PTC Therapeutics (PTCT) 25 Apr 242024 Q1 Earnings call transcript

Company Profile

Participants

Jane Hanlon	executive
Matthew Klein	executive
Eric Pauwels	executive
Kylie O'Keefe	executive
Pierre Gravier	executive
Kristen Kluska	analyst
Eric Joseph	analyst
Dingding Shi	analyst
Samantha Corwin	analyst
Joseph Thome	analyst
Huidong Wang	analyst
David Lebowitz	analyst
Danielle Brill	analyst
Tazeen Ahmad	analyst
Kyuwon Choi	analyst

Call transcript

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Operator

Good day, and thank you for standing by. Welcome to PTC First 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.

Jane Hanlon

you Quarter us Financial XXXX thank PTC to Results. and First Therapeutics Corporate afternoon, for today joining discuss Good Update and I'm Officer, our by joined Chief today Dr. Executive Chief Business Officer, Financial Gravier. Chief Klein, our Pierre Matthew Officer, O'Keefe; and Commercial Eric our Kylie Pauwels; Officer, Chief our will call statements our current based Today's on forward-looking expectations. include in statements. moment call, which the to a posted take with the slide on our conjunction forward-looking our Please review Investor Relations website contains

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

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

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

With call that, over Matthew let Matt? Dr. CEO, me Klein. to our pass the

Matthew Klein

on development Good Jane. results the to progress you, pleased I'm our afternoon, with joining an you our all you call. provide update quarter to of share XXXX and and programs. first financial for thank the Thank

be As and a number execution value-inflecting year. a we of milestones XXXX have for discussed, will important of year PTC, with throughout the

and milestones. revenue of all achievement clinical with of and off first are strong great planned quarter a We regulatory start to the

entire had We portfolio. performance. commercial quarter. by across with great commercial Starting driven execution a the

DMD First was $XXX quarter revenue million. franchise and revenue $XXX million totaled

will CHMP that during Translarna European to additional thus, opinion shortly. time this provide the will Kylie details we Europe. revenue in time, across has authorization. adopted resulted continue from the revenue that The Europe. And Eric our team's on performance withdraw the and authorized yet commercial our not to commercialize DMD Translarna first we to ensure At efforts quarter Translarna Commission remains optimize

quarter. of the important regulatory achievements a first in had number We

submitted and bring for we European marketing patients schedule global to we standard towards sepiapterin sepiapterin potential the the around for the EMA the planned, NDA a an submission no MAA As step of third The sepiapterin important to than on as of remain submit launch the our planned we new later application care globe. is authorization to quarter. PKU

by the based As of generated emphasized liberalization. the majority of to PKU reported PKU PKU vast served data is recently date, deal great Director in the patients by there not on are Milberg, available the Lisa experience currently Executive of enthusiasm the the therapies, in sepiapterin including for National the and a community diet PKU Alliance, patient

placebo-controlled from NDA The data, confirmed resubmission of In predicataxian. also submitted for we which quarter, of first BLA slowing for on based Translarna the an the Translarna ambulation. XX we for vatiquinone resubmission submission Study will for benefits the FDA long-term and and treatment the results time Translarna and data from with be the loss of registry on to NDA an first aligned and NDA STRIDE

results from include the XX-week data the trial, with mid-XXXX. the MOVE-FA will long-term extension. along trials We The those vatiquinone expect portion submit to placebo-controlled in open-label NDA NDA of from

submit XXXX. late expect in We NDA to this

and PTCXXX plan XX the programs. update cohort subjects whom to in interim Turning initial the results the data we of summer. results will quarter. shared share study PIVOT-HD data disease We include clinical development This approximately from to our last of second in biomarker from patients Huntington's on XX-month

including biomarkers CSF protein score, signals lowering, data understand endpoints. blood functional Huntington allow we tolerability as the several of on volumetric on Huntington identify as well changes mutant profile motor PTCXXX data total will data Specifically, for and MRI favorable the XX-month on early disease These levels CUHDRS. as lowering, scores to will longer-term SL well protein CNS as share and and us total capacity clinical to safety activity clinical of

for of XX-week update quarter Huntington include larger a second blood number also changes. Stage X X will protein The results subjects, and including Stage

the ALS highly of to report compound enrollment registration-directed of patients trial in is Lastly, in on to the year. pathology. be fourth demonstrated and we targets schedule ALS remain in utreloxastat stress a cell relevant of specifically paraptosis, and results the ALS for death this Utreloxastat cardinal top completed developed being quarter first to line pathway that oxadata

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

first schedule in to in of achieved proud and am the achieve planned the I of team's closing, all many objectives our XXXX. our We In important milestones quarter. on execution remain expected

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

Eric Pauwels

Matt. Thanks,

Our global strong XXXX off footing delivered $XXX has kicked our customer-facing marketed products. revenue team on in X and million has for a

on for Upstaza focused is on commercial globally. sepiapterin U.S. growth as within launch executing preparations team the Our in diversification our and portfolio well and as current

our DMD global in while and North a franchise in had to progress geographic quarter, Africa, expansion markets America growth Asia and the Our East future and region. our continues Latin Middle the robust Pacific

the resulted delivered to strategies to U.S. Europe to which in $XXX Translarna maximize of from protect Emflaza revenue and the continue our business successfully revenue We million, in

pathways ongoing. receive that continued this local patients treatment. are work Europe continue team we revenue treatment of named Translarna access sales $XXX to For to Translarna, achieved to ensure quarter. for to and continued country Evaluations patient million The in in

by new patient loyalty to patients significant with million, net which number the revenue continued Emflaza reflects a starts of of was Now generic face protect $XX health in the strategy our brand and were Robust from providers brand on EMFLAZA. Quarterly to entry. initial care turning Emflaza. sales driven

with advocacy for Emflaza care DMD work groups patients. payers, pharmacies X- to and the exclusivity providers, X-year-old the closely and We reinforce benefits continue to while health specialty to our of reemphasizing value

I product of current our launches. update progress Kylie? future Kylie ask to will Now and the new

Kylie O'Keefe

Thanks, Eric.

to recent and submission the of the as additional in later following and XXXX. well We sepiapterin regulatory submissions FDA in as Japan EU the this the year submission continue plan planned our for of in Brazil NDA global to MAA launch the

stated, Alliance and is significant amongst unmet There of these specialists, recently As for the the patients to National sepiapterin metabolic geneticist, of recognition the significant PKU patients. dieticians there Lisa of Milberg, needs. unmet widespread is PKU meet Executive a PKU potential Director need

we Kuvan from on saw unresponsive numerous significantly patients As those better well in trial, potentially AFFINITY patients the as on PKU do sepiapterin. and as controlled classical to could

extension for see continue phenylalanine control the patients levels treatment we the recommended of to their sepiapterin daily still Importantly, long-term effect the ongoing protein on intake maintaining durability dietary and in increase to allowance while ability of beyond study. affinity

have burdensome for a patients have PKU quality the that their reduction waiting restricted like to improve combines to sepiapterin potential groups through been advocacy around a shared liberalize patient of world life. diet incredibly fee and that their ability therapy PKU efficacy

continue We believe opportunity. to in potential the billion plus $X global

Upstaza. turning Now to

and gene where continue brain into directly results. only we approved to transformative infused The the therapy first see

treating we the the As In the for administration U.S. HVA in quarter. the to needed new readiness U.K. as the from into and team this of new X common identification, the filings up that weeks our Europe, hypotonia. to and as study in FDA customer-facing underway, as we is treated access and treatment production advance patients U.S. prepare data in after February, cross-border Upstaza, launch we the well as presented additional to symptoms incredibly discussions In Matt mentioned, submitted was improvements excited dopamine patients and to and patients and AADC BLA of reimbursement treatment are France at the well IAS Congress in regulatory approvals. March in preparations bring F-Dopa to PMD such Globally, showed GT-XXX center translating CSF, patient in significant continue uptake and and much

in Latin Moving and to Tegsedi Waylivra America.

growth both and across order across the the shortly. for purchase patients for identified excellent this In franchise anticipate receiving group make to group and we region. treated in completed a order continue Tegsedi new Waylivra, delivery with We progress Brazil, purchase of patients

Our strategy regulatory both anticipated expansion additional continues for geographical products. approvals planned for filings and with

set first a and portfolio continue for Upstaza. off XXXX will are geographies global the diversify across coming well financial conclusion, strong deliver Pierre call trajectory launch to and our sepiapterin over now quarter, a update. turn we execute for as our to for and have and our to strategies well to a Pierre? as positioned In I robust

Pierre Gravier

quarter for DMD additional afternoon Kylie. financial the of share Thank first now this refer results. highlights of top million, million. Beginning Please was including first XXXX. $XXX to issued with I'll of our details. line quarter you, press franchise revenue for release $XXX earnings the revenue the Total

franchise. DMD million. net in quarter million, while the Translarna the Starting of $XX net product was revenue revenue Emflaza product with $XXX

The the for reduction in $XX its quarter and of R&D Roche, Moving as first XXXX, achieved million global earning stock-based high the XXXX, compensation in to first to focus $XXX First prioritization composition was million differentiated expenses about Non-GAAP expense $X million of R&D noncash resources expense. compared revenue to million excluding royalty year-over-year million portfolio reflects strategic USD for in continues XXX for programs. the $XXX potential million of its quarter expense by revenue our noncash of was excluding company $XX R&D quarter stock-based PTT.

totaled equivalents noncash Non-GAAP strong the million the anticipated SG&A for expense, in sheet call costs a $XX several XX, the years, strategy compared to of stock-based expense $XX reflects million reduction of as in and XXXX, XX, of expense $XXX to with compared stock-based quarter to reduction million the operator turn the balance on noncash will first execute to over sepiapterin And lower over securities compensation workforce. milestones was our $XX I provides XXXX, launch. marketable This The for cash our of the Q&A. excluding XXXX, Cash, expense. compensation $XXX quarter December million Operator? million excluding million to achieve first $X the for March now including in the the PTC employee XXXX. of next resources and result

Operator

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

Kristen Kluska

unmet specific there's you'll being us about On length where think initially, at multiple see highest if market uptake need where But of the remains. specific segment PKU, gone you've segments I you the times wondering, a with talking I'm strongest. know

XX, weren't to critical So actions one thing a market our in under initiating is we example, really that for be

Matthew Klein

thank the very much Kristen, you for question.

we the including a we position as opportunity. about the long-term bit see and us to detail that have set the sort this really data we're all large of both billion-plus what really segments. opportunity data We lot a into as I'll key a talked to a why as And we address how go strong AFFINITY penetrate. that's about tolerance segments those well about the as let see well of being talked extension, Kylie initial market from such more from little $X positioned

Kylie O'Keefe

Christian, the Yes. for Thanks, question. Matt, thanks, and

to going an think immediately a segments. are different post near I the key quickly. be think all to I number at across I penetrate as Matt looking term, And highlighted, segments of the there opportunity think launch, truly is in we

I means to a have failed PKU, opportunities, that at think one potentially then, be those allows and of we includes Kuvan diet, talked a really [indiscernible] highlighted. We the to about liberalizing past have we've I to the the that KOLs is on Kuvan also have their able that need, so poorly to of of think look could the their unmet high dive on truly controlled. Phe patients previously heard deeper we've that did that last as patients them And those in classical and reduction that's this because past, year. something have ability important. Kristen in importance we've in also and reduction highlight KOLs that talked also Phe therapy as this, naive, deep are medical But levels that even the since about heard those other highlight deepen number on

sort the the billion. believe segment, of hard will to to particular it's achieve segments able a near but be term, that's at key we're why pinpoint So in looking and of sort we those we $X

Kristen Kluska

at you as remember upcoming Japan reasons, deep one already nice about what and regions you've other your a patients decisions? don't and around to the I but key you the of where available, Brazil Europe dive, I'm as map some about as had identified available that therapies And Okay. either those filings there's respond think talked of and they whatever well done wondering for or were work that

Kylie O'Keefe

different regions. Yes, absolutely. We've the done across consistent all work

As existing have infrastructure we've place go. and about, teams are to commercial in talked ready we those

upon treatment getting patient excellence, ready They're to there. communities the to execute out of they're and know be upon centers patient So they're the launch, they'll those segments. visiting

that's information of what collecting. substantial different similar being the we And to So that, across in all that's But also have we're there's collected addition market markets. a pull.

drug. coming people and PTC there's up So to wanting gearing the

directions. both from So coming it's

Operator

JPMorgan. with of . Our the comes next Eric question line from Joseph

Eric Joseph

up reconsidering his expecting on perhaps in opening might any have when potential pending update for picking you were the month. following sort with CHMP's recommendation sequential authorized, with as how to should whether progress of visibility up Just performance FDA? to And And If be that. of Translarna. then your the guess on extent light maybe a we advanced to quarter? decision? comments in Commission European Do regulatory in earlier saw second perhaps quarter decision I about last thinking you EC the on remains Translarna come the pull-through they're we the just

Matthew Klein

much very the for Thanks question, Eric.

business as in withdrawn. would about the usual be Translarna right? opinion opinion commercializing adopt remain talked that has That XX as European in of January, have that obvious, Translarna highlighted that some would opinion typically, days You happened. not fully Europe. terms it as the authorized Commission With CHMP would We we been in and the following

make that have to little community, a efficacy what the leading to until what considering considering commission we performance was in we're significant Translarna unprecedented the withdraw Obviously, And as are as we'll And very long a Translarna be that and we talked bit be discuss surprising the data for to look, on to physician week of business Translarna it it impact about, opportunity the terms be a terms lot expected that, the has would need bring in we mutation we've to while this looking therapy quarter your record team to this in of We of look patients revenue to to patients a it said available we sure be and was safety for hear, on which at forward again the matter been live by the that many here. the do fact in Translarna authorized. possible. the continue that as to see can. in patient usual tremendous But the commission medical as Obviously, or update patients of Europe, Europe of not first strong second the in alcom revenue, may they in we and we'll an as closely. situation was question, wait further is unmet working based on based meeting. -- would that notified continue a and

and quarter place number continue. on in a As to protect the protect Emflaza Eric guidance and had mentioned call, for our if need protect to we business. will the U.S. we of will. through revenue strategies strategies that brand the there is this Translarna in and a Those update will The efforts continue

Operator

of comes with from question Next line Kelly Shi Jefferies. the

Dingding Shi

ALS associated prominent proptosis, mentioned of do inhibiting the in . of subgroup I'm a MOA a you the curious proptosis with wonder ALS is kind what ALS outcome for trial, in through genetic biomarker artruloxastat more expect patients? patients? of you And treatment

Matthew Klein

recently being cell And stress described diseases, neurodegenerative cell of, is what that thank relatively increasingly related a why about death it's Kelly, linked is very genetic in as to ALS. including is is ALS. for idiopathic patients. to pathway genetic and question. Theraposis much our pathway enrolled the both not with a a you And important uteloxistatis specific any common really response and it's trial this ALS that's inflammation, pathway

nongenetic or targeting any of this So one advantage approach is common cause genetic we're to objectively ALS. this that is of

Operator

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

Samantha Corwin

from the view U.S.? contributed of potential perception was any patients, on commercial et [indiscernible] this might of the Translarna U.S. I any cetera, and data what kind Translarna heard could additional be wondering on provide have the the their color you And with of and their much quarter? then physicians, how in if you to the revenue EU feedback

Matthew Klein

patients give And Thank very just for more for about and of much a DMD means I'll for second been with it pediatric Translarna known therefore, in unmet there U.S. significant there's and you of brief for physician turn community to over as terms well for data Translarna a your years, for stability a patients to I'll expert the for of amount you significant questions. potential that of important for there's little and patients the there's Kylie, turn not meaningful in a that on of is that over for anticipation and FDA loss delaying then bit we've the vatiquinone Translarna Certainly, patient patients, the ambulation on remaining detail. the the great make amount availability well approval. revenue. is interest given number need in comment, similarly what's more an in a vatiquinone collected question, I'll a unmet on Kylie of in it demonstrating in that it's in need the appreciated to the community. effect color incredibly community really as and what MUF have significant time

Kylie O'Keefe

for Yes. thanks, question. and the Thanks, Matt,

I around year. quarter think revenue, pretty consistent at you consistent starting that first Translarna, your the of look the last shared to with contribution year XX% with from European last around for pretty of it's We've it's represents it of of QX when Sami, QX and first total revenue year. this question,

the the have market. Translarna comments, Matt access So remains in while continue in an going upon it the incredible that job ensure plan. patients to executing to as earlier plan team into quarter his in to has And was I said done think the

in U.S. your and So contribution the perception around consistent. vatiquinone and to there Jumping FA. also second question is about Translarna in

absolutely accurate. think what Matt said I is

for mutation DMD nothing patients. think I still starting with there's Translarna, nonsense available

get think in I high older as those a in And And for still look a [indiscernible] older the marketplace there's fits FA, so to you medical patients pediatric there's for vatiquinone then And well. understand place how at there's physicians available the when patients. are there. And need to into unmet starting patients. nothing

So the we've versed positive seen Translarna well board, on Physicians in feedback. very U.S. across very are the

patients drug. are there, the on And then the as a we're there's too. positive FA, with there's that As community said, coming on feedback number that of engaging

Operator

question Thome from line next TD of the come Cowen. Joseph Our with

Joseph Thome

long what measures, like the Huntington CUHDRS. going maybe Looks that measures, time be enough on interpret do study. seeing TFC to show expectations we're on a these to on that the QX bit question, for to AADC this see those you should Maybe maybe the benefit of the in think? we you apply talk the on Maybe your functional some be therapy. can points, might little second Maybe just them? time committing TMS, the data and those Europe? follow-up we when what how those you data, first progress really Or about efficacy we're gene learning And are U.S.? the to there in you are reimbursement Can

Matthew Klein

the Joe. question, for Thanks

We much the are to later looking second HC quarter. very data forward the in readout

our glimpse be As as a functional to get talked at that CSF, to, summer. this Huntington effect, pointed you of total for will the protein NFL the And XX-week at total we opportunity capacity. levels, will on we including and levels scales, brain first the whom XX-month this us patients motor volumes, score, allow share CNS from time, and in looking biomarker out, about, initial share clinical data last group data

or see home the and data of and CNS to be I run able given signal given think this XX the then And tolerability see scales. for XX after number biomarkers really of whether standpoint, And of then said a from that's some months here, signal XX approximately we the the months. safety I on to think would patients. be our is activity, able patients, be we're duration a small to on trend continued of any the on or clinical a looking

AEC and/or you really let U.S. And CNS setting to turn that's how scores do looking give And biomarker success at either how it us signal Kylie, think the tolerability here. the so that having over reimbursement I and activity, we're for stage Safety more some we're that's clinical on color patients. me on to want

Kylie O'Keefe

absolutely. Yes,

place those with progress see agreements additional favorable pricing Europe, reimbursement be to continued have continue and across come we've shortly we treating countries. and into in to we'll patients and negotiations So

quarter, the in As additional -- and the we we talked in in in France, treated care. about additional patients health U.K. through patients cross-border

space. And that's continued success this so in

of and of is in with things unmet you the from sort budget of about standard I recognition think a AADC available. we treatment small and there's Upstaza. of market, an how need learnings that care one some U.S. the of If and view, come no results the away There's high think treatments there's a disease-modifying we've from absence condition recognition with discussions of that talk recognition a and of of transformative of walked to point the the of with impact ultra-orphan a these therefore,

we expect so upon forward able And us together, this do U.S. be to into carry the to and with allows launch. negotiations. put pricing to across that move favorable reimbursement And

Operator

next will line from of the Abrahams come Our RBC. Brian with question

Unknown Analyst

This is John on Brian. for

you can the about European If withdrawal may than follow Translarna, suggest the Commission on the talk buying back seeing side, you're the on Translarna ground channels more or does in more on you opinion, more gradual anything might So pattern that orphan weeks? recent for even are in that CHMP expecting? rapid be Europe seeing decide from you're to

Matthew Klein

business it continue take expect is numbers be physicians to are the that I advantage Yes. usual. to Translarna are here. And and as day the and on question, that we market, of the unchanged Their to as Look, patients Thanks for goes case. prescriptions we really every Joe. want saying said, that fully the seeing, and really the without

Kylie Our teams, pointed ensuring focused that as happens. on out, are

that ensuring therapy. we're So to access patients can

event the course, to might to of the in mechanisms we've to adopts opinion mechanisms leverage Commission in working been the to in be country-by-country the also background provide that a continue that Translarna EC place Of Translarna available basis on put

Unknown Analyst

I more. regarding to on if with the in carcinogen get you visibility a the soon? you Just not? filing wondering parallel it. meet Are FDA study If squeeze you or the file could Got will to whether anytime one can scheduled

Matthew Klein

to of last year NDA fall, and agency. don't that last submission matter with submit efficacy we meeting to pre-NDA there have carcinogenic we clear the We that -- formal part other package be of say it Joe, done with And is we We the the data did contact We discuss We've that that a expect on had to of the maybe was meeting. was remain QX that in relative review. that safety track Yes. still and that support expect plan approval on the bring and June. support the QX, but that the in study we meeting to that had into that expect to timing study. that in we will completion said them would formal every of our place, completion on in the mentioned be very a one study. we agency We little But don't based outstanding early to correspondence. a

Operator

line Our of with next Wang from question the comes Barclays.

Huidong Wang

have questions. just quick I very

in is then Europe? The do be on when regarding first withdrawn regarding you one is PKU. question Translarna Translarna sepiapterin Europe your And expect Based second with status. will in EMA, discussion

to that is one how our have mentioned usually does you patient that population a you PKU. part classic across the sepiapterin the understand target PKU us any you precursor cofactor help initial think understanding I Can a and hydroxylase. be not wanted is could that

how patient will does classic basically in have like be the population? not would enzyme So PKU an helpful sepiapterin

Matthew Klein

question, the Gena. for Thank you

meet nonsense mutation remaining therapies Europe, And April the European status to days. the intact in with of physician clinic As efficacy current had sitting late we've we're the status safety that was Commission would first have they're supports the support and DMD, here the opinion in regarding commission matter. notify typically, the from on for discussed the in Translarna this Clearly, Europe, that the across not within opinion lack into I of planning the XX taking and January, to that and of Europe. patients Translarna alternative the adopted patient We to occurred. has outpouring community -- consideration week authorization question adoption the in discuss which in assume the for your data of in following the

So matter closely. I think they're looking the at

no have the enzyme, of and that life We to On X,XXX or Sepiapterin function. no children micromolar the have some their going the prevents second and is, severely who therefore, small That is is from classical therefore, functional studies PAH means PKU of day a also time, expect Translarna. them sometimes they there's a phenylalanine mutation and phenylalanine remainder BHX, be adults level what every you in fact. the mutation have which said, as very control. of it's greater we at to precursor Obviously, that if basically stabilizes future. of called misunderstanding affected on has point diet your to update using pointed And we're to we A have ensure why the enzyme. PKU, as having usual, definition I classical seen But classical a They're patients, in per continue has could an those a hydroxylase to at both the the allele And access patients classical time we've get function, for have that business by lower, think enzyme. which they any our which near that regarding seeing that number be question the patients. a documented in And believe have patients confirmation until enzyme. as than is PKU that's they what's a no a second point the homozygous which very mutation. minority of patients liter. is is It the of cofactor in a in of but function, significant small chaperone patients PKU

actually of trial AFFINITY production mean phenylalanine a reduction PKU in the of In subset was the we in than fact, the observed of had greater population. classical a that overall magnitude in patients, XX%, is

phenylalanine. included sepiapterin seen able mineralize classical that signifying as can being diet open label an the in we've we've patients participate of and role to the -- these and extension patients defined to PKU important that tolerance certainly, Phe maintain as patients. So patients their going play is PKU control for protocol, Similarly, classical

of to classical really and patients important most speaks that all have patients a the severe. of including potentially role who PKU, all tend So to sepiapterin really with the importance those to be

Operator

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

Unknown Analyst

you lines given into can sepiapterin, you are patients what just like or years of phenylalanine, us the give there to for would For you plans collected remind And the meaningful patients? effects for that? results And on then under data X translate old? that's younger those confidence time starting in

Matthew Klein

AFFINITY Thanks, X the include Jeff. did in study. We patients under

than in phase reduction run-in Now and to they study. initial patients randomized, authorities the to open-label regulatory through the into went the a directly the had run-in had wanted phase. XX% extension we be but greater those But go patients not who

have actually enrolled we directly had in were into long-term started X, we've we've who So additional under who patients that AFFINITY patients extension. and

So getting and over age under age number the what of And of of efficacy the patients what is a phenylalanine. of we're patients also X. X. seeing in data tolerability We're exactly safety terms seeing we're in and we're seeing reducing in

patients, to of potential full So PKU data again, sepiapterin ages. classical patients the range and of nonclassical of and all the both benefit in the support continues then

Operator

of Leerink next question with Our Partners. Schwartz comes Joseph line from the

Unknown Analyst

for more wondering readout was This little a Joe. Jenny about could you for ALS. XQ I bit just is talk if on the

clinically give I think kind insight give could the be? Can past to meaningful study some would change that And functional scale what you in us for that? be will an support in idea regulators of of a registrational. looking ALS into you the said you've rating can what us be

Matthew Klein

rather. the much very you Thank for question

endpoint having endpoint, difference on been that and of registration are X.X we study meaningful study is primary which to threshold with a study the to controlled ALSFRS the well ALSFRS designed treatment that. between we which be effect, scale, a the placebo the to point study would agency has And and and detect to the study, we We powered being for the the the you approval. clinically powered being X-month what success here have traditionally relation FDA, both said, be significant the this placebo roughly the group. as believe CARDINAL be, the the define designed our We in came So from primary strategy, endpoint terms scale. on roughly direct, feedback of benefit as statistically

ALSFRS. believe to have the if significance we able statistical on we're So

We other to including believe that mortality, where secondary in look will scale, respiratory that position we, course, approval also, advance have of disease, capturing of for U.S. for function, will the data ALSFRS course, important of supportive aspects us the we including endpoints of

Operator

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

David Lebowitz

point, of a this or recently really felt To have Emflaza you the approved of presence of generic significantly

Matthew Klein

David, much you question. the thank for very

talk of little a about we bit to dynamics. market, the in the talked seeing we strategies the put Kylie Emflaza competition, more preserve what a of we're As about call, terms in bit even in the a lot place on

Kylie O'Keefe

Yes, absolutely.

strategies put the the the we think, answer I David, said answer business. think -- team that the to first been in that. testament quick that place And they've the protect I has no. And as to executing a the that's is to quarter, in upon

in of I think the think new the for strong to is continued both physicians. seen a patients incredibly quarter. testament Emflaza benefit and the to that's and first by message continued new starts been the I really large patient us an seen loyalty number and a starts, And we've what's patient

Operator

the from comes line question Danielle of Brill James. Next Raymond with

Danielle Brill

brief I have two ones.

updates the specifically ratifying considering ratification question EC on of Any First, delay And EMA second the feedback Translarna on in that this the clarify is the because possible about the it expected EU. Or EC believe the Is in -- on approval opinion is then CHMP procedural? EMA. on meeting was was this XQ. the to of that called I conditional in front? is the feasibility a the decision? status not in the week

Matthew Klein

Danielle, first XX for question, discussion for usually was meetings. It's instead across states, then having exact the this lot what your done and written occurs motivated writing. pause the That agreement we days clearly member beyond is questions. of know by we procedure that was that Usually, XX What live discussion what days. meeting procedure, of we're typical Yes. a is within On typically typical that They're procedure. a allows nature the we the the a say say. is written the EC It's called the a can't was by for discussion, that adoption point. Thanks, ratification time can EC.

decision, atypical. are to EC What things are of adopt the now that in that ultimate the lot on a terms going of means whether there's that matter So the the back really what decide. to the can't expected say what has and observed the to We had and what is beyond whatever far procedure CHMP, we what say can typically we're we according was that gone not happens. typically opinion,

meeting question to through conditional from that study they scientific we had looking side, time. older performed from well this of as that extension. the of thought data had we experience on the we years feedback have a procedure, them. where had in advice few the we from we FA we different a Phase could the And on the recent gone study QX, at made your with have FDA with did -- open-label forward that forward they with from the EMA course, well that a a back moving previously discussions as NDA are as the not support as long-term open the indicate based the move terms We're authorization In FDA shared on book further study extension got after vatiquinone, MOVE

Operator

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

Tazeen Ahmad

different sole of used up been least it secondly, what's confidence be not the One patient been having has sizing? for to at can talk population, EU maybe biomarker Can Huntington, endpoint? might on versus And of just terms recent so the on that point, about then the agency, U.S. us based you in be FA. a the with that you've brought in Is conversations how that an topic level the far as end in discussions? your

Matthew Klein

the very Tazeen. much questions, Thank for you

and lots in and the age applicability And in treatment In regard territories, population a we in the U.S. affecting beyond important delay indicated obviously, talked data from of really ambulation. terms population that time about similar has of that lot the roughly FA, in both -- for in of Europe. there's populations the under Clearly, for sized patients prevalence the nothing it's XX, supports with of to how FA effects need just that population we potentially patients We ambulatory So all and observed correct? of your to see question believe important second their fill I in age. important, in is ambulatory ability the population, under Is same also XX-year-old as with roughly [indiscernible] regardless the size. for was on effect pediatric HD our the have an but an the patients, biomarker the an patients endpoint. unmet

Tazeen Ahmad

That's right.

Matthew Klein

with So the regarding and the pathways discussions had strategy. biomarkers -- endpoint we yet have the accelerated biomarker role agency in not

for like looking I that the leverage Alzheimer's disease, approval neurology the diseases seen Huntington's the particularly has III clearly takes disease, collecting like to efficacy a been pathway accelerated Phase that division data trial many think definitive agency, neurodegenerative years. we've we're many, in

we drug. the used of potential then obviously, a the systemically We in NFL protein in for course, the we also approval the have of no HD, be. And biomarker that think which bit levels challenge for precedent with yet that is has important CSF. particularly including HD biomarkers, the several study and first offers accelerated in I what for believe Huntington there's would administered like peripheral things been PIVOT talked the the case and about Huntington ALS

data efficacy activity. for think certainly you the the there data of do hand is asked, of based of with the biomarker agency's on would path should agency appetite using what collect once a or as So disease, an we in data to just in what discussions we to therapy what the establishing have and be be we begin but have biomarker the data role plan Huntington's recent

Operator

from next with of line Choi Sachs. comes Paul Goldman Our question the

Kyuwon Choi

well to the contemplated and first I the plan AMLIC. wake regard thoughts follow-up just design then recent either made -- a just changes of may your discussions on any regard changes as study sites is what there? the the ALS. second, to safety CARDINAL and been In or the PTCXXX. trial recent have progress And my maybe just or PHOENIX and Can regulatory to have you on your HD Have on and and implemented study side results based also any just results U.S. with you there. the that on are just the two, statistical from comment PHOENIX just with for

Matthew Klein

question. the asking for you Thank

of analytical regarding terms -- also in in see variable. change their patients been who well enrollment. of the of specifically, as which understand to ALSFRS. as died death both and have implementing quite score together the When understanding difference has first mortality the the then scores a and in want analyzing they we Your on continuous ALSFRS for CARDINAL. about time interested wanting rank talked interested the clear something They're mortality. changes score approaches as between to called following to of placebo information treatment question also the been way understanding we And timing joint in in a and FDA in may and test a They've is very what

our results, So that have designing the advance with to get if ensure approval. see. appropriate of the analysis that ensuring and the well ensure course, study We positive, registration to directed agency consistent agency's change what towards that would that we when in be the so positioned plan anything analysis it we drug And we've, to taken to the we'd with they're that be closely was the with they work the doesn't plan guidance that want regarding

HD your PIVOT discussions on question Regarding with safety the data and from agency.

safety disclosure, asked the quarter that will the the for has then to in partial data review lift data we to hold. prepare we order the second have As agency

available. the So we look to when forward to submitting those agency data

asked Just the tolerated. as weeks. continuing a data subjects to data had data only a number following patients. drug to data, to we've of a Well, see we're clearly, on at that sufficient be reminder, the And to would of the of on them agency confirm provide shared, a last but number the XX-week seen they X-month position what not us review to as we've X-month be XX-month data well going in we're provide XX

partial to together, agency in and So hold submitting putting that a future. we the near package the it look data to to lifting hopefully forward

Operator

this Klein to hand at remarks. to conference Dr. time, questions. back like the Matthew And for I'm further showing closing I'd no

Matthew Klein

all very call programs. excited our you revenue all the first about of both Clearly, the terms joining outstanding the today. quarter, and Thank of we're performance in for execution in across

to forward move and look throughout this well updating all remainder to success of as you the We year forward. are the positioned we continue

all, good evening. a thank So and you have

Operator

And this for your call. concludes today's Thank conference you participation.

day. now Everyone, wonderful disconnect. may You have a

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