PTC Therapeutics (PTCT) 27 Oct 222022 Q3 Earnings call transcript

Hello. Thank you for standing by. And welcome to the PTC Third Quarter 2022 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session [Operator Instructions]. today's recorded. that advised be conference maybe Please

I to your like to conference over hand the speaker would now today, Kylie O'Keefe.

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

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

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

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

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

pleased to strong Good and joining PTC’s everyone call. thank share afternoon, Kylie. I'm quarter for Thanks, results. you third the

pipeline to with advancing medical our marketed We and are revenue continue and strong continue unmet on deep to growth moving forward our XXXX to strong products significant of milestones. treat Ore are demonstrate needs. execution therapy and we diseases new broad generate

of doing focused PTC PTC and and them our to seven programs therapies Today, to on of stakeholders. development diseases so, is to in create unmet has rare with commercialize medical at products high five need. bring innovative disorders, value significant all treating develop and mission for Our to patients and discover, marketed

continue expect therapies of robust build two potential state steady we deliver to to therapy years. a every new We three pipeline one that at to

treatment our DMD the to total achieved on XXXX. $XXX Moving XX% revenue, third revenue million of in performance quarter. increase all million authorization marketing evidence an positions increase performance of $XXX world are go was in XX% regulatory a total data as year-over-year. I the allows detail we of results in to to convert registry over quarter in will us long representing We Eric DMD provide quarter, STRIDE to from marketing the from the robust strong and raise commercial this our portfolio, a our a the term of revenue The the guidance. update on end across Based more Study well a Translarna. into continued shortly. the XXXX guidance, low real European like continued XXX to standard us our benefit now increasing from This authorization. the as conditional well

in the first the X CHMP authorization EU, September. the European half of lasts for the The opinion have And to several variation regulatory in such, as we XXXX. Agency path X convert we expect planned, typically the Type Medical submitted For in procedure to Type as variation the months. a

filing request Turning submitted US. clarity an to of the to on meeting We path the NDA. FDA gain a the to

filing, along with as to with neurological from using for along diseases Amylyx in ITT of could support the approval. recently that evidence of has with discussions are XXX Study a in substantial follow While not we the to support understand studies this XXX, FDA feedback seen results Reata. evidence population and with NDA evidence Clear we've other rare effectiveness, evidence does planning provided the provide whether initial confirmatory Study written of approach agency examples confirmatory the up trial

track Let being first geographic turn growth patient markets to the market leadership the is has patient switches, in all me on Evrysdi splicing driven major become by and and established naive and starts Rapid market for label expansion. product now is SMA. our from to marketed platform. discuss global leader

based XX% addition, pre-symptomatic In Late Medicine earlier for months The expansion label rate approved retention in EU under year, by such age, with from majority a months treated to satisfaction. of efficacy is on sitting the Evrysdi study the patients SMA this of The is the FDA key treatment. which that submitted standing the be and half end label safety of the year. expansion use with data interim filing has Type last XXXX. a X variation months treatment demonstrating XX first and expected walking Roche in the before the of a milestones, of approved as two Agency in Evrysdiachieved showed infants already after pre-symptomatic European Europe RAINBOWFISH to in with infants newborn, XX

And Australia. our next PTCXXX. global before, currently we to European move and and compound, study with active our Program Disease Let the Huntington's in many splicing countries as is said PIVOT-HD me enrolling now

the expect first Matt into more go XXXX. the shortly. week We will trial PIVOT-HD from of of results XX portion half share to specifics the study in

remain in months. candidates robust three PKU important the or XXX Vatiquinone the Our second of over quarter Sepiapterin, results Friedreich for fourth XXXX mitochondrial XXXX. of quarter of quarter associated ataxia first the in the nine clinical previously on in next Vatiquinone to in development target pipeline seizures in to in drug and towards advance disease data We continues commercialization. achieve

also Blackstone enough state, therapy every steady build This are into support to our announce least at will financing new to three one We to mission our in can pipeline deliver that years. with we that at Sciences. programs we entered so have collaboration two excited a strategic Life collaboration

so to and transformative create our And patients that stakeholders. continue globally for we bring medicines value can all to

funding. part Emily dilution in $XXX million will of million capital provided of initial with cost, additional many the new partnership, the our product to sustained With build pipeline, Blackstone we financing. the advancing our in years to for option our for details growth describe of strong As $XXX platform continue for growth products low in an an commercial come. marketed and low many

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

Stu. Thanks

the drug the and We of of product. additional drug the across in the comparability I'll FDA meeting to product Based of our asked Over Type third had discuss between registration bioanalytical October support trials all with an in on the the the C several next on of start a discussion, make of we BLA platforms progress ongoing from in our package. FDA details the to expect used for months. directed course has to quarter, our and Upstaza. submission data commercial results and nine six update clinical submission for studies with continue

request and now submission working are in BLA first We expect the address to with currently the will we this of half occur FDA XXXX.

those subjects population endpoint primary sepiapterin, study PTCXXX to enrich demonstrating run-in in quarter. share a To for randomized. two Turning target and sepiapterin in the study only We PKU. likely which study reduction patients known the weeks six week during by sepiapterin of of phase with The is includes directed results to as a APHENITY our blood treated responses trial with to remain for with are are potential levels. the the this responders, ongoing phenylalanine X randomized previously APHENITY fourth trial placebo-controlled Phase registration on of end

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

to Turning platform. the Bio-e

study MIT-E primary controlled a ongoing for three previously six MIT-E Enrollment randomized the months and aspects utreloxastat of CardinALS MOVE-FA MOVE-FA fully CardinALS continue Both as trials the have quarter month from placebo disease-associated The in subjects. rating to capturing from morbidity of from study XXXX trial a MIT-E in in the first as registration the of ALSFRS target XXXX. the the of patients. enrolled Subjects global quarter for secondary with will of ALS. change controlled the known endpoint MOVE-FA XXX second other with now placebo ongoing endpoint and is in placebo. The then and utreloxastat ALS two well PTCXXX receive We disease enrollment we be are directed of trial with results mitochondrial expect trial the study for and is Friedreich trial in six as seizures the with vatiquinone, utreloxastat, disease of ataxia, mortality. to X:X trials approximately is scale baseline for or and trial study one

platform. splicing our to Turning

for As Australian we and are PIVOT-HD the we level to shared, sites. study to US PIVOT-HD asked dose and data the European is enrolling support recently the additional Phase actively the PTCXXX has duration. at our FDA in X paused as proposed Enrollment trial

which dose, and reminder, potential findings be milligrams, is dosing. trial and As month milligrams from levels, to XX PTCXXX X initially of includes milligram dose X XX two controlled Huntington's milligram patients, the placebo on based with in a the PIVOT-HD study XX third and a disease the a will

we CSF study, approximately plasma ratio XX% and at lower studied at on plasma HCT selection the milligrams We've the achieve of the that study. Phase in You we’ve blood the brain. PIVOT-HD will dose since treat milligrams and X. exposure why in of desired selected of reduction XX a the levels X starting HTT HTT the both we protein been the levels allow based PIVOT-HD XX healthy relationship volunteer lowering dose to a dose patients MAD XX in percent HTT higher recall asked we the in CSF Phase in exposure still of approximately lowering reduction XX% dosing in at to and trial observed X, was dose levels could and XX% approximately a milligram have XX% levels observed In the Phase to to us X dose. as for Phase X blood milligram the potentially The blood that X:X.X, observed ratio

the to lowering plasma the of protein XX higher HTT exposure dosing. an we Given expect even have to XX milligram of CSF exposure, level would milligram of observed at and CSF ratio X:X.X

as Phase consistent to desired protein of lowering target demonstrated to HTT brain. patients, patients in volunteers milligrams observed PIVOT-HD X HD milligrams XX in was and ratio to healthy in very achieve the level. the If with brain at is XX% have Accordingly, plasma may given dose to the our CSF lowering X milligrams of dosing milligrams XX% selected in what we HTT X, the well XX in XX% protein starting HD stated XX%

patients potentially can Australia level key that the that have confirm XX ability in in course dose is needed. PIVOT-HD study the includes study. CSF to Europe of closer XX-milligram dose ratio to month plasma and study a exposure to ability protocol we X:X, the the the levels to approved in study if for Of also higher the We PIVOT-HD

controlled Overall, study and pharmacology and our phase of neurofilament XX PTCXXX focused near exposure, look chain XX initial levels, including on the as in two placebo followed portion volume study across results of from protein the HX We XXXX. as to a very are progress terms includes future. programs, phase week brain to MRI light on biomarkers, pharmacodynamic design, parts. focused HTT CSF of on controlled PIVOT-HD forward and several In excited results in nine our levels. forward about In effect, changes development look CSF PIVOT-HD by our placebo of month plasma we continued well CSF sharing sharing the from week studies and

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

Thanks, our high to the commercial is It number to our the and medical see progress a is Matt. of bring patients address to eager unmet pipeline exciting worldwide. from to treatments needs innovative team

was yet delivered XX% $XXX another increase Our outstanding quarter. has global million revenue in an DMD of the customer year-over-year. team quarter, facing

Our key revenue a driver continues and remains to diversified. and robust be geographically franchise DMD

result product we with growth the raising to million, of guidance the Starting third revenue DMD quarter million a which are our XX% net represented was revenue, over revenue quarterly last year. As $XXX consistent to for second strong the $XXX quarter Emflaza, million. XXXX $XX

Europe, the supported drove million for starts which the patients revenue third third team driven Translarna markets was which for based in a Continued continues future compliance increase access, is Overall, Eastern we Asia revenue high in growth This XX% to continued quarter, globally product make and to additional Middle be all progress which with And Pacific, XXXX. our East, delivered favorable dosing by execution appropriate Emflaza our quarter in well for continue drove Latin importance into Translarna as $XX net DMD good US, growth. expansion America, and represents by over in of strong strategic newer by in regions. weight of as new the the growth. diversified. patient the markets

Moving to our launch activities. and Upstaza ongoing

Germany. Following SS the of for approval treatment in was in the Upstaza AADC Europe launched the deficiency, meeting recent IBM at XXXX

actively with the strategic is on team executing initiatives launch. Our all

first identification our patients program additional treated cross and already XX access commercial anticipate in data XX via upto date, of the have early French results and will patient clinical to up submissions safety we France for QX. Germany, as commercial from this and access Treatment the we pleased early treating programs are well as We others to on patients on readiness border confident years from healthcare. older. follow and the on continuing at Italy Upstaza HTA and the treatment is that European months AADC that in with preparation year first treating out have accelerate. under we reimbursement efficacy dossier key only obtained for deficiency are the Based and to we carried KOLs patients track, and support feedback for strongly surgical the center focused approved are as durable further Patient also is markets centers. treatments And

$XX $XX and revenue in quarter in guided million in fourth to million Upstaza work have this from France, Germany to to towards continue the We and Italy.

in America. Shifting gears Latin

team additional be progress strong, particularly over order advantage innovative Our Furthermore, the Ministry fourth Health following where an patient significant areas administration from Tegsedi Waylivra. classification self group in identification continues the quarter. anticipate and to of In to competition. Tegsedi continues the Brazil, Tegsedi, with remote a this is order We group first year. purchase to purchase delivered the the in receive earlier we for

patients. which with the of technologies to Finally, drug Commission access discussions of continue incorporation will CONITEC, inclusion in hATTR simplify Amyloidosis National progress the to essential the the list, Tegsedi for for

important treatment. grow continues Ministry in FCS For our This to identification which the base received Brazil, Waylivra, pleased in our are the for quarter. group anticipate continue of across patients we purchase to of for treatment FCS. to the progress an patient Latin and the Patient fourth awaiting we order Health to America is first from we deliver milestone have

If application reminder, globally be the treatment approved will for treatment this submitted for very anticipate for our in last the in this first we indication. of a and particular this in approved, for Brazil will approval December, As Brazil, a year. ANVISA FCS. the exciting an facing Waylivra of global to approval FCS this a for teams. first conclusion, time later We customer PTC for mark In and is decision Waylivra

successful foundation We delivering strong are focused even an on XXXX. and the laser for a finish XXXX to more setting

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

Thanks, Eric.

highlights, future I revenue like transaction includes Blackstone up with on Before turning a consists to puts the equity. allows moreover have position $XXX PTC $XXX financing to X.XX dynamics. in and of financing million of to innovative in third SOFR also near and term plus debt secured $X an term at describe million dependence to year This to of execute The quarter our opportunities pursue senior we a accelerate financial closed that would billion pipeline. bullet $XX market seven This additional million upfront. strategic just without strong with partnership and This Life to PTC Sciences.

that XX be term $XXX the accessed the close. loan months after million debt includes in first draw can another delayed in Additionally,

financing the a agreed credit includes from line total opportunities. upon mutually business million $XXX Lastly, development potential

Importantly, mission will to investment and the secured our breakthrough We a including deliver to commercializing Emflaza, be loan continuing Upstaza, collateral therapies Blackstone Sepiapterin globally. by limited limited term bucket, of forward on assets and Translarna, and look to developing Vatiquinone. by

the our release refer the results. XXXX, review million $XXX with third an royalty and additional DMD issued Total XXXX. to revenue for $XXX from minutes of quarter a $XX franchise Please few a results. Beginning for third Evrysdi take surpassing XX% of sales primarily annual third Roche for the top to million, detail. $XXX million were earnings revenue of net from now million $XX revenues line of press I'll quarter afternoon quarter financial driven of was by of milestone over the Evrysdi increase This product million. this

million to revenue $XXX total $XXX includes quarters includes guidance million. our of million. also the million therapy. million from million XXXX revenue to from narrowed $XXX guidance gene million And our in and This range we $XXX revenue three to $XXX to was recently from DMD $XXX Our first million revenue of $XX from our million million launched revenue to of raised to have $XXX Upstaza, million $XXX $XXX previous $XX consequently,

cash expense. based XX, recent securities million Cash, investment and report $XXX $XX XXXX. of marketable December of approximately to for the equivalents XXXX, $XXX quarter for additional non of compared programs were Translarna year-over-year. $XX XX, net forma our advancement the of expense year-over-year. revenues to September of million cash growth Emflaza franchise. our million basis. third on product in R&D in FX million revenues This representing and for in quarter based expenses the $XX of $XXX have now million, what million compared based in XXXX XXXX $XXX clinical as noncash XX% noncash expense. million compensation pro excluding growth $XX the XX% pipeline. Non-GAAP excluding based expenses R&D year-over-year compared of We're product million million expense quarter year-over-year to XX% Non-GAAP as DMD happy $XX balance to million quarter of net despite $XXX million million for compensation of compensation $XX the a stock excluding reflects compensation to cash Turning of the to were financing growth XXXX, or approximately excluding XXXX. in noncash were was and expenses revenue headwinds and totaled third third third million The research but increases been increase stock had to third $XX growth quarter approximately at stock compared SG&A $XX otherwise the stock XXXX,

for the over call operator the questions. Operator? now turn I'll to

Markets. first RBC from Our Brian Abrams Capital question comes Instructions] with [Operator

Brian, This taking question. is on thank our for you for Joe

provide the more a you you feedback could data detail Translarna, to the this and address issue? on what include to do little in Just initial plan about package

we submitted did know, FDA. a was, you the meeting What have as we request to

So clarity on the we path get to can NDA. the

this one, XXX with that support Study FDA planning We're from not the substantial understand the then initially therefore -- does provided effectiveness. of with on population follow-up a feedback evidence So evidence Study along XXX, the in confirmatory agency and written the in lead while to us some whether to could written other studies, discussions approval. ITT the was to not the evidence

So of I can really where initial case conversations some involve need some it And medical unmet patient community. have that, back program, the long there we particularly did saw submissions, agency, of history of which with have conversation. feedback, expect I especially where think, usually the the rare diseases, conversations and they some then in they interests in a to is and of forth, initially at the as clear from Amylyx the neurological with fact the that written especially the most situations. like been live and and we that submissions, seen NDA disease and with patient the given companies recently of some the weren't Reata regulatory the recently, the other can [presence] we've has example with really And accepting companies subsequently similar you and think

the beginning, here, the inning So this in we're looking to and is we'll forth going the them. back of with game be which -- first

with Our Fitzgerald. from Cantor next Kristen Kluska comes question

with late opportunity of your I reaching Blackstone this deal BD pipeline, So with upcoming mentioned And successful if Emily well. you stage here help potential your sustainability? that as and frame you know could self think expiration commercial potential how some opportunities, do towards

give a you for outsourcing? about as health future looking framework as well balances the innovation, what and might general in be you So can

and As pass I think started we it. all

combination product both that's we're our the as is As of the to I years growth be we've to. development which well to pipeline, bring of accelerate marketplace been both been, a to And able those development, drug and business use plan building to building external has processes. our of through every what two those going we're combination both as to a three to internal of discovery what we said, and

great to or term our able way later already look this we for that opportunity commercialization and molecules increase the bring are we well accelerate, add have do us near -- in to assets later at assets efforts to also additional we Blackstone that and stage commercializable, as to revenues look, from stage be which a them, and to pipeline. take to So indications gives as have to

extra So you Emily, to do you anything add? want have

I'll just reiterate excited this about deal. very that obviously we're

business incredibly As We terms, development. you to drive attractive to existing utilize pointed look to our strategic further and favorable to for for to the innovation and access growth some allows out, provide it to business want look this commercial drug at create we obviously infrastructure. capital those us of business to flexibility really And leverage opportunities development opportunities. does financing of will development

Upstaza, with confidence And is I that essentially is comfortable check surgical off before the submitting? guess agency items? you and then this procedural that And you now cannula the to what item the for need gives last the

you Matt, to want one? take that

was with that have prior So specific been the the product issue to experience there using brain the to for had the past products cannula the our children, in that. And be desire for a us of delivery the of with addressed. was no into issues experience the there cannula the drug appear

data be of with the able that delivered showing of with the provide procedures included the that device compatibility so device. to testing that drug children importantly, those course, a set safely surgical can been and have was we the of now benchtop to both the And data the where product that then there of them

included which support the of comparability regarding in want, material In to the additional they what in just the some the well provide samples analysis, to ask positioned are clinical be from terms we is BLA. of batch additional data

that submission half said as and the can data forward first move those provide So the be in XXXX. confident to able of package fully we the we are we with

comes question next Eric Joseph JPMorgan. Our with from

are that to PTCXXX. to US that allowing a at recruit the in suggests on part I'm US higher you could progress revenue of guess of like Just to are a be recognition on Upstaza. clinical with you [Technical and resistance or the therapy resolved of that some if European the new launches, impact data gene sites at I countries dose, point all have encountered Upstaza expressing then reimburse confident to wondering other following overtime. follow-up I'm Difficulty] so? kind as from that moving pause the that secondly desire how cohort? it curious sounds And readout, how you if might

So Eric I in asked a the bit were out think XXX. a the -- I Is think you going you question and was on your But higher right? to little dose. ability that to go

Not would you go you're you if able US if to able only need to to mg, to point to I resolved US pause the go you -- that time, to if to sites, mg? XX be at the have that, guess, would go at in time the XX that recruit higher be

durations sites, the just and in the remind countries, maybe in I'll everybody, the and multiple desire. that multiple for levels milligram, PIVOT-HD X outside we So global milligram. milligram, at US running everyone for that up and of And everyone the remind dose the start XX study includes that to the XX is a that's just the and

of all regulatory throughout in So bodies through Australia. went those and Europe the

the that. terms have outlier there -- only the -- of the was US that the we sense in in So -- so it was

the forward moving with And parallel, is work in really doing to the and as are we'll as now get are what study possible. So is the priority agency then results to get obviously the we rapidly -- program enrolled and top concerns. address their our keep we

our subset view, results that the most from point thing data in concerns that get do show from we we so of the there. the the And likely from of to would terms them of be a could

our doing is all is doses we're within what move within the good -- nothing of those And incredibly group to than view anymore able goal moving news is Australia. telling the are rapidly. in Europe complete that our quite is and data. approved human to we actual clinical There So we're that that

is of it try Europe. But priority and in patients in result to rapidly real will that as complete really as subset and to So obviously move forward. possible -- bring move we the is it our to

about permission there, able the is we'll go. Kylie So in as able being consequence don't but to terms we and able European pay get of in Matt these you go can need all the result talk as the the good terms do in to of other of their news Upstaza, be a you sites. Then to be trial to maybe

correctly, your And was a heard the understanding in sort you I Eric, moment? just if the having discussions question we're at contracting around of, from perspective, Europe

revenue going been for upfront, paying there's go gene to your that might products. pay also, might those than structure prefer go all rather that phasing some is, forward. discussion and countries But fact, Yes. paradigm a might also just some upfront that some therapy apply if impact if in, recognition, I'm it how wondering in a pay

durability because focus really number payments. we've allows us technology in I think obviously, Europe the that that. of strong clinical the is assessment that benefit clinical in past, moment. term also to package, in and as perspective, for I about that treatment with our as benefit transformational important important talked from have, under these think of we're data short and of the durability this pay the you things the of to years that's to we for a package that's for agencies one across upfront Upstaza with to as discussions, for follow And have health emphasis the you pay at but discussions single think believe not performance efficacy what's safety the And that that safety on but And the think patients we're I only have not And think package data. up it's is up. just the we continue going And the that I durability or put in strong our we all XX perspective. the be that, really with coupled we we also what have I go can able on we shows patients,

next comes Our question Thome Cowen. with from Joseph

data, any work you ahead And kind guess Kuvan, SEMSA year came to the from if I success of you What's end there submission? submission, need Palynziq end we given of generic study, of all year? need the from I the Kuvan, the like there. into there's guess can of in pivotal a positive of then the is in look that just it’s PTCXXX out that this under the BioMarin CMC on Maybe here ahead the lay this study context at out conduct of

and is, of this XXX is because PKU. and better one excited Kuvan, it's it it concentration, of patients. more to far it's the you're be in of which, And in because end And Kuvan that even of before, really able where part pretty we've for a can was and to effective really the to effective. that more that’s makes that it shown advantages that not it discussed is patients get think you at bioavailable those about higher We're far of day see -- I the sets

treat So the early blood. of that I patients, is to we're phenology reductions and the -- exciting reductions types greater show the is that of able of more think that's an drug results so within aspect

actually I that mainly that hasn't is can patient that because doesn't been what patients. there's there, actually think and we're is is it drug in it for well huge a shown, target And that and because population those going show we more can Kuvan it what -- already patients work, treated. So that the remember itself, to within think

real and think else So do want there's advantage Matt, add terms to in of you how the I something enrollment there. PKU it’s progressing? a commercial

data for need FDA, will answer the with the will that No, to I world package PKU. in pointed your of mentioned, manifests And severity, Stu be tremendous the seeing the patients. other classical as the we're participation significant move components that, expect the medical a we enthusiasm of including unmet in for ready. positioned say all as out, And would PKU levels and well clearly just towards patients question, Stu and positive trial the forward around all to across be US

question comes Lebowitz Citi. from next Our with David

behalf on [Dipanjana] is David. This of

FDA Translarna, see that about So when you [Technical that study says Difficulty]

when back one to next switch we'll on. Maybe and there. she's go lost we think the then I

was I there wrong. believe something

what this? at isn't XXX, updates along says Translarna in the analysis wanted with hear can Study world a they substantial ITT about benefit ask population? So data when to primary FDA the And there when are that we real benefit I set in the was or further looking about

they pretty So they the a is saying of mITT hit really terms what did population. you of they the -- feedback a took not -- took took in view rigorous that

we now population about -- on confirmatory. would approval So to the get that what with evidence the go along with instead want is the talk ITT

conversations once and discussions. where and able approval. is, were Amylyx to this be Study when we can pathway with of discussion, conversation. of And series could that's did So see out other back actually able along we that a an have the where have you on expect think, other clarity to can there's studies, have the from minds changed after to And population of evidence and the the get I think in changed what ITT you approval we those there, that were look, on this that I confirmatory examples Reata see about particular XXX is you to minds on again, some like certainly look there's and way with evidence be

question the last one maybe And PKU program. on

levels? in So liter what phenylalanine in are rate XXX patients have micromoles than of greater responder you update upcoming who kind per expecting

We the this what per with level the study, in of these in information study able micromoles not what the in in seeing than subgroup the what greater and with blood that patients we phenylalanine historically to have classical importantly rates levels we are XXX been in and of but any baseline phenylalanine expect also across to only X,XXX there we seen patients, phenylalanine X have emphasize responder liter X haven't we demonstrate of has Kuvan, response on in Phase market PKU full liter, than with than patients in which greater what to patients, responses baseline no spectrum again those per of Phase been terms micromole where levels. other specific saw provided was we reductions

seeing PKU. to full those fourth by the So that. of sharing that could effect spectrum we impact severity data well have the as we forward -- look look And different robust of quarter as the to again, well across we when end genotypes forward baseline as

with Xenakis Alexander from comes question next Truist. Our

to the remind Can perhaps, far or are approval, we would metrics question types on authorization? you story number patients anything the price One Translarna to we go us Can we And Upstaza with ready the do appreciated? the does patient sites full get at of In will and marketing change as in you revenue, Europe. be remind that you to forced or in providing you launch, as as if second treated that Europe. all in and providing? Does -- us marketing story you are on renegotiate you considering as change then full progress will if that be go, what uptake in additional expect or identification of be full you addition active get treatment?

and to Europe in have a approval. It's we identifying conditional been to Translarna. transition about to the For bringing pretty interesting, a approval patients from good full them out

have number we a patients already. large So of

to also the prevents five the don't think maybe Eric conditional to transition want such, of it from comment. be I think year, full open, may we and every in you to report HTA terms years. the I And approval us have just

of which helps every full approval a HTA marketing will actually by think course, I own the their but country they has only evaluate assessment. systems

have, combined, STRIDE pricing and along much think conditional current our that discussions. And previous and add only all strengthen with a that so strengthen I structure not results, continually stronger new approval along the with throughout it'll to those the -- cases, in international we believe many the really XXX of affect corridor. studies any forward will current doesn't but add the We Europe too give patients value and us proposition position even

where this of does certainly as in at best So we'll maintain that we value data this five price possible And said, regulatory our essentially, primarily, the Stu point it we to while strengthen a file leverage continue ensure is proposition. don't the year all for to every to years, have time.

And metrics would then on the be Upstaza launch, any additional helpful? color future on

get the metrics were we to think trying was I major that revenues.

comment anymore you Kylie, want So that? on do to

a I working to different and full work able first globally. as patients ahead we've possible. And we be think steam team the the with these to achieved have past, patients being as and access the identify commercial were schedule in number additional do a And early we done good ready French a been countries. treatments has having ready ensuring the And treated to soon we commercial the these have in able that crucial centers to Germany, quarter. France this treated We and our of think identified, is I and program. of patient we've that number this surgeries under number go as centers as patients job able in the patients anticipate to in surgery. were we to of I the Europe schedule the we year shared have And have a go to to approval, Italy, fourth think across extremely pleased able already treatment being of to

environment to that. So to we that patients access the market have do be and treat we're able sure right to making able the

shifting -- focused the we’re perspective, that from revenue. So towards focus

we're hope on million achieve to in do to and achieving we revenue this have still anticipate We million track said to that $XX that. or $XX year,

Prasad Raju from comes question Blair. with William next Our

to FDA get I Translarna. some on regards with the clarity just want to strategy

you clarified? if you'd something the it into do, to run And today? or to would then supplementary it guys study this be not or a has there's meeting, of interaction to stands willing nature, What request the been kind understand that it with a that is as agency? try type really get trying meeting, approval just the and that like FDA be B A did So going type of or to request we'll of set something data support or have

data population, if all statistically a we critical data make relatively one is, in way a you is ITT of of patients, the North sets that in function, the a a a and data difficult what's of to and communications lung ambulating, getting do certainly like minute that -- nature and you been we the we so actually things ever comprehensive the couple of the of in endpoint person strong A, data a in package our or have that's think that And since think person of I through it, of set that very just to slow it's massively COVID the -- heart is, significant most the a have a results and that matter, it's exchange registry overall on have if that the want full believe start the I'll have saw for been and endpoints in ideas in points that demonstrating study written how pretty not sense clearly, data have can Star, an view. patients first discussion significant with way the preservation is, that. the about approval here statistically maybe it's time to So test, significant then that some and have, six that to to emphasize, is some we function for ambulation walk, XXX set however, statistically changes. we and we recall think and it mention with we the -- I them

for there you is towards approval Matt, that's the to Translarna else way that? add to So want work want the US. to getting we in anything

agency And fully as meeting the find particularly advantage true have and the and a Raj, volley bit and understanding forth just middle would often comments that's that traditional that conservative, of of the in written a you'd are address ground COVID. say I we in all then data sort different of mentioned, I complex the things Translarna as was package. think really back package. package. The the to as and always aspects the is And maybe with are in cadence completely that sure going don't quite you agency of one data get make that person data the

analysis whether the in and of study benefit alone than so from that feedback that the and on first a suffice, the population, we're that written using overall the evidence we now And seems criterion offer, out focused in to agency potential we've evidence focused clearly where interest for Study [XXXX confirmatory clear meet can it’d along ITT have stated that a of has effectiveness of XXXX] XXX held substantial X.XXX, to chance the [indiscernible] are course where loss whether compared that’d themselves be the to disorders gotten of of on benefit of seemingly terms understand evidence pooled rare single a a morbid XXX treatment a with significant rather the Study position history can delays important match of four in the to what key progression trying that be outside X.X in where value showing slowing of manifesting we of transitions observing the ambulation with function. clinical disease, of pulmonary cohort, a findings of loss confirm terms just or clearly in in natural by and trials the P years of in delay we're of that can’t year

then they're with seeing I the sponsors where the are for forth well the been think we the submission. really to and rare could what where what criteria meet be certain other a potential other for to the innings data data is and So very now back and in disorders early series that we has satisfy are of used the and allow

with from question next Gena comes Wang Our Barclays.

just what you Maybe wanted I clarification. comment, to have follow -- just quick a

For initial analysis? the your regarding Study did XXX, the analysis interaction FDA other along FDA your with see the ITT with

fact, analysis analysis submitted, are was seem I we data on of method, we emphasizing of focus that didn't the the and say mITT of did which of and group not did effectiveness. pre-specified that in saying substantial all that that significant, population the the given know, we comments and that X clarify, evidence the to statistical than to not primary be their achieved significant, as criteria all would meet XXX subgroup So significant, greater primary we stating

why on the forward to that's having feedback we think didn't is written set And look I clear again, see with the limitations a ITT population. so clear, super feedback with And and we is of conversation this that agency. it of

Another study. submit after months question the clinical study. the data you to Since you regarding initiate nine is primate the PIVOT-HD non-human FDA

non-human highest have trigger study, pause the other dosed in the the what was any was trial? so and safety FDA wondering could words, just the you concerning toxicity seen In So primary dose what that clinical far? you then

about obviously, that safety -- is, of drug. the when level. the toxicology identify -- then and And to effect I look, is think multiples between to be non-observable thinking So what's important always how have the role

the we give of the really details multiples NOAL. so program, while we from were And don't toxicology multiple,

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part clinic. never was And had I the observed to about nothing an we've we think, And example great in example the Evrysdi, like there with where -- was a seen is be think seen that, what reason of specific finding when nonhuman quite toxicological time. other out patients. seen a or it and be for in non-human X,XXX But in some to commercial species turned as talked primates to we suspected later, wasn't everyone obviously, primates patients that about so in

any right terms and because issues in of we seen try we be try that a safety drug careful tag So and very any and not have any haven't we now.

everything. moving really our think of keep results then up some data and is easiest get to and we to top since FDA. the sites the the results, forward the point clinical it's this enrolled to priority global the a nothing is, study, data think key Because the keep And to and the for So get because That's of the and world, like to here. getting opened I -- subset it our that's around to goal clinical do way at there's talk data. moving trump it's and

with next comes Raymond Danielle Brill Our from question James.

show guess, -- since first, year? if benefit, in protein mechanism next on data US, a HTT the have running I are couple levels CSF is now pushed there it well. to as to functional you're the planning the available early XX I you a study you're that are the PTCXXX a Pivot-HD for And running path development sounds streamlined still? like or pathway a full outside U.S. month shows a then

study biomarkers pharmacodynamic to pharmacology is So measures. just where the and is really XX a placebo focused focused part you, first remind the again, I we've, the the second effects. are on two outcome study controlled And part weeks the think PIVOT-HD on and

we've a mRNA state. on I think to first CSF milligram of exposure think going the CSF. outcome not the perspective, the critical as our then full reflection is to the duration what reduction we'll the also And and the that and basically And changes, -- the between volunteers, data weeks, in be about And close and healthy study the doses what what's X:X of the there and look which observe. a relationship will we'll well. think, from but steady right blood seen And also the XX have is ratio the to plasma be protein a tells neurofilament exposure, year HTT HTT CSF to MRI only going because so And other dose in that that measures critical XX the really we the the protein, us know over range. longer time exposure and I in then ultimately in at X are at that's I provide

sense are So it's than patients it's accepted Europe no patients US, they're and patients. and similar think think the that the in well perspective, in I I realize from Australia that different to in that that important

than with from study you get can States. you that the this get results the that in results United are the So no patients different treating

fact biomarker, to of lot the so changes And volume of world regardless of potential a can think potentially there's this matter a it's CSF or changes in could that don't you blood where be accelerated neurofilament, used would as whether approval. can use we view way that of as for US you We best of by and our way is approval do the a well data in of it where be creating of there the not. viable, MRI, terms the that's HTT any either a potential then there's the combination X. That's or use accelerated I the as regardless and if this for -- you a fact that means the in data. in collected define mere Certainly, measured what's changes patients whether to think Phase measures,

Our Jefferies. from comes Shi next with Kelly question

first MIT-E So regarding the PTCXXX the first they And completed have for what change all would wake percentage of seizures to the from want also confirm clinically the next quarter baseline kind the I from for be already help in Can US, in to on numbers the involved study expectation patients of considered Just the that? observed year. of meaningful? set of you portion? results motor quickly on

want that? you Matt, do to take

patients. on first in Your to PTCXXX the question was the enrollment and pause on the US, impact US

Just US. to contextualize in the the situation

the enrolled for in to any to cognizant that XX XXX month tox of and to to the support include not the three protocol. were nine had sites that to running, the lapsed. we plan the month getting XX such, the milligram amend protocol, the Obviously, dose to so started while US put full the with and situation had very didn't mentioned, delays only study weeks first levels. globally -- we enrollment had we but were we we that patients lapse in not that US patients several month be months, three we month As and potentially sure have we as and make the several the we be in And up nine XX have able second study between results up look could the the patients we month XX full limit that in we that only a would wanting enrollment of too and getting we if a wanted to and many the study as clearance because initiated the there had were

XX the week completed occurred, patient the time pause the has study. the portion had of the -- by completed So

because paced enrollments XX enrollment without certain in full no we there to the we patients disruption. the way So until months we impact was that additional of provide could those are

of with they MIT-E you mitochondrial the of MIT-E to just the question, about in the first have mitochondrial XX% seizures disease, all result mitochondrial that death disease, quarter. these from And are are seizures disease-associated to Turning as expect unlike we with in to morbid the in study course because they about agents XX% the in a respond targeted seizures highly in disease, with antiepileptic often And by as disease. vatiquinone. typical other common results mentioned, disturbances common from usually reminder, to mitochondrial of energetic cause and in children pathways that in seizures, are mitochondrial patients maybe patients don't

a severe you're burdens, who these So children having situation in a severe have including some seizure where day in you cases. up to hundreds are

difference a course, think between for treatment change physicians XX% designed of XX% would a clinical a in these monthly would in of lack of We, be and or refractory we'll seizures most for study a boosts. the daily these seizures, be placebo patients. benefit therapy Given seizures even I significant reporting lowering the view of to

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next Our comes Suisse. from question Judah with Credit Frommer

you Blackstone Or the that And capital? was collaboration. one time little raise the of did financing was quick us of you the the there most this on attractive? and Were fall did your Could genesis transactions kind feel Just this a lap? on arrangement? the other into considered being a to give color this

want take you to that do Emily, one?

this for And several were of competitive royalty in came a long and at debt and lowest further option the that cost frankly, evaluated looking options as options, out is capital This process. options options. obviously, best We secured equity-related, the company. dilution

I happy we very in identified therefore, timing, guess, their Blackstone have have a we comes it structure indications. And other some to able of assets collateral with mentioned, as extend the and you We basket assets deal as a be do pipeline. tailored pipeline in to excludes that to far are limited where holds we our as to -- also want the

the accelerate that be pipeline. the able And so and innovation the develop better to sooner to

credit they mutual Blackstone that on potentially to potential on? it's that that just when development million that just facility, taking you? then you're business And agreement, take you planning ownership need in approve say does $XXX Or will that on mean with

think an broad such mean of part where important partner with I transaction, such really was a that network. the highly we're excited I that's to a respected and have

consolidator royalty work to accelerate allow we our through will We've Blackstone. ways those value our number be for demonstrated upfront So that Blackstone be to agreed funding upon to obviously create with they mutually as will or financing either business with will And to development. a in that us then, efforts proven structured We'll further ability of a work their transactions leverage can momentum. network. yes,

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

get can terms Just a given but of initially, treatment place you goal how in least thinking And in the know if it approval be would the give from where early looking think, have, fall? on is path drug hear market that on to discussion? to opportunity? part extent then CDA, would the written us of the the second sense Maybe Was that, FDA that love you to the Translarna so? feedback about the Kuvan And U.S. is present about talk to question. that did I a they a you're the it. at the chance PKU, would to better to data Blackstone deal, see regimen you it's Kuvan, under where to a is about but generic, do

So maybe Maybe let's then start and start with I'll the pass. PKU.

available. isn't that that the one patients commercial well really be, I I PKU and while as is majority think on clear either there it we of untreated that Kuvan, this remain think should patients with should as or the not but very known PKU, maybe make of think products Kuvan, the I is controlled that well two thing even are well

that's Kuvan. small about are a large of PKU population a actually XX,XXX there's So by very patients which patients globally, controlled percentage of

opportunity is Kuvan of is a and So there's the therefore, with consequence that's that more the the And huge big better that treated, huge opportunity here. a here. opportunity be patients will really

so Kylie, the slicing in patients market, to and you that's go don't? Maybe and want to detail think through about explain why through case. of maybe how And respond and more sort the dicing the go do who you

as this see think I unique a opportunity. really Stu said, as we

and need. XX,XXX globally substantial While unmet there's there's about two patients still PKU approved therapies,

So the those or typically see able And that of treatments. are of in from these micromole past, strokes naive broad there's we of in have severe classical other to PKU US. patients, that those per patients in benefit or the the therapy more. of sense patient, And think X,XXX today baseline around been in Kuvan the have liter and XX% not that the fee are levels includes marketplace sort of

that And they from naive. so therapy perspective, remain

are of Kuvan. to them, failed because they're remaining have Kuvan, poorly could XX% they that or as In patients because tried time. initially many be on addition Stu of And either the that controlled that, not said, over the

benefit naive. X able Kuvan Phase head-to-head was been you data going when main X confidence there's sepiapterin with seen a so that the those the opportunity opening up an has gives that segments. classical not in demonstrate about the PTCXXX Phase was able into X us the And be substantial opportunity study look therapy the patient, to which across X you for PKU from And -- think Phase to if from previously,

even Kuvan, saw reduction on Kuvan. to more XXX opportunity Kuvan. with the were up that We than than were XX% responders And failed XXX% for see in we opening able fee those initially with a Kuvan, then previously those treated that greater

the And for that about treatment So of And that. you demonstrating documentation they've certain different population, if segments. aspects patient across those trying on sepiapterin, of and US, points across normally if you providing done the look it's already requirements that. in data around all for the think obviously for XX% example,

it's a to obviously, I in very market fails. Kuvan on quickly why there's if captured that's it be no naive, opportunity put so mechanistic mentioned the should therapy as reason able be And launch. upon And they

think it upon upon very there's able we unique think quickly to we opportunity, a and we're launch. So capture

Emily, due And for deal, Blackstone then you talk to about the the diligence? want

call date, that everyone diligence on our to the standard correspondence, company. on commercial on conducted assessment. the Blackstone forecast, diligence think attest thorough I our this have due our data can very we Obviously, regulatory

regulatory received they have correspondence yes, in all have that so reviewed our of And writing. we

pipeline the really products. to Blackstone's belief is long-term our of robust this think and and statement potential our I a commercial

questions this any time. for Thank you. I would to further And I'm to Stuart any not back the now over remarks. showing like Peltz further turn at call

Yes.

quarters. So first joining us on forward the year and throughout on this delivering year you moving number of Obviously, made can for let a progress me last of all call all, three in thank what you significant see, we've this and today. milestones the

critical We to and now, I studies we results value all in for a forward continue going really our that these to and ongoing registration number nine six also to think are are look the create of have of to months that directed next sharing both stakeholders. transform the

can to mission develop therapies continue innovative discover see, our hard as rare working So to you disorders. and we're for both

again, the for call. thanks So joining

Thank participating. concludes Thank you you. This conference call. today's for

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