PTC Therapeutics (PTCT) 7 Nov 242024 Q3 Earnings call transcript

Welcome to the PTC Third Quarter 2024 Financial Results Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded.

I would now like to hand the conference over to your first speaker today, Jane Hanlon, Associate Director of Investor Relations. Please go ahead.

Good afternoon, and thank you for joining us today to discuss PTC Therapeutics' Third Quarter 2024 Corporate Update and Financial Results. by Executive today Dr. joined am our Officer, Chief I Pauwels; Business Chief Matthew Chief Eric and Klein; our Gravier. Officer, our Pierre Financial Officer,

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

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

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

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

performance, million total third revenue a our competitive marketplace billion raising guidance all for $XXX and quarter joining genericized of the we happy want in revenue $X cash $XX results closed total quarter, the outstanding which the $XXX revenue, you quarter of navigate with and highlight With including $XXX In the ability today. our million, from DMD call are revenue to reflects franchise. of execution. share performance another our U.S. effectively to Emflaza $XXX commercial million. in achieved I'm third million to and the the XXXX over strong pipeline to team's the Thank to quarter I we million in

for Both the provided to have date quarter, third two Translarna FDA submitted is due to Translarna. the An action not accepted sepiapterin and clinically filing. for the been July from for open-label In regulatory has context study and from date planned XXXX. sepiapterin been the for one patients as action maintaining control ability to for durability their sepiapterin The XX, of Phase the the FDA, protein filing. the The applications III the NDAs increase one NDA data as sepiapterin efficacy and demonstrating study benefit of Phe evidence the includes of extension meaningful significant of treatment of levels. the intake AFFINITY while AFFINITY NDA well we

age were the These to to data can impactful groups severity findings. support prestigious PKU in provide of including PKU. benefit results the subtypes, recently Journal, all published patients Atlanta a significant trial study AFFINITY sepiapterin the to classical importance The testament of and from the

Japan sepiapterin this In in global submission, launch marketing of currently December the JNDA for applications schedule and in FDA countries, to will other in our on year. submit addition sepiapterin the are the including These submissions Brazil. review authorization EU remain We XXXX. to under of support several in planned

progressing plans launch Our as are will detail shortly. Eric well,

in from The X stability as approval execution. upright children application mFARS resorcitaxia extension an subscale submit We testament the the of an team's benefit This significant NDA trial, our on rating disease NDA fourth as results the based in year, findings well will be be will adults of vatiquinone scale the this MOVE-FA on long-term the for studies. track submission for December. and placebo-controlled impressive submitted of to the treatment remain on with the vatiquinone to FDA NDA to

benefit of recently their extension disease both we significant with endpoint progression. long-term delaying met in statistically long-term primary reported, As studies evidence

well a adolescent FA need patients for to safety fill FA patients. extensive data the Given promising as to provide as in adult both the unmet for and children date adults, collected potential has vatiquinone pediatric the therapy

the track to potential fast our HD. approved disease. September, reflecting be for The first Huntington's the designation FDA for of to PTCXXX received PTCXXX program program disease-modifying in Moving ever from therapy

we Type later on meeting an first an C Huntington combine the lowering XXXX discuss to potential of a meetings track meeting, has of to to Type expected the serve second which registration asked the in is continues that strategy scheduled to context to fourth FDA or study for quarter has The trial X serve single complete is the trial approval, PIVOT-HD been of the trial discuss endpoint meeting patients, ongoing In quarter. FDA. second to a results requested endpoint progress as is efficacy in the in two One a for Phase the and in with completed quarter. and supportive Phase we HD C trial accelerated trial accelerated addition, as for confirmatory a whether into remain approval. III in the the now II The well, as with

quarter. for developed schedule Utreloxastat highly compound Turning demonstrated on of first in is to remain the ALS registration-directed targets pathology. to death results program our the being utreloxastat share stress that ALS specifically ALS. paraptosis, to pathway be CardinALS trial to We for relevant oxidative top fourth cell from the patients and line

landscape recent positive need the enable ALS significant CardinALS for could the Given patients. to utreloxastat changes from of the study unmet in ALS, results address the therapeutic

XX diseases. therapy If administered November this to remain therapy the and first open BLA. gene FDA schedule very the of by pioneering to new to we gene our this authorized Finally, action I on ability be brain AADC am approved, team's for treating bring patients. door a proud approach date to FDA therapy the would whole direct-to-brain ever the for

of execute as we many summary, a to productive significant on XXXX have had In achieve we and objectives very milestones. our busy all and our planned quarter continued

performance. over call to Eric? to the turn now discuss will our Eric I commercial

Matt. Thanks,

team third Our quarter, our global across another delivering performance the marketed had in million revenue strong of $XXX customer-facing products. X

based evidence trials with renewal, the the from recent DMD Translarna CHMP's the be on continues for on real-world families million global totality opinion solid and throughout experience. had Europe Our of physicians, $XXX to revenue. strong of and franchise quarter negative a there positive Despite support patients in

commercial remains Translarna and the that in Duchenne ensure to available mutation boys effectively to the we review East of Europe, existing Translarna have to European it as our in as authorized dystrophy pending continue America, continue North African men made is worked commonwealth opinion on of of and with young professionals care long new as therapy those living the add states, and Middle nonsense Commission. Outside We by Latin Europe, with CHMP muscular teams maintain patients the independent countries. health

delivered obtained Health. we In a Translarna the and countries Ministry quarter, the new African group we orders purchase in remaining XX% Middle of North fact, third in of Brazil, the two from the to In region. East first-time

for pending under positioned by is review our to and currently FDA highly the mutation mentioned, to experienced U.S. NDA Translarna Translarna FDA DMD Matt team approval. rapidly potential bring is nonsense patients As very well

Emflaza. turning to Now

Third million. quarter was revenue net $XX

to continue with health the work care brand. to dispense We professionals, closely and payers specialty pharmacies

services team the each with supporting Cares PTC information personalized patient greatly patients. continuing white experience new our co-pay brand and for and from provides and programs Our timely shipments both enhancing pharmacies, glove assistance, specialty

believe Our billion the significant patients, for physicians, the on global families potential revenue, extremely excited has to opportunity about customer-facing from sepiapterin teams are over including we generate and alone. upcoming PKU $X feedback Based launch an the sepiapterin the U.S. potential in patients. of

due markets patients. of to patients We than medical of differentiated disease PKU to to orphan vast and this addressable not population options. in with these high large providing a are look receiving in of has treatment patients XX,XXX the treatment unmet majority current more physicians important access with option reimbursement, PKU limitations new forward the address and needs

and and and the in mentioned, As by under the to for children of multiple steps year, potential sepiapterin with U.S. filing major important global important Further is review under Europe, this launch PTC in and now PKU. review Matt the positioning in regulators submissions in in living adults with bringing Brazil, Japan which Canada, Switzerland new by markets therapy XXXX are are this Australia, beyond. end plan in

our some efforts, provide which have years. several ongoing I been will for launch on details

the We advocacy working understand pediatric with key patient unmet specialists have health around care strong the relationship with and metabolic build dieticians PKU PKU to been and world including community. providers, needs better a the closely geneticists,

centers teams continue a of with develop the to of Our PKU prescribers. key understanding and the landscape treatment deeper many

inborn As screening metabolism most early available of the since you XXXXs. prevalent via epidemiology with reliable know, PKU newborn is error disease the

receive treatment centers world. well-defined the clinicians from care patients around and their PKU

mapped publication of trials goal centers and recent Since these and potential these patients data, AFFINITY convenient centers treatment the key diet a getting markets as more have in health levels the has phenylalanine of clinical We globally. care liberalization. rapidly of highlighting profile oral sepiapterin compound's providers many seen awareness in of enabling to differentiated efficacy treatment excellence, in increased

year. treatment excitement option frequently Our PKU with field-based expressed a potentially available needs, teams next their stakeholders met about address unmet to new their who have have

patients ability important optimism providers. treatment for highlight sepiapterin is and this patients care Importantly, with liberalized health to seek the to earlier options of an new diet, from their motivator

in addition, disease Now countries. over our footprint XX leveraging established in global infrastructure, which are has we rare a

will potential country key We a to access maximize and pricing and sepiapterin globally. reimbursement of corridor launches narrow markets the in maintain sequence

in Our are a treatment disease available, strategy sequences research upon benchmarking current of approvals. reimbursed key and launch of premium on our be treatments based where obtained branded pricing to especially potential indicates are we profile where the rare many options compared analogs sepiapterin initial can planning markets PKU differentiated

at but and reinforce as disease U.S. landscape. Our of payer with managing customer-facing core treatments stakeholders These developed rapid PKU last highly for the teams rare in not facilitate opportunity, billion-plus the in PTC over profile, adoption should potential globally, our the only belief regulatory the understand differentiated the $X complexities along decade, sepiapterin's well capabilities access as and alone.

vatiquinone. to Turning

FA that believe with NDA our course provide who is compelling all rationale patients. opportunity for patients in vatiquinone could vatiquinone patients, the in in tell us believe functional With mechanism potential options its that a for this We parameters excited early December FA strong favorable launch of commercial XXXX. the particular, is current needs adolescent patients profile possible unmet pediatric FA and in KOLs treatment this for They and benefit for well unique safety efficacy submission the vatiquinone's team there year, FA disease. the devastating in along for as treat disease. to significant from of data differentiated relevant highly as action, of treatment are planned

key team on for focusing in neurology of vatiquinone. U.S. projects experienced preparation launch is Our the

and moving Latin Waylivra to Tegsedi Now America. in

franchises in treated and with across make to across growth progress these both continue patients identified newly the We region. good

continues approval HTA we following In government discussions. quarter. purchase in Our Tegsedi assessment with of recent a new delivered we which reimbursement a Brazil, initiated in and positive for Mexico, third Tegsedi capital the have expansion order

to continue all fronts this revenue first on a of execute we year. based months conclusion, strong In X on

with million. XXXX $XXX $XXX to we a mentioned, updating As total Matt million are guidance of guidance revenue

the in U.S. is AADC team well in gene to customer-facing by Translarna, opportunity sepiapterin and thrilled globally Our as products: the new X therapy, vatiquinone XXXX. launch sepiapterin potentially

I turn for call a financial the over Pierre? will update. to Pierre now

Thank third the now you, refer details. Eric. I'll our earnings share for afternoon release quarter of this financial press to of additional highlights issued Please the XXXX.

top million, quarter was product DMD line Emflaza million. Total with million. third for Beginning franchise revenue in Translarna was revenue net the of results. including $XXX product $XXX revenue of million, revenue $XX quarter the net $XX while

Moving Evrysdi. to

Third million compensation in expense. was $XX noncash third of quarter expense, million GAAP excluding noncash Roche, million achieved $XX quarter XXX global to was stock-based revenue expense stock-based the by million for USD resulting excluding in of $XXX net XXXX, of million third million quarter R&D revenue for for approximately of the XXXX, PTC. $XXX compared in $X compensation royalty

XXXX, for million compared $XX excluding compensation of expense. $XX the million third third and was noncash expense $XX for quarter of the million $XX stock-based expense million in quarter excluding XXXX, in SG&A to noncash Non-GAAP

$XXX XX, securities December of to as billion equivalents $X.X as Cash compared marketable XXXX. of September million XX, totaled Cash, and XXXX,

sepiapterin Our execute including strong with over launch necessary provides our financial anticipated position resources and achieve products. next the the the several of strategy other to our and PTC milestones on years,

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

[Operator Cantor Instructions] comes from of question first Fitzgerald. Kristen Our Kluska

got on is for This We've Rick for you. two Kristen.

out Is when Do in about community So update there of potential clarity we'll first come the have of that path that any chance you for the more all, Huntington's. thanks learn C meeting Type on meeting? you for on could forward the here? the a update any might

Yes. Rick, the for question. Thanks,

we're lowering discussions terms the So trial, with surrogate end also, an of around to both the understanding FDA, know in we but there's Huntington endpoints lot have around a discussions efficacy for course, the -- a as point. of excitement of the going

benefit thresholds approval, evidence to accelerated that talked we've us As Huntington surrogate FDA go wants is think we ultimately. lowering present really been protein when specific about, endpoints clinical of about have there what are of package likely a provide associated why And to lowering is of for to benefit. the scientific that

them of in literature, the which studies, rich have in scientific provide that protein a including benefit lower have show were XX% of we've to in So protein. Huntington you our with human disease in achieving slowing Huntington likely lowering significant vitro course, if vivo to PIVOT-HD that to we in been you're update, the threshold XX% XX-month progression. as shown able we And of

year. of sure as have We sometimes the to the expect to community, minutes, with soon broader receive we meetings FDA once And at thereafter have the this as always, that with clarity, or shortly meeting share or be we'll in Street. the these comes of time December as which we including

accelerated of of interest there's PIVOT-HD. a know, approval hearing we lot forward As path in for this about

flexibility in rare Excellent. And more time. space one seen We've regulatory maybe of recently. the disease lot just a

Translarna. FDA XX for given about weigh data STRIDE and have going and submitted of how flexibility? were sort that in Can talk sort of looking the evidence FDA at of these resubmission think given So terms regulatory might you you things just kind of Study the new how registry the been

sure. Yes,

talked NDA things. two on So as Translarna was about based before resubmission we for the

on evidence of Star data across Study XX. the effect tests, overall flexibility DMD And all significant set Assessment, in of enrolled pretty with significant XX%. to population time that First, not. This time actually unique endpoints benefit had is we North a nonsense is or the of boys function broad those population loss to in showing distance, XXX Ambulatory where X-minute in walk benefit significant

the these I really all set think first is significant that an population, effect end ITT points. data has across in this

manifesting In And is the what confirmatory to have is demonstrate of to of and X.X over of addition benefits disease years term on agency STRIDE years. the adequate several Study able trial, ambulatory cumulatively STRIDE a these ambulation loss addition, the the short other likes which as we're are events, single see delay in in aspects as seeing significant to in we registry a registry. function well-controlled in XX of

bring in will have course, package the and So would of package of both to which can the with is be of -- But we any own, quite a its clinical great. on agency and DMD be term the we long package patients, data. an compelling benefit evidence the flexibility of efficacy in the nonsense assessment unique short-term

comes from of question Our JPMorgan. Joseph next Eric

Or now -- the might that you've be noticed a that And overpowered in from I of expectations? -- the and the think -- I have one to that change participant use be primary a recently Was endpoint the Can motivations to on in that fact CardinALS accommodate prior meant combined talk modify We I trial. what what And of to you ranked the or powering updated slightly statistical guess, were impact change of behind some also you you overenrolled what endpoint? would the perspective? sort study. the changing assessment endpoint? ALSFRS also survival. versus about that

Eric. question, the for Thanks

So not your related, not are related. two are those questions two

of the really of disease analyzing a remains continuous progression The the interest way course trial. the study. that outcome which a variable results first the is score, rates of of The ALSFRS is the over

use test, well they However, patients us the the their which deaths that's which incorporate to for occurred will able that in is has account the the analysis, study. as as information study. the course would of decline is that method ALSFRS order known well over the the course it's analysis the Joint to test in ALS, of the of death And as on recommended during RAN statistical die any primary for in like the us known to FDA preferred

the in of statistical in analyzing primary it's really just analysis death data. we're way include the to it change the So a

And on that that use analysis to it's again, like fact, is, from able the if ALS that, of preferred certainly, feedback because this came have you And FDA they they in their analysis information that back incorporate look test see that, to said from subjects. all is you'll the to other plan their the from as reviews drugs, method. really

population primary Regarding the had population run-in a as the talked rate question, X-month observed we've based during study. target of analysis your progression we of a the for about, second on

how this to need X-month ALSFRS they in calculate patients of scale. patients observed in and enroll number So that change group. analysis achieve we to the run-in order many primary the a certain where We study patients on targeted of number points in

a actually screen what the of see enrollment subjects we slightly because in lower had analysis you just population. really So in who the for met criteria the primary yield increase was

Shi Our question of Kelly from Jefferies. next comes

maybe would set this expectations also Evrysdi? beat for could franchise QX also dynamic versus to you of help what DMD the quarter, from into Given the for revenue XXXX, royalties be us

Kelly. Thank you Yes. for the question,

revenue said, us the strong this overall to So we -- allowed year to guidance which quarter, were $XXX was raise as we million to quite this million. $XXX

fourth the quarter a continues in in face incredibly DMD brand the done perform -- generic typical we deep revenue, Europe, the we portfolio remains our about, well. expect see we franchise Emflaza across of both overall, great will of in in to . Teams expect And to the revenues, talked We inline continued expect but quarter-to-quarter as as quarter. with it terms be protecting in from products, strong contributions in job revenue we the the quarter. Translarna, available Translarna stable competition. fourth that Certainly, contribution over lumpiness the have have we fourth

comes Stanley. Hung question from Morgan of next Our Jeffrey

patients OLE? of plan maybe in is like all are going diagnosis? since Cardinal's the progress for you Or characteristics the Congrats you Riad share line XQ, Ahead of the maybe can baseline in comment on programs. time the what on to like This Michael top Jeff Hung. percent into on do

the Yes, for Michael, thanks question.

with seen endpoints. the key excellent line they were cardinals. our as patients baseline characteristics results, finish we'll they to and continuation the OLE, once with top give We've regards of key results the the typically, So into

you Okay. And on see extension to a Or the rapid maybe separation extension like with add like the perhaps the you more think some FA how you for meaningful studies impact Why like do mFAR trajectory color is expected? start there? then as long-term could like on vatiquinone. was vatiquinone studies influencing

I of of benefit a function. we're terms continued is preservation think seeing in what

when group we're reported slowing approximately treatment progression. X the that group. As we the the the seeing XX-week out XX% saw years, study, XX% placebo-controlled an we in XX% And benefit, in progression to slowing we still relative at placebo read

started a X.X the a vatiquinone, Whereas decline. cohort, As patients was X.X natural point who they we had reported the on history in there point in decline.

we're continuous a terms function and benefit So in preserving disease of seeing progression. slowing

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

one I Translarna. and on guess on sepiapterin one

Just in if patients particular, maybe to bit And and that's you elaborating reactions Lancet communication I'm confidence stepping the from providers, the you in maybe the the giving could a some potential little data. research on about after, curious talk more payers payer of the there, in then hear lines you sense for on U.S. And premium might both XXXX? in you any And could steps Translarna, of wondering if we the for the some next think have pricing. updates and the review just whether time Europe? potential

Brian. Sure, Yes. for the questions. Thanks

Translarna on Eric. me to sepiapterin first, second Let the tackle question pass I'll then and over

first Translarna. on So

that to approximately whether the or days assessment of want adopt the course we where CHMP about the So does its U.S. XX are opinion. In of the not you they opinion, in the asked EU, the EU. and following period Commission European now of

And and remains. we've So authorized to Translarna of commercially about, as authorization expect we're to while we further least patients XX talked making provide anything make available hear in that continue to remains period. it approximately And days. this drug course, the at sure for

NDA the of years fact by In in this resubmission this a many been the had protest we've many, filed review. a of the doing a that Nonetheless, terms XXXX, as is over clock bound ago. an FDA U.S., initially that not is given about, talked in

package. However, of the just really the is we expect just evaluation the given which involved what's portion resubmission, efficacy in a

in months. few they'll next expect the to get that the review We

time So as the specific clarity, have any that information line soon can will. as more we on we provide

quite about sepiapterin, comment first publication. that make excited Regarding you're the let Lancet just obviously, me the

who differentiated that action, has how precursor act I think co-factor, in that's second ability with dual its board this. its the fact chaperone a is to we're of patients BHX to variety mechanism significant severities not but mechanism PKU benefit action, across true it and which again sepiapterin about which The are a of why provide of a is testament patients the to allows a element to of genotypes. it the physicians only as really to BHX then very such seeing not effect responsive, are in all important of excited

they they is see been for publication which the need that patients. Lancet significant is meet that unmet potentially what this think believe, a PKU with has the transformative consistent therapy I can

we're Eric about payer Let it and over to a me pricing? turn our bit thinking to little talk potential about how discussions

for Thanks, question. Yes. Matt. Brian Thanks the

payers its of As very of to Phe pleased of With you including on know that goals doing at than on what us unmet market to been a goals. a can is AFFINITY. see research differentiation in of sequencing that in X of Clearly, we're patients have can XX importantly, there publication lot levels time. a the really efficacy we're activity, measured many We've medical we landscape those population close high need, this more engage rapidly. and the is achieve XX% to be actually profile, with based told able and seeing can payer also is that it. lot that the see, therapeutic countries And not a therapy and where of research out

U.S. There to is willing in need the premium as as in pay see and that well a payers unmet a high price Europe both and they're

matter weeks sepiapterin. a see of You know gain literally can of with or they levels that achieve can -- they therapeutic actually the in

branded pricing differentiated, of that achieved including have many know, of having is that been believe clearly therapies So many for these looked are obviously, premium number a can But we've analogs, in we as we at a launched markets. recently actually that existing evolving. and strategy price be our different

to we pricing the is just the closer when announced And we launch. until have final I'll much strategy to really get going say then label. be not Finally,

next comes Eliana Our UBS. of question from Merle

of bit in terms a of of you endpoint the Can feedback study or on like? surrogate, I FDA supportive would meeting? And partner? what forward I from your get and run registration a forward and is guess the what would you range for Phase could there this potential a is not a as about in supportive outcomes III specifically clinical path you that little the where the path of the the about of own Type Huntington not from you the if terms guess use the talk more of look in could that And scenario use potential a your whether are potential around that thinking surrogate, strategy of potential range full look of C lowering outcomes how

which of Thanks we're really what that for to because serve in endpoint. in they with surrogate potential There's to a the protein specific Obviously, the a ask about evidence able for ability a it associated clinical productive Eliana. can endpoint That question, agency shows meets a benefit. to that, the surrogate with discussion Huntington there's have terms reductions optimistic that as lowering Huntington the achieve. for we're is threshold have regarding

disease to we're caused the Huntington monogenetic of a consideration able and itself, taking that. address by but disease just toxic a it's And mutant that's protein

target protein, in at in squarely about example, way very dystrophin protein disorders for a it's think drug I and implicated other where surrogate they there's endpoints have thought in DMD. an the

approval agency for has interest significant of neurodegenerative accelerated establishing disorders believe like the where a there's that pathway Huntington's had medical in leveraging an time. unmet also bit We need takes and disease, efficacy the

as marker disease could is open And to if So time, certainly ability therapy the what as also safe outcome about fits. there's to Huntington trial important certainly that and regardless meeting. a a want we that that an to that for that because be efficacy efficacy that of PTCXXX that with approval. door accelerated protein the strategy an position is to endpoint in believe disease shown a known possible, we're lowering move having do we impact a has to discussion trial be of the quickly for forward pathology that same of the At then

the back with and put possible the FDA, don't these you be could complete discussion XX example, data very the that from or a PIVOT-HD And with with want look we to For we'll ready data surrogate Why set to if revisit meet in outcome. still you first could say, occurs, so spring, good. come the they patients on and that's endpoint PIVOT-HD, quickly doors and get situation the approval where to other as so endpoint, that to efficacy in place be finish the up on you we're and study part want accelerated because started to trial we trial for, say, running in get possible. as an efficacy either the as as need initiated on of than going we line that the can

sheet that to we study. have company and balance of conduct to Phase be build terms In said able position along, conducting the study, that all the like we III

because and have to safe to tolerated. where oral the study for they've who be and study and an investigator physicians Obviously, the didn't patients previous whether as want with have trial disease shown the in is worked groups either really has the that's and in that. the world, in We Huntington's around who the including community, they well to administered Huntington-lowered enthusiastic of has participating that patient be opportunity our study, the and are incredibly data easily capital do therapy, seen closely of team the in infrastructure the for want a part incredibly which now the an to be

approval. or advance registration confirmatory we'll an of trial to able either forward the study to way, -- trial. look do it in accelerated we becomes Again, So being it the that whether the context whether we have

And Great. follow-up. just a quick

you but context any logistical, X requested with around single asked maybe I the suggest? meetings maybe the what combine think this? just the meeting. you this can had Maybe share C is to this FDA, you X to mentioned Or a but that FDA might Type

provide Yes. their review people Again, to going roughly the And feedback as we've the it's meeting overlap as the neuro than this for issues we well with as and request is rather -- have purely it's logistical. collaborating how one proud know, same they We and the involved. just are who where and of think and be you the submitted same reasons, at I two discuss such agency time. logistical division, in meeting other we'd the been same people have the

to that we look meeting forward in having one December. So

Our Joel of from comes Beatty Baird. next question

the ex For would And any your by percent Translarna, how not affected long last be sales those on commission U.S. into potential what future? potentially also current could the of decision? sales

Yes. Joel, thanks for the question.

was XX% Europe revenue in that opinion. the revenue As risk be of be the think that that roughly we've talked that about itself, CHMP the adopts EC would from in at would the Translarna event XXXX, we

Now we individual going will only country other But can in revenue that pathways see and is forward certain that other that EC there's European adoption. commercial make leverage we obviously, play side of product that we've that that possibility the inpatient something or be will the a it's available. that And revenue. said would still out risk at countries on

those would long other how regions? be And exclusivity in

It varies.

exclusivity out many to XXXX. in have, We regions,

U.S., one an last for do expect you ataxia the then question Friedreich's And in AdCom?

submit package. Hard the to know. Obviously, we'll

or out there review. it but at be to submit We AdCom. there'd that priority a whether expect not We'll be in expect would an point to find we December,

Friedreich's importance the And And particularly, should the a of of understanding the with discussions. our ataxia certainly, therapies with deal good number therapy development, have very in great approved obviously subscale familiar quite the of FDA of disease. know of upright have we that already so endpoints. they they Now is stability having understanding a a for there's

this from AdCom, not I'm need ready they've novel first is if at for of they outcome this don't terms would know that to time or have disease an in the But we'll of -- looked in therapy be endpoint. first So I being standpoint there an it.

from of comes question next Our Blair. William Sami Corwin

Congrats quarter. on the

a they step As of to based on conversations the you the of follow-up. you your And your many would copper their I patients classical? have disclosed have generic be would of who through And Kuvan? have payers, with launch drug kind ahead then are for that specific that are on go to early patients terms what in adopters? assumptions patients percentage And envisioning the may of be sepiapterin, of

sure. Yes,

then it Let start, to turn me and I'll Eric. over

be had see we would who've PKU of we and response. would as tried not on population which as the or including not optimal are that been on talked those as classical Kuvan segments adopters, those those nonresponders, So several Kuvan BHX patients early be well that about,

just talk two summer, heard So we KOLs of that that from strength easily detail as a more last of patients all access about try study. And who are over those whether we've into me therein fit those on buckets. to commercial to our how dynamics But want the we regardless play they payer . can bit let can believe that. into segments their sepiapterin based pass data from on and INFINITY Eric,

that. see but is for patients the Right what treatment. medical of seen need a tried, And of an thank discontinued question. payers standards market. get that, don't the Sami, also unmet It They surely reasons. either now, and care the or physicians Yes, current for are see XX% is we've variety failed inadequate of

one, that with So it's and when this that, -- treatment a around a not in taken. from you with is easily payers conversation efficacious get benefit substantial very we number also a there oral key world patients these highly when the markets, can that can of be discuss U.S. from payers, treatment difficult

Second, have rapidly. controlled They're as with recognize adequate premium very quickly pricing And have these not measure with They're treatment. diet. not treatment. strategy because what a do earlier, not controlled can that actually can simply current is they and efficacy they mentioned we patients

of in burden a also patients is rapidly. can They actually that level many measure can liberalization. something They cost. diet how therapeutic isn't can to get very reductions fee measure goals. this the see They So terms

supported study we'll data head-to-head and the so physicians And the community. get received So well access pricing pathway has been payer able definitely to the AFFINITY far to the by rapidly. already when strategy, by everything and be been that know we and has about patients we treatment presented access know talked that we've there, premium quickly

and simple about again, quickly. I To because, very levels can it's very a thing easy measured Phe be step question your edits, think

authorizations Phe and So measurements. on can rapidly sepiapterin get through sepiapterin patients to quickly prior through

has a now, right by response treatment community is the new the to overall, option. overwhelming excited the AFFINITY now very PKU So and been right data have

penetration. has modest $X PKU the think the team opportunity the BX, in billion Great. And assumption pointed was out of that with and I

what opportunity assumptions I that So $X in penetration guess, U.S.? for the are internal your billion to reach

Yes.

especially patients a XX% XX% alone somewhere going important modest believe closer preparing I contribution and Provided U.S. to to launch, now, not them not patients an all yet a there. get but if the right? the strategy, opportunity have of medical to in somewhere medical even currently of penetration, with many right launch, -- product and And while think if of be that support And So we believe $X the -- not on be we're treatment, to alone. our we we're between ready are you on our would be everywhere, say, $X would the on a can first how -- launch between U.S. treatments. is I unmet guide opportunity we all, We're guiding and premium going there's get very this to XX% global opportunity, to we as because billion there's to for we pricing again you which look revenue. you product. in need will believe we closer substantial And XX,XXX billion are XX,XXX get.

Bohnsack Our of next Cowen. comes Peyton question from TD John

follow-up. you history guess And that on sent requested they when Congrats December, then Have I've got I FDA reviewed natural it know the certain the to I on outstanding then submission? what's quarter. stats a Have vatiquinone application designing And given you a for and for any analysis. the FDA? feedback? the strong were the

the what part I'm sure the question that is heard question, for I asked? plan, Thanks statistical you Yes. of about the second not analysis Peyton. your

or readout FDA that after whether on -- you have to back came asking in or if was I any And has the it. viewed not October, the give maybe FDA? you sent the that

Great.

Okay.

the interaction are reports of had from some long-range in show time Obviously, we extension those results we it in. in October, They've also we're all package, terms long-term seen We terms agency set outcome including up items, contents send and studies. a reviewed It some up with October. the finishing in takes all -- pre-NDA of what of had, write-ups summary with and planning the long-term the the that got of format table just outstanding So to write just them we to where in data. of our the data.

plans few the And physical study the earlier extension using ago. data completed analysis later as the importantly, they as of previously extension placebo-controlled agreed analysis the have well we from for long-term years a both the were MOVE-FA to

So primary were change went going they we we strategy exactly to about how the matching knew with over do -- the the analyze being endpoint were scale mFAR data, endpoint going to time. we how doing how in the

align And as We have so agency that. NDA. after them of will And they any mentioned, from some the until don't fully with I the we data. additional expect submit seen we the on feedback

lot the done you how the a makes quickly could That I pivoting then all then the patient week, inspections Great. facility? really -- therapy? manufacturing for next launch the assuming necessary And sense. And to And approval guess of quickly have been identification... how the

Yes.

the we of and November Everything We to quite -- look XX, well forward next with look any about we long obviously to U.S. as standpoint on quickly potential the this and done. So the date being in bring possible. patients week. to remain since inspections from are based an therapy transformative as forward approval for for to went our interactions, inspection PDUFA the able optimistic

of Our Goldman next question comes from Sachs. Paul Choi

I just discussions how about quarter. on payer the of of sort to feedback on external with your what to just multiple very population? is to healthy what in sort has sort payer of currently 'XX? just cash on potentially up here be regard want been? comment I remains coming funding position appetite potential here maybe Can And relative Sky versus here. just Claris ask vatiquinone to secondly, pricing business adult want development thinking for for follow the the may Congratulations the And here. you you launches Matt, --

safety that, Obviously, payer on package be and terms discussions a and on Yes. for there's in. clear that Paul. the of supports unmet We're vatiquinone. -- need benefit say important patients. adolescent package bit pediatric focused getting in these we're I we we Thanks efficacy for data a the a and now have an to patients, look, would early for But questions, the having

diagnosed increasing we know diagnosis are an adolescent. are proportion important patients being field, the is there's as in As who of increasingly young. But pediatric as are and who well, patients

strong, we for therapy patients. being getting and need effective to need safe look And -- and forward for so a that that unmet patients FA to able provide is those

provide But for can patients. as to we a you also believe be raised, adult able therapy we

adult benefit We have and patients. in MOVE-FA evidence

We in disease stability which as well for of was a loss extension only have have also incredibly is studies of safety we long-term we both early profile. long-term as Again, risk which of in MOVE-FA to ataxia ability important of any evidence reduce scale, the placebo-controlled age. speaks on Friedreich which the the and very upright strong And ambulation, progression. evidence benefit conducted course, study, of tolerability the of have slowing important in patients directly ambulatory any to adults,

forward discussions about having So payers differentiated look and thinking the also we on to that a I the how with we really have believe product price that.

restructuring the breakeven. cash balance of of operating of the towards position, terms expenses we've move reduce to last the end the in look, both company terms our worked and year, efforts flow In very towards sheet cash

strong business to only I in of the of the having launch sepiapterin. would situation having a launch to cash launch But not global the balance products, enables we we be us opportunistic the development. that capital products to needing also, have course, love and think and and more about -- of remain to year in U.S. the both

on have have the hand. we we cash And

an we in be We we're have position monetization could the additional the U.S. to, that term. many well we needed we saw we opportunity down a our short are or ability, fits that some the of launching royalty Royalty in if also we where to Look, products if did portfolio. the draw Pharma in -- with into

into ex our also think easily to could that opportunities infrastructure a so now And out strategic commercial business infrastructure. existing about other the need or we very how can and set to fill way we even U.S. think development any development about commercial in help use portfolio

comes question next of Barclays. Wang Our from Gena

I few questions. have a

XXXX. first date, The PDUFA sepiapterin, regarding XX, one July

confirm AdCom. -- PDUFA FDA to be no confirm will want no that there Just

Second question Translarna. is regarding the

now days mentioned XX You the that right EU.

if I know the XX impact in through days, can independent how is Europe? that long So there. questions actual process in when related unrelated you're And steps? the on decision be what walk -- really will take would Brazil, will after unfavorable, the you see the revenue maybe next we assessment under the like And

April. Any regarding have independent back ADC next renew will week, process regarding fingers approval, update our the get keep They crossed. approval hopefully, in Lastly, an you very there? process quickly How you many the And doses regarding already do manufacturing? can how ready you in you many you how patients you to think -- identified the stacked? are ready U.S.? How already

thank Gena, questions. the you for

one. them Let me one take by

the of be sepiapterin the not where is AdCom. on AdCom. XXXX, approval. to tests endpoint phenylalanine for been support indication reduction it publish in that of date PDUFA situation one of July First, the no Again, of trial at expect XX, There's full a can do we we're blood

expect Obviously, would the one agency have X I not an we So PKU, where understands is this AdCom. think to therapies the prove

very data of the question. also is leaves efficacy little clear the package addition, In

think brought I to we to that AdCom. be needed

late to until April, first and Look, it opinions time through days spring, or the second expected made XX in we was and Translarna March the adopt as XX the CHMP. went about January take days that approximate time it this the actually late had back that to the the EU. the line we in to days learned things to is or May Your an us question XX we wasn't confirmation we late from decision not and got East

operate of the for implementation, can't could So I what happens other what depend it adopt immediate -- that implementation. and on and the say it we're XX opinion going to think side they is delay adopt and for days European to going that say to we Commission the it approximate sure says would going adopt it's we're the

In due to the -- not issue. not is come about situation this safety here, this it's has important realize that a to being

get no in of urgency being the market. the there to there's terms off here So patient an risks product

those of of well. see of little it's negative as a provide whole also may following that And think exactly -- bit of in to course, an adoption I opinion. that's the back a have to are and the instructions what happens sent terms flexibility what the So as CHMP then, terms steps of in we'll well

assessment. still a been is unknown approximate minors we So ongoing. of It it obviously, and has The there. days that an is lot independent we reach time XX line. Into Brazil,

We orders, that in for know obviously one any one interest as third on orders we had have strong gotten a quarter. is not a Brazil quarter sign. good in bulk We obviously is to X additional that in the view Minister feedback the from Health Brazil, we of What do in the patients ensure or second There Translarna. remain

been And XXXX. then AUC the a for in approval terms again, we've Brazil mean, had deficiency. We approvals. EU in had we of had ADC, -- I in We've ready. we're

in process the patients doing in Taiwan. globe. And of around approval the We the in U.K. treating have we're Approval We're cross-border

treat the more have well the to next adequate X once addressable and we patients understand will patient supply supply. enough see believe, at again, than we years. the And X than more the market we we like through to U.S., label with look in hand So have,

question Danielle James. next Brill Raymond Our comes from Bongero of

new NDA new NDA an a to Why Translarna commission it a have just quick question. Study data from wouldn't why technically the from curious I XXX? a clarification is see I'm earlier under review be readmission can

complete to agency adequate Study this response XX NDA make an and letter. back the conduct the The The what and XXXX. in that reviewed need fulfilling additional CRL, was was trial main previously the to filed the a well-controlled resubmission XXXX, XXXX. sense? protest. to complete over miles, XXXX, and This identified establish it was that Does now therefore, in of that NDA goes complete issue the issued that to addresses In in efficacy. technically led bonds

It I appreciate clarification. the does.

Our from Joe next of Partners. comes Leerink Schwartz question

and have a one about on utreloxastat. I question vatiquinone

that they by to heard we've be FA, in efficacy. blown So benefited patients patients SKYCLARYS' some while away not more think seem that

How market options that is do and of as as vatiquinone, evolving which reps feel new their might and now how switch potential well patient to terms downstream. you in further with a wondering we're So this or retention influence its about following vatiquinone? treatment for combo how think works might try patients

for the Thanks question, Joe. Yes.

it's know. progression I of clearly, we in would if to no with I and And the I terms upstream is the of mechanistically pediatric in at acts able think given terms where of say, of the always slow I perceptions patients' hard SKYCLARYS with drugs vatiquinone and it's the effects in SKYCLARYS think its disease First, there's we therapeutic option better at always believe in of getting But label. would pathway. look, or But of theratosis least needless for say, think adolescent that -- option a adults, not, to be they're where hard certainly in slowing provide patients patients, scale, significant know can progression, to of symptomatic complaint which also is the benefit not data disease with a we but only the #X ataxia. Friedreich from effect effect we patients

look, one as if particularly forward able interested like got do. and you effect. a adolescents any would therapy. disease the particular therapy, best think being offer it therapy to two more you've enjoy I progression, as a and slow there, time we to to lot likely need to start ataxia, that than will But Friedreich the and do look And need benefits be out desperately FA I patients, and A kids you think, Do adults, is I make sense for thing a combined provide payers to have safe mean good can sorted possible the be much you to so could would them disorder to early look, to one look, would who therapies that it as we that flex a terms have of people well. opportunity possible. the of as the as the slow depression in when be welcome with combo therapies? of I in But such. course,

preclinical you're targeting history or data context meet done in based there CardinALS natural the on supports give trying then hypothesis? anything any And us study patients ferroptosis mechanistically? for a therapeutic that particular you your to you're have ALS, select Is some best threshold what And Okay. in achieve to that can you looking for respond? might to

Yes. lot established that's in how targeting different go. learn But enzyme side well a Obviously, show which the this disease with studies inducing ALS. -- overexpression whether pathway. is rationale clearly of key nutraloxastat ferroptosis about in the the ALS Enbridge for question, ferroptosis It's is we'll ferroptosis target really a very study by of aspect, Joe. if you a preclinical other that on first how There's in could number a we we that have GPXX, probably good is the in look,

significant you of you affect SODX can in augment distance directly You The any stress similarly, phenotypic. ALS. to But a speaks stress oxide able if process ALS. of accelerate the being whole to insults relevant ferptotic edoxaban as for of decline have number to concept in

disease. be aggressive hope We to bit support slow and provide key to keratosis. element. a targeting targeting But by what's So rationale that ALS to of going there's to a could for that is progression great complex data able be pathologic incredibly a the look, therapy

CEO, question-and-answer the for concludes back Klein, turn it This now session. to Dr. concluding remarks. to would Matthew like I

much. Thank you very

to you, we study, taking Joe just last the look and would the all for and not we for a you and being evening. incredibly be quiet of year. joining the question again, the side have of whether all our across next to the the commercial just close of milestones any And execute thank and utreloxastat data for the an forward we sort CardinALS important having We following time. Overall, with me terms performance, of again, good of call having be regulatory this ability level about data are excited the the to that the call, the CardinALS parts and saw questions to strength incredibly joining because follow the Let high all number period teams updated revenue the a where in everyone, study on of share be on period quarter, at company, side, close it getting to everyone continue We're keeping as you for to the ALS just of of in on thank clinical this are as call. around.

today's Thank you This your the in conclude conference. participation program. for does

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