PTC Therapeutics (PTCT) 26 Oct 232023 Q3 Earnings call transcript

Thank you for standing by, and welcome to the PTC Therapeutics Third Quarter 2023 Financial Results Conference Call. [Operator Instructions] Please be advised today's call is being recorded.I would now like to turn the conference to your host, Jane Hanlon, Associate Director of Investor Relations. Please go ahead.

Good afternoon, and thank you for joining us today to discuss the PTC Therapeutics third quarter 2023 corporate update and financial results. I am joined today by our Chief Executive Officer, Dr. based Business and Pauwels; Pierre our will forward-looking Chief Officer, Chief Gravier.Today's our expectations. include O'Keefe; current Kylie Chief statements Klein; Officer, our Eric Commercial call Matthew our upon Financial Officer, materially Please risks take slide our forward-looking adversely call, of Relations as and Investor these statements.Our the statements review that actual in subject with materially contains such posted forward-looking business differ conjunction from on our website which and moment a operations. to can the our to results are affect could statements results

and that, For non-GAAP are company's certain a non-GAAP other to SEC GAAP measures detailed well disclose regarding uncertainties, Information on during in CEO, report call call. Matthew available description we quarterly of Securities will Matt? recent filings.We with you today's me of to Form information the as applicable our reconciliation and review Klein. filed GAAP the and use Commission annual risks XX-K release.With as over to and to let company's report of non-GAAP pass XX-Q our a encourage most the the financial this earnings Exchange

related strong from PTC third focus revenue global another R&D revenue financial to debt totaled million. will to $XXX Pharma X us financing results. Good with announced Evrysdi keeps deal, over us now additional obligations.In royalty to continue in global the I'll accessing sales.The allowed our to million Financing along with on years. with quarter Kylie for operations includes $XXX outlook commercial solid the of us like additional meeting our of agreement billion programs of joining We $XXX our target which performance all resources next milestones thank put with for up revenue on our PTC and Blackstone rights recent call. our capital we I'm high quarter net begin update XXXX an of provide expense Evrysdi and the to revenue $XXX programs. total million the strong including very between an results capital addition, our the the year, the maintains the Notably, to September, flexibility recent million had DMD between monetize stream. million of of This Royalty of development on several $XXX operating pleased Jane. provides This you on will I to $XXX footing performance for structure PTC $XXX regulatory of potential the reductions planned our and to turn robust activities to retire for quarter commercial to you, share and Royalty remaining million and support our Thank programs. shortly.I'd allows the quarter guidance franchise details non-dilutive the to XXXX its differentiated afternoon, provide $XXX and third update deal remainder to announcement million. our as for infrastructure.Now, and DMD guidance to $X our the Eric million, $X.X on on billion.Our total update

Let Translarna. update begin on me an with

expect re-examination the authorization of the to trials, CHMP marketing continued the marketing benefit negative optimistic negative re-examination on us the conditional the on as of with option to in the authorization gave the authorization, pursue the as on by Following C from concerns for of the of solely with full of methodological a address we held sepiapterin, on accordance NDA in to Translarna pediatric opinion January, on raised the European U.S., FDA of re-examination concerns to supported to NDA quarter to data or Europe. the opinions one submission. the conditional allowance authorization and this can renewal opinion. we only.As focus the remain a FDA stated conditional later. resubmission robustness patients. we Translarna of scheduled procedure the by opinion the adoption discuss late clinical PKU adult We the XX STRIDE and the such, from previously with We discussed, efficacy of raised evidence path that sepiapterin and on third the re-examination NDA meeting for conditional the data the with key request guidelines, opinion clinical both The in At days the that is CHMP of potential treatment process to well that analysis.As meeting, pre-NDA conversion the will EMA authorization opinion decided CHMP meeting only Commission of FDA type in the in safety the discuss of quarter.Turning submission the renewal

labeling. submission under our NDA the Section if study from we mouse assess of to a requested the pathway, planned was was sepiapterin decision non-clinical non-clinical the NDA as to when required Censa XXX(b)(X) to risk was that file component XX-week they considered sepiapterin carcinogenicity XX-week acquired study However, not required under the XXX(b)(X) With prior complete needed to study submission.This potential initially is pathway. inform

a application the could XXXX. no occur in of process.We mouse submission to second study review The FDA to quarter, during in submission half NDA in to occur affinity the EMA authorization submit the EU, the during than NDA if of XXXX. expect later data. of first the the strength delay study quarter We no NDA third of non-clinical will mitigates the now process.For submission The the the allows the the way report to submit submission results discuss review continue expect in potential with FDA to we marketing

the to remain X in sepiapterin.Moving and we incredibly commercial ongoing disease PKU program, enthusiastic open billion-dollar-plus the observed Huntington's Given the PTCXXX trial, of X is global the both spectrum for in long-term in benefits PIVOT-HD effects enrollment extension full cohorts. as well continued study about to patients the the evidence early tolerance study, opportunity highly providing as potential the Stage the Stage the meaningful clinical label phenylalanine to of

in only, would support X could subscale procedure we We disease levels. study occur dose in EMA subjects upright dosing months XXXX. PIVOT-HD data which likely partial predictive whom the assessment, safety treatment endpoints, status expect United a demonstrating had update time and of include XX to next of at data months June on safety Type at follow-up This trial confirmatory data we is of to study update stability milligram support in ambulation. clinically with half initial needed will group PIVOT-HD loss FDA, determine year.Regarding We data, to the C benefits could study dosing have the FDA In effects meeting. can be of the several meeting, a MOVE-FA a endpoint, at they marketing stability of to first a including XX-week reported across of meaningful milligrams stated EU. raised upright vatiquinone, mFARS and demonstrated of parallel, vatiquinone's on be was XX-month application A from as sites. the a data enrollment would with hold data for the had good a support in type sufficient an data support advice of approval.In the that scientific stated news, the in the quarter the meeting profile trial if the third data determine MOVE-FA the to existing in to believe lift that we the is only response, trial. we X And six clinical response of safety as favorable XX-month the the generated clinical of safety suggests whether to States, similar with on this the the could requested discuss be enable written address live the the value support conditional the very to written the in accelerated that sufficient are this the while it the the of concerns safety we US NDA a the United they data the by resumption FDA response in we the authorization States.Turning This PIVOT-HD should from At being to written FDA of to see meeting the a FDA submission.As a data NDA needed favorable participating MOVE-FA believe in

outcome FDA in drug we expect were of to informal the have procedure third not intended the We of have that they to comparability between which quarter, still Upstaza, data product provided with said quarter meeting first product sufficient. commercial to time had the at the support clinical we and XXXX.Turning the the drug this an in

over drug BLA data accelerated available States the be FDA the the However, cannula promising delivery assessing as our in by clinical team's expense of biomarker safety conclude growth that will a position financing review of increase scheduled for based programs. meeting, proud that on as possible deal, been study the the many to used turn the in in expect now Royalty support discuss data Eric? conduct a suggested incredibly December the operating we the United to recent on strong meeting said could Kylie all call future thereafter.Let approval pending dopamine potential contents treatment-related of Eric to fronts. along de The shortly the in outcome, our production.FDA to meeting that and the has for commercial as novo the pre-BLA BLA. a we for reductions, planned we submit This quarter. BLA continued demonstrating I'm of with the and from realize saying, performance, ability to the PTC our to strongly I me our Pharma execute

Thanks, Matt.

this and which adjustments, global of totality delivered million be supporting whom on providing new medical of DMD on starts, have turning net Europe, only commercial with revenue around with in suffering the continued markets patients our delivering X% With the nonsense the XX% revenue for quarter, targets is million be concluded our significant DMD mutation for is of we tirelessly their which and our low from reemphasizing XXXX. of compliance, continue net we quarterly year the were our the and meeting and continued growth existing on Translarna year-to-date to strong our to quarter existing for Year-to-date life-changing solid. providers we well that as present DMD boys therapy success continue the third quarter to Emflaza Muscle key in the $XXX again, performance, and deliver Emflaza continues specifically $XXX $XX portfolio and as new has healthcare focus which million quarter. disease, the in team increased in new $XXX important patient young Quarterly dose treatment DMD.Our build was world.The million, has year drivers, patients.Now on a healthcare growth for discuss a expansion.Let this communications our data million to all revenue. important revenues from guidance customer-facing growth third customer-facing in to to franchise. Translarna, the from real-world of our to the global products.Once sales this teams proud Society are continues revenue focus bring The raising and providers Translarna to worked two high an business Translarna, to availability opportunity million. discontinuation, me prior achieved with We impact The growth strong team, is information recently over million to million.For as to Emflaza. devastating efficacy growth $XXX close to are $XXX treatment products strong of evidence geographic was on World franchise Translarna $XX $XXX its and accomplishments to compared

We year-to-date million had $XXX in have sales.

current forms see trends the product community is ahead Emflaza continued States, next loyalty and new the team launches. and will to year.Now, loss future the our of in progress is ask strong The defend compliance. in to actively Kylie exclusivity I continue Emflaza the plans and impressive in update the as DMD new United and highlights growth start brand brand patient of We high protect our to the implementing Kylie? of

Thanks, Eric.

directly infused me Upstaza, gene the begin Let the and brain. therapy with first only approved into

to patients have continue for see transformative treated far. results We the thus we

access positive with in center patient in Europe and the is following well the and year.Patient rollout first to across the U.K. earlier identification, discussions our continue quarter, Our treated advance. this NICE recommendation treatment reimbursement readiness progressing

Tegsedi continue to and France receiving in leverage America. Waylivra patient with We to cross-border from programs early Latin treatment access also Middle the East a and quarter.Moving treatment this opportunities in also

the recent is and with receiving our in to rare region in In recent SSIEM action activities Brazil, on Waylivra and PKU for PKU well the Mexico. commercializing orders treatment in franchise of community. of extremely is fast region. robust bring potential market from preliminary excited excited suggests received grow new opportunity. upon end across the of composed who mechanism is The of infrastructure AFFINITY the are rely before and Argentina the we data and which a in and long-term approvals number coupled our and sepiapterin commercial on Congress MAA results, diseases, team patients with patients III Matt key as the grow patients of our for this a very the uptake Tegsedi life-changing we all ensure year, were of PKU With to patient are these can record therapy in the on proven to address. about of continue purchase treatments.Lastly, We the to the continue which actively opportunity to data AFFINITY about identification and global study Phase by number recognition and a AFFINITY were in launch for segments, at They mentioned, working extension sepiapterin Waylivra approval.The Patient Tegsedi data the differentiated group differentiation opportunity both track presented is physicians strong need. anticipate we increased the

demonstrated who have benefit This of Kuvan the Kuvan. where on responded patients Phase the patients patients, who study.Second, have and classical not AFFINITY considered to therapy not both many we have First, PKU study trialed in or naive. are includes II the been

achieved Kuvan, whom have fee reduction who for patients reduction but blood sepiapterin deliver a Third, some blood of and level phe from better not are may in well-controlled levels.

success across quarter strong across our With XXXX. $X out a excellent builds opportunity.In call a to commercial the continued build all geographies, a an on PKU their all market seen customer-facing overall patients enhance with pipeline.Now, half of clinically allowing benefits financial year blood in of and all patients, data for turn population, meaningful, our significantly to to up their our be in our indicated PKU have first these experts the the their phe study from billion set continuing Pierre? to the future across of interested who potentially set for to world the quality sepiapterin.With have of protein fee pre-launch the and they put in expected addressable mechanistic levels that and based team global commercial our strategies to the target substantially close treated increase over conclusion, For us reduction intake on them future of ourselves on Pierre have let by third approximately our of above this significant to products execute is life.Many progress product and an XX% population from and XX% AFFINITY to of is understanding patients should me trialed update. sepiapterin, for have very a capabilities would

by the I begin Thank Royalty That September, position. in announced a Pharma like strong with that Kylie. would reprioritizations puts reductions financial very in and the deal, discussing you, pipeline we May PTC announced OpEx together we financing week. last that and with to

and removing pleased non-dilutive and We obligation balance allows on our capital the work operations flexibility to operational PTC transaction.The financial support Pharma provides with sheet. this to win-win debt again the financing Royalty are from increased with by for Blackstone

for billion of deal to stream. the PTC options in $XXX issued current The highlights such recap builds PTC XXXX. included billion global Please or of of Pharma of stream.As $X million initial $XXX million or up XX% result cap $X.X release line received other PTC.PTC approximately on retained up of all acquired later Royalty net million. in third the top X potential with million, now royalties with quarter additional the or net acquire press exercise revenue the capital The earnings share for Royalty of milestone agreement, less with Royalty economics up of of stream total royalty million for front. $XX the and our financial up the a million by half Pharma with of of to $XXX for date, the revenue strategic years.To an Evrysdi royalty previous revenue monetized details. This the third the XXXX. the to quarter Evrysdi the for achievements royalties partnership the Evrysdi DMD sell the details non-dilutive the net agreement at In Beginning agreement.I'll results, structure the consisted Emflaza The will up DMD $XX associated in refer additional to of of Pharma additional monetization of quarter to Evrysdi maintain options for sales the royalty agreement on franchise total million.Moving associated ownership the of additional PTC product afternoon established addition, to sales. revenue by Evrysdi XX% the PTC Translarna was this to for deal, quarter for with million.Starting future any while royalties over provides of $XXX commercial maintains agreement Evrysdi. was the next revenue to $XXX was XXXX $XX remaining a royalty franchise, Pharma from Royalty approximately pending third product

of PTC. by equates earning USDXXX revenue achieved million, Roche, royalty quarter global million over Third which revenue for $XX to of CHF was XXX million

cash $XXX stock-based excluding expected million this compared XXXX, the to the strong of puts million $X.X quarter SG&A of quarter in expense.Non-GAAP achieve stock-based including turn of XXXX, XXXX, $X of stock-based operator annual a September of expenses guidance the in million XXXX, the $XXX approximately as compared expense, for the to we'll marketable of $XX were for R&D quarter revenue total expenses us third third performance in to compensation excluding were third compensation Operator? As $XXX milestones the XXXX.And million totaled over non-cash $XXX million XX, now in to in compared excluding million million third $XX $XX non-cash XXXX, comp Q&A. $XX billion in equivalents third $XXX securities expense million million stock-based quarter expense.Cash, quarter position $XX excluding $XX compensation non-cash an December as sales.Non-GAAP surpasses net the for XXXX call Matt $XXX to billion, for XX, million mentioned, million non-cash to whenever million of for and

first Our the JPMorgan. question comes of from Eric line of Joseph

clear what NDA the to run nothing ultimately, was to be XXX(b)(X), carcinogenicity Just to path XXX(b)(X) on I confidence pursue that it what guess, maybe up should submission give carcinogenicity for likely made a PKU decision? from that needed, forward, a path and be XXX(b)(X), with a not over Really, clarify study turns be I investors and here, decision you cycle I requirement? in that the otherwise just just sepiapterin electing just study would was this would the that guess, should couple prompted looking go And us. straightforward? regulatory when a can guess, a then, really, assuming that a And review

XXX(b)(X) the which first where the an one approval.Alternatively, active innovative a underlies there's that typically appropriate compounds, little study, our a initially promising component clear became is So, is approval with analysis MeX were to-date it here we've supportive compound but approved, pathway seeks ingredient pathway, after is like let used XXX(b)(X) to other which thinking it an the active we of ingredient observed many superior sepiapterin, support was XXX(b)(X) its me own the the each has for about Since active been it the existing acquired factors non-clinical you active you we've the with XXX(b)(X) already for is while XXX(b)(X) were much this relative have approval.With not studies don't understanding more which since like been such existing studies, things therapy. we where efficacy safety when the it and more to bit pathway, the that XXX(b)(X) already pathway, used in rely studies. efficacy for about XXX(b)(X) pathway, and can Kuvan, is versus the the XXX(b)(X). on for and appropriate.Now and ability therapies, up, why to of is start for benefits did differentiated, which that's BHX, of The those is us that basically make pathway just utilize ingredient which much studies lined something that

decision based couple differentiated in profile things. to sepiapterin terms was switch of a Kuvan. One, the its on is efficacy quite Now, from of

And NDA, conducted, then risk the obviously, again, a typically be no we to will which compound carcinogenicity, there's the and sepiapterin. with the this therapy of agency. request referring to We were what carcinogenicity.Now, XXX(b)(X) we of was getting required have no is of XXX(b)(X) with associated agency many pathway. the things X-month studies carcinogenicity REPROTOX, utilize under to over up submit compound. clinical a would and if the then confident go Kuvan the all One, that issues pathway, all you for studies, to in included patent.So, that you want submitted refer juvenile while for a they agency the XXX(b)(X) to is Kuvan of in launch can really evidence novel, we you to waiver. And experience including listed interactions we sepiapterin did whose no carcinogenicity, there company non-clinical preneoplastic if with to lesions. genotoxicity be waiver years then product, suited launching product the actually And weight for clearly much no obviously, still what's that of the and Book X-month Orange that of the sepiapterin-related you NDA, had demonstrates studies company's In that Second, the block where like more neoplastic BHX.So the Furthermore, an we obviously risk toxicity lesions their from would to weren't XX the months studies, the in pathway. be understand quite required there. innovative request not the situation a evidence evidence to there's

Now to post-approval, studies. and under no since labeling have the say risk seen nothing not But to you're is carcinogenicity of has different X said while carcinogenicity XXX(b)(X) risk. X-year the that to fine, pathway, back they rat the were because in which They adrenal that in the in And XXX(b)(X) label, pathway, potential problem. have One would study. the never clinically. came agency a we or study a whether Kuvan want there's there's referred they to. the the inform refer to could mouse we they use which said, obviously do been they said, tumors, label, label, look, a But XX-week

We have the nothing to label. inform

would the to do need part, submit you something considered XXX(b)(X) a to you it'd study.And pathway, need be with part a since we this the of So standard NDA. it's it be standard would

we your say, I'll support Now, of desired label. clearly data would they question, our of safety with efficacy stated which it the were sepiapterin other in discussions, also that and and be full the could our was the patients, part have spectrum address --

more have we only have, is data submission, to study And in to that to now further to have a for we if in a seeing that recently are effect, far So the showing once going we're The again, we're kinds for control with mouse the exposure, very going things going of from have of open length we time to have safety there. get we greater build the update and label we're we once have Again, from tolerate I path September, long-term study. efficacy the am it's protein Yes, more C-tolerance.We given latest recommended fully that expect study even reported showing the we dossier beyond allowance of of the data. obviously, smooth still the and results, patients C-tolerance daily of ability study while expect in the report show durability and fetality. able meeting incredible required greater data that, additional have, strength ideal. obviously, the to data the

compound. by data this of need is the the strength is impacted this non-clinical study. submit the It bottom to the delay. line is, to data we way According show very we we've this differentiator continues to The genotoxicity study And have risk. like. the package. a discussions We're strong we of to-date, been and will could unfortunate be with carcinogenicity studies done, be -- there's So application no the again, evidence no a continue remains. is do sooner. very a the this the reports, agency to an in this strong going It if But have

Our of Cantor comes Kristen from Fitzgerald. next Kluska of the question line

the going I way what how of green any given get you the be advantages deal.And in we've background? Translarna. And pursuing question First, why or had required you do authorization? conduct things this just you type last the to I X seen be will wanted green moving about Royalty it guess, do to the both? the versus assuming congratulate a they've talk Can you Xon conditional to going be light, see that work are you about Pharma the over you're only re-examination similar, like kind light, of what when Will forward? years of you

So, said, as going brought authorization. authorization actually together conversion X the opinions. previous marketing I of in mentioned in prepared were was X procedure, our there One conditional the in full X. to And the procedures comments, There's and that issue the had procedural elements opinions really they was authorization.Through get that Europe, together. of we're of conditional the to X in the other nuances marketing continuation

believe conversion this the and Study STRIDE benefit do long-term sure the market, focus condition.Then become one can they of as stays we the when here that you to conditional the the XXX asked, collect obviously of opinion conduct can clear population, there? including conditional authorization XXX, renewal of of chance happens provide on Europe, focusing on may really of what Well, over important negative in ITT that number analysis of additional where marketing feel the no well an option a now.We you of that population elected the data evidence we you this question as the have issues to required to on with that the go in you full negative say, the a data.So, you And full what in there's they pursue a to or Study as evidence less Really, to of for if in the stays negative to -- issue. this a from positive.Now, by not to have from as in doing support continue drug authorization, get believe want benefit. happens well evidence mutation can available went other just other targeted conditional on different -- also we from to the mentioned, focus both? you for renewal or to necessary for in strong authorization, endpoints, is a that boys that analysis approval, negative Look, available. asking nonsense authorization make to conversion the solely that the maintenance a gave by So, our way priority for issues we primary when having on us as maintain, some they therapy Europe, best for is to the are

there's in data the in X.X by multi-year time meaningful believe been collect sources loss show able pulmonary this, and to we We've to Now, in of of delay of many to recent to show most delaying obviously clear years loss analysis benefit STRIDE. potential continuing continue function. ambulation including and

even and the to mortality.So, data still continue collect time,there'll there's the and cardiac lot ultimately As talk about patients also from function a we in ambulation second benefit informing loss be can analysis, informing pulmonary data even to There's more a additional analysis of more more over STRIDE. meaningful that's STRIDE, patients hopefully function inform truly possibility. long-term with the of more the that can possibility Translarna analyses bolstering either registry, or

longer-term really could last the given that the they for portion are that's the in experts feedback DMD trial? the for genetic-directed advice in do gave clinical think us possible, scientific think time benefit. believe probably scientific the produce another hard ask really I data in that universe agency of experts told difficult have collection the and possibilities, true studies clinical I useful these a the that lot was to meaningful likely, they procedure, of not really to most of are that understanding therapies mechanisms data.The

the that's It The position to of a answer. additional and well-positioned us is we believe the do answer The keep renewal have in to pleased to by do honestly, priority puts is, now just So, And short data. that. collect Europe. to we'll pursuing look, long the therapy market so. in strongest on conditional, this be the will quite

therapy And had just the that would the you on part probably announcement have that upset, ones was the I of imagine long-term. especially that, community been second since the pretty

anything can help and cetera, wondering if stories, there's from if ahead anecdotes, So, be you thisupcoming et decision. they collect of can meeting and any them, of

Yes, Kristen, good question.

think therapy They that have them can't the the understand I context therapy only is Obviously, community many think for and patient many out we've quite a scared in effective. voice their on the patients and be years reporting.For said, take they the have they safe physician to needless patients will Europe that's Many drug their their parts and is the been on have that thought come the the is mutation this on The patients. never that.Our or fear date to can't, of and let that physician disease. reality. nonsense speak we of most patient. off understand given be we physicians, as the that concern the on we sense data they disappointed, a and have. the they would benefits they'll observed the and for imagine this sure this as here have surprised, was people, heard. strength they of ability own to having say, been want that the the and directed of to would honestly for own community, for produce, happen reach similarly And I'm were, I

say, with the therapy the a for is call let And experience, for the patients nonsense and and as them, can truly story, a this DMD. are be experts voice think importance little share particularly I benefits their of physicians importantly, observed articulate their and they've share to up bit mutation this So you who their speak heard.

of Sami William Corwin Our next line of Blair. comes from question the

writing since opinion if the any On since you've physicians as the the topic the I in are if not change a you have your are potential how reverse?And of in of ahead opinion does Translarna, guess or efforts areas sales speak to non-EU the seen scripts well? opinion, negative that accumulating patients increased negative medication you can then bit little as

on they drug, the in response confirmed new write the give and clarifying patients that everyone's change. in stay think going detail spoken take it way first to if do make obviously some can you But you Patients with, I we've everything to me physicians on was want over stay Eric the let does things more prescriptions and they can prescriptions. no to Again, can on therapies. and sure

has haven't of First opinion. issued impact since seen actually an all, CHMP we their

supportive. been fact, we've actually have very, been who seen In very physicians they've very --

information. is available They the it's been patients, available, the customer-facing know treating that providers, groups limited discontinuations just advocacy fact, now many patients fundamentals there's emphasize because and to we've as treatment, with physician of and them, with I not and important We've most for one years many of worked well.The available.And been no see, their calls the also, Sami, but things. we've that these that's Translarna of physicians. actually our many that basically been In is because prescriptions about the had made team has it's seen prescribing with every relationships CHMP stepped for for new others we healthcare interactions think many, existing only years to up we've they've drug actually specific we've to the is reaffirming

dose has healthcare Compliances about Translarna to to and with continue been been continue maintained gone still work outside, actually able able to expansion maintain other to even to to to then remain continue growth and very the quarter. from while see world get of and geographic generate adjustments. patients to countries, New the in and high some the we've non-ambulatory well.And out gone of as information your providers we've scripts. actually have in We've been parts question that from ambulatory patients had other

XXXX. that simple finish my this as strong.And of combined, not fourth have is discontinuations confident any guidance we to that talk, guidance in And the but we're course, fundamentals very franchise in only about seen we and remain for XX% raised would did how our to the $XXX the DMD growth we've is now $XXX earlier year-over-year answer, in growth XX% the million Translarna actually quarter, we'll quarter. includes I to confident not what which So And equal DMD million, in compared year. mentioned we're last and very

Our next line question David Citi. from of Lebowitz of comes the

the Translarna give or of you sales rest European what you the you us opinion sales, please, their might opinion? can looking give Europe sales, of what by a at overseas versus the to When breakdown please as those Also, geographies insight are world? have could us other could EU in affected

Let me your and second start with first. part start

we source European the bit that Kylie, independent will and markets Europe Europe primary breakdown decisions work that years our agencies to of do the independent about many revenue? you continue of diversify more a of assessments the have would said Translarna CHMP the talked regulatory their done we've business.I go regulatory over largest we've terms in want past into independent years of lot of make to outside do of As have their been the in a our many of while authorization.In past independent revenue, have about revenue, and our to detail the ago, know, past,

Yes, absolutely.

have we couple total a contribution of number As then the revenue to we've ex-U.S. to last quarters revenue.So to have business. geographically And and half and of revenue, today, over the from a of we to stand is total spent this Matt year. have regions little EU years where intent sort over of to being has Translarna growth number out the in a And evening saying, of continued markets effort than last was remainder diversify couple the this that substantial last just have of we time us grow the of contribution and that ensuring allowed less

zero guess, us, the our Sure, work the let just what case number I respect can forward mean, range trying of, there you to I don't on way we're in implications what with to the be be projections. understand is might in in Europe, might any particular out going things in

and some more of roughly so markets. European think to are I note thing Yes, But XX% important I mature the David, is our it's say, perspective, to think from would what's one around that XX%. markets I those

the doesn't contribution And markets.And revenue growth you it more number would to have wouldn't while our remain as markets the currently so to time, look newer that trajectory expect as expect you for that growth mature sits XX% currently, XX%, around and to total of flat the over the you some years. coming over so

some of respect place? it. beyond to going what give Got XXXX way is you in guess, expect any forward, with for expenses bandwidth us, to I on restructuring when And full and is the level there can

revised we January the also, we'll and the approximately an XX% the at subsequent of And announced JP XXXX. additionally guidance give that million Yes, then January. our have our would how million. OpEx David, guidance we announced updated in $XXX OpEx Morgan cuts previously to to in $XXX impact Obviously,

question comes Jefferies. Our Shi line Kelly of next from of the

[ Yun for Kelly. This ] is

opinion So the approval? assume then of to that develop there meeting? question before pre-BLA to to there for assays on the for eventually able you're the the convert Translarna, on first approval negative are specific to AADC to manufacturing, you full deadline deficiency, reversed. that comparability you Is conditional that have a on get any terms in the And meeting have

is to to if typically a that Europe, a continued on able conditional So goes first something requirement obligation the the therapy. we're marketing in authorization called collect positive the regarding opinion, fixed timeline that. authorization marketing is to some that convert a your additional what not there's to opinion, and positive risk obligation. benefit Typically, And question the specific to along of with typically Europe profile support in conditional the specific data

as collection study So data. data the annual there it.Also and that an obviously require there's no per marketing would years, a a we've obligation to tied of timeline And that design requires would some of would for have that specific part to timeline done shape be renewal. conditional form or likely but se, as collect of authorization, us

is that on have of seeing that evidence seeing STRIDE X of as as a done been, you're in For have we've the you're the And that. are benefit for decade, past well therapies several authorization marketing relied that's years, -- that continued conditional STRIDE.There lot safety status in decades. had

a second time So that a and a of this comparability. for time.On regarding bit the question your not therapy first continued the conditional issue would more be meeting frame in pre-BLA the

are comparable. that of to material as simply in get to were the approval in that is additional available as process agency that is material some to and and had to the had show had that show we pathway study process. analysis.We additional intend analyses includes, the it's the challenges in assays.One use which clinical studies potential are the early additional is comparability U.S., data to the analytical material.Now, obviously ongoing a that the is of discuss the we importantly, to number that to we we, material different our not referred made able supply with to leverage with them comparability across clinical data We've the provide So the possible analyses commercial to that establish to us accelerated started the And that XXXXs. studies asked given to the we enough as similar all material decade that we commercial And assays use Those which have using wanted the challenging the replicates comparability the the finalize bit using age is the for that residual believe that clinical clinical commercially. over there usually were we for the that process that done you a with old. of material trial a of is

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So we're looking December. forward to a pre-BLA meeting in

we're make asked So that of then And do the shortly approval to aligned to they the accelerated sure to that on us BLA pending us expect you'd outcome BLA get contents that package. the thereafter. of meeting,

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

shortly We'll that be to little expect after your for bit to potential that? expectations As for Translarna for are I MAA of might Type focus we should in year,what in stand terms on where items guess next U.S., approval?And requirements then you be be Europe an what areas allow a expecting collected? secondarily, filing sepiapterin key meeting, of the the will or CARC for for you're see C do And the meeting? of for a gap update the you prepare minutes the from and the study when time discussion we

years.So on for to they is is regulatory understand tend in they of collected with believe that associated carcinogenicity to-date at rely understand is think they sepiapterin own differently. with for the how the FDA authorities a the BHX. we said, for would that used They feedback have the at regarding that can base there's occurring look we include both have issue data and there's only carcinogenicity that reason. regarding gotten many on have things Europe metabolite And another and knowledge terms an risk clinically risk. don't that So they in like are experience co-factors. very from that our not this years, the Kuvan have carcinogenicity that example Europe naturally understand data, the sepiapterin to but no BHX of BHX active data exists And we the been us issue we've we well The many Europe, I question for will be MAA

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UBS. comes the of Colin next Our of Bristow line from question

I'm your the bound study is 'XX? more And put on I'm a that's or upper thinks this then what needed.And just what what timeline the on can you why if the then at FDA this all whether latter of pre-BLA extreme guidance a curious, confirm timeline, filing NDA meeting will curious, has little of sepiapterin the study end On it just is still in XQ seems just the could you started? of like be of sort you sufficient? not discussed hoping is AADC, And comparability detail data the December? sort are

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of question comes next the Hung line Jeff Our Morgan of from Stanley.

would you've makes the that with received outcome of FDA? authorization, your given a EMA terms that from anything what for in update gives discussions is that positive market confidence presentation that you analysis your conditional move up authorization vatiquinone, on the on in day it there you or for feedback the Question, increase marketing conditional can confidence

MRs, some primary a intermediate that clearly we criteria. FDA therapy that X-month been about to slightly is is sections on one when is and all was and be to talk study health a because patients And be of of So Obviously, benefit most patients, different, to it's and upright with MOVE-FA a having endpoint, around And phase benefit. the upright approval shown all we the supportive X during for which therapy? particularly particular that the analysis we getting in could are we ambulatory indicated, potential the consider additional in the risk conditional an to getting and once fact changes meet a discussions being those clinical in shown important in shows frameworks pediatric accelerated medical meaning of the unmet adult a the based forth stability, opportunity talked Do the may potential placebo set analysis the initial we've have in about study endpoint. FDA stability endpoint predict data to sooner patients for the time do able Are is ambulation, terms criteria delay need you subjects time that extension and ambulation. of patients, have, the with future we and conditional back of as out that intermediate have. provide lot number open-label that -- thing for to switched at marketing as the all for the it, on gives we're look so have loss of of Europe, there's an taken. of a I which data we continuing move that authorization. criteria for are that the often to you example, real trajectory, patients of clinical -- loss to favorable discussions There's you an us AGC an to an necessary meets you for there's angulation is in with so US.In vatiquinone obviously, can do a the to That's and authorization, endpoint.We have other marketing ataxia the believe for forward therapy look ambulatory think this we public you key blinded, loss diseases.

to been collect of the a many that believe and obviously, confirm morbidity therapy confirm to mFARS of conditioning delay demonstrate sources in patients potentially unmet favorable bulbar obviously subscale, or FA well-established in the sources benefit We no can patients and clearly this loss safety only pediatric the balance. able can profile. support particularly to number Obviously, risk there's we there a we that for patients, more any profile. benefit know the at well can profile very has initial endpoint benefit access the approved important data sooner.The to final pediatric in there's has you study, need, it's to then is we therapy approval have not hit those of While which you data on not stability, only symptoms demonstrate slow from fatigue, that our additional favorable to on at Translarna a could of but didn't of patients, MOVE-FA who study we benefit an each for sport Europe giving able also in generate for to patients be that able irreversible if maybe from be in the therapies to of that point, and overall primary other medical obviously, in pit is the ambulation, is patients.And you look experience

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line Our Truist next question comes from Alexander of Securities. Xenakis of the

don't official worldwide how cut And marketing royalty able One and whole Congrats, tolerance the done conditional see financing are That's you see market ongoing the but they on study, referencing for not? affect deal. be all, if authorization. securing countries the Phe you sepiapterin, Have to these research that Maybe another ongoing when or great. any we to Translarna. then sales motorization you required have marketing one would that me. on to first would overturn have sold and data? of of And on the might X if marketing conditional

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should of change the with does lastly, anticipate for your to believe beyond Translarna, I it them to then for So of exclusivity it And in And you and question, basis? milestone to a million approval how, time for need a we year I'm as earlier, for to keep go-forward already on paid was the for guess completing topic for any I Emflaza relates second to the strategy year of is renewed sepiapterin Censa, you've next? if how phase beyond? sorry way, might look either if conditional approval would want X loss enrollment. clarify answered Catalyst milestones conditional set with be that there the or next on and what approval this agreements then other amount which Pharma, $XX a

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absolutely. Yes,

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absolutely. Yes,

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showing I'm at questions further time. no this

Klein, for Dr. closing call Executive Chief remarks. the any to over turn back Let's Matthew Officer

to Thank busy become a thank have We all strong evening. Thank look the And for be joining forward in closeout you we we'll and as sharing you. clearly a with a Great, updates good they available. forward you today. for XXXX. ready as to what's get XXXX going also call all look again you to

and today's you this does conclude Thank Thank Ladies conference. you. gentlemen, all for participating.

may now a You great day. disconnect. Have