PTCT - Earnings call transcript Q2 2021

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the PTC Second Quarter 2021 Financial Results. At this time, all participants are in a listen only mode. After the speaker’s presentation, there will be a question-and-answer session. [Operator instructions] Please be advised that today's conference is being recorded. [Operator instructions] I would now like to hand the conference over to your speaker for today, Kylie O'Keefe, Senior Vice President, Global Commercial and Corporate Strategy.

begin. may You

Kylie O'Keefe

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

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

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

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

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

Stuart Peltz

us Thanks, today. Kylie. joining thanks And for

has of half detail first a go in proud made progress more this I'm I'll the facets of business. in shortly. out XXXX, of to close we been into And As lot all that our say

know strategy our that no strategic patients to innovative measure We is and drug PTC sustained the like with pipeline to innate PTC will we having a to all and our to over take provide long-term. However, is the revenue deep treatment foundation produce pipeline, of moment that how debilitating both our before reflect At stakeholders so may and a to short-term I'd value discovery at of The drive the treatments fewer development a create diseases importance that all of I on mission to to options. process. have plan treatments commercial continue and balancing for have we're doing. do, challenges

number building all successful forward. are us grow working to commercialization steady towards substantially potential approach so that a state base. of the reach a will and therapeutic This programs that programs, of have new move sustained pipeline our revenue therapies We allow

So This parts many impact much is to of the business. brings have we world. aspects pandemic COVID very us recognize of of challenges access still the additional with have many our in reality fortunate that been multiple potential to vaccine, a across

DMD Evrysdi, arise. incredible However, our based through in impressive call. $XXX these uptake want million we revenue on and to in $XXX this they as franchise driven detail the of will growth the manage into have raising and by guidance we to growth is the we're impressive million will go I be this Emily in DMD our later more XXXX to strength seen this emphasize challenges revenue which quarter, continuing to the that franchise,

let with start me franchise. DMD So a

our to Our commercial the accessible to DMD team hard around has make globe. been working therapies patients

existing the a more proud, geographic over very the continues post I'm growth in franchise revenue of XX% the will almost year. As deliver an Translarna to strong success is with shortly. increase Eric same continued last period the both continuing discuss of an also of launch, Emflaza XX% seven year-over-year saw detail commercial expansion. result, that impressive in years and growth. geographies with

almost starting on are XX pricing in less and the treatment, post SMA markets in US a to to now than in let splicing strong and additional me Now as Evrysdi. market turn year markets we X,XXX patients is early on share platform, starting in see XX% a and conclude approved expect representing with reimbursement adoption Evrysdi also continue, continues growth an launch. the we an this Evrysdi of US, with to to discussion. now see outside these the uptake

anticipated, $XX million demonstrated a quite The the I the I the Evrysdi which SMA Roche the Evrysdi to patients Evrysdi this therapies, infants using we stabilization in milestones approved in study which SMA shows from upon quarter, Presymptomatic the Evrysdi results Japan previously Spinraza Jewelfish other will function energy of wanted as the from the remarkable. treated switching study, commercial developmental including was to and diagnosed increase As also benefit from overall with Rainbowfish SMA, positive healthy as milestone recent receive the same touch studies, payment as find on first with sale. be Zolgensma real world children. and motor Enrollment to after main broader Evrysdi. treated population. diversity assessing achieve in demonstrated infants is Evrysdi data the and the This

in to year, program, excited of second were study. and Phase this the the our PTCXXX from healthy to preliminary release Turning X quarter we Huntington's disease results volunteers a

Huntington PCCXXX. lowering As demonstrated cohort. after a results reminder, to dose dose The demonstrated study the results only also from lowest the multiple ascending in target XX%, a a in XX% even of lowering mRNA, single a of even mRNA this dependent to dose of

Drugs and exciting electrons, treat an during triggers stress excess within from produced is electron transport. havoc this would multiple X therapies completing of This cohort. can that of results of of causes This I’ll the wide cohorts initiate oxidative CSF exacerbates study, and usually our variety plan This in and food the additional additional in as of are cells bio-e clinical in that consequence are therapies a We sampling pharmacology trial. release includes to diseases. the be we well share to novel result new We planning the this modulate valuable process as a on turn next to to mitochondria end. the result a third disease identify cohort the year to that science these platform which and quarter states. our process platform. details before now protein as year, Phase of

We with particular note. have ongoing two important trials

key Our first a compound of from is pathway. that which regulator the this inhibitor, platform our Bio-e is XX-Lipoxygenase

in mitochondrial epilepsy, The and drivers. is value are near-term second registration These ataxia. in Friedreich potential two trials therefore first the our directed, is and

We for well adult a a range XX-Lipoxygenase second have properties, also pharmacokinetic diseases. suited of PTCXXXwith generation neurodegenerative inhibitor,

We are Matt to pleased the have completed PTCXXX. share later results And X will in Phase the volunteers for study the call. healthy

driver. near-term initiate affinity Turning this another program, Phase in to now value is plan study to of existing well our we excited with startup as which globally. registration medical patients a is PKU the directive X highlighting September important XX,XXX PKU year. an vast patients controlled majority substantial unmet Study therapies, are the well large with indication underway. The of activities need. estimated are orphan We're not

well on it in sites a the utilizing our US has community. well world, We to expedited unique PKU and differentiated opportunity commercial potential of and excellence, as to defined an disease is a forward know population, are to look globally. global the clinically and the We to rare focus patient path as centers therapy in PKU development commercialization. both infrastructure for the PTCXXX

therapy Moving platform, AADC program. and to our gene

CHMP This and stop a we CHMP the expect see is fourth a a to in inspection. pre-approval As for the is ongoing, the quarter. process allow clock still impose to reminder,

to been in announce that were has shared, we completed submission, to in have I prior Jersey. we moment the happy the the US, want our third New discuss plan previously with BLA of surgeries to Turning facility gene end to this FDA the therapy this Hopewell, the surgery by which now and additional conducting and recently to submit ally we of advance BLA the will take we're year. to submission, manufacturing

functional, fully manufacture equipped our CRO. XXX,XXX in lives a and of gene development have foot for on square pipeline, validated the therapy facility well our minimizing We thereby external the products and

with the facility and unique capacity, other expertise produce our opportunity vectors. retain As we potentially and relevant excess quality AAV into to DNA a a service entering expertise, revenue companies, manufacturing utilizing this plasmid to and we development stream manufacturing and high have create by have agreements

the upcoming is PTCXXX for pediatric a and clinical to oncology tumors, previously muscle. a our is is Both Results and tumor, rare solid sharing with the in tumor results, DIPG Unesbulin, XXXX beneficial niche potential clinical I and positive to medical treatment forward brain two have unmet and of adult milestones platform. Lastly, trials the wanted solid anticipated for to potential initiate DIPG need results the LMS. the no have options. look to with we rare in few half from trials second two We registration LMS are shortly. trials. highlight directed

Matt? commercially As turn make to to across clinical efforts. are continuing progress for our progress, the to of our I'm on patients update strong that, Matt commitment and over people and needs. proud driven see, an to deliver therapies With their our by our you development. mission can I'll call we to

Matthew Klein

our by start made to want development continued our all I the Stu. across have progress Thanks, emphasizing teams programs.

our with Huntington's platform, splicing disease our program. First, and PTCXXX start I'll

As our Stu Phase volunteer this the X study for mentioned, of we the second PTCXXX year. quarter from results shared initial healthy

we lowering As in MAD SAD cohorts. the levels desire HTT to treatment dependent PTCXXX and of dose resulted reported, in both mRNA

of the these protein Results this released cohorts additional quarter from CSF data in of in are levels process year. third and completing provide We HTT to the on cohorts biodistribution. be will

and key underway, the we by objectives study, focus on will be end The in reductions that the X demonstrating this we dependent levels. initiate is Phase HD PTCXXX planning X protein will year. Phase expect for trial mRNA achieved our for in patients to have Given conducted and the the of X and dose Phase study HTT trial

study more details design once We final. is the will on provide it

the for made I highlight would like platform. Next, program in progress have to we bio-e

children with Phase Phase vatiquinone, MOVE-FA, study have MIT-E compound mitochondrial studies in trial the But in the ongoing the compounds PTCXXX, and our global Phase with Friedrich to bio-e second that trial the X/X completed are actively trials and our the are And platform. registrational recently ataxia volunteer healthy enrolled. epilepsy trial, disease X We X in our inherited platform. two lead directed of we

in remain noted, near readouts term study we these to and MOVE-FA the with value study data XXXX in XXXX. you program drivers. And the MIT-E on are As schedule QX have

to X with I we pre now XX-lipoxygenase we efficacy which achieve PTCXXX that events. volunteer healthy desired a found in PTCXXX, be the study. was the out able neurodegenerative predictable am to clinical studies. developed for with of levels And to demonstrated our share completed reporting Turning have consistent Phase observe and tolerated also a exposure inhibitor we at being disease. well adverse were serious the levels pharmacology, plasma pleased PTCXXX

to at forward CNS inflammation, XX-lipoxygenase, pathway PTCXXX disease the X. activity Phase positioned to Through other a to now and as key Parkinson's pathogenesis stress disease targets Ferroptosis, in ALS, of oxidative are neurodegenerative disorders. and pathway its PTCXXX We move known

provides series and death vitro protection disease injury inflammation cell has in potent program vivo tests. a our Our against in preclinical in base CNS demonstrated PTCXXX and stress oxidative of and

we are study our Phase program, PKU trial, with this directly our PTCXXX registration on schedule the Turning APHENITY to to now initiate III course.

APHENITY reminder, enriching a responders placebo approach population to blind to As Xa responders success for treatment. be the PTCXXX study subjects then weeks. placebo the of of with six is controlled the with receive trial. a PTCXXX These randomized of either to will who probability a or study increases double respond This identify Phase

Following a the the enroll the XXXX. long be in plan label term from open quarter fourth all data to in to study, of efficacy subjects We will eligible exploratory trial study. have extension

the submit now of submission as FDA align support the data to been platform, on has the of the of by package BLA AADC plan Turning our to the therapy in third We Stu BLA surgeries gene to the completed. year. with and noted, the end then tangle

intended the cannulation our reminder, were a gene product. with the to commercial conducted As gain experience therapy delivering surgeries

path the the key area is therapy the brain product this provides directly procedure MRI of the relies gene of the program order that to that direct putamen delivered putamen, brain of to tissue. safely to to the aspects based an is a innovative reach One AADC delivery gene stereotactic direct surgeon google use to therapy. the disease for that In into pathology. on deliver therapy gene the our the map surgical neurosurgeons a achieve

ongoing for COVID-XX, of Let quick update a in study Phase completed Enrollment currently is II/III this a the in expect to previously we XXX. this now year. this trial emvododstat, trial and me by on PTCXXX be provide end

acute with As is patients leukemia. reminder in also in emvododstat being studied trial ongoing myeloid

now global like in from summary, important fully an XXX. commercial Dystrophy I to to placebo Finally, you turn is DMD milestones enrolled study development results trial we the in business. remind the Eric controlled ongoing and many Duchenne XX to XXXX. many with on of This that placebo previous continuing Eric? we move randomized made expect a have incorporates would key program trial. call trial learnings is have QX forward for with we future. the In for will near about our update I to study week Translarna study our are controlled This our over

Eric Pauwels

I'm instrumental strong focused again, the was the on remarkable III proud Phase in our team XX% global of growth Thanks, our and year continued highly in over delivering global both seen We've the Matt. and Translarna our team of quarter patients, commercial resulting for growth franchise With Once for progress extremely execution in in team our with DMD Emflaza another our successful from revenue. DMD a year customer franchise. growth franchise. incredible

drive clinical starts million increase revenue fewer by prednisone helping growth discontinuations McDonald have support differentiation Dr. the we of prescriptions achieved Strong from supported this UPPMD, XXXX. and to is quarter, by of quarter XX% over presented over data recently Emflaza. it's patient continues at continued a $XX new high patients. the New and adherence Craig second executions new In sustain in which

$XX in as due continued high geographies the compliance quarter to Translarna, growth revenue expansion of geographic base, broader of by expansion. this second well existing patient to a driven Turning in the and we performance achieved million was growth as XX% access This quarter, continued sustained over XXXX.

of Europe, in Latin Middle the footprint As patients have quarter us, now continues of infrastructure we treatment. to growth for in we pleased the revenue to be launched have and example been additional Ongoing Eastern America, markets Central markets Mexico. expansion such that successfully as of major for and including and geographic second of launch Pacific and other the East, a growth approval an focus expansion, one following the Asia are expanding XXXX. geographical in in Translarna Russia, our in announce entity Russia drivers key The in are receiving Japan,

diagnosed Translarna extending England. towards making patients. both We patients, progress purchase with America, for Brazil order in the managed security group and XXXX and in reimbursement access continue second increases newly half In DMD DMD in the are Latin to new and making see are we pleased progress we're agreement existing continued treat in Translarna for of good to in

of have awareness, case the continues. focus continues on pricing progress reliever, our be Now patient protect the to identification Latin turning study ongoing discussion teams challenges and substantial in region. to and despite setting and made America, Brazil, COVID-XX disease the our In

basic and we During treatment America education, program the Latin to progress early multiple study countries medical important patient amyloidosis in continue available process, access to in provide to testing genetic this patients. to support bring for HATTR

first treatment We some that America the pleased from access in reliever benefiting are we early have the with of pathways. now Latin patients through

for We are in Brazil. launch preparing

However, registration QX. to at delays in COVID now reliever is due visa, anticipated

disease a the touch of As priority the gene first backlog pending establishment we announced of launch. [ph] PTC’s with rare will preparation for to on taskforce a applications I for the applications. therapy address

progressing we and customer in readiness to and facing their by generated With great testing will to program gene identified accelerate US, flawlessly and announce in approval. rich confident strategic saliva excellence to ability neurological Emily? initiated home EU profit $XXX Emily high execute America has that, million launch transformative Significant AADC am to Preparations their globally continues from occur I potential our call to EMA turn AADC against PTC with that I ensure risk sustained another treatment time are been launch PTC a our And and XX of over activities launch. QX. in at with As and We the patient identification accomplishments achieve Europe preparation final in well therapy made performance pride base is the populations. of the priorities. XXX of the financial for Latin I changes throughout and raising to in PTC $XXX patient. that team centre goal XXX global meaningful to deficiency a genetic anticipate XXXX pleased our produce of take the guidance strong expert to centers after continue the pediatric to now to over update. at screening progress shortly million. over a revenue to the and in reminder, has we remain countries patients

Emily Hill

we a the our progress XXXX, cash robust in We quarter Thanks, to of see second balance position Eric. demonstrated across pipeline. a from year-over-year performance strong and healthy continued franchise. with and another In revenue remain commercial in financial the DMD increase

will revenues light details from to now million million We revenue results. we XXXX, of XX% $XXX increase details. upcoming have review XXXX afternoon revenue of performance program. of of been second In guidance second pleased from issued working platforms refer million. key strategically guidance $XXX to quarter over net product second quarter to the strong were summarizes and our the XXXX. $XXX to Emflaza to partner number for earlier results, to million $XXX to The DMD line sales consistent original for franchise to the additional our I Evrysdi date, with includes for and Beginning $XXX minutes million of in pipeline. $XXX forward top to from press take revenue in look are release these financial for XXXX release our revenue the Translarna royalty million Please the This a XXXX quarter advance the of a results. press raise the this financial of $XX and a the few million milestones

East net the on product Middle as this compared million the success XXXX. and key quarter Central geographic Translarna quarter for A million was and XXXX. has $XX Emflaza of been expansion, product Africa growth $XX second the to compared the Turning and net of particularly in North million, second sales in million revenue Europe, through driver quarter DMD Russia, this of more of region. were now detail franchise, $XX Eastern $XX to to in the

Evrysdi, on is million francs, which update of approximately partner our an $XXX to €XXX sales Roche Swiss reported year-to-date approximately Moving on has million.

As a return also upfront cash million approximately royalties of the Evrysdi PTC. revert to after Pharma back reminder XXX% for a exchange Royalty billion, receives royalty a XX% of sheet. our of PTC retains in €XXX added balance to $X.X until which

PTC this regulatory and also As milestone. monetization transaction, royalty sales of based part retained cash

development SG&A for expenses, of for expenses primarily approval Non-GAAP of quarter service to term commercial quarter, merger milestone with million Japan the million Japanese in of and $XXX were well second $XXX second this agreement in million million of the second the quarter compared non-cash second quarter Non-GAAP stock-based the stock-based as excluding million related $XX.X $XX.X expense, $XX one-time non-cash MassBiologics. of XXXX, the of of near XXXX, million XXXX, The XXX.X to for million XXXX in commercial $XX.X expense. excluding R&D Censa $XX.X Evrysdi compensation we charges decrease non-cash research payment for for in as million manufacturing compensation for compensation quarter second to stock expense, the upon in Following excluding first anticipate $X.X in sale. the were of XXXX. compared XX.X quarter expenses relative based million

XXXX. the $X.X securities June in stock XX, non-cash XX, equivalents of $X.X Operator as to the compared million marketable Excluding of Cash, to December million over based and I'll for as now compensation billion Operator? Q&A. turn expense. call $XXX.X totaled XXXX, cash

Operator

Thank JPMorgan. the [Operator line Instruction] Our comes Eric with first of you. Joseph question from

open. is line Your

Eric Joseph

nice Good evening. quarter. question, the taking Thanks for

choose a flat raised DMD half. now the to to trajectory guidance midpoint for that buy performance. Just down franchise on the the looking second we're actually here, With at

performance Then wondering kind either to So anticipating with us reflected if that risks could the purchasing any I have follow-up. this talk you second or I'm through or a you're results and Translarna ongoing whether Emflaza with stocking? advanced just respect quarter wondering of

Stuart Peltz

call. for Eric, Yeah. don’t why this. you Thanks, take the Eric

Eric Pauwels

Eric. Sure,

had QX. think First revenue of had of We million XX% had with identically terrific for all, growth. $XX last sales in XX in We I year. Translarna, Emflaza, the over million And that’s a quarter. we increase XX%

largest new after at We're reflects markets. now our guidance guidance, America, the team continues major Central from I and revised the think coming done this, of proud business especially despite European be of Latin in our right work some that from all Russia, challenges that I you has patients and years, seeing right you somewhat the in East lot southern to America the high Europe, of of a be we Europe think base dropouts. to seven COVID high patients that is large look right orders, the And expect, very growing and really, year. our in the and of happen northern we're have. second those are already when half of base, what we're compliance able be we in lumpy, now, in significant been maintain But to the Brazil, to growth solid, and Eastern now Middle we've in they we where Latin orders particularly have anticipate can with know, rates that patients base minimizing which

So think I mean, I very confident. we're overall,

Translarna. We have a strong of continued growth

On one had continue we patient front, of ever, new the we see grow. and to Emflaza our best quarters

generated very also with really seeing the that's to real patients, switching data data, a only world And not now, new of Emflaza. we're side. seeing and that are that compliance high. important being but actually patients see data, switching Emflaza this from amount prednisone we're We importantly, And new an data more that's extremely increased is that

very achieve. now where be that's And we our has on think in least it right guidance, upper think overall, pretty continued we would be end I the we're at tailwind. can And is year-over-year. XX% with looking good increase now would now the right what at business about strong, right So a performance, line

Eric Joseph

here, Envisat. potential outpatient just And for on conduct Thanks. hospitalized Do is use you you that? to flexibility The about to trying file is how Okay, regulatory the a market sense of I'm get in opportunity for great. just have separate you trial FITEXX need but a patient? Do do success. a setting? the second do you're to patients, focused the see assuming used thinking on its question Or

Stuart Peltz

we're Thanks excited Thanks. that for question. Yeah, about this. I'm particularly thanks.

outpatient a certainly are so in everybody molecule. the small PTCXXX can't that right. setting, be And Just used to that remind

it can we doing a but hospital right now think, So, used. trial, we're be certainly

an and dual think its of I is so is has it anticipate have it and that it's mechanism, SARS-CoV-X that we're targeting all, I mutating the likely a by all, of targets elicit this, advantage issue. won't with enzyme. -- resistance We of that. much it issue the as mechanism the think a first of mechanism as less DH that second the to the of cellular and don't cellular drug And because consequence

on as -- in that we we based if this use on the in it will certainly we'll that and whole, from hospital can the And think a successful, setting. current successful. So, have so FDA have patient the settings. how possibility with in the we outpatient the both discuss well trial results as to when If

we're seeing it's used has that the in take. simple both So, to to capability and certainly be

a get you as can COVID. soon -- it can You take prescription, you as have obviously, it if get you

Eric Joseph

got taking Thanks Okay, it. the questions. Appreciate for that.

Stuart Peltz

Thank you.

Operator

you. Thank

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

is open. line Your

Nina Daniels

taking question. progress. This expect and for all congratulations the CSS? and should Thanks. you Huntington's our you much for Nina Hi, were update, what is information wondering on just around share how could if the We on Alethia thanks get or for

Stuart Peltz

want Sure. take that? Matt, you to

Matthew Klein

sure. Yeah,

cells cells lowering key the peripheral distributes cells in and and its oral into dose ratio one body blood an that of with said to a brain. X molecule dependent one Phase a So, study, to broadly protein unique that Huntington where the blood objective peripheral the the inside we of with bias was brain to of opportunity have mRNA study as before the demonstrate for we lowering

get one an from brain. the what biodistribution as look molecule, the is on Huntington eflux to really is which to function a able going total of is peripherally and and which obviously it a a that cells fact is of brain And disease. been So, that's that's we're lead able molecule the in at regions the all what's two, the important and do incredibly brain blood designed CNS penetrate really oral on equally,

of CSF dose out peripheral And able at right sure refer work in in done cohort on far to MAD going of all cells so lowering making before. cohorts. that's SAD what -- And we is with get key the we're show biodistribution both we What that we a the we've to mRNA biodistribution the design cohort. Well, this plasma the the is cross in this the the in were molecule happening only And and drug CNS the the can that's that way that's volunteers, or based blood those throughout we to in influence, of the levels and means stays was CSF the One get pharmacology to regions it ensure CNS for is it it the not so all in the to blood-brain blood-brain important look doesn't anticipate not is the in. that barrier, to element barrier, that if incredibly of of whether measuring specifically, levels tell brain. comparing CSF, we the peripheral by -- the levels. the cross healthy

getting being CNS penetration. designed we'll that So, -- in drug we're getting and the levels CSF CSF exactly that the drug up able from desired check cohort, that's the readout in CSF the plasma lining be levels with the end to of box that of

So of able of in the design preclinical key the box that and specifically, drug of levels molecule. in CSF, animal feature we're getting check the studies seen to the these the a the in -- and being we're we've models, readout key beginning flux biodistribution that that lots

Nina Daniels

you. Okay. Thank

Operator

Thank you.

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

open Your line is

Tazeen Ahmad

patients been a This while. where we've I couple, many think been you have It's Hi, a since on much gotten good questions. AADC. patient engaged Guys. quarter you how Thanks my an a in taking is for just I on for afternoon. have one program, a guys update found. so finding efforts

was have I patient in curious, you numbers I if was any the think then addressable could follow-up. today. one somewhere identify I And population you the the on last that have count updated XXX.

Stuart Peltz

Eric, Okay, to want thanks the you take it? question. for yeah,

Eric Pauwels

Yes, Tazneen, lot doing of still we're right a now, work.

and But to As going our time and we're well be you this we're be and achieving anticipating providing would at of to number patients. the this now, have now at really in following goal, pursuing not earlier, patients. patient addressable that time, in look have be when I really, to on we've our be continuing are But have you mentioned we're ultra really, really is Germany, the We're that at an net disease. now. which know, widely not approval, And prepared done you understand to patients, country we cast of launch have to numbers -- launch launch, that point first finding. XXX between the to rare and pretty track is, EMEA our what time, would aggressively reply be and to we've the to right plans is our first

XXX very announced across in So access, patients And the represents of our sequenced when number amongst in mind, according And high we be we've been cerebral palsy, the in course, have number and keep where will then be But globe. can the at right get that like we in we screening of center. of those MS where the Europe areas, reimbursement countries. that risk geographically to of have tested countries we in patients XX program populations, early And over access launch, market the globally. will time to really particularly and access various reimbursement. now, programs epilepsy XX patients countries

just who Europe, sure have really Matt So the and expanding will, neurological as pediatric the we're and that we on centers -- excellence. Asia, now have only in we're the of you our right US, making that that ready. centers And patients really focusing treated not centers said, in are if on but focusing have, binding, patient

to these So be treated patients, make able they'll as have that sure when we quickly as be possible.

Tazeen Ahmad

exists, that I between view. XXXX terms you That's think population total of still your addressable your think guidance patients. your in was And somewhere XXXX last and Okay.

Stuart Peltz

Yes, Eric?

Eric Pauwels

current our we've in --yes, that are global currently a seen is published suggested and program, the of risk We literature population, that --what screening the we've --that sort particularly of in the number. work terms done

oral use an treatment you're to even a of this not and simple product remember, is that like sort going a Now or infusion. tablet

stereotactic and follow-up. So it approach, requires surgery

would treatment patient we than going be slightly So the of the intervention is treatment and more your average to sort of that complicated do.

we what have that finding and the to it's the testing patients believe in in that made important of the these pinpoint the through we program how of in So program, exist, number credibly terms important genetic US strides and we using patients know therapy many patients, of patients that And countries being important. programs finding where we're to spinal and simple that the easy currently know and in are more are really reimbursed, gene we're saliva use. that's

Tazeen Ahmad

Okay.

Stuart Peltz

the Because countries. beauty as we're finding is everywhere -- well in they're different And there. many we're them

So that there. still X,XXX the think good shape us there's pretty confidence in I patients gives we're about that

Tazeen Ahmad

could you. Thank if in squeeze Okay. Huntington. then on And one I

on planning a by end you for are clarify, knockdown wild-type this year? the showing to of Just

Stuart Peltz

at So in looking -- with the don't wild-type both. We that, here. we're discriminate in between

so the So and RAC, that as the yes, mutant wild-type overall reduction of you'll see the protein we -- both since reflect you'll levels overall be RNA the of seeing levels as that level get both them, from well both.

Tazeen Ahmad

what you wild-type Okay. is? highlighting knockdown But be specifically will the

Stuart Peltz

so is differentiation on based we'll think be nice. the the to wild-type two. over, that's be And knockdown I showing the between we'll say what -- able

Tazeen Ahmad

Okay, Got it. Yes.

Stuart Peltz

XX. is XXX

one-to-one So ratio. it's a

Tazeen Ahmad

Okay. Thank you. Got it.

Operator

Thank you.

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

is Your line open.

Unidentified Analyst

Brian. Thanks taking question. on for Steve for is our This Hi.

data Huntington's. rapid mutant or curious tell animal given turnover? could to accumulation on is inflammation models Huntington that neuron of cause preclinical dysregulated protein whether over might a the what the one known models protein And from Another or how I'm any us depletion about Thanks. time. it's of mutant the adapts

Stuart Peltz

don't We any. have

that results HTT I in benefit the models, of that think improvements studies shows in in there's clinical several terms animals. been show in lowering of animal lowering, right

So that's sort know at I in reduction least any that you causing consequences a sense I animal rapid have in the of of think studies. I don't think unusual that

Unidentified Analyst

Thanks.

Operator

Thank you.

line Peyton Our Bohnsack of the with on comes Cowen. next question

open. Your is line

Peyton Bohnsack

details is of taking congrats This disorders when ALS on strong if wondering and question, And why trial could instead of Joe. my on guys. other CNS PTCXXX the background on for the chosen quarter. Peyton Thanks you'll Thanks. for these design? on for on more neurodegenerative provide is Hi, a little more release I was timeline disorders? any or you some

Stuart Peltz

Sure.

then So maybe and it I'll to on Matt. start pass

platform really key. excess that terms exacerbate It’s I in aggregation. turns and inflammation stress, XXX causes a whole knowing the think of think if you and bio-e about on electrons oxidative that leads

electrons XX-lipoxygenase on of ultimately response in things. sense in process. -- key when sopping this turns gets you're that XX -- then interim And to up This a system a is end fix the So the inflammatory of the try when system of emergency is response and overwhelmed. sort

turned other it's happens diseases, as in So, turned it that neurodegenerative why the a used get many as a in important the it point and gets doesn't real I guess things. down problem of well up. But -- be that is all can

them. first but that's as multiples go them. will doing we notion even would through picking as and why up them, look example. the number why a was So, to idea of maybe that, And through Matt you through go go at And we're don’t ALS our be the to of Matt a

Matthew Klein

sure, Yeah, Stuart.

As and has disease in different its that the number the and including inflammation others. so preclinical program on mentioned, is of Parkinson's been critical XX-lipoxygenase pathway oxidative a understanding entire disease stress based you pathologies, and neurodegenerative ALS, targeting including of

a in a in and diseases to others what that and are able vitro So some we've at efficacy done are of show models number of efficacy, specific. models, all is been disease and synergy of different number across strong generic have looked vivo that these different

and of to So the the arrived with data on now starting X selection to based fact we in ALS now Phase three have we And right hand X. move the move Phase with that is ability months. first our

think trial also key disease about are the patients benefit. being to three of course, a disease. to the the support the neurodegenerative to able actually rate see -- time we right identify going over of key change, able when two enough having one, to of And studies dosing. then, in considerations tox month one that have for those being which development, long And show We to so able so to, progression change are are; of

is measurable. unmet between fatal diseases XX a the ultimate and patients a with or function, of progressive all patient demise of XX despite is the disease. with rapidly months and unfortunately, following rapid still diagnosis. which being But neurological of dying over of the progressive ALS, respiratory decline function rapidly course function that majority muscular readily two and are there's prior in One the And approved of that, it's The to needs. are key significant there advantages therapies ALS

change We of over history FRS, of we impact a allows also ALS and robust three the endpoint, use with us on data, have study can a have as is time, an show on to month natural able in both an validated based well treatment three PTCXXX month ALS disease. the to placebo period a that which known group as

here registration opportunity that in month a work pathogenesis study. in importance the was a preclinical an target where the can disease ALS, have then a we what pathway we our So three definitive that XX-lipoxygenase show to is our have disease and treatment effect about understood of disease, form study directed that

So then of in the selection toxicology months data ALS, as to just the the summarize, to three of basically, indication three terms are of first able being do to program efficacy. and being able complete months basically in able move an having and ALS to time, have to to necessary that show to dosing be can collect data the effect, on we're be of disease, we PKPD period

quarter rate designing of But well. be Obviously, sorting development for well placebo, running a a validating as is and provide effects, and It’s going the be disease on action use the So the the previously, establish details has treatment about study months groups a the I [Indiscernible]. We that important of analysis. still full the bearing plan biomarkers mechanism have in that and as ALS used mentioned probably begin for to [Indiscernible] the of dose we’ll exact right FRS to standard the which which indications first of validated and window. elements phase there'll more other we're at ALS thinking progression very we baseline both key we’re the details, trial approval least as And of now, of of been in three design to we're the of scale, endpoints. XXXX. in And out ALS endpoint disease pharmacodynamic process trial.

Peyton Bohnsack

or trial more to Yes. one just is into That’s of question will wait on that. data? indications going you done you very Do plan until with the on Well, quick other follow helpful. this kind

Stuart Peltz

to it's issue is, timing long ALS more be I we think was and this than more term will we've indication think, of get a of GBA, -- that. anything timing spent the having a toxicology. disease as goal short adult into the -- is of I versus always Parkinson's neurodegenerative because else, Our of And matter

Peyton Bohnsack

helpful. you. That Thank Okay. was very

Operator

you. Thank

James. with Brill Our next question Raymond comes from the line of Danielle

open. Your line is

Alex Sklar

approaching could as early Is capture patient pool. share for your enrollment the Phase in looking for thoughts possible if to was should criteria feasible patient be on could from if the KOLs Phase stage as hearing the the any is Huntington's patient question. X? Phase see trial? it Huntington's for color the your I Thanks. provide how Alex Danielle. We've Hi, this for X address Thanks to benefit. to the plans XXX on in the disease not, been on X pool taking And that you you're you pool

Stuart Peltz

Obviously or neuromuscular either that. see, always range that measure, Sure. mean, be they right. where you to can that they statement But early could you make in in decline neurodegenerative get a the wants general you -- where still these I'll there's on some in I are -- that as any -- possible. diseases of early that in you that the as you're of while

if So come Goldilocks, too help late early, that, and the critical at the just too like a help too transparency, takes in decline choosing you doesn't right the population, right? doesn't the it so in pretty the you to end long range early, you. have Too much patient day, endpoint of right hit you like the for --

for that that do of population. to amount a particular population. and It patient the define the fair trying define at takes -- you that high the that's find outcomes the patient have that looking in an in amount So gets you be to right natural or patient and right history declining. some confidence have a be right the what's that inclusion-exclusion time experiment, will then to the confidence you're going you of given And what's have can the is population, that criteria, patient, clear population, you

a in of taking the next. what that. teams about of And were philosophy thinking that's we're the So lot doing

also, -- So want Matt, else, there's if to about. talk you anything

Matthew Klein

-- just I it’s okay.

can is if to biology, with raised Goldilocks enough early we make that you can that have that the sure have you're that over overall that that, because that's change able sure And right, the the is therapy show the sure population disease actually where assuming you disease the that a matter you'll this point placebo. duration untreated, had the want receive And you group approach upstream in that disease of ability the our think affect acting progression. that's of of going especially, to trial, key to do a enough to concept really that here have benefit Alex that still I during the in-force, in placebo but you make the is population is benefits. you a clinical also a to meaningful one, can amount of you show decline have an an now, affect we're to disease progression, the early absence of enough

that we work we've that measure that a always and our over database patients. said. in-house XX,XXX like carefully databases endpoints working at population, been teams we are And KOLs endpoints repeat, ourselves a to moved of those only the collected actually number to think, spending looking changes and external important lot of well very with us one, with it existing the Goldilocks so, let's overtime. factors I -- time like the properties as availing the are of And second, find appropriate as CAG by different get really that We

on patients Phase measures based other going Huntington's protein mRNA. are reasonable have that to our And changed to our of patients X to disease us, demonstrate effect over enrolling but lowering and go and is obviously, who continues Phase from of see We to in as So disease. obviously able X will show trials HTT are work. in, what this you're in be believe trial, that be to the foremost, first also workflows

Importantly, we HTT they what at able reduction and of the in into look geographic to of biomarkers want tie things changes, the to then mRNA is disease, both like demonstrating as geographic say what be to benefit protein.

overtime, right So obviously, patients. benefit who to want are the patients sure in again, those can making template make direct to going to do are so a that, that we have we show the change we and

Matthew Klein

the hand, I'll maybe other make on bit. a But also

based things with experienced I Alzheimer's with experience them FDA, plan discuss some the on And think, with to on. interesting focusing for accelerated going we pathway a the there's, do approval monogenetic about I used, biomarker. better relevant I if even and plaque think Alzheimer the mean, the a disease. think have, practice in you you the on that was for case here

targeting You're the that's involved in the precise protein mRNA that's process.

company you You data there's reduced new extend the soon time before shows that of as see as History know, protein, Natural that levels says, a the onset disease. the of immune

is So discussion there at be the least, had AMA have the well, both that a talks to certainly possibility it able FDA. and discussion we'll a they've about a the as with with Novartis Probably FDA.

good position if And we've endpoint potentially very second for that's what is because the been then a about in work accelerated case, that see approval. we're if study. to with for we the certainly So that talking could

Alex Sklar

for the And Great. Thanks you so much for the taking question. color. thank

Eric Pauwels

you. Thank

Emily Hill

you. Thank

Operator

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

Your line open. is

Unidentified Analyst

a I phase what if And volunteer question. many optimal we that X updates, make two this data doses. Thanks. Hi. doses beyond is, trial [Indiscernible] therapeutic would are? three would your you XQ include? for the from you how have to you second trial, I the those XXX my X Gina. question in mentioned the how is in they share, healthy And define couple XXmg another levels window? the Could can think This have you for the XXmg past, on doses Thanks max and comment

Eric Pauwels

Sure.

XX% somewhere So went and in to a the we XX milligrams milligram to reduction of the after in what to between XXX% XX at XXX were the we the XX% mRNA in down past XXX singular those talked up XX was probably of in does, milligram level, about even to are able HTT. around to and XX% reduction of multiple of dose we've and dose XX we get which, treatment. ascending equates test the days to In cut dose XX way,

if could got which milligram that you So, and really between and were, we -- XX you lowest XX in that so our milligram the shows to it reduction. quite that XX% trial is nicely, XX% dose were served was we -- got

to the HTC level the So, able able clearly depending on of -- mRNA we're that. be of to titrate, exposure the --

other The -- XX what reducing. -- reduction, that in order dose looking terms where sense think, will we're in milligrams we of about already already at I launch, the probably a the between we've is as doing sense, that -- a really somewhere in the we start which CSF, effects food thinking looking things, we've effects, gives food said, at and XX is in us XX% that with range really of going we doing are in because to

of the we're pretty and the be begin So position, will look and HD the at the to biomarker really to consequence HTT there think I patient. biomarker a HD step good looking next reproduce, a at in as of -- in

Just is to we're and CSF what -- so reduction that, said the see just is we that that PKU is the And then study. and exposure a sure make that also, looking we and CSF well. doing at

seen just what the in that would we ensure that what we've know in define is we see the to equivalent So blood CSF. and

in in we're lowering the XX% range. Ii therapy that and dose we comfortable XX So efficacy dose or we're the know with I what range we're them milligram therapeutic, that makes to received the think, at is -- that trying you? we desired low that do lowest the that, that help the already of

Unidentified Analyst

Yes.

cover in covered level dose dose CSF do one multiple the that will cohorts will cohort? or For level that at

Stuart Peltz -

right. We're only doing one dose level,

Matthew Klein

Yeah.

really basically the dose based single proportionality, and only we've this we MAD worked, one SAD dose, pharmacology. Obviously the throughout all level. -- is back but the on this the need the need pharmacology You business simple dose only not pre-clinical seen

dose thing pre-preclinical we've in all the sort learned a dose single verify to think to models way about translates Human. is single pharmacology the right can we've one the degenerative bio-distribution one pharmacology a from very say, you diseases of level our clinical thing learned able that assess we at what I predictive well the what in the and about

Unidentified Analyst

Very helpful. Thanks.

Operator

you. Thank

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

Your open. line is

Unidentified Analyst

Communications Raj. came the in administered ventral pretty for compelling and therapy is ultimately quarter This Nature centre published that function. on academic Hi, taking in to DC tegmental gene There led an a in, Sammy was on that a, our thanks substantia there. area, Congrats out and which of nigra they some paper the for questions. recently motor that in and used was to improvements

opposed midbrain I have Just want targeting conducting PTC-AADC And to thoughts them? study on these your the to then to putamen? regions if as follow-up. you a administration a get And consider would of post-approval examining

Stuart Peltz

Yeah. the Thanks question. for

So where in was a you clinical nigra. -- substantia at in setting done university a said that

years well results as And looked we've we're, much five five as profound the for larger underway number so at going work the we retainer, I it's into after. patients a that shown terms years where you we've the if that a in really results the study, think had, in look that of saw at clinical were in of

So AADC. to in XX also think improvement, submitted with EMA up saw years. to BLA And And of neuromuscular think end have That's the of an XX what the that up we regulatory the therapy the for only the I And I was think year. is process we have see XXXX. by years -- plan I active we submit continuous you neurological we we for results. that can durable again,

we positioned the issue that to to So I think is transformative reason world to. this, we're we to I obviously, that think I spaces think, many in around them that needs structure there's all space retain that patient to the is PTC-AADC therapy. much have uniquely available deeper to and that AADC thought picture think like thing it, other is cases the show that showed a that, improvement, less And I midbrain the is when all get an think we then that is we

think, So think mean, I be would of that really, that have the transformative. results to based be quite shown in quite and shown a based dopamine It's neurons factors. this on the the of on choice really data. I that the active selection are where spacious, number is And was I

feel the or pretty doing other and were we're necessary where bring then gene-therapy we the good requirements patients. So to all events, at, regulatory we to

Unidentified Analyst

from which candidates disorders. little we your thought, therapies, your Got you. separate before, for mainly pipeline felt us much too just what strategy you. development those Thank remind about and heard oncology I a genetic Could target other clinical divergent and rare And a like your haven't you it's are them?

Stuart Peltz

that thanks for Yeah, question.

on the PTC estimated with and US. options. tumor. medical a a patients there's deep diagnosed -- in brain UNESCO which adult LMS. It's Unesbulin, sharing -- which and So, bit few are about heard quite program have working it's compounds, we're the planning treatment as PTCXXX. little plus you And that the previously solid per with rare the estimated LMS update called dive year sort is is rare in are XXX and CITG and Both higher is Emvododstat, for that, is the these what was beneficial some shortly clinical X,XXX trials with program's CITG have that And COVID-XX tumor muscles of we a the of are AML. two a year and about really for in a to oncology well. on no trials an patients one programs pediatric then per programs XXX, United needs is diagnosed results States. exit these of on for is

of been so programs have ongoing. And these

rare clinical So in trials. the these rare it's second We of a much the disorder, anticipate very reminiscence XXXX of approach. results of having for disease half a the

here, these And we're directed quite And results registration with following that. so we trials positive the initiate have about think excited results. to I potential

So by we're AML results we of within stack the with also and XXXX. bonus then -- end a expect

much about these we're what programs two molecules programs, that starting not So be are to talked going additional have right. we and

data. and into talk to about -- them talk to getting Now have you position some

think be quite exciting. would I

Unidentified Analyst

data. you. seeing Thank I to Great. forward look that

Stuart Peltz

you. Thank

Operator

now I'm call queue. no back for the showing further would like remarks. in you. management closing the to Thank to turn questions I over

Stuart Peltz

you. Thank

today. thanks us for So joining

And to of highlights, team we're quarter in is pipeline. progress the continue really of continually our the impressed the that, execution substantial I a of continued with am execution shared look the we franchise. I in has you this really is made we these continued quarter perseverance Also And and forward across of while guidance believe As to revenue ongoing, result the the as progress -- global this our raising for resulted that of year. year, people. this made to second And available. by the full in addition development updates I our pandemic dedication they in that the this XXXX become team the the think the second DMV commercial excited has PTC we've half

and look to forward for thanks to So joining talking also. you

Operator

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

may disconnect. You now

PTC Therapeutics (PTCT) 30 Jul 212021 Q2 Earnings call transcript