PTCT - Earnings call transcript Q3 2020

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Operator

Ladies and gentlemen, thank you for standing by and welcome to PTC Therapeutics Third Quarter Corporate Update and Financial Results Call. At this time all participants are in listen-only mode. [Operator Instructions] I would now like to hand the conference over to your speaker Miss Lisa Hayes, Vice President of investor relations. Please go ahead, ma'am.

Lisa Hayes

Good afternoon. us update the and joining quarter Therapeutics for corporate XXXX financial you to PTC discuss third Thank results.

our financial Joining our me Business Emily Officer chief our chief is officer Hill; officer, executive officer, Eric Klein; on Stuart Peltz; Chief today's and chief development call Matthew Pauwels. our

in review remind Investor that on take Please current call start, with the posted include our will conjunction to on slide we website which Before forward-looking you forward call, let Relations expectations. a me statements. today's contains looking based the moment statements our

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

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

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

Stuart Peltz

thank you the Thanks, Lisa. joining And today. call for

discovering commercial revenue as needs, progress through engine. developing with medical this patients by have these we've We significant novel global made our with our well R&D therapeutics bringing patients increasing commercial growth. built and quarter for products PTC stakeholders value our all pipeline We've as for continued so business by to to create

to further pipeline position of to to more Our patients deliver therapies allows company. strong on a continue grow to to the and promise cash bring us the

SMA. Starting this the of treatment for some through go the achievements with quarter. Evrysdi me Let of

administered in patients, safety access administration SMA older. and of about two pandemic. the milestones during orally therapy but to in a and launch first across profile, to physician, for its ease improvement is launch We also and efficacy the of achievement patients. SMA and U.S. motor function the The are home-delivered add from months whole Evrysdi convenience home its of and clinically not treatment a community. patients meaningful only for functional response as the range SMA Evrysdi very positive The of a excited important COVID-XX had developmental has abilities broad particularly very especially payers ages and Evrysdi showed a strong

As or with and unmet reminder, treatment-naive concerns medical SMA in with have patients burden high there is need the the durability, community who patients who therapies. current administration a of are

that or on reported treated the Evrysdi with patients previously has of Roche two-thirds initial partner were spinraza Our [indiscernible].

We are type patients in and Chile. three. seeing with SMA have in with to bringing all uptake been obtained of There of Brazil, be Ukraine one, To types patients. excellent XX% are treated type XX% remaining globally, approvals Evrysdi to patients across the already progress two SMA continues date, and

by NDA million April also an Evrysdi in addition, appreciate a made decision a our strong the Japan, review to filed was under in triggered with AMA partner for expected We Evrysdi is milestone with Roche. XXXX. In the payment effort PTC. which $X.X

comment commercial increased portfolio, Across this me uptake Let patient commercial quarter. associated on progress our our revenue. we and saw

for globally with Brazil's XX%. XX% to agreement increased of purchase COVID-XX Translarna. net continued Our revenue a and despite pleased securing that revenue We're very including we've year-over-year net product challenges, grow Ministry increased Health the

commercial R&D the about equally progress in but products, You made value progress they will excited we've create. I'm made on can and we've the our see programs, considerable

from pioneer and platform. was PTC as splicing this first developing the small Let's Evrysdi a the start our in splicing for selective approved molecule with discovering with product platform.

that are polio of number a splicing have exciting programs forward. moving We

healthy splicing molecule. the pharmaceutical and in this distributes next first results in platform study of volunteer expected to and the concept differentiated the and is the body. blood mRNA. in bring globally. to biodistribution to correlation HTT from our is results to Phase to preclinical products because we is orally direct them to that I'm there that developed disease. be next which splicing After advanced barrier levels tissues selectivity, these and was performed selective. PTC to will excited program, how PTC of these an allow developing subsequent quarter. patients the critical XXX trial select crossing all in of Similar year. The XXX, the create for of developing the for of report and value enter to from should that are the HT to a effective half plan in allow PTC has successfully Evrysdi, volunteers, been Our demonstrate will compound Analogous It discovered We've by clinic Huntington's eternally us proof of will reduction healthy studies, to doses the X brain very is learned engagement This elements to in SMA Evrysdi Phase us XXX properties the patients. X molecule study constructed with targeted blood-brain and bioavailable highly was be most

in turn Now, From mighty with Vatiquinone we a compound the registration-directed congeal epilepsy. that recently attention clinical our of announced let binary enrollment me the programs. to our trial our late-stage platform, treatment again called

year. patients additional addressable enzyme inflammation with Vatiquinone stress of trial globally. XX mitochondrial are stress is and end. These a and targets of move with are FX As inherited about important We to modulates there to directed of oxidative neural X this from registration FX is results response. oxidative inflammation. children the regulator to we he key disease, initiate mitochondrial levels suffer particularly the excited disease particularly known and XX,XXX transfer estimate to planning particular electron so disease known be dysregulation that FX all leading initiate seizures. refractory disease registration year a lipoxygenase, from the result seizures which pathways stress An these and is non-altered the epilepsy inflammation, neural the also known pathology by of process, results reduce high half approximately which reminder, of commercially inflammation, to Phase from pathology can Previous progression. inducing oxidative directed an study that course

demonstrated third program PTCXXX a need, not Our of study we for not [ph] Phase compared to continues that levels estimated or kubek. medical greater responded XX% kubek. in the did respond to XX,XXX Results when alone, mid-XXXX. more from than to X in PKU, results is initiate an on twice the demonstrated Kobe. drug Furthermore, centrality patients compared With majority previous the adequately studies had in late the to pivotal there PTCXXX reduction approximately these initially be treatment that expect high U.S. the stage studies, as patients to the PKU controlled in

long support to Phase trial. are the conducting I We studies study, term X dosing study want to turn XXX. currently dystrophin planned Translarna non-clinical for the now the to

this the just patient underway look muscle XXXX. of forward COVID-XX preparation despite able first quarter by with analysis potentially now in is to very patients that the samples we sharing therapy. that for our discussion the of of receive to the I'm according completed. XXX FDA You was from the U.S. data biopsy been and our final may to outcome very security in will so approval accelerated recall a strong challenges and the team effort with top-line that happy report study these the has and protocol be the about the Translarna in to the

has are Translarna We to for accelerate excited that U.S access potential the PTC patients. to

production. continuing for Hopewell to platform. We is ready gene The are our manufacturing facility also with occupied make and in progress therapy gene therapy

delays, and expects have we deficiency deficiency, a COVID-XX previously, observed CHMP in opinion the deficient transformational been discussed has As advanced Due and now patients AGC our is program for application RMA the submitted CHMP. first ADC PTC to most review results the been that decade. for have of durable by treat in ADC under half final XXXX. we treated have to where related almost

PTC submission VLA, first the to FDA expects will in half the the also XXXX. be of For

oral PTCXXX a reporting of to over We top-line dual infection, the the profile, a treatment replication Matt that in recently COVID-XX. our potentially anticipate recognize mechanism, immune the a PTCXXX enhanced the viral the We viral XXXX. With of is and key effective call that preclinical phase bioavailability compounds as against Matt. including turn trial The program, confidence half fight-XX. COVID-XX We us Klein also called for combination results in great well-established for for stages both PTCXXX updates response. in of has first that, the gives now potential initiated the for safety unique I'll mechanism and X/X data, clinical

Matt Klein

Thank productive advance innovative to quarter you, from platforms. our We've very as had of we R&D therapies continue all Stu. a

ensure set on that tirelessly throughout this COVID-XX of have initiates to without trials delay. teams trials working development ongoing minimize next our and Our been impact pandemic

transformer study underway in biopsy biopsy report to excited muscle very results are of district until study. now we the Analysis are remain the and for with we mentioned, analyses we the final now biopsies is completed completed. study all Stu As blinded protocol, to that the have profile accordance samples

district As that with a baseline, as collaboration asset increase we expression outcome this in an is in and reminder, study by an primary electrochemiluminescence over the the assessed of developed FDA.

in to XXXX. the top-line five expect districting study have over QX data We from

has employers six-month Primary collaboration includes orphan have assessing with trial have our designation enroll initiated aspects reduction will enroll month the and remains disease a with one in or to XX,XXX trial placebo. a phase, approximately the will other XX scale The refractory trial ambulation The related XX phase activity patients measures by confirmed FDA and The receive period a ataxia placebo. FA endpoints seizure X to a seizures, to young treatment Vatiquinone trial Vatiquinone in one with year followed the of with receive with strategy as secondary epilepsy. ataxia which a which study end This This adults approximately X/X ataxia. in parallel for children was is this or of children secondary FDA the to granted study rating includes pinpoint developed the registration minimum either disease associated worldwide genetically mighty designation an observed Friedrich a seizures, epilepsy. Friedrich This key XXX subjects directly are during parallel motor will that living. platform, EMEA. Vatiquinone we There as number enrollment on arm to by outcome design a and either burden, Vatiquinone and with placebo-controlled of to Vatiquinone is will end. daily design both Friedrich modified mitochondrial for pediatric schedule endpoint subjects eligible enrollment initiate with phase The is bioheat Turning outcomes Friedrich move seizures. study trial ataxia. well The all of morbidity. rare primary phase approximately in children trial, mitochondrial of subjects art front mitochondrial and ensure in

The by the ascending and end PTCXXX, second compound the platform, dosing have our X MAD we completed expect Biwi year as XXXX. to study planned, phase from study in single dose complete

platform, the selection trial Turning inform study for X to this with PTCXXX ascending Huntington trials. safety includes court. our and as parameters, initiate to subsequent single multiple remains disease X phase splicing well to healthy dose volunteers both pharmacologic phase and as regimens to on program those This in the registration and schedule guide

to we're planning the early development done volunteers levels program. in gain measure Importantly, splicing htt also mechanism, plan of as risk proof to mRNA was to healthy in SMA

from the XXXX. the half study have PTCXXX of to X first data in expect We phase

PTCXXX an program. on provide PKU our update I'll Next,

bioavailable in as to is the U.S., to PKU patients needed phenylalanine children oral We phase a double of formulation are XX,XXX and will highly will begin include is both have BHX, term in there directed Stu is that initiated patients. patients study PKU. long support This adults and of effective trial X registration the an for enzyme PKU the estimated study precursor an with globally mid-XXXX. critical blind trial remains the XX,XXX medical mentioned, of hydroxyl, in significant unmet PTCXXX synthetic cofactor a that be for There to placebo-controlled that initiation PKU. the need PKU the scheduled toxicology

year the to the XXXX. expects The CHMP ADC for to the and final in half the XXXX. the patients the of the commercial the therapy related of submission XXXX half first treatment of be to MA through platform to by in tangler with first Turning opinion COVID-XX be PTC procedures our AADC the deficiency completed gene BLA FDA end on delays,

by storm trial DHODH COVID-XX. targets an provide of XX trial. in of of fight of to I'll on is want over Through end, schedule stage actions inhibitor provide the are and PTCXXX aspects at commercial remain analysis I stage complete Eric our the COVID-XX study The trial the cytokine bioavailable, two Finally, and focused stages with consists stage the a ongoing planned its pass Enrollment the of disease. lung both on The PTCXXX replication the second developed now leading to to key first update of on viral interim a in XXXX. safety to of one. the completion being to orally and HX update. DHODH, call treatment year

Eric Pauwels

Thanks, Matt.

Our the with inline delivered franchise from customer-facing XX% year-over-year strong team PTC products. our quarter commercial at across a growth

few discontinuations commercial to of implies We as within real-world Emflaza time importantly, We in QX. to care restrictions patients increased continue a all with reductions in patient the Brazil, we we benefit clinical of XX% outside timing demonstrating U.S. of newly all Also, evidence continue were maintained continue therapy. lessened awareness have the standard purchase from accelerate regions continues therapy revenues to DMD some for in patients have expectations benefits Brazil to Translarna exceed reimbursed product prescribed growth and of high with current prescription group of year-over-year, term demonstrating see to long order Emflaza to a impacted the and of base payers the importance patients bringing the of over pragmatism. access by compelling Emflaza compliance our the DMD therapeutic and where

quarter, awareness entered opinion together are both this securing DMD therapy ultimately the the in of agreement with Brazil's new of Health given one the Brazil's was hard Health of hit PTC team, half bring work important, continuity purchase we for order to is in first Brazil Translarna treatment and the agreement groups We The shipments, Brazil by existing Ministry ensuring of of in Minister one subsequent patients. important that to advocacy we order two a delays exceptionally of this in all The in and the pleased were key which October, and leaders successful patients. pandemic. governmental supply This and is received DMD administrative the for XXXX. with expected and specifies

to Tegsedi. switching Now

tegsedi We year. Brazil, for end in price to the and continue by to engage the in expect discussions the public pricing of finalize

tegsedi For waylivra. both and

these to in patient Latin to see We and success engage programs. in continue America continue in finding

in We filing also continue we and programs Latin engage to waylivra Brazil. a America, as in visa early our on in access decision await

in for XX implemented our expanded pursue potential in Now palsy patient finding moving and XX screening in on focused year. more to to epilepsy Europe high-risk than continue countries have on activities programs agency, next aggressively we launches We preparation clinics. populations, and geographical cerebral reach enriched particularly in

pinpoint single by adapting providers key were These New leveraging potential screen patients. ADC diagnostic needs programs, PTC that at genetic care local local no a We're such launched, panels in to which health country markets. to source targeted also cost. labs access as the is can

patient with key to databases. existing globally are of to finding algorithms partnering we high-risk patient Lastly, Centers Excellence execute develop against

key these with working AADC centers treat actively they to also are ready patients. treatment are ensure to We that

health and with virtual great awareness and useful have will advocacy by diagnosis masterclasses about network.com. the professionals supporting turn patients. and Day, for ABC patient support earlier content digital AADC of accelerated through we Emily. Family our emissions at proven education These presence in to Awareness and In care over AADC call continued the I now informational addition, the

Emily Hill

PTC a Thanks, our to sheet. maintaining in balance business continues while deliver robust growth Eric. strong

U.S. approvals international the of shared, XX the a the filing third the we start with with Evrysdi sale first with are of of milestones of EMA. the very multiple received million commercial strong MAA As total launch. a and in the In the Stu we pleased and the in quarter,

initial Japan Roche overseas Additionally, X francs. for CMA October XX, Swiss the Roche of million sales million On in approximately $X.X from we submission. fourth quarter, the reported received

approximately reminder, royalties return $X.X farmer PTC PTC. a the receives XXX% royalty As Evrysdi which royalties of after of retains a until XX% to of billion, revert

revenues to million third the with of the are which growth million quarter $XXX.X Translarna. revenue Starting products earlier highlights reported XX% our increase show the release royalty The timing Emflaza third and for we the XXXX, the net of top-line includes million Brazil exception financial in Translarna $XX.X Now results issued collaboration of a $XXX.X press and $XX.X summarized total results, XXXX continued today. year-over-year. quarter of order revenue. with in of in delay and million in

now that pleased have order. are that we secured We

this are we the driver Brazil As Translarna Translarna of year first quarter. of X Eric of is of delay fourth the orders decrease. quarter of global and Brazil order the in net the XXXX. compared over segment Again continues expectations growth. seeing received primary mentioned exceed the revenues the the to were million group $XX. to for our $XX.X continued year the outside was quarter product million third purchase in

excluding and increased and we expense, Emflaza are clinical of $X.X for excluding that million for efficiencies was reported compensation PTC third for totaled received in of in the business. XXXX associated and answer platform, compared reflects XX expenses XXXX. third the Growth pipeline. by compensation were investment loss the advancement R&D $X.X the stock-based Non-GAAP SG&A activities $XX.X prescriptions advancing quarter the expense. XX, million net XXXX. million and and of $X driven to were stock-based $XX.X expenses million non-cash approximately of R&D $XX.X third in million cash, Non-GAAP $X.X and I our quarter in million compared expenses year-over-year the marketable the XXXX $XX.X compensation pharma to $XX.X bio-e million as non-cash The million and $X.XX sales revenues quarter This of XXXX operations the net of compared billion royalty for excluding increase compensation in the to with closing million new gene July, increase third compared for and million XXXX, XXXX, the therapy programs cash quarter balance $XXX in of the and million the to third stock-based to in non-cash marketable quarter deal. of product manufacturing research September XXXX, quarter Operator? For patient over expense, costs will to in of $XX.X call cash at expense. Cash, the equivalents continued stock-based million for of excluding commercial hand the portfolio. December third operational XXXX. compared upon for to expand of product cash the quarter reported improvements our operator $XX investment SG&A in continued non-cash as section. start of and due million Net the million question third includes XX, securities third quarter to The September $XX.X loss now our as to XXXX, $XXX.X of monetization of for the of securities commercial in equivalents by

Operator

[Operator question JP with First Eric Instructions] Joseph from comes Morgan.

is line open. Your

Eric Joseph

good Hi, evening.

for the if just Thanks Huntington's You I questions. a taking I could. can couple me? hear on for PTCXXX have

have how humans? First, the studies dose with track could of sort IND now And right I'm in of you effective what I you start many be might you through based sense a you at quarter? in just phase setting? starting? biologically Can the What's escalating do the this And study you the X point? dose confirm? on mean dose-ranging within anticipate courts the have Does would this do on sense your guess, healthy of filed you that to plan that volunteer being findings also wondering,

Stuart Peltz

right. Huntington, the with start we'll maybe Sure

very about which that Huntington of the We've molecule the It'll actually the and blood-brain Huntington be protein. in reduces both first this. the protein, splicing both the again, you the and that it's barrier, project within pretty RNA level Huntington reduces it's hundreds molecule shown So messenger of remind that only a nicely available and the the brain stays human RNA splicing protein. in excited which that to passes is the we're

we've observed we'll the to of the what extent, preclinical we brain, be in models. So we've and reduction levels we've see seen levels levels shown are so -- in that that that Huntington the in that in in and brain blood

able the that levels we we able being healthy to to that to PK So exposures at within within feel the get find volunteers, the want good look we'll pretty patients. be by to

so study human PTCXXX. little and phase And in X bit doing we'll the be let first for about talk I'll a Matt

Matt Klein

Matt here. Hi,

the So we be of end as said, are X we'll going the the phase before starting to year. we

xUS that all obtained, the a We CTA are there share and needed was but ethics so that I was approved. conducting documents approved it had regulatory XX can and not been of to be there

blocks passed Reza excited starting we're we're about So will study the just we those This study be of this course, healthy opportunity the all going. hurdles. phase proof important well program. of having to did as with now regulatory volunteers the X to the mentioned, getting FMA to get get Having analogous of development of splicing in we what as to study mechanism again, and the clan

Eric Joseph

ascending Huntington, it. portion? at tracking Got something tracking multiple of you to as the single ascending as more down in the happen dose? samples early as as far that dose periphery the mRNA collecting or Portion look is be that. on of Or And would regulation to that expect

Stuart Peltz

will The Difficulty] looking it FHP well the about the be [Technical as both multidose. will nice as thing at look

it And so share do we'll the available. then, relevant as is becomes data obviously, we'll what

Eric Joseph

Great, question. thanks for the taking

Operator

Brill Our comes Danielle from James. Raymond next question with

open. line Your is

Danielle Brill

Thanks Hi, for question. so the guys. much Good afternoon.

thoughts long what I'd data that term? treatment couple of that landscape ultimately you scholar and first, announced a I might trial? on in week? And this guess And be do epilepsy on your the how your on any into curious up thoughts couple rock SMA were hear the to then have were a So follow mitochondrial the fit

Stuart Peltz

excellent it that you obviously, in disease, plan and important. cause we underlying seen game we've developer types and patients, about an SMA both of meaningful important. that's Difficulty] really the the cause underlying amount three, produces think like original milestones the estimate improvements protein, when functional SMA Sure, quite [Technical so obviously, it think treats the helps of the and And that one

can SMA that patient having degeneration, concert combination that tone SMA muscle growth therapies and improved like protein, with things that see that protein. with help think improvements terms of is you in be can then when for in useful the other the combinations could long about be muscle Now, you quite but could that in in

anything help patients. So that can

So the clearly, obviously, muscle in and next of And for best this steps program. notion the decided is building really our are what decision obviously the that we of quite terms the considering for and partners about we thinking are what's important. patients

we're three So, about know, type can rapidly it the excited they notion show especially to that, more the power and two, that show better would patients you be them improvement more if than having combination.

Danielle Brill

Understood. the And can the what this competence might now and then, Thanks as in trial have access is in a you Thanks. you remind terms that. idea in for benefits rough just do of provide And that enrolling, gave look indication? to mitochondrial enrollment? what up more you timelines color? for epilepsy any then of seizure Just program, saw you that us expanded little like

Stuart Peltz

Sure.

transfer So stress and that we're points just key refractory be epilepsy are electron to epilepsy remind doing trial. with And oxidative really these does patients. everybody, epilepsy mitochondrial a refractory

inhibitor, [ph] unique a is that key stress oxidative inflammation. and have we so in pXX inhibitor, controlling regulator And detox neural actually a

showing There's excited about there's data, a So preponderance regulator. a is we're pretty certainly key this certainly of that. that data,

talk minor. have known, there's And with so we in about, on early but data some the data of the that terms based that that Matt particular

by end the amount the that could XXXX. and a other of additional be initiating, we completed of consequence delays, any notwithstanding be epilepsy, that so of COVID times think And could this

you to a little go Matt, want So, through bit?

Matt Klein

the for thanks Danielle, question. absolutely. Yeah,

to foundation's what doing test mentioned, of with of COVID sites get by possible. what can a time. is that the possible the also able the that And having strategy end so of be from we've conduct really assessments we closely minimal will data XXXX. have sort emergence with try there's areas within study be of to a ensure we're that to over family prepared that will we're study incorporate this position we from a into for As with may in the allow to expecting working any study impact finally, distributed different sites as networks and wave throughout the we'll of Also enrolled different And the world, number light of remote as of so we and COVID arise. again, been in developed obviously quickly globally aspects protocol Stuart COVID deal

Turning number and on morbidity. over expanded some to indications and related historical obviously of for specific studies mitochondrial well these what disease many as of an study different the or studies the of data, patients as of a these studies at aspects access seizure specifically we seizures themselves impact so we've a in different specific looked and and number time seen is

see demonstrated XX%, related five a or a refractory reduction subjects seizure kids We you seizure hyperplasia, influence the importantly, in a hospital cases, disease of had life, decreased reduction in significant and days. ability died start decrease we refractory procedure as or in the seizure significant a other the seizures over as So, trials occur, typically of as status syndrome In is Epilepticus as some which well mitochondrial mitochondrial where specific we site in status such a these expanded least and weeks early in frequency cerebral of have six, access, treated mentioned, reports these of single frequency reports frequency, to seizure XX% to cross occurrence seizures PCX study patients. that in resolution type And to disorders subtype Epilepticus, eight known [ph]. just in of related a the of mitochondrial investigators a all and first study medications the childhood. epilepsy complications basically in at continue as

but across related when different disease seizures on importantly many impact So subtypes look really, seeking we an you've mitochondrial themselves, over morbidity. we the done, studies collection have not only on of seizure evidence

Danielle Brill

thanks Great, Matt. so much,

details, it. appreciate For all those

Operator

comes question with America. next Our of [indiscernible] from Bank

Your line open. is

Unidentified Analyst

I taking what discussions you the questions. Hi, color get preliminary for the to You dystrophin bit about our data? if learner with a be could trans you should needed? had thanks my think how know, good Have evening, little any agency expectations is Wanted do can for much of you follow ahead, get they from formal a talk to I drug then for point? couple for would happen this approval up. for give what need do go And Thanks. And US? the a us order in you the on the through of timelines if to in production have you

Stuart Peltz

Sure.

we're trial we so complete all XX get and data. So that really we'll excited able be the to that were obviously pretty savings able to patient generate this

that use with and a clinical with is of we've show US them in is really And it of able the was was recall, accelerated the forward for with as well. years, trial dystrophin baseline, extensive the to positive with trial, And we be discussed talked in the the the half pursue safety that this with this and talked a on times the levels dystrophin could the positive result the discussed about on that it so over first the we condition sale the we've to might we've coupled data, move if is increase was that expect we'd this that. the an And statistically in approval FDA, data the with FDA significant data over we'll multiple distance you year. the that what [ph], really accumulated

until the remind with the everything's which all completed in we finalized obviously protocol, completed. biopsies blinded accordance in and now we to So court

would be there timeline, the of first in the half submission year. the again, So

takes protocol. data the that we to with analyze think the So quarter a about this

And so priority us. the a to analysis is clearly

patients positive we have in time. this some NDA forward and so for US, the forward with to and data look been who drug do move think waiting I the And being to really submitting

Unidentified Analyst

on for a limiting on the AIBC, the get of now patient rate factors that identification little right what you Okay. us BOA? how to indication? And then is And of going process bit color are give can the course

Stuart Peltz

go to a through patient team's wants doing you your little on Eric the identification. with, work bit been that start actually let's and on your So

Eric Pauwels

now Hi, actively Yeah, we're sure. Susie. to ultra obviously, this And And working is identify proximately right XXXX really patients. only disease with an worldwide. the rare patients there's

three probably more presence out in from the actually interest had we've of of care master education, attracted we've So a and expanded levels increased class so reach. that program, or things, And we've actually providers. months, we tremendous patient the four digital including a health geographical of what we've amount last rolled has number our the done the

reimbursement. going XX in are to focused particularly access on countries high we have we're population, Now the risk than implemented and really programs where screening [indiscernible]. XX are more know we've And in that

last in You know, health really nicest cost. quarter, and this it's providers no go really called there's a the pinpoint source can this PCC right via to where donor. launched And targeted program we care genetic single patients quarter screening

back disruption of think, movement. this I middle of But and was quarter, we've screened there. being amount numbers last we actually of tremendous during the got upped lot getting a tests I there screening. terms a And of did the terms some COVID, patients of seen results think have in and tested really in out we

quite going it's overall, well. think I So

Unidentified Analyst

to about have on think in to you give stages? identified, us have started been update you're I the early an And many how actually

Eric Pauwels

to access we medicines, commercial We where by we means continue, have launch. I want and to treated. mean, those or our addressable Disease looking ready patients have we obviously to have XXX continue goal time is to the a And be markets to cost have globally, patients those addressable to more first high go patients. in And we Rare are

We're screening I think, more scream on are us we're to track else. enrich results. than sort programs continuing anything to the and that to better understanding and risk which population and get best becoming high better giving of

Operator

Bernstein. Vincent Our next question Chin with comes from

line open. is Your

Vincent Chin

on my for couple progress. congrats very them and Thank questions you therapy. XXX taking A ADC on much PTC the on and then one gene of one

some us could endpoints provide you're PTC Huntington you specifically, beyond on What I with measure thinking how go of level knockdown it? and you what of knockdown? the are the just And what starting color peripheral wise, with I study And curious when follow XXX, a timing next the kind volunteers one one, Huntington peripheral healthy So, for? trial and the wondering additional intubations? if just ADC? would have what's of then kind to your guess, I'm up initial was into on, support for do Let's moving looking you that readout with

Stuart Peltz

sure. Yes,

in that escalating we’re Huntington that is multi both as bioavailable an brain, know, exposures you this the ascending Huntington, right, versus well in signal the to drug the might XX% have reduction, so at the in And in in at saw looking one as levels aspect to selective reductions the measure of it as that what the study, trials. between bulk that. preclinical and orally of in volunteer look specific, that obviously small in to so can probably again, preclinical dose blood spent say, a protein of as in sure protein animal that the we looking down blood a almost one RNA time really we blood up can because you and was studies making right, nice and changes measure. we're In we we to a you can phase what So remember within and the where demonstrate X able look we lot for we're a and correlation for a consequence the blood molecule looked at

Huntington able see to That substantially And so of exposures. the we determine reduced protein. pretty the were lack

in is the brain that in an brain all because of bio-available we the get that or tissues that measure drug level down would level Again, other barrier protein. the the that So of brain of it tissues the whole we're gets see reduce of we depending shooting, nice blood can awkward molecule, orally we on it of gets feel all dial better the this of the you a getting the up aspect sense, so into exposure can aspects it's passes brain.

So that it got to everywhere.

and the of we'll safety, doing is, measuring the reduction what the so protein. you Huntington And know, levels RNA looking here obviously exposure at and be

the HDT. at we up right this exposure So XX%, we defining to can, and what characteristics. is really, an dose, the of think That's reduction define so safety of and is that the looking exposure

Vincent Chin

one maybe see, therapy. the I ADC then a gene and second on

Or And the related year? just you can the to to you respond year this MP to to questions? response that's and has that's then they next provide the delay from little could upcoming questions, measures zero if you timelines, letter So confirm of sort CH submitted questions you've and for. of asked something to FDA later what how a is little early the to this little in bit taking color on where a EMA, a the just compare on if the sort COVID, longer of

Stuart Peltz

terms not also Yeah, no, organizations only that I think fact with obviously, contract many these working you're the with are of CIOs of vaccines. COVID. in stuff also But right, with and working on

within to some there delays done. being to need analysis go of sort this. thoughts that, that In so And obviously and pushing of forward was significance sort the to through been get we've the are I'll questions of let all there this. and asked. And, we that are on do time, order specific you know, of But Matt general the

Eric Pauwels

question. the for thanks Yeah,

some really, as as limiting additional Steve develop review standard And around on So, the data. well regulatory our these of were questions questions that the fairly do to activities, up asking key do some we're assays in fact of our therapeutics. them engaged analyses some some with acids additional factor that help that and including during the other COVID using time are CMOS detailed labs that the details aspects come the for mentioned, of as and was bilateral diagnosis also

of process the hope getting a and to we've delayed soon bit and CHMP to a of in in as to to resolved we so questions what's had have COVID activities expect little we're follow those that's backseat And the possible. we're that why that's HX. and response And related to those take saying

Vincent Chin

all progress. very and you much again Thank helpful. Very on the Congrats

Operator

comes Our next [indiscernible]. from question

open. line You is

Unidentified Analyst

then will Hi, they for a not initial approval? correction timeline thanks thanks. earlier And for about follow your controlled, what controlled doing on, regarding trial step on a more timelines bit just knows, a Huntington's. you so on -- is programs, that FDA I'm trial? from question that study blinded like bit thoughts little blinded taking a for for blind, ABC the that your have study a the comment the a then in study second PQ, And thoughts maybe their you And that came be are do Maybe I'll have suggestion. up why questions.

Stuart Peltz

and why are the controlled trial? bit of to so Okay, the where do Huntington little a in but you're within on trial, Huntington sort placebo not we out a basically, and going

Unidentified Analyst

suggested of The blinded? blinded not that they're sort trial. blind, FDA to them suggested a FDA Why saying a

Stuart Peltz

there folks, though. well. a trial placebo as it’s Within phase are controlled a one trial trial, our It's

Huntington they're a same So, predominantly exposures, they look fact position, that because unusual subjects, that the safety this Huntington, the could do protein. some of can, reduction we're so monitor dosing time, an that, could RNA can though rapidly at the in pandemic to but have and we're also for we Huntington even station, think we way I we not of in utilize studies, at the healthy in in so we

terms volunteers, to patients healthy in in we even did it when very what of safely. and FMA. looking So at sure predominate And to see will both analogous make in

doing of at it's analysis of So period end blinding And through the then look that. the you it. at the

going that and So, there, reduction on, and a drug to can to what study with reduction define in dose able look is be healthy But design then greatest alone we’re that the And we is you? exposure then that with there patients us in Does advantage to and levels. start studies, FDA the advantage, we'll that to accelerate able that bring the the the we right, gives define even we're basically from Huntington's again, Huntington's drug patients. appropriate months to engage causes where these volunteers we'll the Phase going patients to want early very and X studies, the precisely help is to think that help accelerate. we

Unidentified Analyst

very Because doing a Yes, FDA, early study? sense stage. the more Huntington’s a to get in smaller so which you willing do they're do you at blinded trial, weren't a is if

on well side is of the FDA more quality early I it just limited sense more feels if are approve a that willing from was good the as wondering standpoint… getting on patients like So to -- you study.

Stuart Peltz

do in that healthy is into That's of beauty do more [ph]. want these reason you thing you an to adult. studies is we Because subjects could find the really rapidly. the beginning in go studies first first the going The to these

they’re They're blood RNA coming can in, get look get you X hooked sort you volunteers. and And on up multiple in the the molecule at them was studies, therapy levels. times, line drug, path, so It's using -- probably up. doing small different we gene we at protein Phase have -- this healthy they to of subjects, so and all a is a they're not unhealthy

dose So you define that that dose want you then lets think you Huntington will the gives to there but do right more be you this very that rapidly. efficacious. what level, guess you I into patients appropriate the exposure Then there, from the go can reduces get you

for The PTC’s disease of need [indiscernible] no the blood what's obviously, the we question Huntington, that barrier. we really the RNA passes it gets medical protein cell. and -- is fact directly to know that brain blood every reduction significant hits about unmet and that Huntington's So unique of think brain

there's So a the major is advantages think so we this fastest think lot to there. way we this of in and get

Unidentified Analyst

Okay, you. great. Thank

Operator

Young with Our next Cantor. Alethia from comes question

is line Your open. now

Li Watsek

in your perspective Hi. This on follow on Europe. up taking for Li of Thanks just to Alethia. the AADC, activity, on for the about curious especially is call. I want sort launch

early Do that product? a You for up. have one. confidence screening seen you've follow the the in have trends the And I your support

Stuart Peltz

Sure.

I activities pretty we're on. there's launch? going lot bit of a doing. on his been hard team have to image terms on through So a to I ready of this. think working go Eric get in and wanted what defense Eric think little for

Eric Pauwels

especially Europe. many connections screen. labs each a local the lab. We've the key a question. activities, is AADC know and most about I really preferences number in adapted different to we’ve and first cases, Because activate we've and confident how as own -- important country done has their launched to the for thanks course, about of of feel sites of mentioned, thing and book We Yes, lot very using in we

providers. can execute to algorithms already, [ph] begin develop partner [ph] we're of able a them this XX we're patients those process really in programs. healthcare of retrospective helps accelerated with programs. now we treating And the approach mentioned, where to the them. identified various an algorithm-based find Europe I countries with patient also number as XX using and be in cases against And We're using we've to that So and

the thing all the preparing first unique reimbursement. and will standard with a health good to our addition, work preparing a care. the at readiness patient, and are and and be lot not do key sites there's the treatment to out but also would Europe In We're the upfront doing is that the work approved accessing receive continuity One rolled that ensure is treatment ensuring remind will looking AADC vial just of [ph] what's treat site it component of in therapy about care. gene after product you and the as technology. to is of I that We're regard that it be only

different it's so gene for therapies approved And been have that indications. replacing not other therapies like other

So AADC treatment. and its will have first treatment only

in of countries, programs first, and certainly terms process access many we early like where as long well available would as the northern include in Europe, CHFC, many In of opinion, Europe. will who sequence, of garnering which southern positive those Germany classical launching following the sequencing are begin and the of and

Li Watsek

it, dystrophin U.S. environment a study, started the on regulatory And getting bit then little more is conservative. helpful. seeing Got that's we

you. function be see dystrophin you data So with your my -- That be be sense want us will still maybe think question might would to well just Thank a FDA is, Or the helpful. you that agency? as an do do endpoint? discussion the approvable give think of recent

Stuart Peltz

Yeah.

all was -- that and And that was So, really remember, have we in is dystrophin we And without to the it in first, way talked study -- they've to to the would levels accelerated that about they with is didn’t we the that that discussion them if minds XX, over this experiment. has told they has think be the look be when gotten trials do which it about that is on to one the thing they bad sort you after the data that's looked their of they they think benefit. any significant. about has with along data wanted approval for you that what know, we've we that why would efficacy VA, the companies the be talked end the statistically other clinical well -- the day, that we that, did as at extensive I our at dystrophin be the with But be completion the that, both -- and approval. above -- it -- safety data and full clinical over levels, know, that clinical did appropriate that us background approved you to data, dystrophin alone, That's that we they sufficient as at of for done. that of have, the have, have do wanted

the combination have that with have data the So dystrophin trial that nice And both as results be then that's full that I the see think data. passage out be as dystrophin well hopefully, actually are come clinical we we strong data benefit that a we'll that for with that the would clinical approval. ongoing along the have that will we

Li Watsek

Thank you very much.

Operator

Thank you.

with Our next Beatty Citi. question Joel comes from

is line Your open. now

Joel Beatty

for Hi, the thanks taking questions.

The first one is on Translarna in Brazil.

pricing the characterize compares from volume bulk order the For recent you there, and orders Brazil? previous could the to how

Stuart Peltz

Eric, Yes, little about a sure. bit you that. talk to want

Eric Pauwels

spending disrupted aspects but been, of, very, say pandemic. in haven't is has In that administrative only Joel. been healthcare I has Brazil, companies challenging of sort any here. of been really in way a exceptionally -- it, PTT think many all, the of know situation just Brazil. that been not the I we're, The very We has pretty the team country proud Brazil's has hit right first Yes, hard of has can lumpy now managing terms what I'm COVID-XX orders the diverted. orders. Brazil received by And their

and off job treatment. others of added the asked QX the but done that good and and opinion work risk between shouldn't important, order. And we and in be find and budget received, that's leaders, bring Health of patients which to And QX. the patients Minister we've of due us to be constraints else, and there terrific help order orders everything the second already shipping mean, some substantially split So more the to the we about we'll continue a really and I to sort few that patients. with, team to has really is advocacy groups, news to awareness

substantial necessarily the about here order number through we're also diagnosed process. new we that patients specifics, Now, and have that the gone out substantial is was patients, judicialization cover but only don't happy give a been of enough of the not existing what to had number but

that year in patient and first get pleased of will we're drug continuity this very the well. year next to I’m as So to be able patients half

Joel Beatty

year, this Can one. that filing and recently, is question half end part be there's characterize NDA to seems which one the the could on filing? months be needs And for you And help next in Thanks first and year, AADC. hope some completed I to seems between now seems because reason to of be the up be now what like other that the by delay. six it to of the of that ask

of the you So accounts the extending maybe for timeline? know, what

Stuart Peltz

to surgical previously talked -- it is associated we what few Yeah, to little about fourth so patients get pandemic been to more I've want now that lot we expected of the quarter. a the part pandemic. gotten obviously, and after news additional the as math -- talk you it report we're go did a a complete little bit bit to a And the my detail. But of is gig that? has Matt, features a really with in factor

Matt Klein

sure. Yes,

BLA cannula before, we therapy the are got the So we've scheduled talked FDA that precise gene treat patients request the they've that to device into scheduled those we're And mentioned, the of the was to an fulfilling the the quarter, and of surgeries fourth on commercially. for and delayed about items due use area was had you now third with And the delivers to cannula brain. in one key quarter. as gating it the the going COVID,

those procedures. do to expect we So

not we thing that important study therapy here without of of the or the is gene the but procedural safety can of cannula, efficacy the demonstrating gene the to therapy to again, get therapy, right any the gene Now location the complications. basically

and procedures, be the with make submission. the plan from gather move then data So FDA will and sure we're those the the forward with aligned to

BLA able to long-term follow-up durability have about that X,X,X,X dopamine the gene harvest therapy the aspect this the is therapies this with production one is obviously to really to very and it feedback able we're the mentioned, of of a of to with a to things data treated because landscape sure why submission gene aligned durability And the HX to We're we we been that delay and is in you some XXXX. make effect that companies. closely. we and do remain to able of been years move the well that look also As therapy. response. well enthusiastic on package know crucial that are we already. obviously, evolving do have watching of other expect and We're long-term want agency a forward durability we'll package we as very continue want emphasize as to we've clinical incredibly package. I And during in strong patients the of we has That's aware demonstrate to treatment also that, forward the after data from be program that move

with moving on the to procedures look have forward presenting and So to this done, the aligning we with package being forward the other agency to any able issues agency.

Joel Beatty

Got Thank you. helpful. it. That's

Operator

comes Joseph question Cowen. Thome next with from Our

Your line open. is

Joseph Thome

on to a disease submit Friedrich when for expect meaningful online? ataxia, note, for you. on is you then that you're highlight any patient XX are Thank enrollment, that in looking? taking now week reduction you the consideration Thank And the note physicians any timeframe making in a related us severity? gene as around to questions. depending there. And IND updates clinically you ataxia Can FAR On a Friedrich that Score give regarding is there Hi, that you as my vertical therapy?

Stuart Peltz

Start you join Matt, us? Sure. want with to

Matt Klein

Yes, please.

score inflammation one that iron building to we're one own [ph] those couple most One as in the associated the FAXXX, and is which stress a oxidative points. that, FARS studies, clearly over others. a prolonged which period mention the So in be of treatment. a is data a important have we important demonstrated had pathology. shown in XX well three target -- reduction incredibly FA to body of phase one efficacy to a base the significant in two the is as months, And pathway of of improvement on disease And severity. significant top phase to our the and of it's that experience the clinical lipoxygenase we of trial After on translates obviously

treated improvement X.X an improvement on relative history of to a that give X.X an those the was points. of points the natural in example a you FARS to match Just cohort, is of overall worsening which

to So we believe effect that of significant history of relative a disease. is understood the natural magnitude

clinically believe aspect placebo then I and be And placebo that's parallel only that can of the present disease. we is based is And effect on of so the reflect scale. endpoint in progression, we able which, but months effect aware in think, we on, strategy and study, a so course, to that months, a for Friedrich strategy two also key going XX-month EMA what on that aspects the incredibly also because elimination FDA be phase of an meaningful FDA’s function. mFARS, mFARS are that which of experience, treat And and on is The able those, to do we'll the that both phase, expect realized disease not six where the is of and include a endpoint to put important to aligned the sometimes effect, course, well be there XX long at as as effect of have by longer. controlled in or to a others we're we have walk of was an again, our of addition, key able even to countless of own well on as that hopeful activities placebo daily feel with selection that living ataxia endpoint the is being of crucial which desire statistically to we've see again, dampening and duration and we effects saw secondary all significant endpoints tests, demonstrate term is

severity, also of And on while the to severity disease a important the Friedrich mFARS group placebo of disease critical group. range that about for ensure question impact we aspect mFARS and stratify set. obviously, the also a is progression to scores ataxia. have to those therapy understand have we placebo plan we potential only to not your balance that the the It book severity, in disease randomization Regarding can we'll both in and response a between that treatment on rated this

Joseph Thome

And on does when that kind -- the you. Great, the thank I&D then bit ataxia the of just second clarity can be a was little for gene more on give therapy, Friedrich submitted? last bringing part Hopewell online the

Stuart Peltz

think, think we're -- well, on we're and a pretty gene I therapy working precise forward to And as that Yeah, it. the on, move enthusiastic we the update we'll be and more you I give that, the Friedrich center on insulin about able support to still timing. the

Joseph Thome

Great, you very thank much.

Operator

from with William next question comes Raju Our Blair. Prasad

open. now is like Your

Raju Prasad

taking for the question. Thanks

up follow dystrophin on study. some made comments a on the the of Just

up? mind versus So then I the background of is what delta kind a the and have study, follow sharing for just you would

Stuart Peltz

start. I’ll Sure.

particularly very that For of is number no she about there [indiscernible] actually, approval, precise us analytical might but no -- and the in is remember it's important terms to there when sensitive. addition real was Dr. the in said, quite you Woodcock basically, this, talked point and

to of little want terms you and and see a we background, Matt, So the to ECL the level. should monitor above and about where be able are bit that we that. talk what's in on

Matt Klein

absolutely. Yes,

As you said, we recognize that.

over endpoint therefore, at in our with whom recently levels far Finally, And experience we interruptive had we of it those we dystrophin the from know right, statistical as that want because them seeing improvement be as low typically based the most baseline there's on important we an patients. we that to that expression, really alone, have that have on sensitive, expression dystrophin that show want know develop that and of very able was studies, significance for that approvals we're us same we incredibly to who dystrophin based of assay lower was incredibly can to levels capture to need.

we've which obviously looking is low setting coefficient of others baseline assuming more XX established statistically demonstrate dystrophin was different our sufficient baseline, that up potential a increase variation with significant for that and number agency we as in really at ensure for to but that the what in of obviously study well. us, so modeled established over have dystrophin levels power the done threshold subjects eventually baseline this a than And expression, scenarios, improvement of to

Raju Prasad

you headwinds the quarter, some willing like you quarter Thanks. spiking from seeing and the Great. type X; obviously three on X maybe and a you nature commercial into of color split potential just guys that that quarter? know, for looks into Emflaza And of a expectations you kind adopter it -- any prevalence And good us the two franchise, your the X up Or fourth have could the The number. on and the something or from risdiplam, type function give given three physicians of are then is between two the to on disease patients? is early do more again, you fourth that's disease? COVID of going and the learnings anything to being second think and prescribe

Stuart Peltz

sure. Yes,

in know, been good Emflaza. terms portfolio of performance -- the really from So doing that from -- risdiplam/Emflaza in you that the very 'XX really, both we've

even it's through. And actually COVID, get been -- spite in so some the to even insurance because well; has in thing ways, quite with allowing done of company COVID, with more

So the is fact transplant the risdiplam, of Emflaza was orally the as advantage. a production, distributed more patient to advantage Eric good pretty talk think I But through and big directly the really that that bioavailable, can well. about it's

Eric, do put this? continue people to that. on color And a with so want more little you been to able have

Eric Pauwels

Yes, sure, Stuart.

step reduced to Care's is of I'm fourth has quarter. that seen positive. either PCC new prescriptions, our time in which timing. that quarter have strong US a the to in during prednisone, or and a base And on growth of with the very important the year-on-year, involved XX%, think into we edits the so payers the these franchises time over growth previous very quarter, seen to Main continuing lot few all, of the really team of pandemic very there have We've we've for the patients and -- that focused been anticipate XX% well a the prescription strong the incredible that the with mentioned been, very, of really maintained DND of growth that are compliance, really new first patients from we eliminated had reimbursement team, proud and with we over quarter-on-quarter and Emflaza, here on we've Stuart Emflaza. prescriptions Emflaza; from far and dropouts again, have drivers well terms continue that's from I that as especially and great has team position into in response growth we're see time -- have; the high have

pandemic has Translarna, patients COVID very growth seeing certainly and Spain, we newer in for in in think areas accrued France in hidden hit the places them really we even by where in in Europe; hard. the so As are other that Italy, like are we places quarter

we in Translarna, don't say with So now and the see quarter. that it's any that have figurehead think we see to Brazil, we all order -- within to continue safe in I regions, that growth importantly, is the

Raju Prasad

Thank color. you. I appreciate the

Operator

with next from Capital Abrahams Our comes Markets. question Brian RBC

now is open. line Your

Unidentified Analyst

you'll you Steve to my you anticipate will clinical [ph], blood-brain given gains? XXX, to one for diffusion be for this but early progressive question. Thank On this Brian is I'm to you nature Abrahams. treating how see one the just taking you Hi, think a bit, on do touched have to Huntington's; deep patients do start ratio brain extended of And structures? the

Stuart Peltz

of orally the had an small good drug that's that. again, really exposure for beauty very Yes, molecule brain. within Yes, so bioavailable the thanks

that treatment that all So the the honeys blood brain. the were well the observed with we've of consequence in regions protein, very a and as indeed, levels brain PCC with of studies, that preclinical in in correlated of shown lifting XXXX, the

that's you especially you Huntington's a certainly So, always I where advantage, -- that get get -- you brain me, disease a been -- major that can think like get self-death. to that in certainly

and well. obviously that; titratable of all So, tissues PCC XXXX you in fact, so know, so a is, reaches distributed, as and real equally is -- the there's it's it that, systemic; advantage and is

question they point when treat and, it's in interested your we patients, you'll Huntington's So, that. yes way obviously disease; the of in know, toxic patients there And, trying -- benefit we're degenerative no you one sort is do important obviously when believe good patients reducing that's a is I take important. of pretty Huntington's think to of of you're be belief your that I'm that questions, view to predict to from know, you time, because to able obviously,

So do, dermal because would being know hard able you the patients the don't do notion long put of to nice, while study is how be. to

you start we right, manifest terms need think have trial clinical of then something in with to ultimately, patient, we with I because measure. you have, can So

look things know, course, disease. and the of at You

disease, move that from learnings that. the that, obviously of occurs So, not alter it obviously, well, looking the juvenile ultimately, we the only studying early that get something to the and clearly, the the before look is rapid we're types, prodromal show onset, But know to so you you because once as want possible to so can should to you manifesting rapidly, Because, as patient at of patients, ultimately, patients, at more the want understand manifestation. looking and move that's but, more well, you that then well But and patient obviously, you and goes get know, there's as into including, step we're they're both the rapidly treat and in, all then JC that. as earlier first course which know, that you into manifestations, possible, Perdomo.

I that look good know when we to like it's So, the really we're we just at estimate time know, us news it's the we're did going to clinical blood, they're you fact demonstrate you and we effect. you to know, really, an and perspective, this, now be only target the reduction, about is our on learning what think know over from from my that but we're, really we do as see

Unidentified Analyst

you. Thank

Operator

Our next question comes from Gina Wang with Barclays.

line Your now open. is

Unidentified Analyst

our for is David Gina. [ph] I think on setting it's questions. This

one So on question program. PC a

CH-MP as half discussion be on with soon for paralyzed? and current installment of the for then what been year, What's well. reimbursed pricing, the opinion claim has as will first So next I payments consideration as

Stuart Peltz

sure. Yeah,

we this easy done, of clearly, quite about terms an ABC And obviously, want bit of we've it's lot are talk a that and results see done in all really a And of we've drug. that terms effects looking the little compelling. Eric registered. of So, to work to one in maybe the have, the programming you getting the work

Eric Pauwels

there. Yeah, and that’s ready. It's quite

to because go we ready been it's time. a now ready a strong And for have studying have think very because been we've I interesting, getting long we're we And little data. patients.

have who But currently assessment, with treatment. a for data that. we all forward when have pair drug the in with And by prove go than As children we reminder, years, have There to even and a are this approval. and in need. had we need have longer we our with out, anything durability, there's cases are treatment children HTA ADC, unmet more five which to Europe, when available given, patients some early to have emphasize else multiple for is importantly, commercially that years, that going access this, high those that discussions is treatment more for seven no ADC than what treated that early that now goes is this who after we those mechanisms, is try

a from care, technology the standard assessment and healthcare bringing new we're health bringing we're only care. perspective, So, a of of standard

have as will access and narrow Europe go as the number which the benefit have already very we've in and by course, of well of such mechanisms will And across corridor things, would markets, we asked launches then earlier, based is So the each States. pay markets first good but Germany we programs. as pricing the a the early to free first maintaining approval a United been anticipate Europe, I and about Europe, addressable, reflect where markets need country a will to number mentioned willingness of free that in as and to that and have the it our would pricing be international pricing population on different across commercial the certainly unmet its us at traditional have very subsequently

targeted as other and targeting potentially well, markets in I So Asia Pacific. Latin we and those Europe of the many following outside European southern those, Europe, think of would particularly countries be be America will approval,

a worldwide. care, where small at in population strategy product, other will care, established an but based XXX are, not of on So [ph] replacement a patients big this standard time a certainly gene it's unmet relatively which need we make be will standard therapies point our price as discuss burden, that new of of pricing high and

Unidentified Analyst

Got it, helpful. that's really

actually be just question on T he you X could follow ID a attended the up; program So dosing. [indiscernible], a another administering ballot, so

and of of pure and you just the the what's color any the also the can So, some burden on Max provide half-life the effect? like profile PK T drug, including more foot

Stuart Peltz

Matt Sure, to want you go?

Matt Klein

absolutely. Yeah,

can So quite XXX of also medication. pharmacologic which children or the feeding years times for seizures Obviously, a it food, with administered, administered of is that dependent. helpful number be refractory the mitochondrial with disease, drug many three a is particularly well. TID been with a a are dose patients children, young parameters including It's given it's and given disease, treating tube those as solution understand day we've for mitochondrial

and and some drug which through of lunch, is So being given with half-life dose, patients. And able times a breakfast of feeding the two in the a convenient. renderings within form times quite quite it's to drug, though a four tube three obviously, day three day, then, the generated is is between dinner solution course, given hours meals, administer is

based the and obviously that's previous is of imagine again, drug. obviously, just informed on as pharmacologic been And evaluations, this well well so yeah, as

Unidentified Analyst

much. so you Thank

Operator

And next up with Vincent Chin from question Bernstein. is a follow

Vincent Chin

so the in for amenable. couple us, much you weeds of the a follow have thank taking follow deep ups Hey, Huntington's I you're on if

function throughout still knockdown? a made what's you're expect is shown And the knockdown dosing range how I excess which of question But may lessons you adverse guess, way looking risdiplam, sake knockdown so a especially XX% loss there distribution or to for of in the [ph] from it's because, first likely often simply easy is, at wonder, competitor, worry of also I could And to well compare an I effects of minimal again, you second to, point a, brain. the you plan, all basically, a for preclinical the XX% on range deeper the about or go Huntington's studies know, for the you've on preclinically? from plus or is and can little to adequate, how you love imagine dose Is the a a of what The uniformity the based safety some sort mice. into pharmacokinetics, high amount of perspective, be comparable dive program given from seen

Eric Pauwels

for that. thanks appreciate no, Yeah,

to. to between thinks what to therapeutic a And somewhere XX% Yeah, that of three that you've think but results. benefits based So the be with that up the the preclinical associated titratable. XX%, titrate to be was nice an want would results to drug question, and Obviously, community get is therefore, it what does clinical bioavailable XX% orally some there's discussion could you I seen in could you the aspect the on level that good, it

our shooting thing, used benefits but do hard So, in bigger, expectation. be to that's well, know, the accordingly that. effect just humans. day able to some what and think the certainly always that. Probably be a it'll range actually once that but in we'll obviously saying of as and put a humans, that's measure that in our our say so, with, But a until and in plan dose way, be that you're consequence we And know, once of with, expectation. does we're it's see we to for you up, wait similar goes be how you We you it's a able it day will think and to to some we expectation,

Stuart Peltz

Your experimentally. we if -- question yeah, at want thinking that's post had lot you probably definitely I about terms terms of the know, something we're think we down And the in think question In development. you you to of of look can , we can to a go just higher, on you But substantially better. we of we to out we're do range think that Obviously, of go? that sort do that's of, this figure fine XX% about high think to would trying some just even it. can. But be and able But XX% how

Vincent Chin

that I what at don't guess limited plan, I some stages. high. of risky to guess, wise, you I see. And dose end guess then, of sort the preclinical the I by upper go Actually need

all put high clinically, seen that's XXX. with is to you there Pretty up anything works to basically think invasions an that sorry, it That healthy So, very of how lead been on upper work out well. pG some limit would or in volunteers. sort could

Stuart Peltz

studies package, the that Yeah, think be have to data far, nice I that that. you we very supports the doses reduce able in that us, think non-clinical to think for led being we a I so much have adequate want will us able if being

good So we was spot pretty feel pretty this. good, that for a

spent obviously, the also but this is molecule, a we interval, specificity, on time lot selectivity pharmaceutical identifying of on You know, properties.

maintain drugs brain. just don't the get the barrier, the and the know, reason that work within even they some don't that the as within not obviously, for they they they brain get within could you that we think take, can was key a us, pick to of so could brain people You because lot pass was blood cell. heat flux, test there blood up appropriate levels things they flex brain And barrier, of in no goes, if D for the substantial if issues the

so done it we've not does about we that having then it a pretty so, good blood this. property, all make that feel this and Isn't on all brain, couldn’t to gets And eat tissues. lot pass that right the nice, flux of that top work but drug into sure the of they

looking results trial. the we're so one the forward to And phase in

Vincent Chin

Awesome. Thanks up. the Appreciate for the follow taking discussion.

Stuart Peltz

I the but appreciate question. Thank you,

Operator

I'm turn no And you. Thank closing for time; remarks. would to sure Peltz the over at further questions call I this like to back Stuart

Stuart Peltz

despite pandemic. to I'm been I the Thanks that, challenges all the see in calling the today. And that know, PPC well, you performed them team of you access for has thank have as you thank pretty and of Okay, during can how by you. were think COVID proud think discovery able then there's to complete of the biopsy mission the our that approval [ph]. protect many translaminar it us we that culmination we've We the development of teams, so it, the time. by NDA able also to been the the was of about a, of years presented

well. as so And about that excited we're

We registrational starting particular are known practices. we to XXX phase to And PTC trial for the one and the for you've for as and ready for performing honey. DBA we're now epilepsy, just studies seen, initiated mitochondrial PTCAXX

the think potential value excited on stakeholders. we're needs with portfolio really, high commercializing, are we So, drugs built, therapies a we and medical the the where gone lot this, has that discovering all for patients that we've creating and actually about of of quite all developing in unmet really have valuable future with our the to generation of that next

want I to time again some time for that, good with us. So evening. a you thank Have taking with the for spending

Operator

Thank call. Ladies this and participating. you gentlemen, today's for concludes conference

You may now disconnect.

PTC Therapeutics (PTCT) 30 Oct 202020 Q3 Earnings call transcript