PTCT - Earnings call transcript Q3 2021

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Operator

Good day. And thank you for standing by. Welcome to the PTC Therapeutics Third Quarter 2021 Corporate Update and Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. advised conference be being Please Instructions] that is today's recorded. [Operator Relations. to I Kylie conference Investor would Instructions] hand O'Keefe, now of today, [Operator speaker like the your over Head to Please go ahead.

Kylie O'Keefe

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

and is Hill. Chief Matthew Business Chief Klein; Officer, on our Financial Peltz; Emily Stuart Pauwels; Officer, Chief me Joining our our today's our Officer, call Executive Chief Officer, Eric Development

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

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

a earnings certain of is me this non-GAAP of information and regarding to on SEC over Stuart Information use uncertainties, GAAP the pass GAAP release. to Form For risks as report today's annual company's a other With disclose filed Commission, the and as company's recent call well We to that, Exchange financial encourage available Stuart? the measures review XX-Q call. quarterly in of non-GAAP non-GAAP CEO, Peltz. most would applicable detailed our report on during with the Form reconciliation you our to let Securities filings. description and XX-K and we our

Stuart Peltz

all you our quarter. about Thanks, for in. tell to progress Kylie. dialing And thank I'm you excited this

and are accomplish have robust all unmet therapies long-term making to patients to pipeline. medical high commercial our both short for developed needs. good a and commercializes treat across that built have committed stakeholders. continued and creating remarkable been demonstrate have To discovers company we we to As At of a this, PTC, we value progress revenue

efforts continue This continuous commercialization building and new a will it's to pipeline reach state a years study seek will base commercial come. these directed that the now to so our increase products. substantially are ongoing all on of grow in five preclinical allow an it registration our clinical number therapies revenue in The have increasing And basis. revenue develop studies results bearing to will and of with continuous we fruit. We growth us impressive and are sustainable steady that of progress. We

building are enduring sustainable company. a We biopharmaceutical

growth. $XXX revenue. Turning new grown to year-over-year the franchise commercial we This deliver total this for by compared $XXX and revenue, Emily are third to Translarna both quarter’s raising I'm million Translarna. Eric primarily franchise The million in continue was driven has with revenue Emflaza to XXXX, million and DMD of to guidance $XXX from XX% starts to expansion more by franchise $XXX impressive shortly. quarter will the the we that $XXX to pleased go into for announce DMD XXXX to our for million and detail geographic million. patients

later call. of on commercial the remarkable there we're Brazil. to in care. Now were amyloidosis the Waylivra X this not I'd the X or that also would This Portuguese achieved current of is both more need. announce this Category August, significant quarter. show team only Tegsedi, in approved treatment was we of milestone HATTR this pricing. pricing for which Waylivra in innovative patients prevalence higher Brazil to Category excited of Brazilian for FCS. I'm that Due with descent, received is familial HATTR chylomicronemia standards therapy benefit first able the treatments Tegsedi patients given to report and in the And syndrome to the This approval milestones to quarter. this bring was be treatment for go to the is to happy into Brazil over to in Eric a pleased the population. the progress detail like highlight In of

which commercial total over revenue share particular, utilize a the pricing turn our announce agents expects as population in treatment the our well as prescribed of a drivers. launch. on as grow And with of to first me in that responder efforts this in platform. a trial as now pipeline, newborn invest patient let approximately with Evrysdi, we blood-based directed Now product screening, start United is success most in to to revenues in registration which through is I'd just to to discuss of chance opportunity market view our pathway significant endpoint, of continued I'll clear Evrysdi PKU. U.S. the ability to further APHENITY in the initiated a well-defined having that enriched States. is it's population a quarter with And year in outside I'm or commercial SMA an to run-in have through continues this there XXXX success. We in excellence excited analysis In demonstrates further Roche with our like centers XX% value focus the biomarkers approval more as growth our approval XX The and PTCXXX from an detail. called a near-term the countries. increases innovative after our Evrysdi show reimbursement to strong commercial the portfolio and splicing known

me targets trials disease that multiple talk our the Let next stress the platform. and from two oxidative our excess The electrons states. contributes platform about bio-e directed bio-e registration

from inhibits XX-Lipoxygenase, compound response. platform, vatiquinone first this regulator the controls key a that stress The oxidative

study associated vatiquinone seizures. is registration the directed with with in patients The disease mitochondrial first with MIT-E the of trial

And in XXXX. our of the third quarter expect positive basis of NDA. results if would We the be

ahead PTCXXX. is moving in with also from in We with registration FA compounds to affects grateful community, support patients and that and trial, from a ataxia, platform, function. second us the anticipate collaboration now XXXX. MOVE-FA directed disease the Our for vatiquinone mitochondrial drive Friedreich in helping We're generation next bio-e neuromuscular the results the We're enrollment.

the I’ll innovative PTCXXX our the and the to the initiate treatment We able splicing indication PTCXXX of We in and HTT platform of aiming results show are dependent with as X discuss have first the protein, ALS focus for were now on XX% a to trial of to volunteer from Phase next PTCXXX first quarter mRNA selected for dose and targeted reported disease. reduction. both achieve recently We trial. lowering Huntington's XX% healthy year.

minimal and Phase possible X. treat about We are blood-brain PTCXXX disease. this to also has the attributes excited demonstrated and that key These are passes remain into rapidly the Huntington's very We as program barrier as moving influx.

trial. Phase into X Moving the

as safety, Huntington in in We disease will of well the mRNA protein replicate levels Huntington’s and decreases tolerability and as PTCXXX, pharmacology patients.

go we’ll shortly. our on more into Now detail Phase program X

of we Jersey. year, the on to and cutting transformational moving In ceremony Now, forward AADC. recently therapy therapy Hopewell, to that of ribbon for deficiency excellence AADC Europe, look manufacturing we the bringing end to pleased expect our center held opinion by gene this CHMP patients. also the this New I’m still in

we’ve staffed new the commercial art is vectors state produce applications. of and and over progress perseverance the dedication say equipped Our the exciting across and ongoing plasmid the in to past proud we’ve This continued quarter I’m AAV high face to the by who that to steady made hard of the our for all state worked progress pipeline. and of have past quality facility challenges our The proud of XX with therapy fronts. have I’m achievements the very to incredibly execute required across gene speaks pandemic. people months

enduring on company. We be an us to program. positions have update Matt? biopharmaceutical a growing revenue research, development to development I’ll in the and generating the the turn over for commercialization all ingredients, call place that now Matt

Matthew Klein

Thanks programs. Stu. will progress PKU provide on happy our begin of update I our I’m with the program. PTCXXX to an continued pipeline

a enrollment six controlled global registration Phase trial, PTCXXX PTCXXX for of pediatric randomized placebo double our PTCXXX, planned, APHENITY blind As This who responders then in will X adult to directed to trial and running receive space placebo initiated identify the weeks. will trial to either or be PKU patients. in include we

previous PKU, approved primary a the the is trial reduction for of endpoint therapy phenylalanine. of As APHENITY

open-label enrolled study. in a long-term of completion extension trial will placebo then subjects Following controlled be

expect to from controlled end results have XXXX. We by placebo trial the the of

program. now our Huntington’s PTCXXX Turning disease to

placebo This controlled demonstrate of we and study successful one study phase placebo year of with dose XXX HD the in enroll healthy to XX forward announcement of receive pivot randomized week primary each to results patients. HD by phase. moving the Following study volunteers, Phase approximately will disease XXX will mRNA to double a subjects Huntington’s is open-label This a study two include for and of the lowering of TT patients. protein objective in followed The Phase evidence X will pharmacology in our phase X blind safety, are PTCXXX. be one extension controlled placebo levels

of treatment plasma evidence placebo CSF, radiographic and both collect could study the controlled will provide that effect. extensions In long-term addition, we data PTCXXX meaningful in CNS phases biomarker the and of

population discussed to last criteria As year. on update in demonstrate we in natural constructed study based effect of HD of developed treatment over trial our Phase the enrollment look criteria study the initiating We inclusion pivot history of we by course study. ensure X The Goldilocks the for for the were the the month, analysis clinical the forward this whom to this were biomarker extensive databases. can HD carefully program population of and end the

platform. bio-e updates provide our on I’ll Next

for during seizures, registration the enrollment directed study activating trials the in trial our and to MIT-E ataxia, Stu our these MOVE-FA in global the two associated sites vatiquinone pandemic. As in progress the allow am made trials mentioned, trial. we disease continuing have Friedreich mitochondrial teams proud of of are I in enrollment the

from from in results MOVE-FA expect the XXXX the in QX trial XXXX. We MIT-E trial and

Phase X forward patients of we X in reported we now completed quarter, with a trial last ALS. Phase PTCXXX, volunteer and are healthy trial with the moving As

inhibits studies, of and is be pathway including recently in-vitro has Ferroptosis that described In ALS protector a XX-lipoxygenase that disease ferroptosis. once PTCXXX been initiate design potent first of to the we the and We death a is plan a test number cell finalized. demonstrated the of a quarter and to to death neuronal study and systems. have in-vivo it PTCXXX neurodegenerative in series of neurodegenerative details Phase pathology, reminder, a key known disease on demonstrated regulator the oxidative As a X disease. the is provide in pathway key neuropathology preclinical inflammation study as cell additional Parkinson’s enzyme of we’ll and XXXX, stress

we are X commercial ongoing to been for trials update for Phase We progress Eric? the an near-future. initiating making Eric the call on our in PTCXXX I’ll and and turn to look trials, the excited PTCXXX registration forward our over in business. have the about directed now

Eric Pauwels

Thanks, Matt.

facing DMD global quarter another customer has PTC outstanding franchise. team in our Our delivered

expansion geographic We have approval to will our AADC and anticipated gene therapy, commercial across potential launch continue strengthen globe PTC built a the the EMA of our engine. our the strong foundation to will which for follow drive

strong in year-to-date our growth Our a increase DMD continues franchise brings to sales for DMD XX% our $XXX with revenue This million. year-over-year. franchise the

guidance originally are announce the XXXX for pleased million. a million increase to to revenue at are to XXXX to $XXX the of we $XXX that performance, year-to-date million substantial raised continued guidance Based on XXXX, we at QX franchise $XXX $XXX million the million DMD was over Our raising which strong was the $XXX to $XXX in our million to revenue guidance. original beginning

continued patient operational in of fueled Emflaza. a starts, high focus sustained excellence compliance New and growth has

million the of increase in XX% over $XX is revenue, achieved we quarter, XXXX. a which quarter third This

growth We quarter, XXXX. a to this revenue XX% quarter $XX Translarna. achieved million of over in the third Turning

successful can significant diseases, Russia to uneven produce year. to was others. course growth in rare comparison in markets orders of over international driver quarter-over-quarter large especially Brazil that our In large the orders, vary launch driven reminder, this due the This create the like of drives a government in the lumpiness key a quarter, As growth.

received and of significant centralized nonsense large reimbursement DMD obtained patients. a we Russia in order for a mutation As group

markets continued expansion European seeing driving future and and America, geographical Asia growth we growth. important remains Pacific, which Central Latin in are Eastern Additionally, East in Europe, and the Middle our main to

than more boys STRIDE to the for demonstrating benefit patient to the of further of muscular loss compared that years ambulation with care treatment adding delays We share evidence registry, with recently are results is dystrophy real-world of from Translarna. the five excited of Duchenne Translarna nonsense alone. to the mutation This standards in by also totality

in agreements to additions in DMD in and this coverage the significant making we for two see for for label in with an to newly are established Netherlands fulfill We country. challenges pleased of in quarter Translarna broader patients Brazil, treat in diagnosed increases and the reimbursement the are in to for patients Across new Italy. the Brazil the Translarna five-year-olds approval access continued to patients progress both two five-year-old year to DMD include expansion Finland and we patients Despite and COVID-XX of patients the of to existing of fourth now to expect range. Latin Translarna order in America, continue for with

Tegsedi X is classification Market from announce Regulation Category to we classification reimbursement that to current is step are is an the milestone care Latin value price with have that key treatment and as CMED efficacy Brazil. provide Brazil. innovation America. Category excited treatments turning a we given critical in X and Waylivra, in categorization optimizing the successfully Now and in in Drug in Chamber to international allows of innovative innovative than pricing standards markets. for received pricing first greater Tegsedi line towards CMED the getting that categorization

Assessment commence reimbursement public health with for affects inclusion SUS. system CONITEC, Technology study or will provide in now Health name, negotiations We to Brazil's the and access, Brazilian Body patient continue

reminder, is the Waylivra in with was in of medicine in for first are XXXX. We estimated antisense announce by were there Brazil disease. to underlying HATTR Tegsedi a patients thrilled approved August, the available Brazil. As patients as Brazil X,XXX an cause ANVISA to of that the address amyloidosis

the Waylivra potentially is patients FCS we genetic pricing commenced disease significant have which the rare with Brazil. authorities. to other complications. including a disease, approval, discussions chronic a burden metabolic FCS and the pancreatitis for CMED, treatment first Following in fatal Brazilian results in is

LATAM patients access ongoing in I for through first programs. PTC-AADC. treatment PTC’s We on the Latin with now have will engaging now have touch early gene in and in been preparations for on patient therapy finding America launch success,

the with time We anticipate final of identified have to pediatric high-risk launch, progress on markets patient Significant built United after the of made remain is where the which launch. also to globally has patients strong access throughout and approval. continues to center foundation And to centers we neurological activities States and preparation expert treatment of support excellence shortly a identification ensure EU at the in readiness we accelerate been therapy in incur PTC population. screening gene Europe the focused enriched available.

October together to AADC to family much as forward the Awareness AADC and are XX We proud to have very this soon. Deficiency with network needed bringing Day patients commemorated treatment look

looking their teams in our global to strategic will the for has quarter DMD a a sustained yet with QX against another flawlessly third financial franchise. The I been am initiatives. execute strong strong to quarter as the now growth I facing update. call continue forward Emily Emily? customer turn over to

Emily Hill

Eric. Thanks,

see million revenues revenue million the and results, $XXX.X and third now portfolio quarter with important of details this to I XXXX. third million product collaboration deliver milestones royalty Please quarter The $XX.X the look across few press of the earlier quarter the for in XX% DMD financial all press release details. to in continue top-line the results. take continued sales minutes growth third growth third on net revenue The $XXX.X in our to to refer our a $XX.X Beginning $XX.X the issued to have results. XXXX summarizes And across of of strong these strategic afternoon, over compared franchise were to a from includes financial with revenue. We programs. XXXX, to million our total our quarter review to pipeline. release million, Translarna support strong quarter forward advancement continues sales in a continuing position quarter. of for of this substantial We net XXXX. diverse of this commercial increase This product remain we will further

up performance, by $XXX new million. revenue $XXX in of in Emflaza been revenue driving this prescription strong This Year-over-year, to to million have $XX.X the Russia of with revenue guidance compliance Due particularly this quarter better quarter primarily $XX.X and million DMD growth. starts. driven this products from third million expansion, raised franchise $XXX has geographic to we over million for also $XXX to the net million been is Continued has XXXX.

million now million royalties Swiss year-to-date partner PTC PTC. our in million third Roche $XX.X in quarter revenue was in $XX.X Royalties franc, reported to Turning year-to-date XXX resulting Evrysdi, royalty where revenue. for has to of the to

PTC million reminder, received sale $XX this and Japan milestones, also the PTC in the a milestone As quarter. cash first payment based commercial was retained by for sales

million expense. has to expenses for million $XX.X sales were In additional XXXX, addition quarter expense, stock-based to revenue of compensation this, based $XXX $XXX compared PTC R&D $XX in in third an when the non-cash remaining with Non-GAAP this the is milestones threshold payment reached. $XX.X in expected $XX in million of excluding to $XXX.X million of million compared stock-based XXXX, quarter Non-GAAP third million for expense, expense. were SG&A compensation the in non-cash compensation for $X.X excluding excluding third million non-cash $X.X of of $XX.X third million the in the quarter stock-based XXXX, million excluding quarter non-cash for million stock-based XXXX, million $XX.X expenses compensation

and our We $XXX $XXX $XXX as range have $XXX of for the the and the to to million R&D to to compared as XXXX equivalents Operator? of lowered XX, expense December $X.X SG&A year the from will marketable totaled cash securities and September Q&A. million full million XXXX, Cash, for million. million narrowed to XX, over XXXX. non-GAAP now of operator turn billion call I $XXX.X

Operator

Instructions] JPMorgan. first Eric comes [Operator question from Joseph Our you. Thank with

line open. Your is

Unidentified Analyst

Eric. Hi, good Thanks afternoon. This for is for questions. taking Hannah on the

Just a from few us.

there improvement, point splicing Friedreich's entry term, expand So looking end of criteria myotonic near oral it some with with just vaccine COVID-XX over PTCXXX, assumptions you're next repeat Thank just are wondering or other to first time regard any your talk the the be that might seropositivity? then COVID-XX your as disorders able through or to if if you. just prior as ataxia? to And wondering trinucleotide are such platform, respect to you to into exposure powering what the primary relates spiritual a maybe study, and for XXX, of dystrophy about

Stuart Peltz

Sure.

the Okay. oral I'll with start splicing.

splicing able program well. that we've We've one that really have in interesting terms particularly informatics and in that I the as which repeats of we been as quite we platform what of been – sites and think to can talking it's that sort nucleotide us about the the interested the monitor. build the splicing, to been UX allows the identify

And a other So multiple high you say just those, built we of that platform I'll a like we generally the speak platform we at We've said we've ways more interesting myotonic tackle disclosed that's as in well capable looking ones. interest dystrophy talked what able in that have can think use and [indiscernible]. others. be we and to as other previously to is repeats about

bunch But can. a have then been to of be so we've able ability programs in yet. five the we using that of technology interested have probably other our to produce haven't we So we we the disclosed six more and or that these always expression

So Kylie quite over that's the that XXX, think being either Matt pass and in I of on used I'll one. well that to case XXX or

Matthew Klein

trial. I mean, XXX on question the

the looked baseline as than improvement, our an placebo as trial endpoint difference. had days. hazard our hypothesized believe discharge X.X the us is we people And improvement roughly which that saturation to about oxygen ratio which a then to room of to and time of is more study, the median we size two primary XX% respiratory from is data, XX the which error, one which at the to taking sustain day in here XXX us to Basically, defined on based effect subjects, on that then until sustained which XX% was estimation powering of a mentioned, a of were in is of basically assumptions to detect hospital of sides end gives power you So or respiratory type air, brought sample which group, means XX. time which existing treatment

to include terms when vaccination of that or the seropositivity. that previous regarding In question vaccination, don't we your or that any have criteria that's relates exclude

Unidentified Analyst

question. great. Thanks taking for the be That'd

Operator

Thank you.

Danielle question from next James. comes with Our Brill Raymond

line is Your open.

Danielle Brill

of using. I for so presuming Phase would the am And second probably much on XX data. two Good doses contemplating think the lower you mg you're higher It's data XXX study. going thinking the first I year. you the doses the Hi afternoon. questions, next for Thanks Are Thanks. we X, assume guess, of may guys. or then one have do be for to it's dose? reasonable

Stuart Peltz

on I'll Matt. to I'll Maybe Yes. start out, pass it

RNA we the levels helps remember where the states You that saw did anticipate study a and XX down number. reduced steady protein XX would that mg the at up and that go let's XX might mg we in mg XX% to X Phase and we the the we and was did of

the in probably of CSF. initially think that a I of we what as also we've to that. And that to thinking is about bit effect XX% taking able advantage is this targeting XX% what the to we get of would about higher the well be said some the be we're thinking see levels to as way

So us look actually that gives a at really opportunity doses. an of range a to

to maybe pass on to you the on I'll Matt, So that. give thinking

Matthew Klein

Yes, absolutely.

XX. of to start. generally somewhere I'm believe the to the be don't use for up So the the mg – sorry, the well design and that which instead XX% really we We the dose protocol reduction of XX with reduction, will that XX% all range, as which cohort Stuart range and blood plasma CSF been from as we’ve was as exposure Stuart the and and using which protein put we're we give us said dose all is between X, mRNA levels to to come and mRNA the long up ready Phase us five XX% protein said as inputs five would the and protein dose target and out the XX, got to doses, a was details running somewhere exposure until

between first more observed the closer, say, to what will learned leveraging we'll HD we'll differences that molecule. Phase dose. there's If that to of in on get observed any X as mRNA in translatability have be titrate PK, the Huntington the the And information as relationship specific dose, about that opportunity in in So we from what PD needless have we but patients. control placebo we reduction between protein terms we in and phase fact, patients of

the the roll terms focusing start over the long-term looking a In extension we'll Those that XX-week where we subjects that plan said controlled year. of design phase. to the into of be placebo study and We then with data, on are to will And end important on disease, important biomarkers Huntington other measurable the as disease, looking including biomarkers mRNA. changes well well in CSF in the including of reduction of NFL, blood as as protein in MRI. and at in changes as volumetric

In have I on data, once think terms to get we'll more – results detailed trial information we time targeting the started. more

we information future. So in the on can you give that more

Danielle Brill

data to So, provide Matt. until clarify, controlled the after placebo to you portion complete? crossover or you sorry, wait Would year the would one just intend

Matthew Klein

for that we'll terms and up whether patients Huntington of Phase data trial, dose Danielle, important the the able and accelerated pathway we're post share PK, disease study the in the very placebo as the level X relationship this phase a first very part efficacy time PD is I be because is advantage of after approval expect marketing. informally of controlled that's important take in the or will to establishing

that. So important, very datasets would that we believe share do

Danielle Brill

sense. Okay. for so you clarifying. Thank Makes much

Operator

Thank you.

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

open. is line Your

Tazeen Ahmad

I now. how And from Thanks. question do Where the specifically days? of you, Europe, it's are my Hi, new questions. see good for Stuart, you uptake then in these Translarna mature have most a a afternoon. you for franchise about taking product coming given the relatively couple follow-up. of thinking A Thanks that

Stuart Peltz

Yes, sure.

which your why – So should new go which been… to predominantly going think, well. we're still And you're expansion, quite seeing Eric, it's has I been is continuing geographic you been patient do. with quite through than team growth it's

Eric Pauwels

Tazeen. Hi, sure. Yes,

you Sorry, okay? me hear can

Tazeen Ahmad

Yes. Hi Eric.

Eric Pauwels

Tazeen. problems How having Hi, I'm a little connection you? are Sorry, here.

getting at in seeing So we've to also the We're Tazeen lot continued six, where growth lot of many in are we're but a the the quite now product international has that from And come quarter growth have very high markets other main patients as parts of yes. seven of seen dose been implementing We're mature seeing in seeing a also main adjustments of a have in launched compliance. lot years, Right five, markets. and over our and older of heavier. the we're growth the and we Europe least markets.

Russia As recently during able launched came quickly in centralized and also patients. order group Russia. in were And in I we of mentioned, the a get very call, have for significant to we very a reimbursement a quickly

and drugs areas. periods for So Eastern areas Translarna that of dose. adjusting They're patients that dynamic long are continued quite lumpy Latin many their growth similar we many for orders interesting. with it of patients see of are other But So it's is of that are a are – new is number Is the Brazil? revenues coming There America, time. creates what that there we others a bit large which seeing quarter-over-quarter. patients long to – is patients, in staying in little – with Central, Europe on and

up collective years number we've tough around even signed and of payers, the new in is had extremely Europe international some important. and opened a agreements. have with to the fight that for So of And payers some

smaller then more the been that that occurred. restrictive Finland pay the like lot even in shown willing they're Netherlands label there's countries So which and of expansions now the have have obviously past. to And a

the ambulatory. who of We less broadening patients non of years, now children for number patients. and restrictions the beginning two really then of in right as of number are eligible have go on going terms that label expansions And

benefit will, making patients who sort it if before risk are ambulatory. not of are off taking physicians So you decision non drug before

is revenue Russia strongest particularly have geographic And is and one adjustments, expansion, this it at had patient a actually clearly to markets good and today. dose quarter combination our collectively, if main driven really So Translarna our key I in what it of compliance markets. look of in probably quarter,

Tazeen Ahmad

thank Okay. that. for was detailed. you And super That

us And So, to for introduced the coming uptake Emflaza. about my to that? or what here looks your related and competence is that's today, like question and just guidance going to sales from for can Translarna they're lower last talk Emily Thanks. expense say more then said, year in last you why just probably from you estimates? It is, that relative you you would

Stuart Peltz

Sure.

Okay.

Translarna So lot of a it. confidence yes, Emflaza growing obviously and and both they Emflaza both in there's have and been

both want bit those growth a about Eric, talk So to two. you so of little

Eric Pauwels

quarter-over-quarter I working that not as patients million the new a a number high that We've seen only absolutely. We've that again, based primarily growth gone to saw We are I of on in new course XX% scientific prednisone. Emflaza but with adjustments. mean, of of and also very a we've but the $XX we also quarter. mean, dose compliance. publications on combination patients from that that now. data different is naive, who been Emflaza. of superior experience things, we've And but key see many Yes, shows minimized again, to who generated we've have patients switching are lot dropouts, Emflaza,

a base we growing So the of see patients within in of as tailwinds Emflaza, continuing lot those is Emflaza.

I continued work those year, expect keep us many of Translarna, in other we in investments forward we're Pacific, based we are we we provide did confident also geographic the to I continue And to Latin these in the places the paying that and and off to half last in continue looking probably in For expand that Asia America. markets mind a to those think on and mentioned growth. expansion, things year equally and all over is

we're think confident. I very overall, So

Emily Hill

When Emily, Tazeen. comes is question. your And it then OpEx to this

of XXXX. impact it's COVID versus full year I being a of XXXX a result think just really

and seminars expense obviously very greatly we virtually. holding successfully So our and their to Eric's most physician reduced of transitioned team consortiums then travel has

Tazeen Ahmad

you. Thank Helpful.

Operator

you. Thank

Cantor Our next from with Young comes question Alethia Fitzgerald.

is Your open. line

Unidentified Analyst

on progress. This Nina Alethia. and taking thanks for congrats the on our for Hi, all questions is

in wondering And XXXX. of risks are gives the readout third one. do what in think for quarter you We of might trial? the confidence that how also you And another

year. We how on FDA trial the curious the are were next X planning us for feedback to you Thanks. update PTCXXX Phase

Stuart Peltz

– that? go Sure, the want do mighty to Matt, through you

Matthew Klein

absolutely. Yes,

different mitochondrial occurs children that associated is disease about as with a of disease this before, the trial the mighty disease severe and many symptom registration is the So talked mitochondrial directed bit morbid subtypes. highly we've And seizures. a across and trial

as a And based mitochondrial was so this a treatment disease, really and trial seizures in the previous variety we of data who constructed clinical the of children different have genotypes number data phenotypes. with as on of X/X Phase well have mechanistic trial

in previous to we've that are of to condition the arrest We've one mitochondrial what in days we been days to the children following benefit. seizure epilepsy. our we related continued reduce is over so And in status cohort, of the in that mortality the the the was hospitalizations also had were And of XX reduce in able frequency, to course cohort. studies the diseases demonstrate demonstrate zero can That two years and reduced average hospital therapy. specific that epilepticus, which able able to of in three able The on year. reduce subtype on and is that we seizures been occurrence demonstrate hospital and occurrence

and able subtypes enzyme to treated, when we've morbidity stress family again, across not again, all us treatment vatiquinone frequency XX-Lipoxygenase pathology, is of affect underlying children. targets confidence phenotype further we've mutation. looked the we're the again, to regardless demonstrate mitochondrial oxidative and the able across to the what whole been different that genotype fact able the response the we to that the independent the gives So we're that in common specific benefit demonstrate or seizure And these related of pathway the that one that is disease disorders, but seizure across of only subtype,

XXXX. been world. around at the been close world develop is of work to years said, that results many trial course enrollment we repeat quarter closely the part that third trial. the global patient data centers in missed related around the to we've question, your could foundations as with in forward second that with network and moving expecting and over trial. the And that able of children just our are I So enrolling able relationships with you to and if of XX it We've the

Unidentified Analyst

you risks do that trial? think how Just about for

Stuart Peltz

I and the but only in trial, obviously our and with mitochondrial also one heterogeneity genotype/phenotype in not disease. we drug development you leverage think biggest in challenges trials I heterogeneity constructing talk disease, so heterogeneity about and of mitochondrial disease, is doing experience of think many of a relationship. genotype, Yes, And the when years children

And to this That's so overcome to trial. took careful common on And that we subtypes. again focused we and this into all consideration we're of different symptom. designing that that pleased these are

we of utilizing the of So used then seizures, all been those approval the a the in children met different minimum arm a strategy successful of a that seizures subsequent children that phase symptoms the consolation who parallel for that ensure syndromes. enrolling in about worry don't only know to run-in trials also have We symptoms. trial several and have having and has we're threshold. frequency That's to employing epilepsy pediatric

the to any might for most randomization stratifying addition heterogeneity into come In the common account still play, that we're for subtypes.

trial. balance potential the and think good control across design we group. of risks we the we at that ensure in And placebo measures all addressing the what be are those could very both So know

Unidentified Analyst

plan us I year? Got next trial for Thank question, the Phase on second And PTCXXX it. for the how you updating on guess you. feedback just for do FDA the X

Matthew Klein

we'll is I as initiate know. So telling weeks, we be we XX talk we'll results get that you, once the think the also actually well. the everyone after would about let after what be we'll trial, doing And obviously we

Unidentified Analyst

That was helpful. you. Okay. Thank

Operator

Thank you.

Prasad question Our next from comes with William Raju Blair.

line is Your open.

Raju Prasad

with question. you a DC that you'll sense GMP scale Thanks the therapy nature. the scaling and follow gene you some of have and as type things guys of kind plans therapy Just gene a Can I Thanks. of well for program, getting as what of the program? about taking on of facility. compliance that bit opened your know up obviously the for talk recently little

Stuart Peltz

sure. Yes,

square suites and XXX,XXX in compliance of that vectors prepare feet a plasmids we can terms have both had In manner. as – operating well of the the as it's we grow fully where about to GMP well as facility viral and both we do ability

preparation built those be do perform vector that. well preparation now the viral of analytical the is of of well. we and as do only to not in-house, that also capabilities required to quality the as that's own capable to of –the but so fully our team all all plasmids manufacturing to in-house, all as a And able And have we

program as being our going And as well. for for about and on we've other talked all FA that are we that's done. like that Angelman this well others doing So as what been syndrome for

So plasmids customers. liter the and the and team's said the anticipate that liters about external GMP capable in as for potential talked it well. don't be both of yet. doing the past, a doing We'll also functional. thousand I bit as – we'll it time external and – can I of been be We've I facility do fully XX a it be to think from We take in will little make to

And to able space as only so potential we be able also extra a not with of bring some in through bring especially well. in we'll where bring plasmid that, the be sense be work revenue use to in to customers, in but have we'll to able

as as the confident up itself now. the vectors site plasmids of now we've is DNA. and been we're this. So system both plasmids And and both we do And all well can capable is making making running,

more. needs the going and also fulfill have And be so as then we're some on doing, And customers to to grow we we and all do more outside. able have the be

Raju Prasad

you this, the you given generic that you kind regards of if missed think Phase based curious Kuvan of number seen into penetration that Thanks. Delta and see of and of be in patients kind to think to just effect may what or And to you enough do do wondering you registration the of you X, I recapitulate market? to have patients you but convert you're the of bigger enroll – may kind trial? a you've the Great. a on try think go the of over with that'll strategy arms that kind if going Just or kind just you'll of APHENITY, that in need sense most failure, have into the know

Stuart Peltz

that's So and shown point patients, any that that have number while there's it. I on patients on been excellent both an there's improvement and of think have have failed, or never large Kuvan a

doesn't for there's well So number patients. large do patients where a Kuvan of

about able generic, to it. bring no in don't in for obviously have then cohort, be think will enough the And to do for primary a patients we that. to targeting a just XX that So we're to the going everyone remind analysis, responsive people a even bring problem be we'll with run-in to it's patients be this, to

Kuvan of patients done a we had I success. directly up, for and our Phase already it's drug. chance the population who to to set enriched respond a remind everybody high study So just of X think, that We've study comparing previous show to

shown higher greater they we more and saw XX% is that number a effect so had than we've see of And what think and Kuvan. I we reducing responded, a than about patients of patients phenylalanine on

need is blood-based them measure well phenylalanine where better biomarker for which you patients, can if a better. excellence, very We this the There's a large well as many quite population. of number known. a of centers pathways So you really we drug. it's are think approval and think in it's obviously it, a of significant Really, about patient improvement view newborn opportunity. screening It's of defined a

see run-in because the study. this. we'll in the about we we APHENITY then obviously So we're there's in excited And that success a responder increasing analysis think have

need view, high PKU. in medical our there's in unmet So very a

As the controlled not and majority there controlled, untreated well are though available. remain of are patients commercial two products even are that

a this significant we So think opportunity. is

Raju Prasad

Great. Thank you.

Operator

you. Thank

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

Your line open. is

Brian Abrahams

in question differences proteome differences an is might have safety healthy what you Huntington have for and safety? curious, or Thanks Hi, potential transcriptome you or patients at XXX how any models transcriptome comparing by for in tissue to accelerated in samples. approval. for that I'm And And this possible follow clinical you preclinical Brian. Steve our at more and or might have taking speak for long you off can caused earlier hint on looked XXX show does question. there, a building on to on bit

Stuart Peltz

Yes.

molecule as I One upon a small to dependent orally it selective of a specificity a UX patients bioavailable, and us. toxicology. HTT where really we points. the highly highly does obviously that factor selectivity for so I'll spend think an that's differentiating for worked of we usually particular and it's It's and have site. is safety lot, very think XXX, both, that as is I these couple how that for on in specific safety really make molecules say well I are will molecules. So And

to for an they potential we so do And be RNA of review in of HTT terms the of within safety XX safety on we'll the of what be blood to CSF weeks results, reduction, the of be following results study terms Phase the give in still within And controlled biomarkers and open-label X going how well. to do transition placebo that. then doing as going and And the months other actually is as your patients accelerated this obviously levels well. see are we'll going And of for that. XX the total what study they number long-term that the blood the consequence a that we a protein levels the on of the on we're following and approval, question of the review we'll to as monitoring the we're to be study, change that's why a beyond we'll in both

an think not other the So only approval. if that is that What HTT for protein and a accelerated we're well. levels, the we'll have RNA biomarkers at in potential looking interested there's as we've but

the see in a shown we've lot at of few gene changes really we've we transcriptome, alternative splicing. that in what and very cells expression concentrations. in so in only or seen And yes, looked We

So specific. it's highly selective and

Brian Abrahams

it. for Appreciate Thanks Great. that, really helpful.

Operator

Thank you.

comes line of with from next Barclays. Our question Lovering the Gina

Your line is open.

Unidentified Analyst

Gina, Hi, on taking our for this is [indiscernible] thanks questions. for

We questions. have maybe two

you trial. next and you Thanks. my we going the Have think And of what feedback forward also clinical X biomarkers Could is deemed benefits? is good FDA on in question rate. Phase should the how any order contribution XXX QX Brazilian on quarter. got be will from about frame how the run and congrats the about First predictor launch us for number good Russian big

Stuart Peltz

sure. Yes,

we the So we We biometric for think of of and you biomarkers Huntington Clearly I think CSF if as and and in it's to it's protein we that as the blood, MRI the talk disorder of about know as and And clinical, the important. the Both the And RNA obviously, in like well plasma biomarkers. we'll and the measurements will and it's of changes the well. them measurements be and FDA in obviously on reducing, that along neurofilament. disease, the Huntington's so then and well versus animal monogenetic both measuring data HTT protein Huntington's biomarkers disease think with Alzheimer's, CSF, outcome know yet and there's at the show that history well and NFL for terms patients. as CA both that be as not other improves looking other gene then patients we as repeat natural lowered consequence a that levels. function, say, from that as gain a that have that

you there’s and in think, delays of know the in the progression ever show that of you I lowering actually consequence biomarker HTT where HTT of that the protein and helps RNA due that the patients lower levels levels levels disease of is to if reduction gives So disease. could terms the a

the – You’re in result lowering and the RNA the that levels good spot be of that the should able outcomes. protein a to hitting of and you’re say pretty to better that be

and package others, I looking So information perhaps that there’s of volume And then important. at preservation think we of that be coupled with CSF think arguments would the would good pretty a make. other that biomarkers brain and there’s

geographic gave little lumpy, may beauty helps the times. more. at things of is already different bit grow be that, think different groups ordering continuing in obviously a In little That bit a I while Eric expansion that, growth, are smooth terms saying yes, – to we’re of it out the

about quarter to to was Minister Eric, commercial the team And this expect so this do And secure want Brazil working with little talk Russia? maybe while of the hard COVID, bit tirelessly been year. fourth relatively year you Health We with hit purchase. has in a one the of group

Eric Pauwels

Yes. Yes, do. I

Brazil. in was characterize to four to perhaps or quarter, ago have what five very years think I we similar this Russia

So number for or a months. number provide of in and and access these patients a – of a some for provide order of these large single reimbursement countries will the government will will very order

So that provide where adding large particularly the had in four one a group to some Brazil a and two of a we can while large look start for have And easily that And of of a the of lumpy driver was quarter at what reimbursement our least specific at helped revenue of we five centralized necessarily was anticipate orders we’re that have now, you fourth And tirelessly it that in back quarters, as group within creates best patients filling new when year and orders of Stu key Brazil Russia anticipate or orders multiple the order the patients. The dynamic agreement size and started growth. in is us patients one mentioned, quarters as in right we’ve quarter. order getting and or an don’t a that we well years quarter, we in or go orders lumpiness. that for the working ever. this we

the predict. hard tend have sometimes the very number which it’s number not buying in These is months, predictable. governments to patterns pick erratic of to they do of always So patients and

see forecast, it’s hard basis, would of quarter growing to long to say, but you Translarna So of consistently. what be kind annualized on sort in that I’d emphasize like that is what an

Stuart Peltz

what like said Eric before. seeing we’re And

Middle Eastern in East, America, on. Russia, Africa. opened got in Europe, like So up Central North we new just Asia we’re also Latin areas and Pacific, working We but

the patients them a a function, have data top time, are we’re data’s more on we who’ve then at X.X where and keep for we’re patients and just of becoming – we the STRIDE expansion. been kids adding and it’s so about data that continuous like we capabilities maintaining geographic in talked compliance So too, those terms maintain if of getting long in amount that it, get terms as just incredible on better that longer strong the on patients of are of there’s more all years lung Eric ambulation,

Unidentified Analyst

you Got it. much. Thank very

Operator

Thank you.

Peyton Company. next and with Our question comes Cowen from

line Your open. is

Peyton Bohnsack

for taking Hi, there. questions. Good you afternoon. my Thank

you expected are have Can U.S. just submission you with happened? one signed the study. Friedrich months just in a little at BLA I second, high of that just on us before arrived know response can sort the know on they the FDA, us if we let just the any that placebo of submission. prior surgeries bit the a Have study then how there XX remind a of endpoint. in cadence the ataxia AADC And maybe at off the first six little the interactions saw The the in you bit

what this maybe made were to So the placebo response? changes were that limit in Thanks. that pivotal potentially

Stuart Peltz

take to Yes. you Thanks Matt, this? some for of question. the want

Matthew Klein

Yes, sure.

So to make BLA, submission. surgeries, about everything’s then agency we complete meet in the with the were those on collect first talked align place the as going to about data to we’ve package, the and the to the question ensure

yet. We have not that had interaction

the we’ll that able of interaction the quarter first in BLA the expect have be We to submit XXXX.

trial previous our phase study were studies, X of which our based duration terms on to patients to, the as by of we this Phase on In Friedrich XX have alluded lot the months. out move treatment a six you the phase week particularly followed control where had placebo long-term of treated ataxia,

way, statistical months, Importantly, that we that’s which significance. prevented common. effect the placebo six by us recorded And in a achieving

a trial known disease a two to important benefit in that patients two our an study, time, Phase If across, primary you found when the period, had would disease. six we’re that effect a not we But reversal of they on impact. we improvement a that able have trial you the hit because importantly, look over year been even that overall really actually at measure. placebo their at able months, one and see and the also treatments in we demonstrate concept for ataluren meaningful can a outcome of endpoint were That example, again longer X achieve therapeutic looked progression, that over that stopped to is reinforced

therapy. in have when so XX as by along but perhaps study the regards the secondary limited our long-term with position strong not us benefit results, reinforce or importantly, measured the that in but key placebo and the that In a that we we And together, impact vatiquinone able effect, the disease the only ataxia week not put to amfAR Friedreich collecting potential to other progression endpoint no very only that put with effect be placebo of of concern we’re the study. scale, believe

Stuart Peltz

but where that in just was necessarily you’re think improve, to kids I a of neuromuscular people stabilizing not terms necessarily lot diseases saying, you’re – I No, need. learning we’re going you’re seeing

get more better need trial the to within be You able to clinical time results.

Peyton Bohnsack

Yes.

makes just last sense. I one. lot quick think that a And then of

and that gene Now is one Friedreich the there any going gene enter we that we maybe see therapy on updates Are ataxia and to the facility program? therapy ataxia. the were up Friedreich When clinic?

Stuart Peltz

was expected patient getting That animals because in as Yes. first well of XXXX. in obviously on said still one study track we COVID were completed. we the and the hit the first and is we ongoing by half – The of dose long-term toxicology terms

using now. surgical stereotactic gene then therapy we’re be trial. the the Obviously that the be at within pivotal of procedure and where that’s So the will demonstrated safety key right here will product,

as the this that’s ongoing. looking forward to goals its So of we’re as just get going experiments those and possible. rapidly It’s

Peyton Bohnsack

on congrats the you progress. and Perfect. Thank

Stuart Peltz

Thanks a lot.

Operator

you. Thank

comes Truist next question with from Securities. Our Karnauskas

open. is Your line

Kripa Devarakonda

Hey much on Huntington’s program. the question guys, this you congrats so had I is this the was Kripa progress about on me a and squeezing for Robyn. Thank quarter.

you’ve said history deciding natural So at you looked population Goldilocks that for what call you in your databases. the study,

you that of can time the Will think Firstly, already expect separation does talked of readouts? some how we part safety protein in if or you to see data not part is the example, package. it week terms from a part, that that the biomarkers, when the of to be Or like RNA you other do Thank trial, and you’ve controlled the a that for them enough of the XX sort what FDA, you. be the history a need NFL? do levels? what And from Huntington in natural and feedback about see of to are you did comfortable get yourselves we study

Stuart Peltz

the for question. Yes. Thanks

history doing – over the one a database where study in lot – define natural for has community XX,XXX there’s there’s HD the a it In there’s terms over the patients us placebo a of within compare in within that helps – could the we’re history, us natural then of obviously the look first help XX doing, that. it goes trial will to that and define over. that one weeks, control and Obviously at, we’re

So we’ll have not much information.

which criteria there’s DMD. you down a to how a then there’s the diseases similar where can that’s a need see they you So own very the line, if the what of if won’t hard Goldilocks any useful. like, inform changes patients, help really see so too change, there’s see normally where natural time is this – in in happens, true that And area – far if you see it’s not for these in change to of time right? change Because are as now gone, response, a a very it’s they’re to we to very far – actually history, the using what and critical what is types is Matt is has of some us what we’re talked and about many much

population can So pick we’re that but a would that – see you to trying the course that there’s could right to we that you change is actually, make sure isn’t able you you decline, the would measure the where of the – difference. decline be

of ensure So but declining that. gives too those the possibility therefore see trial course the we’re the disease that to early in have enrolling over to and a higher are Goldilocks risk versus treatment helps that that population of the the really you progression, subjects not

we’ll anticipate RNA protein and also but will would as obviously then important, well And we’ll the be see brain seeing CT so biomarkers measure measure of that, we’re and and plasma. we as And that reduction hopeful then CSF CSF we as And volume MRI as the changes preservation the both imaging. in in monitor neuromuscular assessed change. we’ll by of the course look the

interesting. So will that package be quite we think

the will And there short-term the see biomarkers. be go terms we all something pharmacology others in XX to us so in on the pharmacodynamic that measurement the and get of weeks year a to think able for of will, allow the we’ll that longer-term safety will and

Kripa Devarakonda

helpful. That the And program. quick on Great. very up Thank then I question a was you. had follow PKU

terms seeing see to You’ve for are pivotal capacity. trial? any you challenges said you not of at still in are enough easy it’s a the of Or lot trial your patients being full Do the operating that PKU find clinics for that able to enroll?

Stuart Peltz

trial. So we just initiated the

global to the So we’re – doing of have it’s we so the what but a to able But as get we’ll obviously news, at. hard is give you and looking this any think sites, not good hard only we’re numbers well. global, in but be U.S., globally only we infrastructure,

a lot investigators. have We of

those think – can expand the one make have that’s you number I we’re one to and that’s sure things that’s so, is will doing pull if that And through of obviously that. why help that investigators a of get you to patients, so that

So we expect still XXXX. results the end the by of

Kripa Devarakonda

you so Thank much. Great.

Stuart Peltz

Thank you.

Operator

I’m closing no Peltz you. for And this the back Thank at call showing remarks. to to questions turn Stuart over time. like I’d

Stuart Peltz

Well, great.

that delivered has substantial And see, I thanks to our I’m revenue can continue in the really DMD once into we’ve of our quarter, have to life build therapies And to to raising that year-over-year fifth this – – The of in our registration see you growth. around continued we’ve allows PTC the revenue this changing And trial the company. advancement revenue proud today. franchise addition progress what joining again, BKU. continued we’re us in the revenue think we continuing delivering a think as directed initiation So PTC mission progress us can pipeline growth, globe. guidelines, XXXX generating the the I with patients done you made for

to thanks for thanks and So your taking time listen for the questions.

Operator

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

connect. You may now

PTC Therapeutics (PTCT) 29 Oct 212021 Q3 Earnings call transcript