PTCT - Earnings call transcript Q2 2022

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Operator

Good day and thank you for standing by. Welcome to the PTC Second Quarter 2022 Financial Results and Corporate Update Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Kylie O'Keefe, Senior Vice President, Head of Global Commercial and Corporate Strategy. Please go ahead.

Kylie O'Keefe

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

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

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

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

Stuart Peltz

what share Kylie joining us to thanks and and quarter a PTC's year to expect for second company. good results excited I'm Thanks, be today. afternoon, for transformational I the everyone for

which stakeholders. products, products. discover platforms several pursuit our has five Our doing of PTC so, in and, programs develop seven to of mission significant them innovative for generate this PTC development and with is all rare disorders and very to Today, to at been to therapies patients marketed future successful scientific mission. and bring value We've create in

quarter two deliver product pipeline robust that towards new achieving therapies continue goals XXXX. second ambitious progress We at every steady-state years. build a for will We three the important made potential our in to of new to a

growth like significant to in the advance clinical highlight and continue strong towards quarter. achievements We to multiple particularly several achieve revenue to milestones. I'd

revenue increase second strong This $XXX quite our in in revenue the over quarter, which revenue growth in expected trajectory puts to XX% and with is a us in remarkable we me of to strong achieved XXXX. our franchise $XXX position between the total million quarter continue The million. in a remainder million DMD of Let begin growth target achieve $XXX throughout the and XXXX. is good full-year with

relevant significant also reported step an which population. we number a nonsense-mutation to broad forward plan XXX results, across XXX, of results Based Translarna. Translarna patients of EU the and study functional marketing to Study line in Study In statistically outcomes request with important We benefit a on top these conversion DMD for authorization intent-to-treat we the demonstrated authorization. was for standard a conditional

EU, for the to a and look international with we markets. results the other In path approval forward discussing addition these and forward potential to FDA

these strong recently addition, Matt reinforce these therapy and with our portfolio into ever our We the EU European brain. for results into and shortly. In results first directly administered international products. the gene markets. marketed five commercialized value will more is This will the by Upstaza Upstaza detail therapy expands product the proposition third gene in authorization Commission. the marketed approval payers marketing in announced to the go

patients Upstaza PTC at is to quickly AADC-deficient goal as families Our bring to and their possible. as

and AADC value impact our ultra-orphan the commercial takes into of the $X.X to planned between We and million. and with unmet of price AADC Upstaza treatment. population and durable are one-time no is with deficient medical Upstaza launch of is and care. based exceptional including standard This the on commercial payers Pricing for million multiple a well-poised that patients reflects from factors, fatal morbid disease high is of this patient $X small-budget highly the account needs

clinical results transformative Lastly, XX patients Upstaza brings of effect and substantial with up years to durable with follow-up. to value

have We will in in Eric into the through France first more access this program drug our patient and commercial go early on detail already treated shortly.

interim such RAINBOWFISH similar regions. Let walking NDA me to for switch and safety infants efficacy key treated of gears that of treatment, XX to normal standing approved the all achieved on in data updates newborns. is The and across two based achieve to with showed seen Evrysdi Evrysdi of from in supplemental We after the study Their sitting, babies months recently FDA we and with Evrysdi babies. pre-symptomatic months continue SMA strong under approval milestones pre-symptomatic that as age.

podium first range with these a months. the results, preliminary development initiated for to and several could believe a it from pipeline broad study, presentation on has announced across be the of is safety Based Ib now SUNRISE study and next at a PTC truly let LMS. has from agent, in recently patients Matt is year PTC. advanced the that, previously over and of Matt? clearly eagerly results PTCXXX, our With known of efficacy we during in XX We exciting current update. hand to to program out me anticipate PTC safety for as our set X study what of registration-directed to positive patients unesbulin, efficacy all XXXX on of a achieve important and placebo-controlled in preliminary the with tubulin leiomyosarcoma the remarkable. deep and a the transformational ASCO. studies results we dacarbazine unesbulin, advanced having with diseases LMS gene we and results half executed trials Turning oncology an Phase in our binding registration-directed PTC these which and unesbulin

Matthew Klein

on our many I'm continue our that goals. execute proud share to to XXXX teams Thanks, Stu.

around recently on mission from to world. innovative Stu mutation in our to bring with therapies nonsense XX mentioned, are We the to DMD. with Study tirelessly as patients announced Beginning results Translarna, positive working deliver the patients one we

of Study therapy open-label by statistically demonstrated phase is a Study in reminder, effect global from of Top X-minute first significant trial designed boys. extension XX-week walk XX in distance still one phase functional which XX-week one with ITT population a a benefit significant XXX XX trial. placebo-controlled demonstrate statistically ongoing. disease-modifying Translarna As was This to a overall as line on a results the a placebo-controlled is the a DMD followed

addition, the demonstrated tests Assessment ITT the In North Star progression results was a progressive statistically known also run/walk Translarna in function they significant in These but the Ambulatory of X-stair a meaningful timed benefit are XX-meter only and a XX% significant and of unilaterally and as ascend fatal in slowing disease. XX% clinically statistically evidence benefit not provide represent disease of to population.

placebo-controlled Study XXX, analysis XX. XXX trials, is Study over pooled range benefit a the our and a assessments. Study boys a XXX in addition, functional In of three statistically There of significant enrolled highly in Translarna across

achieve long-term of significance, from believe While STRIDE ITT authorization. population the registry, in of benefit the request marketing which results the the Translarna position did EU population XX evidence to conversion the results study the standard pre-specified was marketing us from generated with we to ITT real-world, not significant population authorization one along conditional

In plan NDA discuss in potential to to we FDA with the meet the addition, the U.S. for an

is Upstaza XX-day FITEXX results to quarter focusing share COVID-XX COVID-XX. a the a our to our we to study of approval like double-blind, of on are in efforts gene FITEXX designed placebo-controlled, fourth of EU, FDA Emvododstat Turning would of patients. the the BLA planned study as therapy submission which following treatment of the study for now was platform, on for encouraging the for now I hospitalized year. The this of now

which a rather an As a than particularly mutate. continues targeting is DHODH, or Emvododstat targets cellular to that enzyme the resistance enzyme to is reminder, a molecule as cellular is the likely Emvododstat COVID-XX oral viable by small less dehydrogenase, drug important dihydroorotate elicit virus protein.

benefit disease-relevant days the to of enrolled patients FITEXX randomized a the of early, fever. in to next enrolled clear outpatient the Across five respiratory data relief. hospitalization endpoints, order to XXX to and and on subjects of duration Emvododstat when resolution Emvododstat an the with Given examining we informed there was the changing concluded trend improvement, cohort nature treatment towards subjects, steps. Notably, reduction hospitalization, within infection, across was there of benefit of time enrollment of all collected date study pandemic time decision of on make and the cough setting, review of duration several to including dyspnea a

median majority managed. treatment the will early COVID improvement, the outpatient and As group where they Emvododstat was to now We remaining findings of with a p-value complete a primary are less the XX suggest steps analyses for group for and days These in treatment the advancing of setting, in next the for Emvododstat strategy then cases inpatient in XX as are formulate data study to example, or placebo the in endpoint, COVID-XX. the of plan of the X.XX. potential a Emvododstat an than time in respiratory only important, clear particularly days

responders, trial year-end during study registration-directed response make on other target PTCXXX treated X-week Over results for weeks and PKU, several which XX placebo-controlled are the to run-in study those the III potential phenylalanine with two includes X in continue the our XXX by platforms is randomized. and a quarter, of endpoint XXXX. randomized primary months. are course only of enrich Phase our likely to reduction second our blood a trial we share we of patients AFFINITY to ongoing remain The AFFINITY PTCXXX population To the with phase Starting levels. for with with across in trials expect to study subjects a next registration-directed demonstrating the ongoing from results progress in of treatment

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

three ongoing the from ALS. have trial enrollment, ataxia Friedreich's with disease-associated disease-associated of of quarter with COVID-XX-related mitochondrial seizures Bio-e Turning in mitochondrial platform, patients the trials, seizures results delays vatiquinone in XXXX. we to to to two and expect Due MIT-E in with vatiquinone now patients of the we in have one first in registration-directed with PTCXXX

be enrollment six subjects. receive will XXXX. endpoints PTCXXX Subjects to The study trial one or have study with results patients of PTCXXX trial placebo-controlled the of months vatiquinone placebo-controlled enrolled with second change the a disease is the the the capturing of mortality secondary we The score we aspects baseline in of patients. X-month target in in to and ongoing placebo. Friedreich's ALS XXX to from in of endpoint shared, fully two other to trial CardinALS and CardinALS global continue is MOVE-FA Enrollment As morbidity is of is ALSFRS global randomized risk. quarter expect previously ataxia primary a in approximately

two ongoing phase an XX-week pharmacodynamic our focused effect splicing disease parts, Huntington's consist of on initial Finally, an is PTCXXX include PTCXXX HD dose the This a radiographic X-month global five II potential platform. XX Enrollment our study in end biomarker we by program the portion, PIVOT-HD patients. I The two which from study want a milligrams will from this We placebo-controlled and and followed provide of placebo-controlled levels, to pharmacology and with study placebo-controlled of during on the data in a XX-week our CSF of to blood, Phase year. third data. update PTCXXX the initially by milligrams, study base. collect anticipate will study study, portion will

our continued and to about near our I Eric? several over portfolio. excited our sharing very progress are We our future. forward to across discuss from in the results will development call now Eric of to commercial programs studies look the hand

Eric Pauwels

particular unmet Matt. Thanks, customer-facing rare with address for BTC in portfolio high exciting that patients teams a of for diversified products for commercializing is a time our This diseases. global very needs and

the product strong to much AADC-deficiency treatment achieved to commercial are We approval very to quarter we of recent thrilled and patients. Upstaza another with and bring fifth add to our needed portfolio have a

Our the be support also we as driver to future global growth expand continue DMD revenue that footprint franchise from key our pipeline. a the will continues to

our and launch ongoing Upstaza with begin me preparations. Let

about very initiatives the on actively excited and team strategic supporting recent the in our are We all is launch. approval Europe executing

the French for off to quarter good anticipate We continuing on and readiness commercial have with is a center at Treatment key well identification we further as European first patients in preparation centers. and track this the Germany. treated additional Early Patient surgeries launch Access patient carried are treating upcoming as our is commercial Program. well accelerate to start under out

of access in France, as and range such price in Upstaza future We pricing that to is of European million are via other $X our on factors also The have focused the programs, $X.X markets. negotiations early million to globally expected key projections markets be cross-border which Italy healthcare. in

support and data We efficacy first over of months patients reimbursement obtained are Upstaza durable the safety from will experience that confident years submissions approved we and for XX the only patient for [indiscernible] treatment have AADC-deficiency as older. and with HTA XX

Upstaza. to revenue from We have $XX guided to million $XX in million

DMD. to now Turning

regions. franchise global across revenue robust Our to DMD continues all deliver

the for revenue quarter DMD franchise second was Our $XXX million.

have year. Our was the for access, second product starts, last quarter over team growth States. the to Ongoing for dosing high revenue our in appropriate patient patients Emflaza which the execution Emflaza quarter by and compliance million XX% second weight-based net favorable $XX represents United continued new DMD

$XX revenue XXXX, we Translarna, driven for all a net regions. second million the the increase over For quarter XX% represents quarter achieved in in of by product growth which second

large can lumpiness Overall, government continues globally As a create have patterns. due purchase reminder, be markets. buying we orders can Translarna all key in to robust uneven to and diversified which group revenue

We continue newer pricing the and markets Europe, good approval Middle Eastern reimbursement progress and our East make Latin to with regulatory in in and America.

be markets as of continuing presence importance in also in revenue continues for to future PTC. to strategic expand our potential additional region are We for Asia-Pacific this growth

$XXX from which was over additional to the DMD to continues for franchise. strengthen to In This of million. Ministry position strong innovative was be the $XXX continues order strong revenue drug Tegsedi team our course of milestone million quarter. of purchase Brazil, anticipate delivered identification the to our for we orders America, hATTR guidance a we the classification Furthermore, the patients Tegsedi the in and the purchase treatment. patient important received and awaiting and are achieve following Health IP first [indiscernible] year. the We Latin in an In second XXXX

for Incorporation list with the the progressed discussions in Commission Finally, of of CONITEC, simplifies drug for Tegsedi access. National the Technology which essential inclusion

treatment progress for patient America in now have well. Latin and to For Waylivra, continues we identification on patients FCS

treatment be last year we reminder, December, will approval globally application have mark second successful the and anticipate we the submitted globally FPL. focused for the of year. an approved, Brazil of will will Waylivra conclusion, in for Brazil had and half the first customer-facing to momentum this first FPL In an built half for on approved this the XXXX. for and of ANVISA indication. capitalizing Waylivra first our approval extremely of of remainder teams launch decision the a If As a the the We for be our treatment in of Upstaza on

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

Emily Hill

strong Thanks, we continued our multiple revenue of the across half Eric. advancing XXXX, and first pipeline saw In platforms. growth in progress

refer from combined Given release driven take XXXX. to review to and second additional detail. fund for revenues a advancement net revenue quarter million $XX with $XXX for financial pleased growth, million were now issued over with afternoon the XXXX minutes current capital $XXX XX% results, of space, release results. increase financial particularly top was pipeline. second the strong Evrysdi the line biotech franchise continued the total revenue we of XXXX, for market product our Beginning details our these press million. of a conditions This primarily Please royalty in to allows by revenue quarter the summarizes of earlier DMD I that further are of monetization a press strong this will structure of royalty few Evrysdi, quarter The the the our second results.

million. of our revenue half XXXX Evrysdi million. on from anticipate $XXX $XX Roche said, product reach And milestone annual total we $XXX $XXX an the total payment our of also revenue, net a revenue from sales track impressive from Our XXXX Stu million. when continued to $XXX guidance to million was are we as million Apart achieve first

have million guided to we from $XX addition, In in to million revenue $XX Upstaza.

$XX Translarna to revenues franchise, XX% growth product of million, second net year-over-year year-over-year. product XXXX. of million, DMD growth had our net to quarter now Turning Emflaza or $XX XX% were the revenues representing compared of

now Moving to Evrysdi.

translated XXXX royalty into of million reported sales revenue quarter second CHFXXX million. PTC which Our of $XX Roche for partner, year-to-date approximately

over expenses XX% quarter the $XX XXX% Pharma research of quarter retains XXXX, I'd expense. totaled expenses Cash, the of approximately cash of Operator? for excluding which billion, call XXXX, in advancement in compared The in in in expenses additional the equivalents receiving second Evrysdi increase the now million million turn excluding second and the R&D the securities $XX XXXX, million programs of $XX $XX marketable investment of PTC remaining excluding $XX Non-GAAP noncash total and quarter million compared million of June Royalty a for stock-based year-over-year $XX million compared approximately were million noncash XX, reflect December up for reminder, pipeline. royalties, received noncash as to to noncash XX% with XX, XXXX. stock-based million of to SG&A Evrysdi and R&D million stock-based to the compensation a clinical second $XXX XXXX, expense royalties. question in $X.X compensation XXXX for $XXX of cumulative stock-based $XXX compensation of operator for second As after million expense. excluding $XXX PTC of were as expense to compensation to the Non-GAAP like answers. quarter

Operator

JPMorgan. And our come question Eric first from [Operator Instructions] will from Joseph

Eric Joseph

A couple from us on Translarna.

geographic of you performance you granularity what as considering? we might sort you material that relates out First, the just it should that purchase the a orders like? backing in also if about and marketing on can sort transition the a which authorization to of may that could second then, and provide saw to is recycle secondly, to the group there and thinking bit the wondering have little any mix look Belatedly, both EU, FX requesting we large demand standard be that impact this and requesting should the change unpack little And trajectory timelines, extent half. quarter sales that in the be the in perhaps driven more

Stuart Peltz

want geographies. all a differences think could saw I Translarna saw there growth? little quite great Emflaza, you both quarter was growth and where talk we where a there this bit and But was be about in maybe, of throughout we to where Yes, Eric, growth good

Eric Pauwels

as with particular, sometimes we're all what be and on in And know, product really we untimely. large guidance we we group key growth We But Translarna in quarter, million across never we quarters. large it in a little geographies. seen focusing see continues markets geographies purchase And that robust Europe. right is year in million. $XXX orders this international all and has growth certainly markets really guide this and is what was doing we're that be all robust have very orders is you a to have $XXX can saw actually revenue our And to to across now,

growth patients. really and across continuing new the that's because is different So of markets actually

see programs to high also for continue compliance. We patients. really dose a adjustments would We've added that include

So we're of really expansion. the growing and the also to doing very preserving terms in well geographic base base but

have contributing. markets that We new significantly also are

that seeing global have diversified now. we're robust across essentially, and So growth that the we markets right

Stuart Peltz

important could we're I million. that see going business and I I our quite million to well think to $XXX grow think what's continue that. you to continued We're think do is, reiterating to $XXX we

think with we're I very So comfortable that.

to You talk bit want a Kylie? [indiscernible] about that little

Kylie O'Keefe

Yes, absolutely.

the question with think, So I that are Eric, your reiterating Stu FX, guidance. our said, around obviously, we perspective in

do and U.S. robust so impact we we diversified globally to see an a see continue to a the while euro, dollar And different number of the across portfolio across geographies.

and while confident in we remain an allows are to remain confident and So diversity business this reiterate geographic seeing us impact, guidance. we our

Stuart Peltz

timeline, [indiscernible] then question one? take want And that to do your you Matt,

Matthew Klein

here And marketing of benefit really ability the standard to risk provide the So the conduct to in the authorization Study just idea triggers reminder, evidence the convert EMA. conditional really, it's the Study support one of that of comprehensive our comprehensive evidence part to one authorization. generation was to as from a marketing XX Translarna. conditional commitment of our was XX And following which

and X.X collected data about is So benefit were of And clinical we past study years trial package the then, the collected largest trials have more favorable about endpoints, and the ITT benefit about also ambulation transition Translarna, are STRIVE safety aspect single DMD evidence in safety obviously, in you but of boys in long-term safety over loss functional a really the including function that the data think evidence finally, registry, years long-term that real-world informs we're morbid generated course, boys. of the excited evidence results delay two given which you key again, our in several then, in in to which a provides comprehensive of comprehensive this a in and collected package XXX those the only are delay population statistically of very which when of have of is think in important for not significant XXX loss through the distance Star, North And we results years, on in roughly the the walk Translarna drug. at clearly we long-term X.X which of that the disease. pulmonary also believe of

that end submit where we basically data. initiate of questions profit that the a over conversion. course have comprehensive and the marketing will data, when Study you unfold data in with that may before, They will that's variation requests as the standard. several to the conditional together that we mechanically have to forth. due we of from will Europe way the a said warrants provide EMA about put data believe STRIVE authorization back the months So the QX, CHMP XX conversion The by for one happening This they'll which the data the we two we be is, type are or

typical hard initiation submission. end. till it's think We in but that time MAA it be to say than much of So when be we're from several exactly process anticipating September what will be as clearly will months shorter the the

Operator

next we'll from And take William our Raju Prasad from question Blair.

Raju Prasad

And potential just having looking would months anticipate give more if maybe the us under launch helpful. on could of into next belt year, even a of be that kind you commercial give into your color more going little a you revenue at that how this now markets Can recognition you year? you Upstaza the of cadence sense, anticipate the couple a

Stuart Peltz

do go approval, able of the just Obviously, little then you color, we work a into bit other to be up to all AADC-deficient while our bring places. have goal data is patients. to bit a the And to got of recently then to just maybe

right working in have also and on on being is worked well. or that being So as working going then we're places to on, been different now be market access

pretty well we're drug well in for to to So commercial patients the of both be program expanded the terms access launch. using commercial patient We'll well. as positioned launch bringing as as

a like number of good key in accelerating on like we education, had a surgery preparation patient already and key we one with identification working are center a that of access has surgical said, making is that in have engagement. gone working launch, access we're engaged position hard feel commercial we it success payer a for but factors of The have groups patient paid into We've and the then lot then expanded that. been the expanded which sure work preparation so positions And disease the patient. then advocacy patients and and the there and

program. we The going can we'll expanded particular, by country, followed do where into that other is access first markets, be obviously, international in going be Germany, in Europe to

key And so, for focus just a about if on forever us even and goes really a of think the this you instance post-approval. identification patient [ph], and preparation launch

the think the quite strong launching of global rare to I XX think, shown day, a I countries. broad record commercial get is experienced, track drugs. So into over at a in Team end footprint we've

for pretty the So significant is opportunity Upstaza confident a we're launch. that this

Raju Prasad

the well it as as MIT-E trial clinical first well on like question a the trial where looked 'XX are enrollment, just as obviously, readout then, as us Phase AFFINITY Can two give you you with quarter a of enrollment. on sense Huntington study. trial the And

Stuart Peltz

the PKU Did trial, right? you say for

Raju Prasad

the The the just is in PKU MIT-E then a I think that and trial trial saw 'XX press now readout. I release XQ

Stuart Peltz

Yes.

we're We obviously, are benefit PKU do has that drug trial. excited excited, Kuvan where a substantial want that. pretty Matt, about little talk and that? about over you to so the about on just we're our So think pretty bit we

Matthew Klein

as Raj. of All development of we study, results world end Similarly, sites that XXXX. updated the trials, on with running the we're and the by up, these infrastructure. standing trials enrolling PKU, results XXXX. track the for MIT-E, by on that updated standing the PIVOT-HD end enrolling portion. trial the with we XXXX These data first around children are and are delays. with end and mainly ill. ill. on data the XX-week rolling data mitochondrial the disease and world mitochondrial These moved trial by are as we're quite That's on our PIVOT-HD up XXXX. we to children COVID-related from one quarter and we the global leveraging the are And sites of due end on are three quite sites Similarly, up of disease with the quarter for around And seizures Sure, global study, the to of seizures by then and and XXXX

easily fact, can a pandemic seasonal colds even there's can going them events. In when the and in on, not be and fatal hospital flus put

get been the study into And so there's the the a on lot of and to physicians sites. caution part of kids parents

bit the we're on of target quarter to getting the enrollment the target in delay little results, as have we but to first caused that's trial now a in of XXXX. So said,

Operator

Kristen Cantor our Kluska from Fitzgerald. take question we'll next from And

Kristen Kluska

where next you for guided taking trials Upstaza of So quarter of fourth towards second talk these the they steps and given that commercial the the and BLA data could quarter, you have we're juggling some three be through readiness year successful? potential about filings you're the beyond registrational expecting should

Stuart Peltz

Yes, sure.

that a structure of is team start each is forward moving me the way is, that. little obviously where, Let just such team bit the the on

doing work program, really directed in don't is both get each of juggling, in for is launch each the has and team team regulatory, we their correct. -- of So to ready juggle if registration terms each there

think every to in pretty be like able of moving has the all And each those them as both forward. we one be well that that move all forward. isn't is And that their it as team we'll we Every in one the record you? U.S. pretty the So with track we to this, in help team knock have be well, approved, all experienced at think those I Europe as to to able strong we'll other. wait I of versus one the own of is moving so have footprint to for a And forward. team, us I that goes wood, global Does launch things gives gets think, them progresses and in on as have, the able it capabilities really move

Kristen Kluska

now a from And could to you changed, with I platform? views confidence here, Evrysdi this of away as it that platform your doing lot disease. have lot really Huntington's the a been splicing then, your experience at know or made wondering in of of you're past and take the in there's you've relative commercial to course, But the increase relates commercial work synergies if all anything experience that

Stuart Peltz

Yes.

splicing from view, The the get brain fact think, really every therefore, cases, these part Obviously, could and of pleased that nice with is of diseases. the best the aspect and, the point bioavailable, aspect the for it. the go. of of neurodegenerative of commercial prospects oral is especially platform orally is these to they're probably our we're treat I think, disease. they every that they way to We molecules in

it. titrate could You

or the markers decreasing give protein, have you You like of terms sense increasing the and what's what a amount the effect you either in can of where blood of in done utilize PD with are Evrysdi or PTC to XXX. the we've dose of

the blindly. not bus So driving you're

in getting the you're You have the and of based that look level exposure into important. the is drug exposure CSF saying you to the know level, good the really well. then on you're we this within And as effect brain, And level on that, this that's well, therefore measure what as pretty a know can splicing. getting think

commercial then get that at where you program sort is, that when of good obviously, a fact is, standard perspective, you make a of is the the lot So what show decisions you time. the case this right the same hand. And lets information level therefore, you clinical with is everyone on that know could commercial the what from that dose the the data

time spent honed think really really forward splicing lot where with. nice a of platform the to commercial I this perfecting move is and now product it's a We efficient.

I think get it's to be is commercial multiple multiple platform I an exciting. going to So, going we thinking it's this going think drugs. will that programs really platform. mean, add exciting ultimately be a to We're to that's for

Operator

next Our Cowen. Thome come question Joe will from from

Joe Thome

is variabilities the patients. there? of it What's how that there And of at update them? then, relatively on seizures you're of these patients type patients any in experience? are And the terms presentation, one of the epilepsy as straightforward kind sort identify out to in many are is your mitochondrial looking thinking Maybe

Stuart Peltz

Matt, you want to take that?

Matthew Klein

Yes, absolutely.

So seizures talk disorders. about it's of you morbid when component mitochondrial these and in a common highly disease,

in simple majority as So benefit, reason disease. kids roughly disease disease antiepileptic the most therapies refractory their to of actually that with mitochondrial XX% be underlying part mitochondrial to XX% which with therapies. have part traditional and seizures these as they seizures seizures is pathology vast of children tend their increase, The antiepileptic oxidative the to typical stress of of increase

and the many a able could out, is are So in obviously, And as within these observable the estimate antigenic quantifiable obviously to children. effect. aspect features the you which target Obviously, in group, this They most and, traditional then, of for They underpin stress subtypes. number trials We They structure mimics the tonic. as overall using trial, done Obviously, well-established pediatric with worse. clinical have seizures. important there's clonic. those things the variability. is pointed purposes them picture in We're vatiquinone study with observable a types motor. composite endpoint myoclonic. And study in fact, be of be disease-associated actually to benefit seizures. the have different [ph]. is could And there there many the ways, variability the then, about some be the the variability focusing of oxidative of run-in XX,XXX primary observation on motor underlying of motor seizures focusing of [indiscernible] Joe, we're therapies be an are treatment well, seizures patients seizures, for very just They because being phase two, on one, in of like make children obviously having pathways could -- They design [indiscernible]. measure mitochondrial previous observable the and in that these [indiscernible] worldwide epilepsy this as could design

Joe Thome

And forward in major for order FDA? up that, looking go with follow with seizures in conversation then to submission motor in the maybe of there that placebo just a number terms reduction from you're separation the to on or of is

Matthew Klein

Yes.

the been seizure a the that's a period establish is the course we pediatric we we powered a treatment as And previous XX% XX%. where for are we run-in the the targeting frequency monthly in baseline study X-month group seizures I then decrease based and of on way measure So over epilepsy about mentioned, phase. placebo Delta that XX-day calculated placebo-controlled then and of just what's And as there's to is, observed about a We're a the of have reduction change estimating median in XX% frequency syndromes. approximately reduction. a the in

of to aspects and point related placebo, cases, difference would in XX% of for severe even and decrease aspiration how seizures given clinically I seizures their how disease, meaningful to we're that these and pneumonia of are, highly out how some between of these think death, a and in XX% they're targeting like other children, are morbid [indiscernible] an I XX% while in morbid terms and delta patients. treatment observed will be these

Operator

our take Tazeen America. question we'll Bank Ahmad from And from of next

Tazeen Ahmad

view clarify hear on before endemic the the just outside And I what of a the think all study, met one, get focus. now conduct opportunity how terms you a little COVID. available. oral thoughts do decision I there of your I a can also, Emvododstat and need with to expecting think under-met primary Stu, Would for you on it's analyses make Number love used more And we've the endpoint? to kind would becoming PTC you on antivirals is. areas you if viewing bit number you're product become guess then people your just back wanted from you COVID your to in are next? of what And to expect going to what in of thoughts of to secondly, additional where then

Stuart Peltz

Yes.

learning to see an affect effect in it. you on order to having at the ways are things an started look what come effect we harder both it the best to later. patients that that's this load, obviously the much hospitals, evolving versus So in was in much that going even early were while scene who COVID it viral to something see to of as and look about load. at we early viral obviously, on It became clear affected It's had relatively

decided be order So Nonetheless, forward out I that's difficult sort the study of do something it move and it. time XXX to we'd able and that to was decision, the to to make subjects were actually review was learned think best sites a felt where we taking in to what a be at look would enrolled off in long order in informed to be is is what now trial. to order to when collected be the complete way it. the better in relatively And looking data the best guess because to we seeing early turns and to it do

improvement, deferred dyspnea was [ph]. across patients benefit subjects, statistically when significant a on the trend But time several was relief. days hospitalization, there of duration infection, across a a of treatment Emvododstat the five So all relevant to endpoints, of including to that enrolled hospitalization, examining and Emvododstat of investment were there resolution duration respiratory benefit in towards cough disease time that

in the length group. Emvododstat of placebo the XX hospitalization example, on group days For days, XX

was for is, now ways the in work the in people to think where early which treatment to it's a I setting in suggest as of I clear are this I expect. that the I So managed. is majority of And outpatient clearly, particularly do important, cases pretty Emvododstat what it think they better think, potential think clear the one COVID

work get to what's the DHODH the compounds best the whether inhibit way that get what's that way considering to think and strategy, to to the what's to we with do patient. with we're outpatient So best the best COVID-XX, about the someone else

you Firstly, here COVID-XX. I is question, additional shown we've and I still be up next and in could mutation cellular that they're of clearly rate. there this for because And come that it of proof let will mean, good drugs, see we then a they're think other is because, in think therefore, it's treating me clearly, a some this they're low good is I target DHODH with terms what would resistant, of you, other though, it viruses drugs, need remind [indiscernible]. -- the a be principle for where think The

out for think I a there's additional there. need drugs So

de there viral the what it do, this just forward. we that an where area novo that works good move role indication it you're it's of think a And go way right, was for for need and we'll viral have real and the to this is de that, to your most potentially but knew novo and replication you we we I synthesis a DHODH for need [indiscernible] treatment normally was to And So consider just a position, controlling replication point need the a this for about wouldn't learned enough in way because this best normally in -- was pandemic. that target. that isn't Pyrimidine something but synthesis, salvage the pathway would large you the we the isn't

problems of was like world important the and solution a to thought enough we of this disease the around we'd that if so possible. that part And enough causing be

did that's that. we So why

Operator

Securities. next Truist question take from our Gandhi And Nishant from we'll

Alex Xenakis

us Translarna on wanted And expect? press for flow then support communication we U.S.? remind launches Alex of is This know what we the to both capabilities capacity launch EU also, for your kind the can and we What cadence can of to U.S.? type and And you should in the in the expect about manufacturing to see filing and the Robin. news around

Stuart Peltz

you in Just are general? what or to? referring Manufacturing,

Alex Xenakis

products the and therapy for in you supplying the Do any supply therapy regarding an of issue markets? global gene anticipate ultra-orphan chain sort

Stuart Peltz

Sure.

FDA. the be having think, excited an So talking with the of we're obviously, about end II We'll I and meeting Phase Translarna.

end I talk III mean as talk we that do Matt, next that. Phase steps? give clarity news I will meeting flow -- have on little think more of a get to want bit we'll clarity you about and then

Matthew Klein

Yes.

Europe, of I timeline. talked quite think So we've in about a terms the bit

the expect standard submit end two by conversion of U.S. to to a conditional into We the to type the from variation September request

of able out lay XX the the be the all the well FDA, strongly as of will meet underlying will of the as supports the data in that's plan with of data asked, one for which you to which from endpoints significant benefit As on totality statistically mechanism and have share data to be fact Translarna being we disease that targeting the DMD a first. therapy a functional

previously to forward that, share about. talked look and really totality we STRIDE long-term able data being of the demonstrating with safety So, along as to and collected the in we've registry, benefit, we

path So we to once on share give appropriate. the with updates it NDA, will they that an we have a obviously, the line forward. meeting as move and agency obviously And will

Stuart Peltz

path there's almost been the small path. And is path molecule something supply the we've small now years. therapy manufacturing, There's prongs. doing two XX molecule and The gene then and that then chain for for

handle and facility able site be Massachusetts sure a foot that well NBL in the we of We we this. to manufacture at gene have have of multiple good shown XXX,XXX gene strong to close can which therapy products, as And Hopewell group clinical site we doing or manufacturing a plus another making to we've very manufacture manufacture they've XX,XXX capable multiple square so with is as commercial or square for ones Obviously, supply can move supply it same either feet manufacturing for we programs in time. well. and been forward. have therapy, commercial at and a I It capable that's been can chain think and to they're

experienced and we're only an So, think their of approval really been have under a have commercially in who've capable one suited that's third well team therapy, we and people I gene now right, the [indiscernible]. belt

position we're think a there. I good pretty So in

And excess making with so make have sense out a working of in a manufacturing. to therapy clearly other in we've able the be of small can gene do CRO we facility in capacity built fact, to have that manufacturing. that also And and we we plasmids out companies business state-of-the-art

in as in pretty we're both think as manufacturing gene of the good shape, I So therapy small molecules. well

Operator

And Brian RBC next take question from we'll from Abrams our Markets. Capital

Stephen Mallon

And present might as you forward gene additional This that of subgroup the performed is for for that you FDA a a see accelerated have but Brian. performed? On I'm news curious related such Translarna. consider Study from you for approval analyses question, the can maybe you didn't maybe any analyses path any DMD how Maybe help impact on yet, whether whether recent therapy subgroup share Steve on may application? many Translarna, XXX

Stuart Peltz

Sure.

of So couple terms a in points. I think of

in once you a yet these therapy, of think yet treatment. In treated, that. different interesting terms issues of limited key terms think that can't that with to a to I foundational think capable I I dose, have that of high of the of has. for be the And with you Duchenne used be versus first terms aren't of therapy a there's that for patients. broad of range demonstrated little for small gene Dystrophy think, they're think patient shown I that I and getting bit a at are it's range standard patient have care benefit addressed. population demonstrated view numbers and wide possibly a withdraw that Muscular Emflaza the patients the FDA with thinking not on gene Translarna the are key number in benefit all, the the of questions other and always It'd and I drug there And therapy, age treatments going that in matter, to probably be

where therapy they predict the necessarily data to a get as gene that can pathway this there So, therapies benefit, approval consequence dystrophin I'm patients doesn't gene of very notion biomarker can't functional of how and in to other clinical curious case when that. is you see a the how in bring using that accelerated viable the isn't

skeptical am gene in pathway I So, that therapy. right now personally of

of Study we ITT terms to XXX, the analysis. as the as in the that XXX In population the I well in it XXX fact took think

Star the in in as worsening. improvement to test saw the XX% time as the So XXX also in you terms statistically significance the statistically see well time that and and ITT with population, North functions significant of you XXX or

walk test. analysis, the not So a also all range time pull we analysis when overall to minute both function with you X.XXXX together test, and pooled a of value statistical have the mention significant and Star with broad the P of North that

and terms presentation with that, of there's of actually preservation in show interesting preservation in strong you where data So, so years as consequence the registry able years other ambulation you and mean, five the that results very off of XX% function, much functional the can XXX look get pulmonary and I than see And a that there's getting that we position with you see improvement real get of and being when in this strong years two the when floor have we're what combination ground perspective the STRIDE [indiscernible] a about, a about Study the does data the a almost does mean and one results greater the a clinical a from function. of of we that ambulation, think to in data you patient trial, of with when that strong think off you five a demonstrating XX trial, that. you really you clinical treating think to STRIDE in long-term at when really the what Translarna from showing of translating those shows preservations and pulmonary it perspective,

You to now a results patient. those mean know what

know different of that. North get it's you about Star score. Stars, And North functions then, excited analysis is, pretty with the that we to we're interesting we a other So like a did measure the number

preserve we on well lose only it do say also what time, we they those of top but what functions, did function. not right? So of data, over how that at looking it does Because do

XX% risk we [indiscernible]. the than several looking you at more XX% so by reduce this, on saw, And that when to

a equivalent these perhaps So one as saving in that's to of that. kids two functions or consequence

preservation again, great function. example So of of that's, another

that's So important. really, I think sort of really

at we the of positive population the of think a that thing got most That's important this So in the of I have we day, whole out the population. result end patients. the is ITT

able here. group every large this was to enough study a see and it really, -- So to be

think, I effectiveness. Translarna's those I this results very in think, that Did So study all help? all, of demonstrating really, all a makes strong

Operator

question Colin next our take from Bristow we'll And UBS. from

Unidentified Analyst

on Colin. [indiscernible] is This for

on U.S. in filing PTC BLA question the one have we AADC So

call, in the you be quarter. in as filing So fourth the expected said will the

the more launch you discussions have the any some please details? you incorporate would you on FDA So there's be further the And give us anything example, could color experiences For filing? FDA? for how the And have if with to beforehand? new done

Stuart Peltz

Matt, you want take to that?

Matthew Klein

you for Thank absolutely. the Yes, question.

along, about would EU. MAA obviously, And line. work approval as we the all the across So incredibly said in get the we to the we're excited hard

ways. we're As Stu number in of focusing the a said, this on now the really is submission. And trailblazing efforts BLA

clinical on As to based fourth be package the believe in then year. that, is we that that submission the we believe done and to we Obviously, needed said, is and components with sure forward strong and to quarter approval, those look as agency, for the we've of have the that have on timing part experience able nonclinical to in a puts our able meet the having the position. yet, achieve We all being this all provide we'll and plan to provide not key gone an for very we the have those our make the in being obviously Europe of BLA position that submit BLA manufacturing approval to FDA we of in to to submission. obviously been align in to BLA the through the us able Europe data, data, a data,

Operator

question next Gena from our Barclays. take we'll Wang from And

Unidentified Analyst

to Shan have identified [ph] you before two How have with questions. Upstaza for patients you Gina. start XXX is mentioned I AADC. This for I globally. want

treated how EU by the break numbers you of [indiscernible] label? down have them access? many country many be the how on So of are based And early regions? or can patient or Secondly, them can

Stuart Peltz

your production. milestones for bit the obviously a very of severe disease AADC Obviously, that talk and is Thanks motor or little a patient maybe lacking dopamine Yes. deficiency question. about

So the major the really majority of do missing vast these fall under -- we've identified patients their that milestones.

really we that the label our label the have breadth broad. is of so very And

So we by surprised were pleasantly that.

get this to point so an And that important able was that. we're

have continue as And to continue going higher that. number treated. we additional identify we'll and that and XXX to probably a have that patients get to said, that's translate I identified of we do patients will to patients So

been have and broken But pretty what going haven't going from German found them that get that we're specific to broken to while we're We by to place busy toting XXX place. haven't throughout what treated. our be We we we XXXX patients, We we and countries well. -- to already out in do out certainly reaching have have that as patients have patients I out them think is, we into beyond. centers certainly

expanded also We patients have as scheduled paid access well.

well. the this a as do guided move the million opportunity and that's than the we get this the things and all as a we've million side. the year, a continue this, we to that up grow said we've will $ is quite center that patients, We overall. I as at So surgical from between one revenues really $XX look greater opportunity think and to will train doing think And all of billion line we be what $XX of continue

getting have next and we're think to need in find the all years pretty to the to treat. patients I continue of couple we So patients find excited to more more over

Unidentified Analyst

what by Will to question? gear any like data share PIVOT-HD I your other a such bit data, Huntington, expect you data Kitimat also I year-end. or will should you do May year-end, want what of biomarkers, type trial. little switch ventricle we neurofilament by like imaging second wondering as a just ask endpoints I show beside to volume? kind of

Stuart Peltz

Yes.

the remember of trial on first steady right? we the in -- provide what the at different on the we first and this see and like placebo-controlled a looks dose So is and CSS health the Then that exposes you what the is And to in blood. clinical set the where where between AC Huntington's two cover in the reduction weeks One same trial in is important to between of volunteers in might and state, is parts. to think important PK XX-week and is is effects. looking pharma the biomarkers somewhere to your exposure the three results XX weeks endpoints, we between CSF or trial HDT first the that we XX then really basis, ratio will them, blood pharmacology say remember dynamic that right? saw more the saw part second So focused So do really we're two that's in -- might the the relationship protein right? you of

patients look So, want be we we [indiscernible] But of the then to in HPT like first go see at on, part also it looks as patients. well. want to the it And pharmacology and is what to as blood going PK.

Unidentified Analyst

confirm, you see want we reviewing? internal would Just to collect at XX by weeks. owning going PK/PD are year-end, data some for data so But to

Stuart Peltz

I'm sorry, the I missed last part.

Unidentified Analyst

for you any collect biomarker weeks, going to of are for the XX part, like Last on focused part even first data the is, PK/PD though like you internal data, being?

Stuart Peltz

have we'll been be we as being There's relatively monitor. Yes, is looking and able all everything, probably, to This never bioavailable new. an orally drug.

what doing of the in get in to parameters RM. terms looks in inpatients the we're what knowing it terms of So like right it's approaching of solo trying in what in all terms now, PK/PD, the correct

Operator

Brill next And James. we'll Raymond Danielle our take question from from

Alexander Nackenoff

you to in on not Or Translarna flexibility at on FDA the looking say, This you're there Danielle. touch on and the that different it to do their you agency and the in its the considering through divisions tolerance at kind own? Or bodes read-through just how is some, Duchenne. are U.S. for here? play Alex of reading Translarna Sarepta for it back this I apparent you are that think Are let's wanted considering stands future, dataset better?

Stuart Peltz

that demonstrate patients a we I one if look, study significant population in I population in the XX demonstrates I ITT the think we're well-done that, muscular dystrophy before. in were was don't hopeful walk population statistically set also broad of functional test. But X-minute did North that said I know the think a heard benefit but Duchenne Study a function you what Star the broad in-time in

for first where we're that in and inputs the I is ITT that example underlying the seeing think Translarna statistically with about cause it So, significant versus think show a think population, of it, results the disease, that I the actually that an up multiple drug you a everything if favored -- pop. in treating

shows and XXX think along results And data I XXX the that Study so you're from that with really that benefit. seeing clinical

a haven't predictive is You benefit. view, shown don't hasn't have grown our of yet on that clinical rely to biomarker in

So, I what Can bodies mean, from And for cardiac those, And position is about? goal of years STRIDE we're DMD. improvement where being And regulation, in with so die which that of do Can also five do you to care really pretty think preservation function. showing DMD it of predict I function. along when you're than translates if ground? of see way, have what the then this that XXX in preservation do patient, strong or the presentation you greater saw get and to a in off ground is you And regulatory data able good years for to ambulate? is still of we perspective. the of and you in two pulmonary critical show patients and the the still drug a endpoints patients, STRIDE endpoint the get in clinical you're goes case, a showing pulmonary was right? -- then what When benefit. a the off people, outcomes registry clinical is that we clinical the and data in registry, that you

think we a we case there. strong have So

Operator

time. like showing I'd to conference am back to this the turn now Stuart And no I further over at any questions remarks. for closing Peltz

Stuart Peltz

I us look, to Great. today. Well, joining thank all want for you

that evidence DMD demonstrating that XX adds field nonsense one think the Translarna. the patient -- really Upstaza for PTC results but I And only having approval key the number and not of of I really on for benefits made to groundbreaking of delivering milestones. see positive proud the community. mutation and significant you to and have for and think we're demonstrate the a I this to could think science benefit patients we've which milestone a really of the entire of of Study been year gene what totality been is therapy and from adds progress And the have great AADC year, the from Translarna's a

other so what that the asked, future. that point about particularly that number talked that important that those of with forward look a in we is you registration-directed the is ongoing have to results we you've studies and we've sharing the And in question near are

as on for happy, [indiscernible]. So and developing innovative and continue of therapies these discovering excited see, can the rare you we're to mission

So next our forward an we joining conversation thanks for today to update. look and with

Operator

concludes This a conference you today's disconnect. and day. wonderful you. Thank Thank may Everyone, participation have for your call. you now

PTC Therapeutics (PTCT) 7 Aug 222022 Q2 Earnings call transcript