. Okay. on Thank and XX you, congratulations

Stuart Peltz

Paul. you, Thank

Operator

you. Thank

question Raymond James. Our next comes Brill Danielle with from

Your is open. line

Danielle Brill

much evening. Good Thanks the questions. for guys. Hi, so

Just of me. for couple a clarifying ones

J.P. Is think $XX number for million unaudited reflected million in be Upstaza from Morgan, be $XX XQ? that were XQ spillover guided I at the million. XQ in month the revenues And last you expecting previously accurate? to should to for we $XX still

And C then, as will expecting or written this well. person, request, Thank just one The that you. plan just well? you a in clarification you on meeting meeting as to be Type Translarna are

Stuart Peltz

the question. thanks Yes, for

conversation, Translarna. a means right, be believe we let not it's to going -- me And to so We so, going be be where take Matt? written. to not anticipate call, isn't it's that be will have I to Zoom -- it going it's this And

Matthew Klein

agency has -- request having interaction now a meetings We I obviously, started we'll again. in-personal request live the know and

will to exact details, and to form their the whether this whether phone, believe of once discussion, they obviously, to it's most be (ph) effective but do request get it this be would the we in-person, them is the response. want it whether is the whether to agency. [targeting] another up obviously, live, we Zoom, submit Obviously, know be but that's So, going Danielle, it's up better we we'll way, and

Stuart Peltz

we QX, And remain And we want in. we Yes. obviously, move do obviously, to the what of we those of most terms confident on terms patient in focused certainly, in finding, patients. is that then of, identifying

provide the quarterly launch, in at earnings early more QX color call, more I able I changes within numbers. numbers, on the XXXX to of color very color any how that. we'll doing. the the calls right? more So, think on the of were about we'll terms and we'll there each think We're we're In probably provide be provide certainly don't And

one? to Kylie, have to else anything do add that you

Kylie O'Keefe

Yes. Thanks, Stu.

is As well to outside focus January, on to there countries talked programs new of we at securing and continuing number to said, of said, pricing a as focusing Stu Europe. in different the Europe, audited. Danielle, on number unaudited as and Upstaza identification across patient no to we're focus And continuing about changes reimbursement from for looking as patient on in

according continuing that's so plan. And to

number HTA continue to earnings. we and reinvestment bodies, a provide step look engagement pricing at that we're patients updates of across to payers upcoming different forward strong As in being treat with and to more more and earlier, this, plan Eric able mentioned we quarter seeing to

Danielle Brill

helpful. you. That's Thank

Operator

you. Thank

Our next with question Robyn comes Karnauskas from Truist.

Your lie open. is

Unidentified Analyst

had on a patient Robyn. been what AADC it we for additional me like also identified initiatives but any approach towards the and anticipated Upstaza had on until you Upstaza, Correct first sounds to needed quarter the still going the Hi, wrong, us to if patients that results we're may We to this on if Alex is that. are wait I'm future as question submission of updates for be wanted more Thanks. finding or there BLA into now prelaunch we remind some get turn ongoing Can for initiatives US. launch? the and for

Stuart Peltz

one? Yes, you want sure. that do take Eric, to

Eric Pauwels

a have that's for we Thanks, team doing but the in Europe, sure. that. dedicated We Alex, currently Yes, launch Upstage similar and we're way, of the US question. in the dynamics preparing seeing in the actually are also for a

We have including a focused areas, on accelerated key number disease of awareness.

We have have symposium programs, English, publications also it's of key review publications, and and a that US-based including key in advisory board number peer journals. been

them number because launch. US We're many in are of that and gene talking standard currently replacing therapies We're are the of well key U.S. but a know in of the the payers care. to payers preparing to approved talking for as there design this about we

So, the standard care. Upstaza be will of

an to be really So, approach understand we're this. on view the payer will taking how

at particularly enriched ethnic of a the activities, accelerated and number population. have finding We also patient in -- looking high And type groups.

to and where with ready closely be this the be will to be compare centers to the X and be timed work importantly, time in. bring launch. launch, And at we'll timed target is of patients to We will able of have between our very time somewhere these already XX centers these surgical

US ready Europe, we're of and market, So the product we're we're the following we the the sharing overall, lot launch in learning that knowledge launch a of be BLA have continuous but the outside to pleased that in we're also progress US. sharing execution itself Europe, we'll approval, think I with very and from the the with making

Unidentified Analyst

great. in patients are trials, you're there that sounds same you setting, the that the now That still procedure, results with and what note? seeing match treated results have Okay. along note, you also differences saw find been -- that key clinical early to there And clinical this do real-world early, the of or any in that details clinical Thanks. the the on the

Stuart Peltz

their the it's deficiency and is a AADC gene -- No, Yes. we you've as better we're truly that aspect it transformational or nice talk speaks their where growth of where see of obviously, patients as have they stand -- perform therapy look, the that weren't hold clearly, therapy we changed. over, that have seen see while we and up, in a arrested all some to were patients you and head turn sit patients able up, languages. that have can patient three one And

exciting, been and these So in patients. substantial to improvement continue that's see we

patients part than there's transformational more nothing ratifying on a to -- it. such always say, as and So, of it bringing there's facts therapy we being and drug having

does the for it to them. -- the and for it that's great to community see for being own it's our of family, think family what bringing the just part I patients for

and it, to be a it continues about product. transformational excited we're So,

Unidentified Analyst

Well, question. Yes. congrats, taking my That fantastic. sounds and for thanks

Operator

Thank you.

comes question Bristow from next with UBS. Our Colin

open. is line Your

Unidentified Analyst

is [indiscernible] This taking for questions. Hi. Thanks Colin. our on for

clarification have questions. We two

just planned fixed has us like for like So, and When Part on timing it's you the the on primary give some end for Congrats top delay please more the data. one May? detailed PKU And the Part a data. like the reason of get the enrollment point. have to lot the is X, could was it a X already stuff, on you as colors line first

what behind rationale it. I'm the So, just wondering

second on Huntington's question the PTCXXX And is program. disease

-- wondering, please take have and clarify Could long already that? how on enrollment? US in the the FDA requirements heard And the enrollment, We you to specific of are terms any dose data? started on just high it will portion from like the also, you updates get yet have more

Stuart Peltz

Yes.

I think a -- if little I muffled. it was

So, -- an go reason I really the the the think completed study, when visit, is asking that's was you're that -- so the completed, last end aspect maybe why in when occurs the think just data study we're of May? the last then are can I and I Was think so it the question? the patient patients it and that going that's And clear, of bit little the it's happened to you've it clean get is that at as patients all nightmare you the of study analyzed. so And end air to as always ultimately be it and a in. control -- rapidly what bring we're of a many way, number and there traffic you really at is end possible. a of close able as patients in the get the can large of in as was

and And that the close wanting also to to trial, but wanting the -- so balancing, sure patients make makes from multiple we're got that that it and trial. physicians patients get in into

And so weren't that we anybody they don't anger that trial. of part of it the

the in patients. this particular, lots plane always -- from precisely you the end, going where the to do always May. would that's be we a difficult to to timeline And thought time so why it think was And at moved more that's it's it. one you're land had And of the little at much sometimes

little but from we've It's leaders a and lot the longer, we a nothing people a key bit -- the of gotten opinion it's have -- investigators. from patients lot of all

at it. because the we good in feel and we So, small change, well worth day, was everybody so the have the it it end of the study, about

the think end don't day, I everyone comfortable we're to of amount well that's -- worth small of -- at what the get in. with. change I and think time the So, that's

data. terms collecting in now and XXX, -- of we're that So

the when plans going we what we'll know are, done And it do think to data to and FDA. to and to we're said haven't do, haven't let We that. say our back we're go we set we've but up everyone clean when we complete this, that yet But going -- don't we plan when the to I yet.

have else anything that? Matt, I on to say

Matthew Klein

X that non-clinical other study right, just the and all to we the agency -- a Yes, the the have agencies. of had was at even committed at And is allowed things feedback additional from support reminder, was the we're said, to and reviewed a simply as came that process on I dosing add the that this XX to of just said, the will And at start data couple other a countries as the us able milligrams a duration, asked which and we would year, for think data. start data we some clinical and year reputedly to collecting what XX be milligrams up that, do for it. Stu watch FDA to as in the we

So, comfort for from dosing that US. real the and data at give patients duration can the

the question higher of XX-milligram regarding your dose, cohort, initiation of that started In the terms yet. the hasn't

said, based protein understanding we XX-milligram reductions to data at we effect, pharmacodynamic we're of XX regarding really the milligrams and of in and in decision were milligrams, fully milligrams CSF protein position look decision Huntington what Huntington data-driven a to to at the seeing to Once make like from mRNA X be X reduction of of exposure. what XX then a terms initiation make have in that terms in the is we'll on cohort. a want -- looks an that we in peripheral As biodistribution

having so, decision to and look future. to And forward near help in inform those that the milligram X XX initial data we

Unidentified Analyst

Got it. Very helpful. Thank you.

Operator

over any questions. Stuart to Peltz to Thank no further call back the you. like are turn closing I'd for remarks. There

Stuart Peltz

operator. you, Thank Okay.

where I'd your like wish from see, joining PTC, as thank at closing, the well can years. of today we Especially our and product have, where for stand for always you all you We're accelerating to In our today portfolio. and excited XX call we revenues about about excited strong that.

data three the XXXX, including have in studies. half first registrational four of readouts We

the updates forward you look we're and cylinders, coming over So, all clearly, firing months. more to we for on

with you thank for that, And on staying and evening. good

Operator

your for Thank participation. you

now of You disconnect. Everyone, your rest may enjoy the day.

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