PTCT - Earnings call transcript Q1 2020

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the PTC Therapeutics First Quarter 2020 Financial Results and Corporate Update Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that today’s conference is being recorded. [Operator Instructions]

hand Head over PTC of to today, your Therapeutics. like Kane, Alex Investor now the to conference speaker I'd Relations of Thank you. sir. Please ahead, go

Alex Kane

Good safe. results. I discuss that us everyone quarter afternoon well for hope corporate PTC and doing staying XXXX financial Therapeutics first and to and updates is you thank the joining

Chief Matt our Officer, Emily appointed Hill; Joining me our Officer is are Financial call Development Eric Chief Executive who well Business Chief as Klein Chief on today's Officer, and Pauwels, Stuart Officer, Peltz; and as recently respectively.

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

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

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

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

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

Stuart Peltz

early. safe recognizing responded everybody staying COVID-XX we team update on incredibly PTC the seriousness an is provide the to I’m of healthy and Alex acting as challenging how I joining you the COVID-XX thank hope quarter. today, Thanks, crisis, and of proud amid has time. that us for first the these and

all understood COVID-XX including and that threat implications In and that individuals of late health viral of PTC, a understood expertise included of the up February, task epidemiologists areas experts, we force the pandemic. physicians, public set the from

to from health and the home. ensure equipment to safeguard of that a effectively This and to refine work necessary safe. continues meet processes productive force the immediately to team have allow and they implemented and safety remain us This plan task our employees that

second have arise aspects well force manufacturing, our task critical our on assessing that research, supply could therapies. clinical business. our a to focuses access chain, We programs, as the business force to This issues task with is ensure that continually of commercial as

revised have businesses functioning. in mitigate are how continuously they to We strategies potential our issues well and assessing

services working now teams together treatment. ensure Patient the and are patients necessary access that to have to engagement

open employ to what also strategies of created at force the COVID-XX have is think to strategize globe. what mission through different we whose We as around should places job locked in a task world look the needed to begins scenarios next the be and successful third multiple down. is to Their be is being will from

adapted have teams teams of commercial these As our a and are to patients result products. Field with of execute. have the engaging driving physicians, our awareness identifying efforts, continued and

they Given doing digital the of personal number remotely a using ability for limited so interaction, approaches. are

Our programs work to forward. scientists continued in laboratory also the research drive have to critical

they that including put to environment. allow a in staggered have so interaction, work can We shift place, safeguards in reduced safe

ongoing treating on the sites. providers care slowed or COVID-XX all patients experienced and have hospitals industry closed in companies and as health trials As impacted clinical been focus have and have planned

response Overall, The the will talk effects of impacted about organization's program. gene more COVID of in to Matt I'm have proud crisis. therapy COVID-XX regulatory programs. filings also our these

capital sheet on of than strong position with first quarter. of balance $XXX as a have the We end million more our

capital about allocation. prudent and strategic both being are We

begin at manufacturing XXXX. facility of impact, clinical this because deferred example, of that will As in at expenses capital COVID's we and certain now anticipate an have our facility material early potential Hopewell GMP

commercial SMA patients the the first will at and that we And home. beyond. I potential keep can business us the have and strong be for place, both time, products global the well-positioned, therapy. in crisis to These at that infrastructure PTC of have patients in team aspects our to home administered also want include highlight and opportunity delivered commercial that for an other the

In disorder pipeline have with rare we diverse multiple addition, catalysts. a upcoming

now SMA anticipate me first X X focus the We the both pivotal FIREFLASH for the broad in SMA. Let type with for and recently starting quarter, study a progress year. with during on label the an disclosed SUNFISH later and pivotal approval made patients SMA patients. study we and We results the in X meaningful positive the type this risdiplam

positive of and Part the increasing X the stand. and study. developmental functions Earlier XX-month its over, this demonstrating are SMA study SMN results and achievement week, Roche to FIREFLASH partners roll we the so X Foundation, quite importance systemically motor results protein the and of sit with of the shared pivotal Part since ability milestones FIREFISH along our The exciting,

XX its months study endpoint the The results highly milestone particularly P value with the these with enrollment these exciting. that less X.XXXX. met significant. without than and at primary met XX of was patients are already Considering XX age and disease, X.X sitting of progressed was of statistically infants support months XX% and median a patients had

The safety data safety consistent were profile known with risdiplam. of the

infants even CHOP-INTEND, all a and baseline XX% CHOP-INTEND a median improvements XX observed this survival. Part Importantly, to This X change this were in Part met patients' than key walk all by greater XX of with assessed showed secondary stand of the HINE-X event also showed months. study its in from points. X endpoints. ability and baseline study from HINE-X improvement included as free in to

the component infants of treatment without bouncing XX% XX months maintain One swallow. patient with patients ventilation permanent and of X milestone, in of After key risdiplam, the Part XX% developing the FIREFISH were walk. were XX% alive. a to ability to of ability reached

compared benefits as As a call, is function as to Dr. bulbar Servais pointed molecules when reach out reflect other on to the brain therapies the stem. this well ability particularly the may impressive small Roche

and SMA type compelling. with older the support. Overall, X that these the feeding X, is permanent type history median all age of XX.X infants or death SMA require the natural a than results highly In contrast, clear are of in XX-month it's months ventilation

PDUFA recently, additional communicated date of submission support FDA the from August to in and SMA to to XX due extended range we widest submission patients. for these is risdiplam inclusion anticipated data The the access data broad As X. reimbursement of SUNFISH the of the Part the risdiplam of to

Importantly, to has issues no the review. FDA substantive during identified date the

the risdiplam upon logistic environment. Roche preparation careful drug and has with launch working assured current its is supply. Roche's of providers community COVID-XX partners to ample supply its ensure the proactively SMA in drug

commercial access early Roche's pre-launch several initiated. programs part of As efforts, have been

to access for countries United SMA type announced has States and it open that X was to the patients. program patients. early expanded Earlier include this year, SMA European type been X programs Recently, were the

received to risdiplam. from the decided COVID-XX pandemic. Roche program type the amend type to X to has to response has in be patients a current X treatment and for whose been interrupted patients Importantly, these requests to of allow consequence direct receive as able

that for critical try unmet requests an the the is within need risdiplam more COVID-XX These exists shown they navigate The home be patients for SMA significant need through even oral demonstrate to is for population. a as that taken to at pandemic. therapy

administration, important available only SMA for respiratory would therapy patients with visits for that require concern In fact, be complications. risdiplam an does clinic not the

a for risdiplam broad a expect robust patients and that approval. anticipate following We global range be of competitive launch will product the SMA most

platform. blood our compound being systemic of was it it's the all is arising Risdiplam is from so tissue. first advantage can get that the splicing to One that compound in and affected

and SMN SMNX, mRNA is to risdiplam's pharmadynamic measure advantage protein tissues. the ability or on blood effect second The accessible other easily in

Huntington approach in in in studies The where We plan As are approach studies candidate you we same splicing its program. this and program may from an in ongoing. healthy activity toxicology risdiplam approach same will utilized in to the Phase be successfully proof-of-concept other development a used IND recall, PTC-XXX Huntington's is of demonstrated disease, use PTC-XXX we X volunteers. a

Let me with to a our franchise year-over-year quarter now growth the quarter. $XX commercial million. our with had DMD of first revenues efforts strong We first in turn

sales. sales We are in Translarna reporting in $XX.X U.S worldwide at million Emflaza $XX.X million

positive in we the following to quarter. addition, see first continue In weeks trends the

I Nevertheless, further financial until hosting business months. and guests. our the in on that time. and withdrawing cannot clear York New also effects COVID are Analyst of duration on an COVID coming commercial impact increasingly either want this employees becoming have the be health the guidance to we Therefore, or Analyst the in our at about our Day. XXXX It's of Day in the would the of safety we revenues, severity predict of understanding not the mid-June we talk of best City over interests

first and platforms. way shortly deep will and dives to this in platforms. webinars into will posting to be data, the will which update the all a the which programs you productive We'll We switch meeting multiple programs occur. our We be on and hosting we believe deep dive do postpone our

to turn Emily. the now and and I'll over call Eric Matt

For Matt quarterly update. business team the give of the Eric, their program. on now will the update, Matt welcome and cover Emily and to more the and the financial we calls. status them you debut on first clinical update on a executive will will Eric commercial

it turn So let to Matt. Matt? me over now

Matthew Klein

environment. even to exciting our at clinical into forward look role of join the with the time team clinical an the to step I'm Stu. thrilled light programs. to in management upcoming progress current Thanks, I It's PTC, executive and catalysts, continued our this at multiple

of our with clinical Translarna updates for start the timing programs, terms dystrophin XXX, will In study. Study U.S. the I

require site third elective current procedures, now completion the has data timelines, still patients delayed to biopsy. the the top in XXX final anticipate a on reporting closed site's is study COVID-XX, Based few study muscle the line study to has Due quarter. which we

for in the would that accelerated in significant clinical our expect be sufficient statistically results in combination We data, XXX approval existing U.S. with

As site, endpoint with percentage primary measured ECL or identify suited dystrophin dystrophin. to XX-week as The proteins, collaboration FDA, we [indiscernible] that is by aged from an highly with ECL. single developed well electrochemiluminescence close the a particularly and and liquid boys this such enrolled nonsense XX sensitive, validated study assay seven based that two a linear baseline is as to is large mutation. highly change reminder, years In

our now small platform, Stu for detailed Turning risdiplam, to our this first results exciting splicing from platform. the molecule

will Our include clinical volunteers healthy Phase to X inform to program with start remains both pharmacokinetic order initiation multiple single safety These disease next in ascending studies year. studies the and in track Huntington's dose of of end and the regimens parameters. splicing to on prior in

will epilepsy reduction refractory in of in Friedreich straightforward registrational selection RNA initiate the e-platform ataxia in and trials expect due COVID-XX, now support to our relatively to bio to achieve we target be PTCXXX in range We and those quarter. mitochondrial potential the quarter Huntington levels the Shifting in these studies third expect to XX%. fourth

trials, clinical we for once We will studies sites. sites can to patients from travel reopen the ensure and available of safely these course, and study and once initiate planned are study can

The Phase track for which as quarter. X indication, developed Parkinson's in the trial first being to start GBA on is third of PTCXXX, its remains disease

have oxidative the inflammation mitochondrial platforms. discussed that pathology. the we XX-lipoxygenase, complementary platform As PTCXXX and that a synergistic hub key regulates CNS previously, other with enzymatic target and PTCXXX our Bio-e disease and is underpin and stress

PTCXXX as a seizure hospitalizations. promising efficacy has number inherited been clear as favorable with related over seizures, XXX well of has primarily on and had And signals, morbidity, the children mitochondrial reducing patients, with as PTCXXX patients refractory in patients demonstrated disease, use access studies safety tested well and disease. in compassionate seizures as expanded impact in mitochondrial including it's

PTCXXX trial X XX assessed demonstrated severity, history score. Phase in age Friedreich matched bar a validated in statistically and natural the stage significant relative disease at by impact ataxia, as controls the addition, months, on In

for data of development disease. target the its mechanism PTCXXX, its preclinical action Parkinson's strongly and For compelling support

a in targeting important Therefore, current in microglial pathogenesis, modulator simultaneously to be driving XX-lipoxygenase The us pathways, therapies XX-lipoxygenase factor known only target pathways. disease a including very is alpha-synuclein and of pathways activation, to aggregation allows multiple these target Parkinson's development affect oxidation multiple to of of many upstream key while and neuroinflammation. one

and ataxia therapy COVID-XX activities. associated our has platform noted, one will currently multiple respect labeling gene Syndrome the programs, anticipate therapy be With therapy and quarter. impacted program gene our as filings IND updates, you Angelman onto to impacted timelines. COVID-XX the delayed at has IND We gene Friedreich Moving least

to in filing plan we as understand an update we on programs. timing better ultimate COVID-XX the of Now impact these provide

commercial deficiency program, are the by be been procedures XXXX. in activities for COVID-XX. hospital The deficiency of continuing remains of patients. submission remain These study track. use canola elective expect gating second factor cancellations Moving to submission the BLA AADC now FDA will the procedures have for the the of in on BLA and our due to Therefore, manufacturing the delayed of to half we AADC the treatment

AADC the final still EMA end related to in in despite program was Overall, January As the submitted catalysts opinion for delays, of reminder, impactful timelines the deficiency clinical of COVID-XX place and a months. the remain the and in the year. number for the MAA exciting by we expect coming CHMP

to the call update on the now pass Pauwels Eric provide I'll to commercial business. an

Eric Pauwels

[Technical future difficulty] us, PTC. am the launches a comments, of important we executive an business to behind year, to Matt's upcoming development revenue With delighted with management Thanks. quarter remain first selective products, generating I at and team existing milestone build on our join prepare for portfolio strong continue will this opportunities.

engage sales care webinars advocacy to as With our the worldwide activities priority calls, that necessary commercial DMD business, supply. our host the is team not treatments continue providers, patient significant platforms date. leveraging have have virtual driving with disruptions and virtual side drug DMD, well we of educational the podcasts, we promotion advisory by awareness top groups master personal on class on Currently, from boards. to And health disease ensuring to limited by through COVID-XX, patients as seen payers and local

We important growth. Additionally, maintaining with and for especially the existing products. and an terms environment, role current of had playing our both DMD patients Emflaza, about in information quarter is social in patient media first seeking new Translarna a even patients solid in our

second the continue in these growth into efforts impacting the diagnostic last see patient educational And months. several over and New the quarter. driven business positively was by part efforts we to

saw growth we continued diagnosis and in globally. QX both positive in and trends prescriptions Translarna, For

we we ongoing to continue purchase discussions with patients seen we of Brazil allow region, have in future the of will successfully that In Translarna on Health disruption. increase. to patient injunctions number secure the In with EMEA maintained And seen the have LatAm adherence compliance in and Ministry orders. to minimal region, access have positive have the

of the Cinergy Importantly, we real world highlighting and efficacy impressive Study, History long-term continue Translarna. from of the results DMD comparison to highlight Natural Registry Stride the

case and For enhanced of team Care's and with the management significant improving fully in has Emflaza, impact our starts, XXXX. new U.S. quarter reauthorization commercial been Driving PTC customer patient efficiencies and and adherence made compliance. and service first engaged patient

and commercial to from therapy. ongoing continue next therapy of expect quarter bridge these this new time assistance In of to quarter. [ph] transitioned patients on the moved And the We more programs, improvements will first through patients prescription faster rapidly. year, commercial previously patient therapy

physicians addition, of see the continue In to benefits differentiated Emflaza.

Cincinnati publications, loss such Real multiple demonstrating benefits by Hospital Continued Children's including the recent compared data the laying of better from World prednisone. as camouflages ambulation Outcomes, to

to let's Now, turn TEGSEDI.

activities pricing and now launch with quality we expect is as in indicated Brazil and the the progression which first in the current well the our in with in environment, differentiated despite progressing at-home of are completed on quarter. second be Brazil, still discussions label. only therapy disease life focus approved slowing Our TEGSEDI to

identification prescriptions patient growth new quarter, in in and first continued saw the In we LatAm.

the our prevalence new LatAm, second capabilities polyneuropathy anticipate on Waylivra a with core of hATTR has therapy. year this in Brazil ANVISA continue reminder, of Waylivra to in patients the [ph] worldwide. for a amyloidosis Building half filing SGS As we patients and identify largest

Turning to activities progress. continue prelaunch to deficiency, AADC

XX We have current by care providers adapted attendance countries. over multiple with education from master XXX over the finding virtual initiatives, in to including patient and classes health conducting implementing environment

path We towards Less European "The meetings. also also rolled finding Road early multiple out We've Steering a held Traveled" program for called, diagnosis. Committee a patient

state viewers social to websites. We utilizing videos, on directing symptoms focusing launched campaigns expert and recently media disease

patients virtual identified diagnose patients meetings with as provider new epilepsy teams healthcare palsy clinic. push cerebral in be and to continue our Finally, to forward

our add based us base administration hold. patients allowed our of maintain current at even take as commercial products the to patients, back quarter, and to new COVID-XX started home Looking of the the pandemic

operational Emflaza we the a and As new and to near-term, of anticipate of Translarna result, continued improvements. near supply some the growth patients, -- base patients both the continuity existing for continued in with

will review Hill Emily progress. over Chief to I to the our call financial hand Officer, our Financial now

Emily Hill

congratulations Eric Thanks, and promotion. for their to Eric, Matt and

have prepared We a also tailwinds first continuing the and the well afternoon. highlighted our on are such But growing management the DMD into degree due quarter with this environment, XX% you of franchise year-over-year the feel to regarding we to the the We have from positive quarter to happy we strong guidance the lingering to uncertainty April. handle current happy COVID, and of financial as reported We're duration withdrawing impact for XXXX. team. have with first are

have today's thought throughout home products, multiple platforms and you a timelines diverse have of to have across to administered the reflected deal resources prioritization and throughout the needed. great We're call, to shift As portfolio assets given pandemic. we heard COVID-XX fortunate as cost, ability commercial a of

of Strategically, programs, where for can future biologics we've we forward commercial to with some that programs Friedreich cash through expenditures identified It impact AADC move more doesn't gene clinical commercial uncertain capital capital some production trade-offs in intensive ataxia for such and Hopewell be time. as us as and conservative conserve deferring such deficiency programs push our certain facility allows our therapy products. this as manufacturing we the near-term

reported Now minutes this press XXXX financial our details first I review quarter $XX.X to results issued of Starting to of and of total with these details of million a our highlight first results. financial quarter We first I in afternoon line revenues The $XX.X summarizes take want XXXX. XXXX now. will earlier the the release few top for quarter revenues to XXXX compared the quarter million in total results. the first

net in new territory. product geographic existing were compares for as of quarter. million an driven continued This quarter as expansion first the the million revenues sales Growth products well XXXX. $XX.X to increase markets, for into net $XX.X by Translarna was in

the to first product efficiencies and product operational we’ve improvements first driven XXXX, the revenues earlier. For reported sales in noted for quarter in million net for part of $XX.X of was an million quarter compared Eric Emflaza, XXXX. Growth of the net increase by $XX.X to due by

Bio-e were $X.X first in Non-GAAP compensation million in for $XX.X million non-cash stock-based XXXX, XXXX, excluding $XX.X $X.X for compared programs the as associated the expense R&D quarter increased gene in therapy pipeline. first the million non-cash expenses clinical excluding R&D million well the increase expense. of of quarter stock-based to of costs expense reflects and advancing and research advancement investment The as with compensation in platforms

the compensation to Non-GAAP SG&A for the in stock-based expanding million quarter compared $X.X non-cash first investments million continued expense of commercial million our stock-based portfolio. XXXX, $XX non-cash for excluding $XX.X first in support million $X expenses including were XXXX, our to reflecting commercial activities quarter compensation of expense, excluding

million XXXX. the to of equivalents loss at XX, Cash, March of marketable $XXX.X compared to $XXX.X first and million totaled million net at of for first XX, the $XX.X securities Net XXXX. quarter was XXXX, $XXX.X XXXX quarter for loss December million compared cash

share to former wanted I the from up, the wrap our that we a sheet. details of shareholders of balance completed I this Before with Agilis week liabilities transaction removed

As gene owed the part of probably future of you our we therapy as milestone. acquisition Agilis aware, cash are platform,

cash exchanged those pleased subset for cash have are to stock. milestones a We of and PTC

PTC for cash receipt early therapy X.X liability, the cash. shareholders AADC The to a in PTC in approximately cash the future especially gene of advance Review Priority This XXXX significant upon of and XXXX milestones of milestones former XXXX Almost approval of Voucher. the Deficiency to we milestones stock of milestones the receipt pay XXXX expected in those owed and platform. while shares were $XXX $XX August include our of XX% time-based elected million BLA a million and an of exchange that million alleviates Agilis XXXX

our to start session. Operator? the will I question-and-answer hand now over operator to call the

Operator

RBC the line [Operator question Brian of first Instructions] Abraham Markets. from Our from Capital comes

now is open. line Your

Unidentified Analyst

handling is that And combination the study the prior Brian the your just complete gathered the I therapy additional need from COVID you AADC to on on [indiscernible] guess about you of study how This gene much the to Congrats then able a been Hi. the progress tonight. all situation. of pandemic? make following? and what a little data types I talk do bit you've to wonder progress if for in could of

Stuart Peltz

Yes. Hey, question. for thanks that

we you why next doing progress what clinical making So the and step. with yes, Matt, through trial. were what -- so we AADC actually were talk do you don’t would

Matthew Klein

Stu Yes. and Thanks, question. for thank you the

intended received mentioned, feedback data around young of towards commercial XXXX, As had in additional of cannula from the the patients. you on the end we FDA use the

efficacy the on and of demonstrated years, deficiency AADC and We've a to in quite EMA benefit of of also we program clinical As our XX evidence X safety to track able course to durable Manufacturing profile. years is as strong effect the in submit were a strong. MAA impact January. have continued as the we reminder, with remains well continued

two that the elective early key the the of was cannula. end likely gating quarter patients with to so treatment be end treat the before prepared were to back, pushed first item and BLA hospital the the procedures due had the been QX. QX, the And children surgeries, those of for of to towards have closure for we commercial young sometimes possibly We identified done

so we those that, be to we data BLA for And the surgeries submission. continue once able from and preparations will have

Unidentified Analyst

just we to made time [indiscernible] -- it move So just the a so little on. take that forward pandemic [indiscernible]

Stuart Peltz

you Great. much. Thank so

Operator

Alethia from of next comes from Cantor. question Our the Young line

is open. line now Your

Eileen Maysek

Thanks taking the for one first is This guess there. Alethia. for is questions. I our COVID-XX. Eileen Hi on

up data to type changes Just matches do SUNFISH And any then from for seeing data, the Translarna? Zolgensma then manage how there and you the wondering, FIREFISH set? analysis, present then buying patients? second, risdiplam And for to SPINRAZA a do in you is you subgroup Thanks. how to And keep around patient? access are patterns the data, particularly think X plan in for any age, for

Stuart Peltz

you. thank Well, great. Okay,

the one, with as start well. Translarna, we'll which is for maybe matter first on that -- the So

saw I and you as in from we have quarter. Emily we the Eric think actually we both So And talked, good first numbers known. I could -- see think a numbers the are continually

or to in coming how see you I up bit want about think next in what's patients quarter order. a And been able Eric, we distribution talk for So to you ongoing. be the the feel two? little remains maybe have

Eric Pauwels

Yes. orders. deeper of advocacy Sure, even patients both our seen we've what been Translarna for during Stu, and providers And pandemic. in growth and the seeing groups Emflaza. actually in we on the question. the happens number DMD, with health thanks relationships any We're continued actually decline products cases But actually in and establish is, and care able some with is payers to haven't

need. But I That sort to case centered is Emflaza. both for a have to a now upticks. very we main have question monthly to of they months, is bit, in strong and current but many Translarna the of Emflaza focus been we assure patients establish asking able of few we multiple able think ensure for it's that have really supply. may any have the been and have been tick-up the really them most adequate the our importantly, U.S., And of to have DMD supply quarter. have provide in we right they patients that significant. supplies of Translarna. And of adequate management the haven't vast that the a seen in on continuity there little we've not been establish with base Emflaza that currently connection around all our generally, supplies resources and are people a major we -- If patients XX-day team asked getting sort majority But of patients

Stuart Peltz

Yes.

Part efficacy sense also CNS that well, that this in FIREFISH from molecule And despite both both And the in RNA, recent other a I able there. the and to best-in-class the we and the was not that seen in we relative products contextualize so you with older show changes to we you SMN saw is will risdiplam are was out the is only but how to as really feel see And data risdiplam proteins increases potential. the think, we in did it's relates risdiplam do our X, the even -- the comparisons do the that age, as as And contention Part that that, durable well that And question efficacy the see what compelling but that. could that. of occurred in periphery. that X, when we're second

really So that's actually critical.

who've as a not only been we in about whole we of CNS talked is the also only take is SMA, you was not the don’t affects of the body then ones it a disease. much the you it the an becoming it's but periphery. as that, it's to we're other think showed treatment, [ph] And advantage And Well, clearer able how as consequence risdiplam we That I nor and immunosuppression need that. -- the is show but is critical, has for been tissues do that a well. another clearer take to intrathecal itself. that, injection really need taking drug. We're

related there. safety and very to So there that was think drug we've And that's proud findings in far a major we date treating placebo think patients X had that we're really advantage. thus that then what patients from we've seen to where no we XX also SMA that so was trial far broadest old, had three years trials program clinical one, we've in and the two controlled

adults. to older pre-symptomatic have also We

So naïve pretreated. from to

efficacy well the in and we placebo breadth that that SUNFISH risdiplam a a controlled that both of the as program have shown of trial. really demonstrate So in trials then as the

So were compound on say data in follow we and little which that terms did it's a thing of used, think asked immunosuppressants contrast years liver old. systemic it's three that the years X corticosteroids X well. for even can months. just what durability are by one therapy, you of what terms potentially really and not the on terms there's populations. keeping needed I all drug confounded think that beyond therapy that than patient when We of that, for for you gene you And need to data limited think be there's greater injury both there safety more warnings, patients and in greater concerns then than in were then And approved for in

have So with on you patients, greater in taking patient observed you continuous all old. think lumbar a been in is and data then think administration difficult meningitis think that the compare we the population, We front it's been have the the sick risdiplam take nusinersen trial even hydrocephalus there's you of done when that patients safety burden it the the say, scoliosis, more if we has And what that than where and X have of that’s and lack punctures. than and accumulation molecule. at whole there again, consideration you look best-in-class years of and into

Eileen Maysek

great. Okay,

Stuart Peltz

the had is Then I think question you SUNFISH? one which other

Eileen Maysek

Yes, the subgroup, SUNFISH yes.

Stuart Peltz

I'm on as know, data you That, time the sure it Yes. over accumulates. be we'll of posters having addition

Eileen Maysek

Okay, great. Thanks.

Operator

you. Thank

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

line Your open. is now

Tazeen Ahmad

guys. so much for good of been couple us on then Stu, on updates to you able on number to taking a programs. Can a course my you starting X,XXX of of how one Hi, AADC, you've past, a more had you the is that And that patients several still today? let's of many over thinking identify. questions. that give couple around, have also give years. the hope as us patients number next find you're whether worldwide refresh they're Maybe patients been on and have say, you a the in I afternoon, to Thanks other

Stuart Peltz

Yes. Great, that. Yes. for thanks

the than said in we meeting. as -- yes, that, XXX had XXX in identified J.P. about greater Morgan say I'd January, So we patients

of continued we're identification. now a the environment than bit some that that more COVID a said We And to we a still do previous. I'll with now have talk difficult that. And the numbers obviously we is in matter have so consequence We the patient issues But really comfortable as of search. to and COVID is Eric obviously -- there. think in about right it's

current in at don’t bit how doing hard environment. talk working we're the maybe little that. this still why we're you a so And about Eric,

Eric Pauwels

Sure.

hi, So, Tazeen.

I identification efforts, direct has to right now the that, patient a to right lot had we're adapting pretty safe it's this a impact COVID say think now on environment of but environment. doing and new activities

XXX is As country Europe. than And actually in or XXX Stu more our that's the get this U.S. whether launch, patients said, identified point. identified we've patients at first to by the goal

of XX efforts substantially. presence We about called epilepsy as health increased deficiency web to a patients program initiatives, received, out Less care down started was really diagnose We a things now as how in in clinics. physicians where we're right had and this online QX. trying a sort symptoms, on had that very, have our when recognize clinics little particularly to We Traveled" of very in and care providers provider had it particularly class bit, shifted master various XXX earlier, to different to palsy countries. cerebral And well that rolled slow helped areas well we we Road to number health AADC "The target in understand

quite the to the number of opinion we've In a leaders with those actually to identification. we in And classes reception Steering AADC from good virtual quite few -- had Committee addition a executed European education out we’ve rolled surprisingly of meetings. patient key that, had and really help

noise educational keep given done. COVID-XX, clear activity had fewer are said, all have still doing virtually the being are that we're of a up patients and with Now, seeing that bit that an it's But -- impact level. patient to quite tests physicians

Tazeen Ahmad

a Okay. maybe, Stu, Thanks that. question for on And Translarna. then

you're have biopsy? And received scheduled does study April, the June? yet end biopsy it in if, until what patients a not idea can patient but or March that say, difference or get of to measure For was give a that it don't to they make us dystrophin, percent meaningful any up you an of getting doing let's May

Stuart Peltz

question. good a that's Yes,

So -- of that treating be sort We of get open, able gotten, to able should and patients is to to were once able say come will on to don't are running waiting -- last right? probably the this terms somewhere there. scheduled accomplished. be We to don't around the longer. biopsy for real a we we're back I'd and there's a getting -- think be we XX% They interrupted in of the in there. all So be this sites the approximately we've them think to problem, -- issue be real and biopsy took get to able and

think be So will we don't think that that deal. a that’s -- big

Tazeen Ahmad

FDA, you've delay? that discussed something with And there this Okay. is

Stuart Peltz

general, know think in I having sites they are that issues.

they areas. I because know directly, don't sites down, talked in especially put if just out closed them things so about -- metropolitan already know we've And but to they are actually some

case recall X the particular actually So I and a from mean, XX had that might you -- month. and Sarepta they this month case, in

real as issue. a we at look this So don't

Tazeen Ahmad

Okay. you. Thank

Operator

comes from Thank you. [Operator next Citi. Joel of Instructions] Our question from Beatty line the

open. Your is now line

Joel Beatty

the for Thanks question. Hi.

should to as be launches, anticipate So there payers will open SPINRAZA? therapy, payers risdiplam could also that only could that a cover combo as it or we situations monotherapy, be paying Zolgensma for like or

Stuart Peltz

of SPINRAZA. And start necessarily, to that why what don't mean, the can you’ve a alternative they to got protein. think let's risdiplam and actually think make about. this understand clearly the sort the think that, with with all couple SMA that I both make pretty splicing transcript. because -- RNA to Translarna promote When We was occurred nation, SMA of maybe our So -- shown -- to I points I remember we've -- appropriate the actually of into to you

guess from is that risdiplam a who trial patients treatment, type to does now make they proteins. in appropriate in with being RNA patients patients therefore guess Zolgensma, getting X know a who from be have of now get competing In job of the order -- order there as need to them risdiplam good who is we So that. -- the that are that type are -- and of terms open-label our Zolgensma our very My feel additional case and on getting we well. as in to able get are trial, currently that we'll want

get risdiplam do be know when would as they we of want who we don’t well would how or to light of, durable this. So how get especially that anticipate the probably there'll patients patients in variability

Joel Beatty

favorable rapid risdiplam. more there anticipate the then for dynamics more of on other have And little would what more launch question rapid regions? a of a sense. that type help certain factors one Are I than launch risdiplam? And have you bit Makes have regions region

Stuart Peltz

-- to world, the charge rapidly and will mean, get U.S. moving first, commercialization, But want to Roche but think first that is that charge I for. then going U.S. certainly of the they in is they're the be of country Well, done. will we go -- will in in to -- obviously the want the I Roche say really throughout

wanted make covered previous precedents I the it, there think to about was SPINRAZA where actually Zolgensma. that were question on and The after when other was also I point

with So probably notion the I be think, anticipate we risdiplam certainly that of given goes would that [indiscernible]. along also

Joel Beatty

Thank you. Great.

Operator

Thank you.

from of Our next comes Company. the Joseph line question Thome from Cowen and

line Your open. is now

Joseph Thome

my you Thank questions. taking there. for Hi,

the of sort early are gene aged Just the my there question as on then months those one therapy manufacturing study years. it to X clinicaltrials.gov how any second And for on XXXX. Can you together. would patients tied in just delay X first of slight articulate maybe DMD initiation studies in a this impact is, like looks GMP Translarna is

wondering, potential Just is label? here a this to Thank show the or and submission you're to you. U.S. what ex-U.S hoping related

Stuart Peltz

thanks. Yes, Sure.

just already Bridgewater of So material in thinking manufacturing GMP the terms about we when site we're so -- Hopewell was toxicology COVID, site. the making we've level in really started already

So and we able to really based in on it we any being -- would be down slowing that, anticipate do it way. don’t

of have consequence XXXX plant opening no just by months. is get in environment it forward the there'll it pushing the and So -- be for a a so this the four as we current delays of we lot will that

of tucked real that thought it so already the So access a cash, issue up because amount we didn't was might we our do think and created we site. in considerable concerned it well a we Bridgewater

approval can gotten that that's towards. sorry that get expand the able really we've with to it -- that done in report and terms -- to to we've and we’ve we U.S. they get earlier I and the and the the just extension was years, Europe, bring X the months mean, In [indiscernible] -- to get just X work to of That -- so the people mutations label what actually so did be in to

So to and earlier to that's earlier get continue patients. just

Joseph Thome

if just Great. some at and presentation could you're having And aims I changes of I body do ASGCT, kind of AADC. one endpoint. outline data the more on then, and see scale functional

of they interested what the are at the going each of review one physicians terms how to or regulatory efficacy maybe endpoint? them look holistically. most look at Is in, these are important, and that's different In they kind

Stuart Peltz

Sure.

So, you to want Matt, that? do take

Matthew Klein

the Yes, question. sure. for Thank Joseph, you,

package, we're able think we scan about the show we -- when important to very significant what the data first, PET AADC a think dopamine increase the in it's fact patients. there is in production on I all that that is

now as you endpoint the including that the head that first we're data patient important we're past of evidence in that's really every compelling as That's whole, targeting the pathology the what Then a of the in look data. biochemical mentioned the very of in primary the we're benefit long-term seeing underlying some show So at there's fundamental in critical patients. we're five some that you And with that out we're data shown defect. of control, even XX sign to very its to able effect treated. close addressing durability abstracts very is years, strong, That's package years seeing the compelling disease.

and function that of evidence in and is that motor in sustained. of the those of the that we're favorable defects defect biochemical all appreciate correction disease, correction one fundamental will patients; are we're the and those the two, physicians believe addressing, seeing we data key totality effects, across the what So the board effects

Joseph Thome

so Great. you Thank much.

Operator

you. Thank

the Bernstein. Vincent Our from of next Chen question comes from line

line Your open. is now

Vincent Chen

for and the all on me. taking for thanks progress A couple Congratulations questions.

have far potential to like sounds impacts. been [indiscernible] just elaborate on slowdown one COVID the It COVID. in related impact. so or largely the the on, pretty given basically The withdraw been COVID-XX imagine to wondering you’ve and I further related guess, resilient It little so first a a impact if to could bit was made was is what far franchises have revisiting I April. guidance you seen

in you're going of the guidance then worse better to products demand indeed to the for of date? just second will months, pulling sense -- last whether slowdown And want to end in one the market there. a Yes. things get return pause couple were get QX or to forward. is seeing for over Just I whether course Or April any of in case is

Stuart Peltz

Okay.

after know. they say are we drug feel in where how the of of and long-term, terms uncertainty you that’s the the -- yes, more the just patients it where the in maintaining goes In question, -- on was already really pulling things and would or in the guidance to of So short-term, quarter going. a the because good new withdrawing good first the don't even pretty at I prescriptions. using of on slow moment, on it --

slowdown. like bit So of you saw we a to a it put maybe a bit? little And around this want maybe, wasn't Eric, --

Eric Pauwels

that with top Yes. Emflaza I Vincent, right the now, mean, line, question. thanks both Sure, very in the of in we've terms for Europe I are the still. Stu. seen Yes, mean, encouraging trends

shipping As base see QX. most I the a all, on have growth are a which still we good there. more patients increases positive to seen, of not patient are in asking diagnosis is very is frequently, been We've existing decreased because as little had at the as trends mentioned, for prescriptions The And there, seeing and those basis. continue month-to-month we new patients new -- kind any trends although product. mostly we bit haven't and now, base, patient patients physicians but we've have and patient the new good protected

it's been have of some the a been percentage. still seeing addition back we have few And in to amount that have Emflaza, There potentially an days we're everybody adequate but requests more uptake. and and requests supply supply. make more We there patients for gone a sure of realizes small have XX to for

of is now, cautious. in If now be or of Stu to pandemic, unknown. of shipments think far partners shift patients to -- that some would growth mind, based the that or trying patients right indication there's more no time, various might mix And first be results we're markets. countries potentially if we with Translarna have losing a we're the because payer pandemic this our I there well point quarter business. that seeing the strong But I XX think disruption continues what give close disrupted down. in what our about in just to of will is as at in And and highlighted, the back as Stu. and that some in and go it direct to Keep on we were concerned is we're I there's as unemployment so that

Vincent Chen

one quick Right. that point. And follow-up maybe on

on how in more between the Emflaza were If -- price a paid and who price were the in if you Take that are you payer to on see patients on commercial the about on Emflaza how mix, government way, net if U.S., average? might meaningful insurance? price think for paid insurance impact shift patients patients another does differ the

Stuart Peltz

simple CMS, Yes. higher it's is, right statutory Well, the basically the -- now. answer rebate is more which from is which

there of would XX-XX with Our about more be Medicaid. commercial current gross that payer shifted sort thing shift to would net mix obviously patients because is onto payer majority. our the If moving

to programs, that covered. have So and of for have pretty far, And the children haven't private to have seen quickly. insurance actually disability be that. a DMD in in programs they go and states And seems payers to do number patients if that we move from general can very families good then Medicaid addition that,

there, statutory we again, the would have payer pay So, that to only would be to mix CMS, the shifted. rebates effect if the Vincent, think I

Vincent Chen

data other guess, in treated see the there you let territories data calculations used? the Translarna And study. much using ongoing studies have give an draw patients terms see. like you is size, whether just number upon you experience, in is you done Translarna with how and me patients ask was in I how of study. dystrophin, from ongoing powering terms just the clinical could where there us are on increase another for a I of your sort dystrophin did sense if estimating the in see from to of or just I of were the from U.S. wondering one, likely effect What -- Translarna of collected in

Stuart Peltz

want that? we for -- Matt, on is Yes. there and just answer Yes, based you what you some was on based We other a look got we else That's some to thanks and that it to good making well, assays the as and sort like Sarepta there. of saw question. do studies want that that anything at that. did data assumptions from

Matthew Klein

XX% would expect with would power, effect. the to a that be we to the course, be believe achieve sufficient we low very, baseline Translarna very show have which to we dystrophin that just Yes, thought levels And, the able trial be we of expression. XX we -- in of subjects

Vincent Chen

-- ask, variability? and size just When was do XX% the say you you I can effect power, the what

Matthew Klein

Correct.

at running As base be other said, Stuart trial data we'd simulations. and looking

Vincent Chen

Okay. Thanks all questions. taking the for

Operator

Thank you.

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

line Your open. now is

Peter Kim

question meaningful do my questions. This you in Thank anticipate Hi. risdiplam year, expect patient first is -- -- expect much Gena taking revenue question some guess, or I for do you you the on us U.S. could impact OUS? my the whether do second patients my to generate on risdiplam. initiation how enrollment of is you Thank and this for very to I EAP Peter Wang. guess, COVID? opened rate And you. question related many EAP remind post-COVID sort pre-COVID was the to changed? of and enrolled and note been has is when And sort have you

Stuart Peltz

Sure.

So maybe revenues. picture the and big on COVID

picture, So I have will COVID, I -- think people I I in about to ways make I do sure patients. on think get drug the think thinking start how some big supply

some that a in ways perspective. So think helpful launch might commercial from I be

would Emily, terms in want revenues royalty, you I that? talk that maybe bit of a to about be little think

Emily Hill

risdiplam in and for to year. year Obviously, more XX, with $XX Roche this potential significantly in have on we the tiered milestone teens up the on PDUFA to up earlier programs. expect mean, to up collaboration next August and the royalties have submitted I'd I mid U.S. them million this having we in ramp this Sure. the our now year MAA the with

really and only can as potentially of the being be rate as some the COVID delivered think impacts advantages that and home. those far ramp I therapy revenues, there's SMA at

Stuart Peltz

patient is discussed Roche say call lot think and then of a has previously, they But numbers. have I what Well, don't chosen on interest. the think did the I what they And that.

then they bringing also in already continually so -- do Europe set the and as that U.S are well on the And both well. patients are in to that they're EAP it already in, as as up

you U.S. have So It these EAP that in and countries then program. over programs. sense the allow in to a was all be EAP it'll Europe certainly a global, in in

Peter Kim

Thank you very it. Got much.

Operator

Thank you.

William comes question of line Blair. next from Raju the Prasad from Our

now open. is line Your

Raju Prasad

any of general Thanks aside have go the -- off how Early Access Program, risdiplam, want too, of for expanding additional scenarios any you taking have it what details the you general there if you and or is on they And the around just commentary patients in they’re Roche are many to in, when that apply, expansion patients or do are bringing already types then anticipate stringent don’t rather, On what mentioned, criteria? have questions. on Translarna. you’ve I spin how one is on from do any presenting JEWELFISH And data? more -- RAINBOWFISH do

Stuart Peltz

well type EAP X Sure. into now for who program as added the on EAP are probably in Yes, program. too, And the is to they so that then, to therapies to to SMA. an as the presentations important [indiscernible] yes, that’s they’re And be changed that have also an planning patients point program other allow the they’ve get well. update

So, patients based intrathecal on yes, say, so need injections the can. really who based what they get, or physician is. think the certainly can't It's patients, on that naive they discretion

So COVID is access. obviously limited making

it. nice a and it way is this which get home So at they really take can

hopefully also adding Very So number who transitioning though. in risdiplam. that's patients this interest the to are of strong to

Raju Prasad

the you the have many just a general kind idea on access program, early in getting versus any are of wise? percentage how Do Yes. EU of U.S.

Stuart Peltz

hasn't disclosed yet. No. been Yes, that

Raju Prasad

and you But have orders, QX, you commented revenue of on on to American guidance general a enough Okay. And Translarna. similar? going call. then recognition just Latin you've know is the usually, this it one from I pulled on obviously, wondering, be QX bolus pattern

Stuart Peltz

Yes.

think maybe comment that. I think -- so Eric could on I

Eric Pauwels

sure. Yes,

a -- injunctions saw year, can increase. last recall, had Translarna, we've patients. But are nice patients ANVISA diagnosing been we positive And received to and We first prescriptions. new the coming approval from for America, as you the only new these for therapy we to base building quarter, patients continues not of that regards patients, with of very Latin in base

with have regards new with With Health we base, working with because second towards securing working purchase of Minister a order in meetings, Minister the in to require to parameters. those of Health now shifted group and relationship COVID-XX to those the right get and purchase ongoing orders. all the ANVISA lot negotiations communications strong of quarter. a of Brazil. we we our We're on growth to that a have seen with payers we've So We've virtual continue the virtual-type that very

some as of an increases of exceptional government get goal our right is kind to order continuous of resources. we've diversion goal pandemic QX, our is, any So acceleration seen number kind barring secure or of patients in of and the now, for

keep You small relatively mind care got disease to bucket that the comparison is in in though at budget. cost of rare overall the Minister for Health centers health to

can Minister hopefully to there. some of those is and very encouraged secure that -- going order it's So the on we orders. through continued think we continue obtain an negotiate to are we again, and ensure But QX of and work to things growth, patients these with pressure continuity the are put Health and we in

Operator

call I for the time, remarks. would to back I'm no showing further this At to turn questions. Stu like over closing

Stuart Peltz

you I all PTC. our remind at end the March that at to want was want I look, to joining the Well, call of for of anniversary you thank Okay. today. XXnd

with forward of and make and history, treatments under look evolved. developing to living thrive, of really the continue environment. proud And I consequence patients be made a hard team. now commercializing the progress all company to We're I'm back so in our for to discovering, work gratifying see long And and the that this we've has positioned rare upon well dedication even -- the able with uncertain It's even disorders. I of think to very how push and

you call. joining to look you I soon. again, see thank I for So the -- hope forward all

Operator

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

disconnect. now may You

PTC Therapeutics (PTCT) 1 May 202020 Q1 Earnings call transcript