PTC Therapeutics (PTCT) 26 Feb 212020 Q4 Earnings call transcript

Company Profile

Participants

Kylie O'Keefe	Head of Investor Relations
Stuart Peltz	Founder and Chief Executive Officer
Emily Hill	Chief Financial Officer
Eric Pauwels	Chief Business Officer
Matthew Klein	Chief Development Officer
Eric Joseph	JPmorgan
Joel Beatty	Citi
Brian Abrahams	RBC
Vincent Chen	Sanford C. Bernstein
Joseph Thome	Cowen & Company

Call transcript

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the PTC Fourth Quarter 2020 Financial Results Conference Call. [Operator instructions] I would now like to hand the conference over to your speaker today, Kylie O'Keefe, Head of Investor Relations. Please go ahead, ma'am.

Kylie O'Keefe

Good afternoon, and thank you for joining us to discuss PTC Therapeutics' fourth quarter and year end 2020 corporate update and financial results.

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

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

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

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

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

Stuart Peltz

all today. two Thanks XXXX, to our forward Kylie. trial five to In bring and including pandemic, clinical development we the we with initiated COVID-XX directed have on for joining thanks therapies moving for significant progress through commercial. note. pipeline fronts trials, And new particular Despite registration research and us made patients

strong with Let continue see Dystrophy geographic Duchenne global expansion. growth franchise, the to and we Muscular me begin

were XXXX in sales $XXX franchise DMD million. approximately Our

new $XXX the total Emflaza and million, increase which labor is and from Specifically was annual a patient revenue in Translarna totaled it the by XX% revenue modification. annual last US, of $XXX expansion, geographic for million, year, driven the

the quarter. For example marketing Russia Translarna received in fourth authorization in

turmoil purchase these will Translarna COVID-XX, secured into have Eric details Central during in expansion America. and by in continued go shortly. Eastern we additional the and drive group Latin and owner geographic Brazilian We Europe Even a for also to caused

and discuss also the recently clinical results the the evidence, these world we As along to plan OXX data four a including FDA. from Translarna existing the five real of with dystrophin study, totality reminder, results we reported

long been who waiting Translarna possible. been as Our quickly as to for to always US therapy bring goal this have patients has

was which an Roche SLA for has approval Moving and splicing treatment, benefits the particular durability our distribution. partner for to Foundation. with milestone platform, a the tissue important Evrysdi vacation XXXX Evrysdi groundbreaking of is a benefit broad PTC, in SM all our of the and

of As in and strong XXXX. that uptake show consequence in the of benefits a continued the treatment Evrysdi to US choice Evrysdi Roche it's become of and the a will expect

continued With the expected near approval, significant this approval followed term year. the be Evrysdi by European Japanese should growth

The million to of partner XXXX balance our is XXX% revenue royalty the sheet. deal reverts of to allows Evrysdi $X.X receives PTC receive the to on which royalty Another structure and with royalty once monetization put our accomplishment regards of XX% $XXX monetization deal, million. approximately

molecule disease. advanced the the from PTCXXX most next of validated platform Our is for Huntington's treatment splicing

reduction blood an orally Preclinical PTCXXX the mouse as as brain. crosses reminder, of a As small is is barrier a whole cerebellum brain molecule demonstrated clearly back in results the cortex important reduction and model, regions HD the HTT HTT critically based protein this and disease bioavailable mRNA levels. brain reaches cells in disease Huntington's in PTCXXX the a all and dependent that the on of titrating is striatum dose

lowering, of lowering be tightly can HTT degree the controlled. So

uniform to exposure, lowering tissues the brain PTCXXX addition distribution, whole all analyzed. achieve In and in HTT,

because multiple single in dose ratio currently within see we one and analogous to the So blood. in it effect CNS just the to healthy near blood between cell. brain important one and X in trial ascending in the a Phase exposure and PTCXXX volunteers. shows This the is is what

This demonstrate the allowing of will drug cells lowering. results same proof in in to with allow concept the was of the demonstrated successfully where us quickly study. measuring approach and be healthy program, mRNA in will within was This the select to us activity. in both volunteer risdiplam dose HTT blood, We that levels activity used protein the desired HTT level

same We trajectory the Huntington's in expect the disease program.

results expected are of the half We forward XXXX. are in very program look about excited and first to that this the

to final stage for registrational commenced. platform, and of turning trial the neurology the our second XX Now has pipe COVID-XX

SARS the functions cellular viral cytokine is less enzyme to dihydroorotate As small likely and of PTC-XXX DHODH. that viral a of PTC- advantage dehydrogenase molecule a targeting is a inflammatory both of demonstrates cellular with mechanism XXX replication an storm. protein enzyme antiviral a that drug reminder, by the calm TTC-XXX oral dual action effect. anti it's and X or The inhibits instead Covid elicit resistance. targeting

normal The have the made ability on there focus treatments effective resume is continued a the our key. COVID-XX to treatments strides significantly life been developing lack in and So vaccines. great development has therefore of hampered of

let our me Now e-platform. to bio turn

global We XX,XXX prevalence at estimated have and epilepsy of the is global is are mitochondrial initiated epilepsy with note, prevalence patients. is in two particular The study. one mitochondrial rolling approximately patients registration of both ataxia directed one in one patients in trials FA and Friedreich and XX,XXX

Let platform. gene touch our me on therapy now

to is year. in first the during second Europe second therapy to AADC for half of expected on track for is the gene quarter submission of with initial also The occur Our launch focus is XXXX. patients our this deficiency, BLA which

has the the a years single gene XX PTC therapy. AADC of durability in a post treatment, crucial gene to AADC As up consideration patients. data clinical meaningful is a PTC changes therapy potential to transformative that dose has a AADC robust effect in deficient that demonstrates for produce reminder,

discuss Now me PTCXXX. for let plans our

potential well differentiated therapy unmet medical majority patients and we're As as a vast there's excited are about high estimated a clinically an reminder, of to address and XX,XXX global current of PTCXXX the therefore this prevalence not need. therapies addressed by PKU the

our call evaluating Matt, treating stakeholders. all on year. for Last exciting will key turn our to We in continue many registration year will clinical create substantial XXXX. We now for the updates programs. milestones. year, I'll called important the anticipate start of trial mid a Matt? APHENITY, this we PKU for to That value achieved PTCXXX an over

Matthew Klein

Thanks, Stuart. build XXXX. our to comments achievements I on in development on Stuart's want team's

of many past PTC challenges We navigate worked to the have hard the year. at

oxidoreductase have continue across our in been for a the reactions, transfer oxidative of to to Bio-e XX diseases developed are and called perform electron key that class lipoxygenase. number targets known planned on to compound focuses Now start lipoxygenase, are regulator platforms. XX of multiple to CNS excited of and platform targeting I'd being like of stress oxidative of note platform. a the reductase. Oxido deliver Bio-e development Particularly enzymes special This implicated oxido with enzymes family on and by milestones biological first important the a reductases important has the platform inflammation pathways stress functions. diseases.

we As registrational vatiquinone Stu initiated Friedreich and mentioned, have epilepsy, mitochondrial in two trials ataxia.

data, in pediatric safety exposure duration the extensive being has vatiquinone the reminder, of patients, over XX years. longest a particularly with As

year. had For that show subtypes. the ongoing with seizure endpoint mitochondrial in Enrollment the third trial, vatiquinone and a on XX mitochondrial the study first clinical reduction placebo worldwide. in across seizures motor secondary other differentiated for underway results randomized positive next morbidity. improvement activity These disease morbidity seizures, quarter children and endpoints primary studies related seizure of indication, vatiquinone seizure and The controlled centers a the potential clinically give of the is a to epilepsy the that capturing aspects mighty mitochondrial mitochondrial effect The of are related confidence demonstrated has at expected epilepsy, is enrolling study epilepsy is observed previous trial multiple us patients. data

to excited mitochondrial very children disease. are to We therapy bring this with

disease particularly that quarter is strategy endpoint with and disease rare, EU. in affects primary The anticipate meaningful the was to from the The ataxia, in results second is Phase inherited Natural treatment History vatiquinone progressive the X a endpoint on severity, system of ataxia compared authorities Friedreich improvements is patients. in Friedreich is change developed in particular trial we key change neuromuscular secondary fourth in X of a significant from the which consultation week placebo over to began results the known last trial, registrational XX and that in ataxia months. The scale. a FA as endpoint trial trial heart. demonstrated In a support is We deliver can Phase or XX FA Friedreich in randomized bars. the in nervous modified living, assessed baseline previous of cohort a controlled baseline effect These regulatory scale, by daily XXXX. ADL and the The enrollment trial M This US year, reading activities study. MOVE-FA

from underway Now treating Phase and a neurodegenerative multiple splicing cross our the PTCXXX blood need brain orally region target X given ascending is was volunteer to vatiquinone developed and both trial designed healthy treat let Huntington platform me regimens. disease PTCXXX small disease, is turn to molecule advantage to ascending the that the significant single avoid The of dose disease. for specifically and is molecule bioavailable to brain, the designed includes efflux, every program. our barrier effectively an

data accomplish with studies As five we half a Phase not were HTT and MAD X from mRNA includes In to cohorts, cohorts, the volunteer of and each to placebo of with mechanism year. blood both and have splicing and reminder, study and study. is capture are protein typical splicing six healthy trial and this in study Phase risdiplam study, this to is monitoring designed of data active expected dose placebo proof establish two similar we in what SAD blood to three and to guide the X of for healthy to we'll in each key subjects. X the SAD be also MAD mechanism the dosing The studies. The only to the proof data, study, Phase pharmacology this first in levels PTCXXX five key safety but gain expected and drug selection the of ability are subjects. is six The active cells able measuring to future CSF concentration volunteers two

for this quarter to CHMP turning we PTC-AADC second FDA platform, remain and in year. the submission the BLS opinion Now therapy gene on our the MAA on the of schedule the to to

before we our progress human therapy FA program gene and In end. are to addition, in continuing first expect dosing your

clinical PTCXXX that continuing Turning stages the unanimously the the data We our planned, completed the recently interim safety of and the of second of study. as recommended COVID-XX was with enrollment DSMB announced the first stage study to trial. reviewed

XXXX. in advancing initiated X In second forward initiate by for the We we with of the PKU are have the programs XXXX. in XXXX, to end look the enrollment on trial data are to patients schedule in second the data whereby XXXX, APHENITY additional of half already PTCXXX mid we and stage expected Phase in expected

call building XXXX. in And to mentioned, Eric medical when successful these PKU need. the to sharing summarize, on business. remains development execution our therapies, I'll look more updates forward To despite provide programs programs existing Stu high we commercial the to detail important As clinical now on in our a available. unmet turn of

Eric Pauwels

Matt. Thanks,

We that the innovative are very global, deliver our pipeline, of diseases. commercial which neurology with to excited rare with can we and progress multiple expertise therapies footprint existing stage our is poised in leverage potentially clinical late

with generating significant Stu Translarna continue growth year- As DMD in revenue. had pandemic, see DMD of Despite highlighted, strong and growth the to the in both we the of over- franchise challenges Emflaza year franchise. XXXX, the

ages mutations the high Translarna, and of million and patient expansion. was allowing patients, continued geographic updates, For DMD label compliance, to access growth of broader ongoing the due $XXX revenues we XXXX. space, nonsense older, in saw only recent treatment The for expansion the two

DMD therapy in of approval to in Latin we XX this With globally, progress. Translarna available is and Russia XXXX, to expand America, patients, QX, the see in the mutation use which Translarna bring we now over continue of In good excited countries. are non sense recent to

pandemic. Ministry we Translarna two the year, the shipment Health October, patients. last were Brazil's non in delays we administrative mutation of by that excited Ministry Brazilian in ensure shipments of base exceptionally DMD of and both patients. are into announce purchase new order growing the Brazil's agreement including a last QX, supply existing Health for Brazil by reminder, to shipments, entered important the to sense hard given of last a continuity This specified received hit to agreement As was both governmental with The XXXX

further from in to year. coming see this growth patients the the continue half next Brazil region order We the and new in expect second of

is bridge $XXX year-over-year in XX% revenues We free which only Emflaza, a lower to a for approved is DMD and of programs, new increasing PAP older. which on moving patients, primarily from ages compliance treatment of patient driven and and corticosteroids first and discontinuations. in all two starts, reduction million growth, saw the XXXX, charge Now and

driving clinical publications real Importantly, healthcare to benefit patients over world is from XXXX, we growth their providers. continue strong emflaza's prednisone, to which new see now prescription supported treatment seek into by of switching

continue Translarna franchise expansion is We Emflaza in with XXXX new to geographic global $XXX revenue Translarna. this, and for million. guidance to for million expect on DMD for both our and Based for patients XXXX DMD the growth in the franchise $XXX

Tegsedi Now Waylivra. switching and to

with We for in continue CMED Tegsedi pricing Brazil. discussions of

During we program patient to and genetic testing, needed. process, education, this continue provide support medical as

continue Waylivra, success in For we America, Tegsedi with findings to patient engage and ongoing programs. in these Latin in

to a Waylivra we region, and await XXXX. in engage in expected on which is the as also continue QX We in the access early Visa decision filing Brazil, in programs

therapy Now PTC-AADC in produce that AADC. moving gene AADC to has to the a meaningful on potential changes is transformative patients.

up improvements demonstrating and muscular highly pediatric diseases deficiency a no modifying fatal morbid and shown treatment. trial, available. in gene significant disorder. launch of clinical with gene therapy, is As expected preparing patients approved AADC which AADC XXXX. is therapies In for deficiency, neurological to after half a durable during PTC the for our to reminder, occur years currently is for and are first currently continue neurological There PTC-AADC to second XX therapy Europe

As is Latin XX countries as our pride global take PTC in XXX access rare time Excellence activities Europe Patient over throughout of are by efforts, of underway need. US, accelerated for disease pediatric to America. programs screening identification neurological launch. and patients over with teams products preparation patients finding also I to facing in in these part XX of Centers they of customer of the continuity expert and the ensure to continue identify

commercial our We our on the lives continue to expertise science of disease expand neurology, upcoming patients transform AADC in and success deficiency in with launch translating build groundbreaking worldwide. for rare to the

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

Emily Hill

Eric. saw our Thanks of strong pipeline. growth PTC XXXX, continued multiple and revenue across platforms In progress

press due due label driven by a these now deliver XXXX review quarter creating and by the guidance. of net in on this executing press year $XXX.X to and this high compliance; for few potentially our line Translarna Please to updates, the for as details. year. by end full summarizes $XXX for year for the $XXX factors, issued financial year, US results, royalties year and new XXXX results Emflaza our million fourth year XXXX of value milestones We to reported primarily $XXX.X I on million earlier to the charts additional and to expansion details are milestones was both primarily franchise. full the compared release afternoon full fronts associated will with three million the total to DMD take XXXX. approval revenues as a number geographic the we were patient's increase access, launch. results. This Revenue and The many the million to Evrysdi minutes growth revenue our well XXXX. driven and product for broader financial Starting with top refer our driven growth was compared global Translarna

approximately This product reported $XXX For $XXX total XXXX prior year for which growth. XX% Emflaza for year represents full compares over- million year- net $XXX year. revenue the from The we product million the to XXXX. revenues franchise DMD million net a was of

market invested transforming and created is by PTC's research potential assets. care initiatives. to value its Our million and allowed revenue cash financial million PTC into diversify risk its anticipated is development and not product The million has and a XXXX PTC other cash as one franchise platforms the stream to with guidance for having agreement $XXX patient DMD purchase tied currently this position support reflect flows royalty $XXX RPI royalty This cash any to real future guidance future $XXX revenue between contribution. does of has being

by from XXXX. the revenue $XX.X related triggered million receive potential The in potential of increase payout upside, ability in is the to majority royalties retained to to milestones PTC in million to interest year. and receive RPI milestone, future We the recognize tapping periods. retain future in retaining the Roche prior consistent Additionally, $X.X Roche, were cash to collaboration the due an in increase regulatory at three and its XXXX, future sales regulatory from from that billion remaining has flows primarily rights $XX.X the

$X.X Non-GAAP to FDA recognize royalties we year the annual $XX.X due expenses entitled $XXX.X for XXXX, XXXX. stock full million were $XXX.X non to worldwide are in also approval We net in R&D August, of for excluding the the revenue compensation compared as to in full on XXXX, expense, Evrysdi million royalty sales. million in based million year cash

$XX.X cash in in R&D and clinical pipeline. the based platform, the Excluding million investment Bio-e of research and associated non costs advancing with reflects therapy, compensation expense. increase advancement expenditures gene in increase splicing the programs stock This

the were AADC in expenses operator million $X.X Additionally, Operator? as agreement million I'll of billion of December pharmaceuticals. $XXX.X time cash $XX.X based program SG&A $XXX full XXXX for related to compensation the non over the million, expense. $XX.X compared expenses the in December related non GAAP our full of million stock non as excluding acquisition million commercial be year cash compensation based session. start and cash We $XXX XXXX, compared call full million estimated million to year equivalents approximately the hand the $XXX.X and one XXXX. SG&A manufacturing based increase in totaled XX R&D to marketable lead million and Cash, expense. in between excluding MassBiologic now XXXX, question-and-answer with stock securities $XXX of therapy of XX, to for million charges anticipate compensation expenses includes the to expense $XXX.X $XX.X our million R&D And XXXX, to $XX.X Censa gene for deficiency. Non-GAAP stock cash the year non in excluding to

Operator

Eric Our JPmorgan. with Instructions] Joseph from [Operator question first comes

EricJoseph

me. for couple questions. Thanks And just from taking the evening. a Good

CHMP to walk and initial First launch And me, Europe? of could sort then through rest the also, with of patients by about Europe. older with as Thanks. there about the you thinking what's compared little And in in challenges guess, at risk the population any assuming proportions just infants? benefit delivery decision. you're been just risk be it benefit just for talk distribution? the that sort -- Has the about pipe us you varies Perhaps and how well. What the Are Can thinking I bit launch guess a to I positive thinking regional age? countries, what of in either excuse anticipating are profile? age identify PTC-AADC strategy stereotactic today the in XXX should described juveniles with of

StuartPeltz

Eric. And call. thanks I'll over to Thanks, I'll Yes. who Maybe you and the to planning. turn talked for more about commercial start Eric, it then

see years remind So saying, the really just developmentally and they thought that if therapy a like treatments transformative these patients changes went do meaningful patients them. a not actually, that think standing ultra be I when to I'd gene that's and and transformative disease modifying everyone, by you arrested to AADC all clinical and can like able stand their pediatric patient in in this, of no differences really of move, we a that showed very to they sitting, you about capable from that developmentally the approved remind orphan move the patients, just deficiency. think lives a they head were therapies really There And we and make that start are rolling, up, SMA where being have to really have therapy, probably we available and improved, much I think patients in roll is these and where that that for was is form severe gene are there we to of they walking. of arrested And really can that morbid fatal for for die nothing just really in highly the life. that few over therapy hold and trials, within disorder, unable

a saw consequence this. So we of as progression substantial

pediatric clinical So older which groups age saw five in of XX data that follow may And all and children years, improvement transformative continued showing years for this them. five really, to adolescence. results, including years we highly serve, we after are we

as very We to a from didn't a saw nice any consequence the the it. of we surgeries. So surgeries challenges of anything related really benefit as see standpoint consequence a of

ability it's we packages So we of biomarker durability, one to that where changes are valuable see. think a we product, continue confidence can in see clinical the because of dopamine think we believe you because the packages, best see highly levels of the to

really And so actually think I what that's powerful. was is really

we've are been launches, and working terms to what ready of identifying ready getting getting And getting patients excellence. of identify, centers in so on obviously,

Eric and launch to a through bit launching about we're so And talk the what you don't doing why for potential that.

EricPauwels

there. the Thanks question Sure. for

outlined have that a of all, activities that in Stu of number most regional First key we importantly, preparing rollout.

an are educational genetic XX driving So screening increase now we included testing, who those have in programs. disease programs

We've XX over eastern vast Europe, will and country access products. of and terms we know reimbursement in Europe of central virtual and ultra LATAM, and disease done and -- in there for in Now the webinars specific number well in majority central as global symposium. other countries, be rare where gene and things as a therapy parts places products other

But going the comes specific, European by and opinion key mechanisms is Europe. in Germany. the of areas approval first will once from and activities key are to in roll would launches preparing, be of that our have Latin only and not the will of sites, of finding Programs number immediately been follow as those many preparation of as as treat patients one excellence doing of advantage we've mentioned, expect, where the Stu but marketplace American, commercial which making take other areas the world the aggressive education. in of well sure full out And as that patient the with the the areas, the I extremely, a them. of and would you in as is to we how the also Early been And prepared and say So terms terms regional centers well Access we're in level of final is rollout

Italy, of full America point. others, So France, based Northern will then of Early advantage countries confident course, We're Latin And we in and well, Programs and we in anticipate Europe, like take -- Programs approval. Access at Access Germany as on Spain, European we're and Scandinavia the this would and very Early finding, say most we've important, be that number in an large I'm time addressable anticipating programs will we've implemented XXX I since access patient these and of would excited launch. a gene the seen of countries reimbursement to acceleration And to where in of at have we have we patients these therapy.

Operator

comes Our with Gena from next question Wang Barclays.

UnidentifiedAnalyst

on David the Huntington questions. is the have you program few my taking This are I that for for for Gina. Thank a questions -- on disease PTCXXX.

are questions the First dose. just around

half So levels you're can healthy testing in life dose and And the frequency? volunteers? you what dosing what's discuss

StuartPeltz

Yes, the this to into it's so for remind cause PTCXXX, a of that, we RNA role that an intron that so just to everybody stop splicing there's the codon. thanks have induce premature

and designed degraded. and the you So all the regions an is it's so it. moves to then out toxic reaches is make not of the basic orally think that so would of for the can protein small well, of it everyone, characteristics and that brain, potential, that out many the critical is just so protects things itself be, so rapidly but molecule one we pass many barrier, it get the the brain most importantly, potentially blood that property as crosses a efflux brain brain blood barrier; RNA And prematurely will stopped, reason major are that it brain. things is the it's don't And the that is, of this, that I but that's then bioavailable, the

made property. pumps and do barrier we've critical within that, blood pass to that And so the a that many that, efflux so PTCXXX there's sure that's stayed

therefore is cells you level know in and in the within brain. that the the blood what see whole you you So see of what the

XXX all cells so it cells then see animal what really lowering that a the observed think clearly and brain. the And one parts be levels of Huntington to the what on it I degree passes that level, analyzed, brain. its was between the of that barrier. ratio And tightly brain that cells tissues of brain of distinguishes one therefore HTT all achieves in lower in the models, HTT that are because shows in we've uniform so, we the is near that PTCXXX exposure. And in it can the And blood, cells based shown all titrated, that's controlled. the because the exposure And of blood in reduction within

And see brain, we in very a so to blood we the the measurement same good indeed levels That's have show. case. so

So molecules specificity that saw that what on is in on bio distribution. primate. trials that it of kind that our we when the that from seen of, about experience the in for we think on, working the based understanding was obviously, And the based levels we've we splicing, did, rat, what in mouse non human the we did, obviously screen

we in on clinic. on we that molecule the that the the animal begin we levels would did of with. moved we And what of people in drug the the saw that, date results the levels that predictions chose based really the selected we in we how then is see is models, and So we would

what to and low is CHTT think rate move I think to choose where that did that. dose, continue it this, start we be predict on identify how right we we that's the we were based lower So within move and the up regions

to doses go levels were that the a look helped chose changed. human we how and you? for into that's what and So David, is have

UnidentifiedAnalyst

Huntington's I the question for your what of that, looking helpful. to to on in you next select healthy very follow the HTT patients reduction level disease trial? are is in Stu, for up dose And that's just enable Yes, volunteers, guess you to

StuartPeltz

Yes.

a do multiple this we're to going So dose is the think ascending way doses. we and about single ascending

to the a it's we're that number dose is a single measuring the so fact capable the dose we we And a think one the in that can doing that characterized know equal steady great that's And of the dose state of what have And And brain, what both in knowing ascending seen exposure the to that's including a of primates. exposure, time. in and of in the that the reduction. get gives small know really, that's shown to ascending the act. I RNA molecule of you've so we of actually, in that seen exposure, we've really a in very we and be able what's this what able gives is have us and major over move across RNA good blood level advantages we'll characteristic and be once changes multiple brain. so forward, and is non identifying, that And see animals, we've blood beauty that that then all human level the you the where the to and we targeted the of dose,

very So that. knowing comfortable we're

as we're we the the measure reduction is, people measuring levels and drug RNAs we of blood. what the be in will doing of So as then HTT in

define a good response have we'll that we dose very level we that dose can can what So we take want.

terms So to benefit. look and starting in that. for we're at clinical But we'll reduction probably that think a of within probably XX% looking then about

a if there you some in myths look in without terms think are of years data -- you clinical at substantial see results XX%, I demonstration. you patients in that where nine when out they lower have disease it there, greater of improvement terms

lower go data should as identify able capability -- clinical look more starting so. think the starting at look But -- that good But what that. even we'll a we're a pretty to it's Does we if not? learn that as and correct think go to can also we at be can we to there's if we're be have I David? at XX% looking or we we would that But lower be So benefit. when able that help to lower XX% titrate at But the go that have level. starting to at dose if define we also to a We suggests we're could we're the do that think reduction further, the you we'll can dose. will

Operator

Young comes Canto. question from Our next with Alethia

UnidentifiedAnalyst

Hey, for Thanks for update. guys, Alethia. on this is [Indiscernible] the

in on the the quick Huntington trial the study. Phase where the on study? now? follow you Just moved to us have portion are a Or of up to you just program, update Can give net you wanted X

StuartPeltz

as of So dose trial, we've looking the you SAD at multiple been go the about, obviously we've both in talked we're into single they doing dose already trial. so the terms ascending trial, right, the know, ascending and the

been those. obviously So doing we've of both

the So have by within we we'd of half that it first the -- said year.

So it's been we've moving underway and that forward.

capture, And the so just safety where designed right, remind obviously, to to everyone, pharmacological that's and data. it's

placebo. two The in so six study while single I expected, have cohorts, active up active up -- study that to said the a and five And we'll is half can be six active is have have dose drug each drug it could there subjects who MAD includes again placebo. patients with with six ascending to to what with six cohorts,

XXX the We're And levels the without study, and forward cells. Phase in moving blood in and it. everyone, ultimately so X excited of drug any about protein in dosed things what just issues. monitoring actually to remind the is messenger have been blood, we're obviously, pretty then HTT And RNA concentration the that the

We one as in as have cohort CSF. that well the blood PTCXXX will measure levels blood levels will

healthy reminder, studies a as subjects the volunteer and don't Huntington's these are disease. Now, have

be about the the of have pharmacokinetic blood, don't I have people proteins is that target wanted CSF, to characteristics if at and measurement the we of go interest to sense So the we would to end and to what you'll exploratory to to in that the in that say with is able HTT the to define shooting of purpose dose show of the for. know think see correlate TK be exposure studies day, we're level us be do the expect CSF. what that talked the allow The going in to And need. of levels proof to higher those to that reduction able we we're and mechanisms, able determined what dose splicing PTCXXX, the

the going and able healthy SMA I accomplish out risdiplam what beautifully. in which subjects, these the think analogous really we these So did worked trial, are be really to volunteers to we to the were results when

single year. data planning So dose, expect of multiple and of studies dive in have first we're we that track of half the to ahead with within -- the have and data the that the you talk preclinical you to PTCXXX both the share And, further ascending this the will course, the ascending a have much will we're of can for that. deep of on see for we the the so have that and that dose science data to

UnidentifiedAnalyst

can And Okay, now? thanks you BLA terms file for us you the are the just What is on study, in on to question color. And quarter. an second vatiquinone are then my you give second of the just where wondering update remaining AADC, just in steps? right for

StuartPeltz

a MAA about of year I we think know little the right about everyone, quite Sure. to want this already the year. already, remind the to now? where second and we transformative for just you and in Follow expect this talk a post XX treatment. to well. is and we've durability in both therapy, a seen want patients are Again, a And it. Matt, CGM gene does about opinion forward what EU this it you we've the remind to that are quarter bit just bit talked up moving obviously, we

MatthewKlein

sure. so Stuart. thanks Yes, Yes,

the gene CE patient about talked ataluren to experience intended Mark that been the which surgeries therapy, And wasn't the matter factors why ataluren we've an demonstrating with gene before, to with is was a is BLA of delivering mission population. getting our of it gating ataluren the obviously; for before, many submission. use conducting specific the gene MAA it to therapy was us of the As issue therapies It's in commercially. used and in intend one just our safety additional this Europe delivery we of

those a were number with of The once two additional studies and as those so BLA reminder, We therapy safety on conduct one there schedule. plan we efficacy ataluren well, surgeries just mentioned already move Stu we've and two again, went with from already. forward no product. of evaluate FDA and with given submission. surgery, over that then align completed extensive years that a or complications of gene surgeries the will of recovery to accumulated the And actually and data the procedures safety to have the surgery, And is data are those collected these the are

Operator

with question from Securities. Karnauskas Robyn Truist next comes Our

UnidentifiedAnalyst

everyone, thanks, our taking for questions. for This Robyn. Hi, on [Nin]

data, just all are you on the question guess where going you whatever or And there. And back SAD maybe passages so data it's the the it when present first to around dose get the present be from just Huntington's will from cohorts study? that's cohorts the can few you I child enrolled. so has

StuartPeltz

what head first to past, in both, we're SAD that. the share both to obviously, a have the what going have for we've have deep and the we're Yes, so think studies data of diver I half were planning the said to within on track we what MAD year, is we the of and

So if level point would point, have on at year we -- the if when share time. that get We'll we data that. to get of the with where that we we SAD MAD by the the and both the to end that you dose

UnidentifiedAnalyst

that where guess dose where one Yes, be more idea then therapeutic may SAD in? that the sense that within as you may study? MAD therapeutic And then dose the and the have if guess or a may or fall where question question, well. I I And first, last one an and idea fall

StuartPeltz

again, good what would we're doing, we're of we of we're think, a be, indication the pretty defining in terms had -- the we on and on based what I dose. Yes, based that data thought

been response have both in shape, it's and the we'll moving the good dose to splicing, in forward, a where been, we've dose the measuring, think we'll terms so, of then curve be is. pretty be so And I able

picture a a we'll accurate gives able XX%, to we'll we to or to year. or move that, you know, half pretty by of dose HTT, both at able reduction that. a very of the and exposure this, forward level be as XX%, a good exposure, and first to have prediction we'll the of in between give half safety, So particular the that as you on based and to half able this think of, the of we're of then XX%, in I position the, the be and end dose, MAD the sense and going good of the by SAD a say first tell I of And to to the as pretty first terms went traction what leads well want the in be said be to

UnidentifiedAnalyst

know risks term be titration, greater I be not that guess able I more helpful. there's safety a then safety XX%? should my know do around And measure may you comments reduction, whether would sorry you there guess, Got it. enough, the than with guess, question on come XX% will That's your beyond longer to going regarding to any how have sense with that on I risks you push won't enough child

StuartPeltz

Well.

of now So as terms that, that is we are that I the people Leo for there that. findings I about think is people or of safety of data consequence what shooting we're are that a either of see right way one -- in who no -- of there's around plenty this think have

So I don't think that. terms a there's of going be risk in to

when a of genetic factors, if of you view, much much know can you like you isn't of is that. what going data well, that early We of level that, it's a you point how embryonic have at beyond data a them, clearly mean, that. the when look lot have low after tell development, you lower, really from of says After I most some there you're normally how say, you of during terms you isn't that but that the if there can do XX% things In I of pretty the important need? normal. go? lot

of we the effect? compounds other we're XX% and able see And where continually question is use virtually that to be brain, we're the down And do there our of at define animal of able that. reason to exposures be to models How are versus the where to every see reduce cerebellum test can the reduce humanize with And the every cortex could can would within in go include within achieve in think tissue titration don't we mouse do able with have where tissue so any HTT HTT, being of far capable that level the brain. able might some certainly studies ways down, we what we So really to thinking you XX% interest. will that want its and we that body, animal a in actually to human I some you level? and levels if be doing uniform XXX, that them

we we've So the work that know done. from

least that's actually to that at that that build And to do can would certainly I we'll we're think now. we we're XX% think molecule. groups suggest been I working that, some with to But not. so right able we to for the achieve. And be to shooting target be can obviously or able get -- if we've And be a data go able lower

Operator

question comes next with Beatty Our from Joel Citi.

JoelBeatty

Hi, thanks for the questions. taking

using clinical biomarkers ways assess the you're throughout First, are is important PTCXXX in to data body, generating knockdown the how it there for uniform assess or clinically? if

StuartPeltz

both done in levels Yes, reduction and seen is rat and show within blood extensive there the along other which I primates. think looking goes reduction the tissue clinically, with the reduction that that we're I choosing that mean, we've we've the biomarker we've to been types. hydrogen non the of at And mouse analysis able with human

So that's protein, so in to set, good a knows the and the pretty be just everyone biomarker, going right. that HTT obviously, is that's intracellular

where the that directly looking there. the blood mouse So models. we're good we're we've of other are marker cells looking, in seen level within We've seen tissues, all the analysis, when cells we for and in we the do reduction worked

that able So that reduction Does we we the correlation you, based be blood. do Joe? see the know, RNA to in protein levels. and what direct on we'll And help

JoelBeatty

remind then you And payment gears Could maybe for Evrysdi. what Great. switching Yes. milestones? additional us

StuartPeltz

What?

JoelBeatty

Milestone payments?

StuartPeltz

-- Emily, to give talk oh, But pass going today. you thought it I milestone, I'll yes, sure. were Emily. to Oh,

EmilyHill

for Thanks much. the appreciate I question. you Joel so it. Thank

are correct. So you

and all our milestones $XXX retained -- million milestones about royalty Evrysdi have largely. do with in of we is have remaining the after monetization, We for the

it's sale upcoming the milestone and first for would million regulatory, $XX shooting $XX be term, So in And Roche then about $XX in commercial $XX in near sales. million sale in based term the milestones for most the base commercial million upon total, EU; the potential in potentially real $XXX Japan. is sales XXXX. million million

Operator

with Brian of line the from comes Abrahams question next Our RBC.

BrianAbrahams

questions. on so program. Thanks my for Huntington's there. much A Hi, taking the few

brain what's you Given was those how two to of HTT about or guide are about guys move MAD protein versus cohorts looking into at in your wondering I known What's to mRNA thinking turnover, versus just you barrier And ratio? of decision you running the going whether think additional timeframe context the about the go three? And patients? changes? blood adjusting you then as might not of how study, Huntington's protein known when to as study, you do sort impact properties CSF plasma the how how past the into be to the look patients Thanks. in thinking overall might dose about

StuartPeltz

Brian. thanks, Yes,

we in reduction cells, So obviously, and HTT levels, in what will, primates. equal similar levels the the we're that that the see mRNA within -- within CSF blood when we'll that we think what we'll in that would see protein blood, in level that CSF to the non taking concentration also human drug done and we've the the about the we the be in in anticipated we're see

ratio confirm So we the just we'll that going to very be seen telling previously. think to, to we've able so that's be

think I to good, potential a that's we'll that. least So have at see

what that's the and in terms terms in that So what's what us going to would of of be. help exposure defining is

the you that point way and protein, could important level think actually And you and the I to life a PK able terms that's see in us so terms depends of the on the it good half up pretty RNA and you then bring lot when protein, of of, protein terms and PK, in RNA. of reduction equating of the RNA well. that's in really sure done the be But for to that to sure correct way a of be one as could look think to elsewhere be at that make the

proteins, has brain some that a the then do to we'll barrier. and and obviously state, steady they at multiple be the handle with in on both a to ascending so that -- model so in RNA HD, multiple we're and what to doses they blood reach And and doses, trying RNA in what able be HD way, pretty to happened have level, the the able and we ascending looking good is to leaky get a know

I to. think that's referring you're what

but So PK another think I we so model to into -- inpatient. we'll them consideration as take in that that something of have

BrianAbrahams

thanks so much. it, Got

StuartPeltz

I issues point, just efflux, for Yes, so making great. the of And issues other we the don't with any cause sort have was shouldn't that us.

Operator

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

VincentChen

on questions progress. you on congrats much couple for Huntington's more very you A disease. on Thank taking for the

events of experiments, your how RNA how Which is run the And get? modulated? how second question to study, the doses MAD a your how splicing much preclinical thinking were volunteers toxicities? that in the then were other risk PTCXXX? identified and RNA-seq is And first sense were target much what's for modulated off by ones needed assess is The ongoing about many the healthy do out many

StuartPeltz

I so -- more one think Yes, the first question you the could time? just say

VincentChen

preclinical they the In were modulating? and identified RNA-seq how many splicing PTCXXX? Sure. were RNA they were that much were experiments ones Which that by you did, other modulated how events

StuartPeltz

in little maybe general. so the it's about talking worth Yes, compounds a bit we optimize how

think point. I actually that's an important

looking selectivity way So for point do when And pretty is the us. optimization. important in for a the we

we food molecules, and optimize for property. the efficacy also small And screening potency, do and begin, in with high which we we and so those we pharmaceutical the screen the them throughput then fibroblasts them, in Huntington molecules selectivity when for

we start for much so But be out much less very the select select look to selectivity we can. that may is So molecules for as selective. building

than select, that, the of other, just end obviously show that And we seen a lot ability instance, specific a works contrast that the window understanding questions consequence sequences, and the understanding, than selective built competitor than you up, for the on a it's was and the these I SMA how where the mechanism, they molecule one the we a dive the is for the that. And and time that. new our deep molecule the the both that to consequence in molecule in about be versus really think, deep HTT, to I of and really the we And selectivity at able to different them, more SMA. really we'll RNA-seq, that the with, animal there for that's some substantially tell SMA do the within say specificity learn So alters the specificity build between that HD potency, so the time. build have in selectivity instance, that of time coming in, selective that and for can that one of for of spend in more a we've and in safety day, was models, found the selectivity of of asking, be dive do during to more molecules the we'll properties, can sight molecule strong spent you're that's discussing we've -- spending lot pretty the far the probably identified a the differences favorable in a that's the time in

VincentChen

doses do you off would of simply was in question the need? volunteers Actually second run I how sense your for that's of healthy out need much risk what's how expect many how If study how dose those you up to out much very show ascending would guess the the get? and disease do of helpful. slicing I run to quickly part multiple assess think the target toxicities? you And

StuartPeltz

day XX that by So XX days a followed period, doses obviously of observation. are for dosing the multiple ascending

unique don't that's molecule any different the the when so for the be target to some those other remember that's in time -- could toxicities, why us studies looking we or toxicities help specificity. folks and I for for I think -- do it the and whatever and could the I the what toxicities, spent all are we then target defining look safety but be off splicing of key splicing we're is, it and think toxicity don't is, about that's completing that also the And toxicity for selectivity off look reason that versus the doing the at make can dose, studies do why move toxicity these you or argument got the to will you of toxicities do identify to to to to and other to forward. the there's targets whether so terms able something And experiments, toxicities long-term lot to drugs be versus any it's about we be in experiments something able all function, -- community these but risks listening we we to toxicology that safety understand there's want just don't in other other a

and standard factors, is things. no people very a approach it than I stepwise splicing things in fossa other special is transcription this do. other think I that doing wouldn't make here try there's targeting the different versus kinases, other and cases, something that of

Operator

Our next with from William Prasad question comes Milan Raju Blair.

UnidentifiedAnalyst

the is of for delayed any syndrome, therapy, you question. said previously, your program ataxia because this Sammy taking my Hey, on update for Friedreich gene Thanks and on there's and therapy gene I believe COVID. if curious programs that I Raju. was Angelman were Angelman

the scenes trial using recent secondly, ataxia? FDA just And Friedreich Just wondering the therapies terms you for then the are will that you if the used same other the endpoints you. plan that's in how for for things guidance that thinking influencing if those still How guidance you receive CNS be case, do on behind trial on? of And designs? diseases? Thank gene do for any there's or going tuck-in

StuartPeltz

Sure.

talk dosing effect I about on that. have do Angelman really progressing there And on related But gave think out. the more on therapy of before so that and really programs And timelines that. syndrome. and push happened moving that delays end. think But on we're yes, don't this hard year past. So about the as I some, one program gene human talked there right. the in actually we we is working particular we'll FA pandemic during COVID Similarly, get you're the that we're are probably forward things We've hard we're the closer. the its been that pushing we're

we'll we as So over and do time this. talk through

want this? maybe, other color do to you so Matt, And any add to

MatthewKlein

Sure. the question, Thanks for Sammy.

neurodegenerative just and some very diseases, have therapy not for I about of -- there. think of It's quite in the our surprises have development FDA lot FDA I product and guidance over on a So program. device development using lot therapy. first, you wanted they program sure making talking think of a obviously the having need even also gene the device of to the And and the questions for the communicating consistency obviously tackle groups, standard the recent guidance, guidance sort therapies time works about comparability throughout and your read frankly, that they're rigorous to spend Friedreich control importance the ataxia I maybe device gene of

all where which the the is that So experience results broken function. dopaminergic therapy products, the And from precisely very pathology development. gene and one key general key from, therapies. component thing our gene our which key delivering gene we're is the think to is we're ataxia other delivering ataxia program, different of Obviously, way development, cerebellum the approach and is we're the the the of we're the In obviously to that one CNS that taking area a component a important tailoring, I a drug in programs, disease. neuron disease in deliver having our Friedreich cerebellum. specific the become for to a of therapy is that, all one we're learned CNS is therapy second, obviously Friedreich our ataxia, therapy the targeted gene really really that really anatomic few benefits to with AADC not with areas able gene product so then of example, which we with specific things around along and It's things the familiar quite guidance

obviously the also to which have only by And it doses, your regarding not think vectors. a so risk developing now we're for gene the therapy and excited And this obviously actually allows us it gene therapy, lowers exposure targeted program, Switching to lower to ataxia. Friedreich ataxia look, doing Friedreich to manufacturing be I questions two therapies incredibly to be burden. lowers able

gene is as and [Indiscernible] CNS We has targeted in which broad as have our program, systems, organ therapy our dentate small the well nucleus. distribution molecule which delivering other vatiquinone, oral

a these complementary for example, as deal attacks. a from ataxia nucleus, mortality if disease great heart whole while brain really pathology think, approaches, source I Friedreich of a risk a body has are and the is it's resulting morbidity think significant dentate whole the it I diseases also disease, and of

to our complimentary we And complimentary. a of is So we're in incredibly able approach offer excited both view be to these therapies way.

In terms of selection. the development itself and endpoint

of I the established the was community as daily great a needing that is as of ataxia around history, activities the working rigorous very the scale. advantages Friedreich think history, natural rating scale particularly well by as community natural disease of well living as developed one the the well at

in long-term therapy gene on natural therapy the there's the human so studies, therapy obviously, that about effect important of But have on the biomarkers, think disease significant endpoints of to development we're to therapy disease we'll we looking appreciate be endpoints other impact ability obviously, certainly both imaging that those to trajectory. as really be gene of gene So in also a our disease. other significant know captured the gene can where we the history, to biochemical program, are we trajectory first a our to clinical exploring mediators, also modalities, and

Operator

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

UnidentifiedAnalyst

Gupta] Hi, you taking [Neil Danielle, from guys. for the is question. thank This on

How affected HTT of models identify? a was the for that had XXX the reduction you other given And XXX. Or by dose? nucleotide factors question the on any uniform if piece? the reduction animal length On could in

StuartPeltz

for Thanks the question. sure. Yes,

actually, the beauty fact that that's So I thing achieves one really think that is the PTCXXX that it uniform really pushes exposure.

XX% cerebellum. when the the we'll a reduction within So striatum, in animal see in particular, see, in the in for a blood, analyzed, we and and in reduction instance, models, cortex, XX% reduction HTT tissues in look cells saw lower we brain, XX% we if

all So ratio brain. as well. in saw within within a as uniform. And I good, true we said; that's really, the blood we right. a one it's And that's lowering amazingly had what tissues of that's in demonstration of But to terms one the

but to be, Huntington look, have out disease know, brain So Huntington's that see that a turns cortex, I because, and when whole and you disease. actually important, as about striatum people talked think think, I I you very

very see on of mRNA and that, dose based that our brain a the we'll cortex reduction emphasize important the it's HTT, within results, cells all protein to dependent striatum, that preclinical in includes So and cerebellum.

on have see determine the that ability we as for again, well. gets that was this that And true to I tissues that reduction and the be it's everywhere. since other the we I we measure SMA, well true I so occurs this is same think that here could as then in really point critical in in the blood, blood the end, that And they're can exposure, we going based -- as ratio to is it think within think,

UnidentifiedAnalyst

Right, thank you, that's helpful.

the and any in variation that gene of terms repeat on In HTT like the within HTT itself effect the HTT. reduction? Does the nucleotide have

StuartPeltz

No.

UnidentifiedAnalyst

-- this Given

StuartPeltz

And again, that degradation the the molecule, termination because it CAG that's -- in gets a leads does the intron, that to, in, there's mRNA. it's -- the the of that's the and rapid consequence as normally repeating that. of of a RNA that's doesn't is spliced is piece working way different of Yes, location. into doesn't of within axons, this but get intron pseudo that a the spliced in within near matter, It's premature an RNA the get not the

that. it's this; that time the then this that in are types. be symptoms, the before for And they take. good So symptoms, because looking advantage do that be have no it's with that that for so all it to can be you imagine to we'll over you anticipate the over patients, manifesting easy in here would we'll therefore, and those can because be able well, only there available, patient issue long for patients will of run is time, see that's is that beauty we obviously buy And would initially

that's I So think important.

so So is axon question HTT expansion. of occurs the degree the pseudo at incorporation of regardless the that the end day, of

Operator

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

JosephThome

question. for thank taking Hi, you my there,

Can amenable make of you after PTCXXX potential differences Parkinson's with curious are XXX as what when disease sees advancement indication? to healthy target LO? see Thanks. XXX us, data platform, -- an VBA, a these any the the to both looking Bio-e kind the can of on what tell more that you and study? -- XX one for? that maybe Just we And volunteer into of you And kind are then Parkinson's

StuartPeltz

sure. Yes,

it's have And number where So terms people, GPA, taking of we're I advantage think be a part a different now. for molecule. something that's versus obviously, a long of that going what that does of for probably what we're doing, an lot it would bioavailable different like term again, in properties. for of orally it's and populations different, an said, Parkinson, vatiquinone indications, patient it's a in you And

perhaps molecule different to properties. it'd we with So different have be best thought a

sort the population. different the value of the it And has size patient in of a sense so for that's

And so molecule. Matt, trial a about? we you have of thought what where of it the do was in important we're to want bit thinking talk little another and terms to at we're

MatthewKlein

Yes.

the for So, Joseph, question. thanks

we understanding what's dose gives has So the we've focus Phase been where also exposures to behaving humans for making what as saw molecule efficacious the ascending the able the that completed the multiple is single we're sure all is in preclinical -- and in we've X it's analysis. And been preclinical really studies which work that we're study, the the in you standard, model volunteer the doing in the in studies, pharmacology healthy done. the really identifying the in studies. as that fairly we've be studies, and we and ascending and XXX And mentioned, been that pharmacology, here of dosing looking safety us studying dose process to was that the level those

lipoxygenase, we study know with as we is would bit necessary activation, that exposure level want the safe So of liquid background, you've and delivering pathways, is key have has Parkinson's microglial which seen be alpha synuclein, be important of achieve pharmacology in dose now and a walk the to as a the target to consistent base effects a preclinical that's number predictable oxidation stress. to XXX away which disease with XX this that such governor oxidative pathologies, [Indiscernible] of from and mutation, known independently

to all do by simultaneously, effect we're what XX so able so of below, charging those pathways. And is four

level that see a then what side stage brings do an exposure we the and move with studies want with to So the demonstrating the work, we'd in be that extensive of seen have a in to development. Phase position that we've X up what forward effect. identifying dose amount we again, of And preclinical in matches is next study preclinical

StuartPeltz

Matt. Thanks, Yes.

can really extra because right, oxidative the as electrons XXX. stress, think are I important really well there that into here is oxidative diseases go that we point stress, multiple

having to go valuable. So With think what's different indications. going another other we molecule to into be properties,

Operator

to closing Thank questions. Peltz turn call you. further I any I'm Stuart not the over for will showing now remarks.

Stuart Peltz

sets and many this many to you Thanks And for we for that us today. strong may and of value thanks. as aware, in upcoming I Rare we've hope us just where stock Days [Indiscernible]has we in as a a a Well, reminder of for reporting XXXX, this been serves work, this where company. where Disease be creating been we're lot oftentimes patients, as and joining our and of the up how the is do And how Sunday, going this really XXXX. ultimately, see for take milestones us you that benefits it

with interest question, good the particular, look Again, to to in the evening. X read look programs So and we have, thank that forward to, a we're out, second forward interested excited about everyone's the and us. in Huntington's you that the sharing for the disease Phase know we I you by been joining quarter. we within see Have

Operator

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

now may You disconnect.

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