Earnings call transcript Q2 2023

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Operator

Good day, and thank you for standing by and welcome to the PTC Second Quarter 2023 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] Please be advised that today’s conference is being recorded.

I would now like to hand the conference call over to your speaker today, Senior Director of Investor Relations, Ron Aldridge.

begin. may You

Ron Aldridge

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

Matthew Officer, today Executive and Commercial our joined our Officer, Klein; O'Keefe; Dr. our Pierre Financial Gravier. I'm Officer, Kylie Chief Pauwels; Eric by Business Chief our Chief Officer, Chief

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

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

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

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

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

Matthew Klein

and you Thank for Good afternoon strong expectations and second XXXX. I'm quarter share thank you results our call. for joining in continued performance pleased Ron. to the PTC's

extensive helpful new be to PTC in warm will extend the his First, continue the I would finance tomorrow. like Gravier. advisor welcome CFO, to strategy we healthcare incredibly I of with experience a and to Pierre confident years of I'm our working Pierre and pleasure had have as build over several

This of the total revenue quarter quarter represents We million. another with strong our of revenue second with begin $XXX over XXXX. growth second XX% growth me revenue. let Now, quarter of achieved

over growth. strong million which in quarter a XX% to XX% franchise $XXX year-over-year the XX% a achieve XXXX. would performance XXXX in robust puts to the million, This position guidance $X $XXX revenue of represent quarter totaled revenue to quarter second second our total DMD us of Our increase billion

team's strong than is now our globally. in with continued to XXX commercial Evrysdi approved In addition treated more countries patients performance XXXX

achieved approved. as where expected is is countries in access Evrysdi growth Continued is

In will growth. from infants now opinion positive addition source revenue potential CHMP, marketing pre-symptomatic authorization EU providing based on the another of the Evrysdi important recent include the

we between review OpEx As our discontinue of strategic between our in expense million now and anticipate and and announced $XXX a gene and and $XXX decisions of SG&A year full guidance initiated million associated $XXX decision for portfolio previously reduction. versus other we million May we preclinical our With therapy R&D, million. programs non-GAAP, shared the prioritization to $XXX XXXX previous

to XXXX. May annualized XXXX In impact expected our $XXX addition the of portfolio million approximately anticipate of in on OpEx decisions savings we

our Moving as including we studies QX APHENITY pipeline, to and data our very PIVOT-HD clinical results study. from from sets strong busy reported was several

May of trial announced phenylalanine with met our start significant APHENITY that patients. PKU me highly with of In blood study the we Let and we reduction in meaningful sepiapterin results. clinically primary statistically endpoint

and baseline subset demonstrated of PKU patients. in of Phe overall population classical in from reduction analysis primary the Sepiapterin substantial the XX% XX%

target Phe reach in micromoles guidelines addition XXX less In of levels than to patients liter. were with of vast line majority able the US per

year. FDA the these fourth and submit With granted the of is been scheduled requested hand, which FDA to a quarter. has third NDA in data pre-NDA an meeting strong with in Pending for we we quarter feedback, this expect

strength about the commercial the APHENITY global $X incredibly remain billion-plus for sepiapterin. data enthusiastic we the potential Given of opportunity

PKU physician excited for deep as Professor our community need patients In presentation, medical PKU recent large unmet fill potential the the in dive sepiapterin addition to about emphasized is the Muntau for Ania worldwide. commercial persistent

of PTCXXX XX-week in cohort. XX-milligram XX% of patients, met. Huntington levels all dose-dependent treatment PIVOT-HD this interim analysis protein reported the protein key we disease of mean Huntington data lowering of June dose the interim data lowering Huntington's objectives summarize were with cell for from trial. in blood resulted PTCXXX to in Moving To

targeted levels with level. lowering up of in at greater in exposure to cells. of suggests HTT resulted XX% that This Treatment be X.X:X protein of XX-milligram a plasma CNS the occurring the ratio dose exposure CSF of possibly to could brain

Importantly, PTCXXX reports levels groups. lower with SAEs, overall tolerated is and neuropathy no of no PTCXXX no an trend NfL spikes, towards treatment treatment-related treatment-related CSF well NfL peripheral with in

study. key X And these of the Evrysdi early continued our With on will we disease into time the PTCXXX the effects further Stage biomarkers. longer-term interim of about data patients of we XX-month can which with to subjects enroll splicing forward HD initial the power X support to platform. learn results These treatment along look the and we more the of encouraging success continue data course treated at global Stage PIVOT-HD of

vatiquinone, in May. we MOVE-FA to the from adult results patients Moving pediatric in and Friedreich reported trial ataxia

not clinical benefit did trial demonstrate including MOVE-FA ataxia, unmet Friedreich aspects meet its large results patients whom need. of evidence meaningful for the in key While on there the a of did trial pediatric medical primary remains of endpoint the

the treatment slowing of data loss of to particular, benefit benefit the time In demonstrating the upright potential mFARS in section the long-term scale stability on vatiquinone's support ambulation. a

Given to potential these for patients a profile of with We there requested patients, XXXX. results FDA, plan approval. determine to the and these which FA the were of meeting fourth with a FDA path to if results, the is scheduled in the quarter and need remaining Type for vatiquinone C pediatric the is unmet share granted safety well-established pediatric we medical

CHMP the We support authorization submit of Translarna. the European standard to C the the conditional expect authorization in of a additional this Type to the could the marketing NDA request variation we X third for that second activities convert in to date collected an a month to resubmission US, totality half Beginning XXXX. regulatory number meeting Type of plan expect In Translarna, quarter. for the we with to FDA to discuss data opinion

the between clinical comparability commercial and the on quarter. from awaiting Based we product. timing third feedback, we Upstaza drug additional this submit the of feedback in Upstaza bioanalytical on to can a submitted BLA are we FDA as the we've analysis the expect data previously support shared,

proud Overall, our both of R&D our I incredibly successful execution teams. continued across commercial and am

performance half our to for commercial billion. And first in strong sets meet future growth. Our APHENITY $X and continued us positions guidance well the to from revenue position $XXX well data of the million of us PIVOT-HD XXXX

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

Eric Pauwels

pipeline is facing It the around and treatments team much progress see these of new Matt. to future to our eager Thanks patients launches the needed customer to and bring our product of opportunities exciting the world. is

focused has global executing revenue. in team, to quarter built the strong which significant accomplishments team quarter commercial on customer on momentum facing of in delivered our of growth another strategy We are extremely and proud products. portfolio of The continues the first our the of the is accelerate

continues America, strategy growth Our global geographic Pacific. in and future Northern build out foundation robust markets Asia expansion in East to to DMD to progress regions, Latin our of of Africa continue and the franchise and and CIS Middle continues be while we

our DMD to $XXX first $XXX strong a updating for $XXX With half Now, $XXX and delivering let and guidance Translarna an to is an from are $XXX up compared to million revenue me for million. franchise, growth franchise million net XXXX. turn XX% in of be which the DMD impressive important second quarter million quarter, second the revenue XXXX to Emflaza million our franchise. driver, we DMD the continued to

patient achieved new which in see remarkable same to we in over Translarna, across major in starts a growth continue XXXX. markets. is revenue XX% we For million the from growth occurred added new $XX this and all quarter growth Growth quarter, regions being

large we orders the ongoing in of As the in in Latin Central lumpiness to government see Europe, markets mentioned Middle previously, regions, CIS quarterly our the to and regional revenue some East expect throughout and of unpredictability due year. Eastern America, particularly

turning which net the business of Emflaza. fundamentals Now quarter Quarterly in is over XXXX. growth solid. $XX be the revenue same to to XX% million, was The continue Emflaza

to see progress a important compliance in momentum continued which we continued trends continue the new second appropriate quarter, year along focus will weight-based the start access. as the We high broad in through and provide dosing patient forms on number of strong with

Now, I'll progress current update product launches. on future Kylie? the ask our new Kylie to and

Kylie O'Keefe

Eric. Thanks,

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

We continue first quarter. patient our including across rollout the in second in treating Italy, Europe our steady

to Pediatric Neurology European continue treated that the have Prague. results Conference Society, for recent patients we transformative far, the We the in see thus shared EPNS at we which

the European New centers continue support treatment of excellence being internationally treatment opened to we of as rollout. are patients, the

opportunities programs We second other to and expect patients early Europe, leverage XXXX. also more throughout treatment the treat to both and markets half cross-border international and of in continue access

Moving to Latin and America. Waylivra Tegsedi in

Brazil, order improved strong we quality as of Health. continue treatment the We our Ministry on from profile, polyneuropathy patients. delivery of ATTR group establish In for of purchase the Tegsedi clinical of life the hereditary second of and remainder its choice completed to based

well identification, We continue responses region. to as on treatment as American patient across the see Latin positive robust growth in patient

Lastly, PKU the extremely opportunity as discussed Dive recently excited the we Deep at sepiapterin, are about presentation.

mechanism differentiated substantial upon looking forward the a APHENITY and are With action differentiation the customer-facing approval. need of from both this the patients, strong teams bringing unmet and results, physicians PKU and therapy to to

US we to global of call, activities, launch our team. key leverage This step the Japan pre-launch global market commercial closely prioritize our Europe, established to is global first actively able the markets. strong were sepiapterin the and followed have key at the and to working internal now discussed international team we infrastructure As bringing launch and we by other with

the Dr. proven track the team achieve to commercializing as over rare physician market is billion. and Muntau by outlined $X poised opportunity of With diseases, PTC's in excitement record

rounds achieve well of we an guidance. across With ambitious XXXX all commercial commercial conclusion, products progress set out quarter are our for across excellent half geographies, team. all and revenue In significant the second to our our first XXXX

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

Pierre Gravier

team the to it's an I to is PTC to our XXXX. known press how have Please CFO. you, second to of tomorrow. patient-focused issued team second PTC Kylie. my join release, skills pleasure with I bring quarter to company highlights additional a the quarter this saying, build of and Thanks, by for afternoon several the am, my refer a and working the provide to earnings PTC details. privilege such It years, the I continue want advisor as for thrilled with be as following to begin

million. growth $XXX of the compared net second of results. DMD revenue by geographies. net quarter reflecting revenue all was representing growth second product the second was revenue This Starting million revenue with Emflaza $XX Translarna across line revenue in XX% million the quarter quarter of strong over quarter consisted the top to XXXX. XX% with in performance Beginning of the quarter and $XXX XXXX, franchise million. product of million, franchise. $XX DMD other of of for the $XX Total driven

Evrysdi. to Moving

CHF $XXX XXX of revenue million, which global $XX for about million Second achieved of earning equates was royalty quarter revenue to million PTC.

the mentioned, Matt us XXXX puts $XXX As to billion billion to expected million a revenue an annual $X.X in second in strong milestone quarter Evrysdi $X $XXX when million including guidance position surpasses performance of revenue. total achieve

million the increase $XX were compared compensation expenses in second second R&D investments of for in $XXX the non-cash of Non-GAAP $XXX comp advancement reflects excluding $XX non-cash year-over-year expenses XXXX million in the stock-based clinical quarter million quarter The XXXX in million R&D for pipeline. additional to stock-based of excluding expense. expense

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

the the R&D non-GAAP $XXX from $XX $XXX full million anticipate million end December as as totaled XXXX compared XXXX. the million positive now million quarter to and cash data addition SG&A mainly of cash marketable the the result of XX, of we sepiapterin June earlier, agreement. year first based from equivalents securities the readout on for due and XXXX Cash Cash, by $XXX restricted expense XX, and of as of to $XX $XXX a approximately increased noted million of Blackstone million. Matt between As

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

Operator

from question Jefferies. first comes our And Shi Kelly from Instructions] [Operator

open. now is line Your

Kelly Shi

how you the Translarna how taking for you. My anticipate question great order should is in Can you a the QX we did so question and comment large mentioned in on from sales government and QX the the for Thank regarding sales? first impact order future benefited sales government congrats you Thank my QX Europe. quarter. QX on

Matthew Klein

QX. question. let the between some was I'll revenue. very the it another and quarter you thank much of more strong Kelly, on Translarna Eric details differences provide for QX Obviously,

Eric Pauwels

the for Thanks question, Kelly.

that consistent it's we've It's First been years, growth seen geographic the And mean actually occur nothing past invested of franchise. pleased generated to new the million this of DMD expansion. with and you've as we've in we're know really $XXX year-over-year That's time. at that I we quarter-to-quarter all point think very will I in in quarter. last compared XX% year. lumpiness really

But Latin regions timing the these since and those as CIS in East our consistently size is we've Central Eastern of in regions of seen particularly expanded unpredictable. America, in the government large many relatively and orders the orders Middle orders Europe and the and we've

However, Western the week-to-week US, the month-to-month are orders and we've more orders fundamentals And that to and still will unlike Europe predictable say tend continue. would from which of the and the timing be those I received large same.

to Our business grow. base continue even in those markets

starts patient discontinuation of levels dose rates have very adjustments compliance, and New low occurred. high

the to orders more closer year third will and at get achieve if we've in will we quarter. to of in defined end as We new the guidance guidance. fundamentals be the markets level be anticipate point well that reflect likely that. for we'll place. time upper the The second And really of those and will confidently closer adjust markets size able of the third the timing as to the you in as the both quarter, this in and and if the mature raised solid So our you orders and of are half

year. around remaining So timing on now, orders But we'll an DMD second color tuned very just update more the the a feel confident throughout we in the good the in of purchase and stay franchise have bit group Kelly visibility and very little provide have we for as half. QX we right tailwinds more

Kelly Shi

I Thank And two you also much. quick have follow-ups. very

First, out for quarterly reporting revenue? trigger can start Upstaza's you any to guide PTC

Matthew Klein

Kylie, want about? So do talk you to

Kylie O'Keefe

Thanks, Kelly. Absolutely.

We about I guidance spoken to guidance very of clearly treatment patients we've that to those as launch. last into from think rapidly provide was ensure that So delay a while that did providing we expecting we year we the intent the to treat patients. indicated with weren't launch expected

of we've As the in first very it's months said true forecast to XX difficult previously understand the launch.

and to that And a of more clearer so seeing registrations we'll provide that into through pricing how move on particularly ex-US a Europe garner in country-by-country be reimbursement gaining as clarity we're and more much and clarity. we period we able and a process, throughput, understanding being more number patient granularity

that question. hopefully So answers the

Kelly Shi

Yes. one Thank one may. last if you. And the I

strategy on analysis curious have done can like maybe to group Type the what share of filing, meeting ataxia whether focus with a regarding patients? kind So of C a improved and like FDA also in you genetic on just efficacy group Thank marker and you certain you. some some and also

Matthew Klein

Can for question. the clarify the for C thanks Type Kelly, indications? you which meeting

Kelly Shi

FA.

Matthew Klein

FA, yes.

fourth ataxia, said targets disease the for Type And be one about keys talked about main to of upright take important of it not is drug FDA data transition didn't meeting on lose time think goals would one and to key ataxia Friedreich we the in and granted disease was hit the we progression loss the ambulation. very to you drug the the That time a slow slow endpoint as strong development the of the the C primary of point any disease morbid in therapy number to particular as a aspects in the had So that subscale. of if When and patients of from will we the any we ambulation. Friedreich requested quarter. altering disease we the while place and as of that stability seeks that of try that to

of to ambulation. of has shown be time mFARS key loss of to subscale stability upright The been a the predictor

it least placebo. done strong And endpoint loss delay ambulation able treatment we've some of perhaps that, between had signal a eight show should and work we've analyses So an endpoint points two pre-specified is difference particular effect a we the X.X subscale at some months fact with of more do. to that other component about of showed been and in primary that, a to the that that to translate and of with fact that one and the as of

of FDA magnitude intermediate we effect about fact And the upright significant delay statistical an we've on the able an we which able loss to likely able where point able loss to significance to a demonstrate we a of disease transition ambulation. able endpoint is the are clinical delaying been to ambulation. to and showed strong of demonstrate that, key can clinical be a long-term regards -- that of path come with approval that we're provide therapy really the delay we're likely and stability to talk benefit FA to to longer-term of So goal potential accelerated morbid

the has discussion regard towards again in slow a set the very positive directed forward FA. So the ambulation the vatiquinone have that that pediatric pediatric with aspects Obviously patients. And key to now primarily which we strong approval that data of agency. to study very particular record safety we strong with a goal look in was but of really patients then having is with many included that's patients Skyclarys adult data a loss of ability to

Kelly Shi

Very helpful. Thank you.

Operator

And comes you. our next from Corwin Sami moment and next one question. And question for from Blair. Thank William our

Your line now is open.

Sami Corwin

Thanks contingency approved? CHMP And question. as follow-up have I taking got And you question. why happen supposed pushed? first plan in have to then my any thought guess, originally that opinion there I a clarity for is it guys. was Hi, the to if I there Translarna any doesn't do May. for get

Matthew Klein

more and pushed how during quarter. typical that was into and timing forth such of turn together process Hi, the timeline was third type as back XC put based the and for Sami the of one the Obviously, a at HX thanks two end forth on CHMP the we there HX. the back question. initial That Actually is questions variation. thought

of marketing marketing benefit really conversion standard to is to ability the of So, knowing states existed able statute we being conditional remain confident authorization here from as highly the authorization European the bar the achieve was data our that that confirm that to of placebo-controlled in study X. benefit in voys the based expands now we only And that which XXX over at course but registration confirms XXXX set a time have it. our first on not actually

that's a XX the different population the in North variability a statistically Ambulatory well significance all-comer population benefit shown showing as which endpoints including and significant on The Star timed function of is ITT test. Study number on as Assessment indicated

the data of confirms body a evidence on is builds really is this the but that it. not only So charge, which

Sami Corwin

about And of the million guys capital cash, through near in about you then earlier commercial given have terms how your regulatory term versus in products submissions you in Great. on $XXX and either are deployment be your Thanks. franchises research going that will kind thinking stage focusing of pipeline?

Matthew Klein

Yes.

about we've year through the talked us infrastructure and other are us next capitalized the -- lot I products. to take and place launch PKU we having get to well think as us PKU talked in to Sami, launch. this the to We've about a take

a development We also and have discovery robust infrastructure.

capitalized are the So next to one we're and programs occur to the expect and forward all the prepare the pieces for move well in to that we launches well there and so positioned two years.

Sami Corwin

you. Thank Great.

Operator

And next Cantor next thank from comes you, Fitzgerald. our our from And Kristen and question moment for one question. Kluska

open. line Your now is

Kristen Kluska

Pierre. taking questions Hi, everyone. and Thanks for my welcome

and pre-NDA questions what just for looking main here. will wanted feedback you're First to essentially be at what meeting ask the PKU

Matthew Klein

question. Sure for Hey. Kristen. Thanks the

standpoint And created components efficacy analyses. need how through in together what How do CMC a package. line safety of the often efficacy terms the going checklist pre-NDA package analyses structure the So database then are safety from non-tox and NDA. the of around are obviously the we meeting the put clinical standpoint of focused is with we integrated

that and with have submission our to meeting. the able align are gratified is So to it's granted. in be approach pre-NDA agency quarter. Obviously, quite we'll forward the the a the move we expectation fairly with meeting And to standard fourth

Kristen Kluska

substantial platform, of how And maybe that. we've to experience wanted think splicing you Evrysdi along have is ask adult off the launch for But data? in here. you're sense community in kind that the you especially disease. that with with with if some just based about the from talked given you've Thanks leaders the to from there Okay. the feedback And you has your then, often of any early neurologist with been with community, synergies now much the Huntington's a heard pretty the particular space question progressing overlap the initial

Matthew Klein

to can Huntington But mostly juveniles. SMA, a turn be more the then successfully a patient us of I'll I recognition Kylie it the it about physician obviously oral just to obviously, I power work compound scientific provide absolutely. both but as of this. how is in to with would the for is over whole as able and disease. importantly for there oral an Evrysdi Yes the we're talked with a splicing complete our going and give Obviously initiate platform. bit a detail to and to and of compound pediatric to on little was And develop Huntington optimize, adult communities, say being bit in as about in broad discover, such well -- color general, now we SMA adult a lot blueprint disease able splicing brain

And both for Evrysdi say of communities clinical as patient that ability color trials Kylie, of only the splicing physicians. on as the ways small the and has meaningful the we out deliver a want how of in about physician more it well safe the but to recognition experience to to thinking with and and is us molecule path you many results. don't would think working an oral provide I getting in any set and this because I not also the doing be able deliver about platform we're effective if know power

Kylie O'Keefe

absolutely. Yes

that our amongst all physician sure learnings physician, different there and of business I specialties making we're the as across team sharing in what overlaps said, I that very take seriously, a as would think are making what things we which we between teams. is of our one well where commercial Kristen I sure do addition we take call to as that Matt add target aspects think points

that of obviously consistent as Huntington's the look and it's we of specifically is movement parts to our within if business. sort between So disease has overlapped other But adult. said the more neurology subspecialty things of is Matt one Upstaza at

we're making it's overlap of there are not an so components And overlap. but sure entirely some

we're that having we're points so different teams. case sure touch the And making where in we're that that that sharing consistent across

Kristen Kluska

again. Thanks Great.

Operator

And from our David question And from next comes thank Lebowitz Citi. you.

Your now line is open.

David Lebowitz

Thank you guidance is to much When in half? would second things decline very the work high conservative in my at half. actually very for taking of revenues most the the the Duchenne Are question. for -- is the year for expect just or second implication that guidance we a end you possible assume full the the here the we that of assumptions out that making think should the and decline year by XX% well as in could guidance differently progresses,

Matthew Klein

David Eric the Translarna you I'll guidance. in I that about the year Obviously the been and growth the how we're a for Thank Yes. about talked thinking achieve able for about a and question. franchise. had much very total bit half dynamics let the bit talk much reasons how we've in to little growth second

Eric Pauwels

of to think continue I growth. we Yes, first all, see

think So of trying really these established have in that is we what lot are purchase where orders a countries to group to inconsistent. patterns relatively ordering ,David I we're modulate do

unpredictability the the can conservative orders in size the being the And of and a certain bit we're timing so certainly areas while of play.

which the European have Middle few unpredictabilities. some their Eastern and as Central from in last markets terms started the Latin to patterns markets are countries in continue we and in particularly the we've American fundamentally orders markets still the as well of new of these where see much Eastern and what sort But ordering stabilize of seen orders all markets years to in there

have because of And size orders very such of fluctuations of these the Brazil we the strong. of a areas are timing, fundamentals markets as between still But and quarter patients amount one the the patients large or certain can next. that the other key in the have number Russia and number

that quarter-on-quarter given we growth. fact very think And seen I the we're have positive

as have think mentioned nice think we I tailwinds. I very So, I some

or I going guidance going think it. the to confident able set we're growth. exceed continue achieve And we're very out see we're to to we've to that that least be good that at

David Lebowitz

that. very you much Thank it. for over PKU. to Got just And jumping

Given the a I the protocol. And pivotal are Phe adding of diet utilized part of you trial as the the trial the know diet. OLE into portion that the

diet patient, on can to perspective, requirement a the not or for be labeling as labeling a allow top will a include diet? the a serve a OLE of to ultimately From component

Matthew Klein

Hey that highly also as an confound David we don't the to it's a a diet. that the different the the we've your many trial is and of placebo diet diet. to so trial And key the maintain of good sepiapterin liberalization patients about an importance phenylalanine and the restrictive the ways. question. on that factors being in all holy allow stable aspects context the patients. control takes for obviously talked obviously, enormous of the a ability Obviously, and the our lot PKU families having question. in of in we in differentiating results. It's incredibly stable many, diet is have to is their you important it of able of for Their -- really of understanding effects liberalize one sepiapterin we diet within the relative referred But know is Obviously, to grail impact levels still

and seeing both tolerance We we're as RDA tolerance in signal data deep early of the the thus Phe and that future the far -- a as come are in the be commercial that at we in at levels of the forums SSIEM seeing have the face from few Phe patients. first the intake protocol. continuing to weeks data the exceeds forward -- case back those to continue dive that through data to look that extension, future. in And of PKU that open-label go that in we patients as set in that Phe saw more data presented And well we're many sharing in looking as Phe and we're the readout more in in

their stable about but to them patients. of much sure. is in would very what say to likely the drug in not we're patient life how and and the therapy but that important since uptake talked is reality life possibly all value what But that will in to so protocol liberalized do diet nonetheless an have be necessarily oral diet some to not everyday physician they is. be in component daily more allows not interest The And ability that control. But there. tolerable perceived patients finally able control, liberalize obviously, prescribed, is It being label so going is and that only diet finally will in the what their to impactful, provides just which increased have the is diet

David Lebowitz

Got for you my much it. so questions. taking Thank

Operator

And you. And next our Eric JPMorgan. question Joseph from thank from comes

is open. line now Your

Unidentified Analyst

This Hi. Eric. [ph] Hannah for Good on is question. the for taking Thanks afternoon.

Just of to FA presentation wondering study. be a maybe from see fuller might update we data able the III when or Phase

are if As present conducting and you additional a wondering plan FDA to there's analyses guys Street? the just take considering to to to the these

Matthew Klein

for Thank Yeah, much you Hannah. the question. very

the able achieve scale. obviously, we But on to upright quantify subscale the important loss the subscale. endpoints number components prepare stability difference the data to to of positive the we the based regards -- the of expected benefit results and around the a stability key likely of a analyses obviously, as progression two top from we done the including being line mFARS, slowing particularly we So the -- in have bulbar ambulation secondary as study able additional long-term upright well shared on the the of and were number with

a that some sharing So the in investor would and publication a more part we're future in forward we as the going to those of or those look do to -- work to community. showing will be that available presentation analyses either continue to area

Unidentified Analyst

little then be a question. a Okay. this niche And more bit of may

study might for just So for the diet from specific patients? that physicians is physicians with wondering and your there of would liberalization PKU, increased conversations patients, considered to a just achieve or tolerance be percentage that amount meaningful both clinically Phe and patients

Matthew Klein

we burdensome and nicely how that as I think Professor patients. for I would is highlighted our restricted very say dive a in highlighted Phe diet deep Muntau

gratifying and quality incredible be for in obviously physicians, but intake an increases So patients life. -- even important both slight for would in of benefit their

that looking protein patients an as at would individuals incredibly data beyond would data able our anyone Of daily have hope recommended be to to of get unaffected are and standard of course, allowance exceed able think the are even because seeing in for maintain a we're far I when that those control that what and their diet would powerful part still therapy. that

physicians to us. gotten data. excited the us that patients Muntau that for about they've very something and in observe other are far excited been about. herself very be Dr. Obviously, said have so So as to encouraging And able this seen what very it's has she to

Unidentified Analyst

Okay. Great. taking for Thanks the questions

Operator

from And one for Stanley. our thank you. And more comes next And question. Hung from Morgan Jeffery next question our

Michael Riad

quarter for This questions Riad Michael on Thank the and congrats is Jeff Hung. on and for taking the you progress.

favorable have US presumably timeframe. you a if So in see close all regulatory your launch programs in late-stage outcomes them we'd

OpEx the savings So $XXX thinking an annualized the for immediately launch so about million company the And in if you especially would in how context option? in much. them Thanks was XXXX? is launches, preparations that simultaneous of multiple

Matthew Klein

questions. forward would thanks to opportunity. look that so for Michael much Yes. We the

work these infrastructure? do as more would color for to building let welcome to bit for is that. patients. important that that. been Kylie well we We're provide more well We're set have activity the me little but color do we little Kylie to therapies and just short say get you up We that. just for a as a answer I'll quickly possible provide let funded want around as this always

Kylie O'Keefe

Thanks Matt and the question. for absolutely. Yeah thanks Michael

said but neurology capacity launch. focus the we'd about a talked a we this and and and to includes metabolic. think have, have lot quickly problem Matt really both And infrastructure capabilities up As team globally strong commercial that as potential welcome gearing obviously in place on I a PKU to definitely for we've very is

And we the place sometimes need that to be launch. patient takes that to at all advocacy we day prior to payers of done groups In to to what's a The KOLs lot often necessary of start on up with we work two relationships are has with that for understand successful build addition understand ensure don't of launch. trials one the that to relationships to years obviously team ensure ongoing launch. while build to

And to upon or good there. successful require resources wouldn't a NDA regulatory be positive in it ready movement discussions towards be and submission. successful to we're So additional position infrastructure

countries colleagues and to unmet metabolic definitely in I more know with able these patients many of in to a of medical the neurology So, And speak are are the the need bring spaces. world around high lot than go. to the we for XX ready therapies to have footprint excited we and to I that be we differentiated opportunity

Michael Riad

It of That's maybe last on bit down comment like the Any from for in rate on quarters. then little a should expect? helpful. bit been trending few question. one commentary All what last royalty right. Roche? Could Super. really the more a we housekeeping Evrysdi a little it's a you And seems

Matthew Klein

Sure. fill that in? want Kylie do to you

Kylie O'Keefe

Absolutely. Yes.

So every between the royalty XX%. from year to for rates X% Roche are tiered

is on basis. that annualized So an

with they're year X% the XX%. the up tiered every at to start So they and beginning

progress tiers. we've the So in from We're million second different tiering of third near almost we're the through with royalty and the towards year first sales that half perspective tier throughout CHF they the seen as year up the rates. up of XXX of towards the

I down. basis. But If that's, going wouldn't anything up. they're an annualized say go that on So they

Michael Riad

much. so the for Thanks Thanks clarification.

Operator

our Thank you. And Brian Abrahams RBC have Markets. we from for question Capital next

is open. line Your now

Unidentified Analyst

Brian. This is question. Hi. you Thank on for taking for our Joe

possibility And authorization CHMP renewal? Translarna back conditional to follow-up. a potential Just going I market of about Is outcomes a with some remain have there Translarna, that the the with the you talk the on opinion? can can annual of

Matthew Klein

Yeah. Joe. Thanks for the question.

strength I we're data authorization time to of to data the do authorization. registration. talked said to about obviously standard the confirming. conditional convert in we're to requirements as variations And the ability existed And at that the submitting, meet building of the we've ability So the our of and the the given two confident already is on that that type earlier,

the want portion and control that basis open-label was XX-week XX-week were the open-label if placebo-controlled, that's the the the longer-term, as portion of the of XXX we analysis placebo section request whether obviously decide they part CHMP possibility. which data, a analysis to portion the collect conversion on presented Obviously, of continue formed the us that's of to to that included in the

a STRIDE's an of totally on direct loss having that points disease impact therapy -- that's of of CHMP say, the want the function say really able convert It two pulmonary real-world key loss data the DMD measurements possible. ambulation to they of of to and morbid great. more is also we for transition the Could fact first the long-term provides we're for and time capture benefits the

there conditional So they now that say the for as mean business possibility is would absolutely that usual which with continue authorization Translarna. course of for

Unidentified Analyst

this has to And was on the time And Got touch Thank pathway, I'm to has been the additional meeting it. to mistaken, the helpful. from I meeting very scheduled? requested meeting not and Is FDA requested? response there to typical can a the get Type back also guess Thank if also That US been you. you C forth before back how expect is have once you much you. month.

Matthew Klein

in our not resubmission the recent of came and meeting three pathways said, now XX. the had a discussion division. from US, placebo-controlled particularly that XX the we the with based Yeah. about Study potential so Thanks Study NDA division we request we X, given data an on the to volume only have And Within Joe. Study talked where studies,

data of most long benefit, of the we've to of over that translating function. been clinical course meaningful are way that's loss STRIDE recording time in to benefits recently long-term that the mentioned, those which of the possible significant provide importantly I ambulation the But evidence pulmonary and time trials loss very of strong the

the get can we C about and have some we'll how resubmission. of analysis data including of put discussion together any for we mechanistic our The would generated agency an the why data said, meeting be suitable that years well, package we the over Request don't, Type all together, a that NDA we want.

As mentioned, not we yet submitted have request.

or is be typically is a typically in days. let the couple respond within to Type C that XX will request request date for a the following roughly We of if submitting meeting granted us timeline August. to know meeting And meeting meeting They weeks not.

Unidentified Analyst

Got it. Thanks clarity. for the

Operator

from Colin one from UBS. comes our question next And you. Thank moment Bristow And for our question. next

line now Your open. is

Yihan Li

taking is Yihan on our Congrats question. Hi. This on the Colin. and for thanks quarter, for

our the So I on PTCXXX guess HD question is program.

in lift hold, there's A from partial data already feedback So submitted the data hold? additional FDA have if yet you any FDA the if the of well the for the clinical just to wondering safety potentially terms as Part the as requirements to US and

the part dose so second same the escalation. also And the program, on for

brain. low like noted more reduction our at be determine will data XX% data And HTT XX% seems on some doses like your it the based further to you previously of very to reach So likely to targeted for the goal escalation. you XX-milligram dosing need

data you could clinical escalation decision? when just we set please So next this so your for to wondering, the need us see you be will the FDA data, kind team? update? potential might to to see holding let with And in the much. what Thank dosing conversation the this expect you know And of will make included

Matthew Klein

Thank was that in coming FDA observed to partial in you clinical we have No data regarding very on were PIVOT-HD that and first drug Obviously, very US. much the of very question the tolerated. demonstrated and evidence in see the June the neuropathy, I'll events, for adverse of safe first first the the the questions part been other serious presented pleased hold start no with Yihan. cohort patients no peripheral of that the through NfL therapies. well spikes we

strong as overall we So hoped could record the have as as safety for.

are we to Obviously continuing monitor safety.

of XX-milligram at data support cuts it dose. And also mentioned XX-milligram continues to the that We they X-milligram continues of and decide we support an independent to have as on study the do continuation that also is. if meet so that we the the as escalating indicated that based into part to and DSMB would looking to would

our still data ongoing we'll process provide of is update support We and time. obviously argumentation the That have US. study appropriate the and at the the submitted and to the safety reopening agency in an

on question the dosing. Regarding your second

higher exposures in seeing targeted to of You're rate to X.X:X as XX%, -- the cells. were correct. well the to we're range again approximately we within gives of in any believe the we that lower of with ratio XX% plasma both blood blood CNS within CSF We reason protein we a XX-milligram of based to that us what XX% dose observed believe as reduction as on in huntingtin of shared, terms brain

And we on are collect XX-milligram the continue is range what cohort. X-milligram so believe we and is in dose the to data said since we

want. over of will that the some data if cut we second we those XX-month dose available. are confirm the at that level the And biomarker we will with data Importantly once for present later part PIVOT-HD

use time to that going XX additional probably point level then any And we'll of decisions, that -- higher dose milligrams. to inform regarding

important and is in that's such provide taking able clinical ,and an an effect, that dose provide stable can position important important right molecule course, be oral steer patients. being incredibly benefit engagement us development could forward And peripheral that with factor Of a potentially level note titrate. able for safe, to program that as to pharmacodynamic but to not have biomarkers, this only to we're information protein, to incredibly blood we in important regarding target it's huntingtin

as now continuing five So, we're for with XX. the and

data when And already if we need go We and And update, on when have the XX-month approximately had the look like. from that nine us that the will terms as we data support I months to. that DSMB in it will provided of update. data be again, has we to mentioned their next dose, okay three-month based update decision and biomarker what the expect to decide higher

Yihan Li

That's helpful. very

just just quick – sorry, clarification. one So

data? you provide patients Stage So two do nine example, terms three the you just asset -- will Thank for program, the early will you the Stage from be in months trial-based additional of next or or

Matthew Klein

Yes.

good So, question.

in two on expect update the the XX-month early the months right? A few on that right? three-month And then June, go, So to data in data, first that Stage obviously, So to Stage a data able those or when provide patients Part will we the and we data available. first updates to we additional data then the be approximately patients nine have we'll presented three those from X-month have obviously, more

the will the we timing more that clarity that, yet provide given we have haven't on time once We precise and for date.

Yihan Li

Thank so much. you helpful. Very

Operator

next moment one for And question. And Thome you. from TD Joseph our from comes question Thank Cowen. our next

Your line open is now

Joseph Thome

questions. my and companies It Hi, on here. like patients the success one there. therapy approval of varying ability to are seems having you a the US, good gene Upstaza. Thank for afternoon. treat after various in Maybe taking

can which is you potential during of to anything do US to year? prime of I approval, I to reimburse half hopeful payers sometime guess, update kind ready come upon expect be review the will next So there the process Upstaza in back

second. maybe then And

like I was Blackstone going months. what at the $XXX couple guess earmarked you that by there comes the pipeline depending next BD. for with contemplating in get are on, I million deal, like Thank you. of are end We months? you based over know using looks ever next to all up the few regulatory the this what the of on believe year in feedback your previous

Matthew Klein

the you for Thank very question. much

patient motor able that function providing that that a preset be walk get where physician the course increased in ability virtually desire therapy. we therapies dopamine incredibly no on how on detail incredibly payers. let that The in we're gratified treatment Europe as obviously, some I this give in the will question of But add studying Kylie the Upstaza, terms US we're first with market. those gratifying. gene and both we with continue I'll so included market substantiating dopamine study as an and make fact continuing no part sit of in well of one to data get And Taking see and therapy. Canada we the a US. we're effects to itself we seeing and therapy. strong see again, this hopeful, then very and is it them It's is We're much It the that as the does who treatments to crawl that have as to production, the to that of kids did transformative and sites

Kylie, do bit about talk preparation So U.S.? you for a the little want to

Kylie O'Keefe

Yes. Absolutely Joe.

of have morbidity need payers to mortality queries of the to they engaged and that U.S. date. therapies disease-modifying obviously well And with and do of around AADC are understand team engaging standard the happens types of might the that we what put this the ahead front we of of available do ahead are in what education be us. any especially some impact. or in the high to roadblocks with are And of there lack what that that We the launch and been of work U.S. process all out care, has and actively is and budget disease rates approval obviously

which therapies. lot front in short-term, also short-term, As of not transformative companies does with in and durability, data is not other put but package How in the long-term? have only of your payers said, of perform but Matt treatment in the in gene questions one that the well just the data with a a strong also

well confident very data been feel totality We Upstaza. package received by in very, proposition And has the there for U.S. payers high we a has to have the very and pay. willingness that been around value

Matthew Klein

in in regarding important more portfolio discontinue decision as the your appropriate And the and do and preclinical months. part of add we'll be BD move more you obviously funding Joe can and some to on gene towards they a second the -- Obviously, prioritization. a on therapy question want lot focus the an our learn done strategy opportunities? more to And about of own coming Pierre, lot obviously, strategic color that. we've programs, BD work

Pierre Gravier

Absolutely.

aligned So our company strategy as opportunistic being the our Matt about expertise our and BD. said and leverage well to the is on team and capital

group options proud comes very structure. partnered have life strong capital to as Blackstone, always to expertise there we a to evaluate As on our science we're our such it funding it will be and comes with with

Joseph Thome

Great. much. Thank you very

Operator

for for Gena And one you. Barclays. question. Wang from question next And next Thank our have our moment we

now is line open. Your

Unidentified Analyst

on It's for Hi. Tony Gena.

more Going could back what you but I looking for? you range, add of know you on in protein? kind XX% there cutoff to specifically threshold about be kind of be just is specific you talked CSF might the XX% some would Huntington's program, potentially for of looking terms to color a

Matthew Klein

there's that. lot many disease discussion many biomarkers position, that means where you much it study. about been for how Yes. what we're of important Tony, Look we of a and in a protein, measuring it the there's CSF very thank do question. the Obviously, we're in measure things like Huntington and

sign obviously thus measuring We're protein. which has neurodegenerative We're nerve terms be important safety of to potential of also been a trial. the mentioned in PIVOT-HD any which injury, we've NfL, see CSF disease in far in of measuring I shown very obviously not marker

Huntington's also an as to authorities But that that is huntingtin a protein stages detected to disease first by use. well What [indiscernible] are at including marker broad has levels an look neurodegenerative neuronal efficacy injury in been approval It's can not less CSF. the indicator do look disorders important early of at bit mutant the interest as neuronal in are accelerated that there's that efficacy. it's CSF regulatory so huntingtin an characterized low from NfL We we and there's detectable that of protein characterized. in loss. by no know

level over neurodegeneration the a course However, CSF. of in in of huntingtin mutant progresses, as protein there's the time increase gradual the

neurodegeneration. of a tells that marker is So that us a it way in

believe a less CSF they're ability is CSF. such and huntingtin true when the injured it is the that to if of favorable are during that and we marker protein they brain on they spilling if less effect the we likely is and into an coming protein from nerve become cells huntingtin that that cells So having CFS protein the lower course die are killing the is injured, process from important

So we about that's is understand say to it. what all that

if effects is of you stabilize number it that's disease increasing good. is the trajectory market specific time could at worsens, challenging. say I a in But change to to obviously CSF goal over if than other don't think obviously, look protein not we with think is I terms on way of marker very think able an ultimately exact threshold mind. that has but meaningful about being a of what that Quantifying lower the have a bit is any anyone disease our you in as the and

reaching be And points we'll going as benefit as will steady alongside of dose also MRI the Huntington's But important at that lowering. we state the what at the for an call able are well as disease patients. CSF we're in provide before other terms that early we're protein the we that together that able NfL in in important markets such to to on achieve paint time did picture to cohorts that in and share be volumetric of will those PTCXXX we look observing know very ultimately XX-week lowering

Unidentified Analyst

Got it. you. Very Thank helpful.

Operator

next Thank Bank America. you. Ahmad And from of our Tazeen question comes from

Your now line is open.

Tazeen Ahmad

in $XXX in does to shift savings about XXXX about EU to might closer that as scheme you mentioned could number of your the commercial reimbursement, described million there before, would room the What be for now Thanks. And of the of Hi. really what beyond opportunity, of you're a a take from at approval what annualized then we kind and look terms upon or so portfolio? an or have you for that as far you benefits additional matter change cost clarify to closer things? get you standard grand just practically as either not cost at savings said

Matthew Klein

question, much very for you the Tazeen. Thank Great.

the more every go the But from conversion talk point think don't importantly commercial standpoint the the a first renewal. regarding about to I'll your perhaps I type question to and On Kylie of let through you process year of getting regulatory the the impact more a that. have question, is

Kylie O'Keefe

Yes.

very easy question favorable to it the Tazeen team the maturation not think to securing favorable of years with which a but of pricing is the has And conditional answer the pricing and in benefit, really to being what corridor So maintain your I product, approval. is secure remarkable not access do. over able done job a market of around only

the it upside to we're already a comes registration post I don't access. think see door discussion when from the and true countries upside that a in yet. pricing countries launched would I where that There don't in But some countries the to a think huge have is with benefit the market we open are perspective that smaller conversion. we

Matthew Klein

our Tazeen transition really the cost terms to of about Great. savings. of we further in Look regarding time on And leading we cost. at to that question of ultimately reducing in data and talked were OpEx potential CEO focus particularly were to really PKU we looking down then one the had as as wanted focusing the second your our our things the positive we reducing that we a and at position and start studies with opportunities

time involved in obviously therapy in realize Obviously We XXXX shared we spent team and gave that savings in have expect changes is into in million obviously of those impact this. of the to impact we've preclinical we on a shared bit the a in planned programs. greater the headcount time a gene limited impact decisions the lot why reduction cost of than looking May approximately the that a So, discontinuation these in spring associated of made that which which in an XXXX. cost to savings XXXX the late reductions we with $XXX

also going portfolio. Of be course to to are a going we at continue position in where looking the we're

investment Looking case seeking let's at the to at opportunities savings. that's to well return ensure that cost we're not when on advancing say programs that as have opportunities look a reasonable as

this to $X year. of that. revenue We're million We're looking excited about at billion $XXX incredibly

forward be as position forward in move billion believe in potential a the of programs one breakeven which the we towards looking where expect we point thinking an We really we our program over $X about day can should and opportunity we're launch profitable. ultimately future other market and the be to PKU moving being

of strategic we to very program how OpEx to we about to spend. continue thoughtful about and about that capital think we think do our when utilization be And have and

the time forward. build you're be continue something So, to do going we that as correct. company to is This we over

Tazeen Ahmad

Thank you.

Operator

question And Sachs. Goldman thank from Choi Paul from our you. comes next And

is line open. Your now

Paul Choi

with side on congratulations the commercial on afternoon to Good Evrysdi. and first question My you. is the quarter. Thank regard

of wondering first early just the that days Your I'm the decline you partner could on Roche basically there market maybe comment milestone the your if reported trends guidance for XXXX. a since sequential launch. embeds given

Huntington's. question also on second My is

you. clinical with to hold contingent in clarifying enrollment discussion regard and there? upon pace FDA of Just factoring the enrollment progress with Thank the just the how you're pending the

Matthew Klein

Paul. Thank you much for your very questions

the take me second Let one first.

trials of along in the US, were study a get having the all outside -- but of countries that the said and enrollment world. can around to enroll in development conduct So, trials started this that we've position a in we organization terms look that's benefit run global

in We saw you the as have And to this deal amongst patient in Australia as a well study enthusiasm in up Europe all participate a and running trial. great in in well countries number countries the Canada. in there as a of physicians as these is of groups and of

in slow readout without in to-date the study continue to I in we're U.S. pharmacodynamic anything that US we're the down participate knowing will very to But participate. participate in that Obviously, the to the it showing to patients well-tolerated study. there to the desire going that enroll study the to sites safe for doing having to the to in if the of only that reopen position heightened will US and say the drug the opportunities shown the be in desired the physicians is effect patients of interest. data interim obviously We're is be aware has and

Regarding Evrysdi, do take want Kylie your question that? on to second you

Kylie O'Keefe

Thanks. absolutely. Yes,

patients driven of we're continued seeing in factors we're think I is as of treated. see board. as Spinraza from a total therapy-naive by across continuing continued being patients Zolgensma that growth coming being both switches by It's well Paul things And driven the being is different Evrysdi this treated. the the with number one to and

XXXX. have fact we're most trend market the for increasing you've that to with about and global world of -- leader end be they also markets market about Evrysdi-treated or are the an the Roche leaders also that They talked expect patients. be And global market ex-U.S. also patient In recent the starting in major to seeing leaders to US, the infant are around heard CHMP segment this by talk the expect opinion, they sorry we continue

will purchase quarter-over-quarter government-based is and the us to different Translarna related is Eric you one the that to international have business. directly what is that's do of the large with see And it to that lumpiness they and throughout think so quarters throughout call business. this I you things that Like that lumpiness. we with the Evrysdi, see what see fluctuate on which orders consistent touched expected

into little seen QX we of I XXXX of XXXX rest year. bit in a from the think but QX throughout continue to growth the lumpiness with we've growth of the expect

I you for position in a good million that think we're $XXX touched milestone on. the

If the year you half through the we're first at past look halfway. of sales

sign. that's a So really positive

we why from And $XXX XXXX full that's milestone. revenue guidance perspective remain including with million that for confident that the so

across that well. So just business see our Translarna lumpiness we as

Paul Choi

color. you Okay. for Great. the Thank

Operator

I would over questions. closing Thank now am Klein remarks. the you. I showing to back further for Matthew call to no like turn Dr.

Matthew Klein

forward productive proud second and us we achievements many second look you for extremely in busy XXXX. call the our of Thank on today. I'm half of again to and the a quarter joining

all joining and call have a So thank for good you the again evening.

Operator

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

now may disconnect. You

PTC Therapeutics (PTCT) 3 Aug 232023 Q2 Earnings call transcript