Earnings call transcript Q4 2022

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Operator

Greetings, and welcome to the Taysha Gene Therapies Fourth Quarter and Full-Year 2022 Earnings Call. At this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded.

It is now my pleasure to introduce your host, Hayleigh Collins, Director and Head of Corporate Communications. Ms. you, Thank Collins.

begin. You may

Hayleigh Collins

Head the Good this with corporate rare and afternoon, conference Taysha's team you. Relations Director with welcome I'm to also I excited be to overseeing I'm Jaguar Gene experience diseases having and XXXX financial Therapy. in quarter potentially Investor and appointed forward full-year recently bring update previously help rare Ever Collins, medical look served fourth Taysha Corporate to in pivotal results Communications. at life-changing Hayleigh will in Thank a activities. working Taysha's to of patients and communications and call. with similar high role at to needs. time join therapies unmet diseases corporate

financial full-year is through quarter the Earlier today, results website the Taysha for XXXX. press release copy issued and of available this on SEC filings. and Company's announcing fourth our a A release press

are President Financial R&D Taysha's on Nolan, CEO, me Sean Nagendran, Officer. Kamran today's and Sukumar Joining of call Alam, Head and Chief

will our a prepared remarks. question-and-answer We session following hold

actual Taysha's differ runway of forward-looking as information. also those growth, TSHA-XXX. expected or on to of well to and This future facts product cash today's relating are opportunity property, alliances statements making product statements trials statements, potential be results, to the forward-looking those call, and cause TSHA-XXX risks that and TSHA-XXX will implied may not candidates include forecasted results development clinical from intellectual of the call may market we matters for of discovery statements strategic commercial such of and Taysha's statements. that plans other Various data may or candidates, and therapeutic existing including and timing clinical results the regulatory contain and relating note forward-looking for materially stated our These operating in clinical and Please historical and and programs. as

regulatory results of, controlled ability obtain limitations interactions parties our activities. candidates, and research to third timing conduct and substantial requirements clinical dependence uncertainties patent to for risks of alliances our of or and funding upon our the and product for include related relationships, to owned strategic by discoveries, trials patents protection and our These our development third-party the by imposed other

For the a list and ended the December and on description the see and have that Exchange face, the that for please Annual Form Commission filed our risks uncertainties XXXX. we including of Securities we reports XX-K with Report XX, year

broadcast, is required undertakes update events time-sensitive statements call only conference no this as XX, XXXX. date of as except of of to accurate may that be Taysha obligation call, revise live this the circumstances contains by after to This March securities information or or conference reflect the applicable law. date forward-looking any

our to I Nolan. CEO, to call would that, now over the With turn like Sean

Sean Nolan

our everyone Then you, Kamran Alam, remarks of R&D provide our Today, Thank call. update Officer. providing the Chief financial followed quarter then from call questions. fourth our for conference XXXX. programs XXXX a on Nagendran, update Head results Taysha development of for closing and a by an and welcome, update Financial opening I begin corporate Hayleigh, provide up will I clinical will outlook full-year corporate brief and financial to President Suku and will before

effect. and actions a lead clinical The taken year in positive our progress programs earlier having and two GAN this are with improve syndrome execution to expedite Rett

ongoing and on REVEAL further our to both For we track submissions this details adult execute study. data Phase will timelines Rett here year available remain our safety in and Suku II I/ syndrome, regulatory provide for initial TSHA-XXX across shortly.

from recently Type the constructive to our B II currently approved feedback no with received We disease the meeting. ultra-rare end-of-Phase follow-up an GAN, in regarding TSHA-XXX FDA For treatments. questions

trial interventional data findings further that the completing history new this discuss share Suku there preliminary and path detail are and compelling forward. assessments. to analysis some comprehensive appears a the in further we will electrophysiological review of to discuss believe and biological We Again, are encouraging a potential regulatory the we including intend from functional, natural The shortly. with ongoing FDA

remain In patients the syndrome bringing new milestones on year achieving neurodegenerative GAN our Rett ahead, devastating and mission the treatments these we to and focused work towards our in near-term anticipated programs continue diseases. transformational to of with

syndrome will and to turn provide Suku? in-depth of our now Suku call over a to GAN the I more discussion programs. Rett

Sukumar Nagendran

and you, everyone. afternoon, Sean, good Thank

First, start gene therapy TSHA-XXX, on of program syndrome. the will with our I Rett an for update treatment

Element treatment miRARE of the to TSHA-XXX MECPX miRNA-Responsive syndrome. Rett expression an reminder, innovative or utilizes regulate of platform cellular designed a Auto-Regulatory the As for

orphan TSHA-XXX been adult from screening granted and the orphan Phase Commission. and the patients rare Rett I/II has in has disease for In the from designation designations our FDA received syndrome, trial drug REVEAL drug we and potential European available initiated the pediatric initial patient provide we recently with report data, on the half in remain for clinical track update year. XXXX anticipate to to We dosing the available on on plan thereafter. safety, TSHA-XXX data first and in of quarterly the first primarily clinical half first patient of first

will Rett submitted protocol as the second to the REVEAL included further of expedite year, our to continue amendment in Importantly, allow be a syndrome trial. to intend young in the with which patients patients old study, years we half enrollment. dosing recently as we XX we believe In

For submit in UK pediatric TSHA-XXX Rett mid-XXXX. in syndrome, a for to with MHRA plan CTA our the study patients to we

submission the an IND in FDA the of application U.S. We year. also have planned half second to

turn neurodegenerative has disease drug designation pediatric TSHA-XXX no let's which or pathway. drug for been has treatments is orphan TSHA-XXX European granted Now the regulatory established orphan FDA and and the Commission. ultra-rare the from rare with received treatment GAN, designations of approved from to to reiterate indication an

regards submitted to CMC comparability the commercial In analysis. manufacturing amendment product to process and recently submission detailing manufacturing, a module FDA we our X drug

B meeting, primary scale formal II at minutes. mentioned, Sean the in clarified the B such the also challenge in the FDA due executing Type a The received a the also controlled nature endpoint study meeting ultra-rare questions II enrolling design FDA primary Type as As end-of-Phase end-of-Phase only we Taysha's follow-up discussed blind GAN. our efficacy trial, FDA to of to to from feedback response MFMXX, of acknowledging and double setting relevant in randomized the while a

to self-evident a the alternative regulatory in FDA is utilizing is trial such, objective meaningful. that effect consider is large and As controlled designs a open study demonstrate treatment to flexibility setting measurements and to clinically willing relatively

plans data of We the the a and interventional review including and assessments, FDA. our ongoing completing future interactions functional, electrophysiological are comprehensive trial with inform history natural for will which biological from

progressive is a observed ambulation common that limb and ataxia, ongoing to clinical second The of decade. in loss gait and patients with assessments analysis manifestation MFMXX the include as GAN functional of leads by Ataxia often

functional analyze of nerve that aspects and GAN Additionally, and structural nerve given we degeneration the experience deterioration patients over optic optic and continue retina acuity time. of to visual

including layer effect images several and electrophysiological assessments thickness, nerve activation there nerve is spine that skin to nerve brain relatively a objective clinically self-evident large conducting and cell is also potential, are and whether to biological muscle meaningful. and responses and treatment MRI We ganglion nerve sensory biopsies, retinal action fiber and determine

TSHA-XXX to regulatory treatments agency the further of continue plan second patients to regulatory approved established the the registrational ultra-rare to regarding this reiterate or no with potential in quarter discuss dialogue who pathway. potential meeting forward a intend pathway submit We XXXX to FDA the We bring with the path to request have disease. a collaborative for GAN formal

I to call to will financials. over discuss the now turn Kamran Kamran?

Kamran Alam

fees. million were Thank were $XX.X development XX, XXXX. for the for XXXX, of $XXX.X expenses $XX.X the development attributable was Research $XX.X XXXX. XX, three manufacturing million XX, ended ended a XXXX, other material December million in decrease months decrease million compared and million $XX.X months compared for $XX.X million to you, a and in ended full-year December December XX, research to decrease and development December Suku. expenses to and ended raw the full-year the million $X purchases for primarily three The and Research license

non-clinical expense by December in research decrease a and were the decrease development to ended also to a for third-party the mainly XX, XXXX related million expenses studies attributable million compensation toxicology of decrease a development expenses $X.X expense of The of development result December research for and offset was trial million and ended XXXX. $XX severance and in $X.X in XXXX XX, headcount. fees, $X.X and in year million as clinical Overall, higher research lower higher and partially lower expenses year

headcount and and administrative million were to three expenses expenses December $X.X three General XXXX, $XX.X ended for December XX, General the million XX, December administrative compared million months $X.X consulting reduced were $XX.X compared in year $X professional approximately expenses for The million XXXX. XXXX, ended $XX.X XX, for ended $X.X million ended the for months the were compensation the to million was driven by Lower of expenses offset XX, lower of XXXX. severance by year and primarily million general of and expense. partially fees lower administrative December XXXX. attributable to decrease

loss three Net December for share, XX, to months three million, XXXX ended compared or loss per December as XX, for of $X.XX $X.XX share, XXXX, a Carolina manufacturing per net $XX.X million, in Carolina the non-recurring the a looking non-cash, we was or XXXX. $XX.X ended of recorded charge the impairment North for for In actively we are the facility. process North buyers Currently, facility. November million manufacturing related $XX.X the to months

full-year full-year recognized compared million, the revenue per for of Astellas Net offset months as share, for to XXXX. of the three for XX, or to $X.XX loss by ended share, net XXXX per the December Transactions. the XXXX million $XXX December a ended XX, was or net loss XX, loss The was December million partially $X.X related $X.XX ended $XXX.X

million resources XXXX, the of expenses requirements that $XX.X capital XX, Taysha support in continues and operating planned its December equivalents. of As to cash current cash cash quarter company first had XXXX. expect into and will The

return his Sean? will closing the remarks. Sean to I now back for over call

Sean Nolan

am few study potential of progress during screening Thank amendment our pleased initiating programs with of first the should lead the have in further patient with the and first Rett including you, submitting adult enrollment. the that XXXX, two patient Kamran. made we protocol I expedite a months

the we constructive to in the the regulatory formal meeting support assessment from feedback potential forward. request the received FDA a For data are further analysis second by GAN, discuss preliminary quarter the encouraged the and to of comprehensive path

Our across for syndrome XXXX. look and Rett focus milestones execution delivering throughout our the throughout updates further programs. and GAN will forward providing to planned this year on We be

operator our to the I begin ask that, Q&A will Operator? session. now With

Operator

you. Thank

be you. Wells Zhu now a your question. Fargo will proceed Thank from is We Please And with session. Securities. [Operator question question-and-answer our conducting first Yanan with Instructions]

Unidentified Analyst

our for Yanan. question. is Thanks for taking [Quan] Hi. on This

you. your now study? is for And DAYBUE five would syndrome question that design? to count do how is for include program, syndrome approved particularly XX, study my UK that given into affect the So Rett DAYBUE to acadia's trial you on Thank in so your the clinical patients Rett aged enrollment, need

Sean Nolan

question. planning the that. for much We've [indiscernible] very provide perspective our some you like to to thought I'd ask Thank Suku in and on Suku?

Sukumar Nagendran

I of could Yes. potentially think, having the which from for at it Thanks, further a also federal increase newborn of that This and an getting approved, approved discussion state I is and the disease question Rett, would of very the I number is available state level the patients amongst in, also disease incident increases patient number asked is by screening, a I could communities, population. when state to which and disease that important the comes identify the awareness patients terrible the important you physician patients for the What standpoint. and some like serious increase prevalent an think, whole result Sean. because accessible also it think product trofinetide

be I'm real are but what who interventional is disease also plus sponsors could trials. for the community, collectively doing a whole us like So this saying for

have will that and are naive TSHA-XXX, where trofinetide, and Now like treat – an patients the the question treat disease, approved to is clinical action we product of and product different which optimize impact that your would treatment healthcare this can once and such addresses root other obviously the like ideally cause the progression significant about have our of we our product very that this the you whether address from mechanism of to – patient regulatory disease authorities FDA disease. on the show communities who providers

in and we we Now regulators that well, which may the would to caveat a product, that there is is further the a product is designs take basis. there have approved for as hope, evaluation possibility be team anything something to a that you'd you have combination this add? is Sean, arm future. on is case-by-case my considering else the it happy But and has with protocol if as is discuss like an but already ask, the to the always

Sean Nolan

is to is, opportunity some profile, on add take thing there's adverse for given be able the I other the that may patients only The and not of that, it. product the would the drug half event life stay of

could Zolgensma call So those with or of, that access. addition non-treated patients taking you've potentially a be study we got from pool you even either is wait of And wash to that it, would medicines. patients the other SPINRAZA, experienced with willing out

keep U.S. that consideration We've we'll eye UK. and it our able and So trial certainly we Canada into clinical of on given scenario to active impact including an deal trofinetide other geographies patients enroll great having. in is we'll planning remain confident that the in the be a designs and for the

So thank you.

Sukumar Nagendran

exact symptoms to to more label, but rates I have point. the the that add the I've numbers, are discontinuation forgotten high can GI the trials due up. if if look trofinetide Sean, may one in And at Also, look and you you very

don't was of think trials. even XX% the some anywhere I XX% it from, in know, to I

on So have Rett patients that even significant is this with for being more impact available may though product syndrome. available patients

Unidentified Analyst

Got it. Very clear. Thank you.

Operator

you. Thank

next Whitney Our is question with with Ijem your question. Please proceed Canaccord. from

Whitney Ijem

Thanks the question. taking for

all So looking endpoints, just biomarker, are along things And selected that additional you are are those things curious, those functionally way dialogue cetera is, on, of at. been focused be study? FDA, you're for of look use kind or I'm a or lot goal you that listed hadn't out with GAN, support that of and back And the two the analyzed calling data use at to patients that et in were or to one my of the to another now natural that potentially kind part history? then to to continue there data design will comparing filing question is the

Sean Nolan

hand in is first existing a to This question. of to the take that's that, first free part Suku, answer I'll wanted and opine. So the of feel your data database. I

meaningful patients many and years history before And done. some they there has there's in been the – assessments were are have what treated, time We've treated. a XX effect that of that objective further on been course through that remember of these to the there and So were data the systematically is if demonstrate and the points doing and going fully or data patients augment multitude many, Suku’s comprehensively see been patients of all all is, GAN. are suffering that can study metrics over to it visits additional these the natural and assess got can treatment clinically cases effect and from that of measurement team then the are

question, Suku. the approval that very on I'll forward this answer after think formal seek put forward. this a request meeting will data we second to what existing the with appropriate to the we is idea our we have view that fully pathway And could second – and analyzed be be – about quarter to dialogue And turn the data. trying well FDA it would this submit The quarter with to would your over we

data it's would haven't we've far determination clarify or said? what we're up. and yet, line starting you thus made final the gathered we encouraged by Suku, but how add that I what a to So to

Sukumar Nagendran

Thank you. Yes, Sean.

as available highlighted, an patient ultra-rare very no more with age. So were population, general, of treatment, the years you they in patients than disease are it's six treated and – small

it it's us talked give disease in functional, other endpoints. a we FDA given a it also to request And efficacy these at think that a is comes work, data the an in meeting modeling we the many second Sean that and observed for information the some we've enough to lives, path But and what dataset and we out, back patient's in go the we've as this should year there no pointed were dataset doing is a really make especially that NIH could to that best of ultra-rare think what the difference it's looking quarter that as with further treatments comprehensive on comes biological available. forward when analysis discussion all disease designed, in previously slowing electrophysiological to protocol about of are when we're and So MFMXX. endpoints

as for to hopefully and optimistic hopefully cautiously data with the FDA move the program NIH, forward. the question. the and collect So go we Thanks then all this work

Whitney Ijem

Thank you.

Operator

you. Thank

next question. with Our proceed Jack from Please with Baird. your Allen question is

Jack Allen

to option Great. are the And for here. meeting could love there. sound Thank GAN ex-U.S. is as about I'm the you medical the with you approvals have like are what's meeting optimistic on stick insights to the I to about taking Marks who It any accelerated you specifically wondering thinking it I'd so based for that request the how quite question. FDA? much where if sit with turnaround at? speaking to and Peter you an been meeting recent opportunity? things update going get And then for Astellas was relates the as at you GAN, does time provide a

Sean Nolan

Yes. I'll Suku, at take and please stab jump in. a this,

to have everything with I history this answer who Marks sure bill to ultra And based there, feel understand we fits program, in obviously that compelling we data. Dallas. terms well comments about a possible itself a to of Peter both deal made is was to we tremendously. is, he obviously a mean, lot one we're to that lend There make to We may the the The be about has that And your relative the some one. make is make MDA of is from premature that I the as flexibility good interventional can the headquartered first, point case. definitively going put we say. think to was for of question the it's about that. orphan not how your diseases. Conference FDA as do like also Dallas. in We'll package buzz together that natural And and to the last on but in statement us we could

over, over now we're with watching it'll if of to get many There's other new the And taking And the might on time. taking some will additional course – the Witten happen. you outlined in like, all his flexibility what questions Jack public kind Jack? line… of leadership remarks just, do see there Peter's of two as want hopefully, you we and from mind Celia Suku, to can you're on

Sukumar Nagendran

of lot asked. the is Jack, repeat you that can question you a there because question

Jack Allen

and much, had other one. Astellas it does I relates you could fit surrounding how Where so maybe the or I following much. on think? Thanks if you're questions But where Yes. that that in to were so the GAN? to option. touch I And limited and then we're know potentially still the path thinking apologize. as does just forward about would for Europe, I ex-U.S. Thanks GAN fit

Sukumar Nagendran

you that or me take like to you do that? want Sean, would to take

Sean Nolan

I'll Yes, take it.

now the have the not resources on And want we we're ex-U.S. not U.S. Jack, of all the are so On piece, now interactions on efforts, right had the right with our anyone energy overseas we focus U.S., focused additional on – since discussions. and we to initial

Astellas that or that, would to it where is, But to whether really they relates a that. option not we're we As in option detail is the the say time too I want and evaluate Astellas haven't to opt-in. the has still about what window provided much

we're have. I have this likely it make stands to that they decision meeting we planning reason a FDA think upcoming to after that would

mid-ish we the decision the not submit they actual would And for to make want that year fulsome to but time around more again, planning have or we're can't quarter. whether I is second a opt-in information have speak say that meeting request meeting. to I the on on program. the Hopefully, they Astellas, when So would in

active So it's a very still opt-in.

Sukumar Nagendran

more question the on Jack, times. turnaround one have And regulatory you

request, think the I that'll depends it B, type of right? turnaround the or Whether that's we'll have D. C, we the know, it's you decision A, As and meeting times. a Type make, on to impact And

FDA, days anywhere XX the the type so… on XX know, depending Some to XX from or of requested could as you meeting be from days

Jack Allen

Thank Great. the you so much. you questions. taking I appreciate

Sukumar Nagendran

Thanks. Yes.

Operator

Thank you.

with proceed Gil question next & question. Our Please with Needham is Company. your from Blum

Unidentified Analyst

our Hi. market the about TSHA-XXX, ex-U.S. questions. Can partnering and Rohit you be This program? would talk for for to on Gil. open just Thanks. is taking you Thanks for the

Sean Nolan

when and about XX,XXX right? say Well, the is I the U.S. in the significant, that talk combined. EU think opportunity factors, patients I we there's approximately would

of are So There's there the significant. treatment identified. infrastructure the there is study patients clinical opportunity good sites, over terms centers, well over in

encouraged into one the So opportunity It's of submitting clinical the we're the there. focused our by reasons UK. in efforts mid-year over we've CTA that

of aspect, that to trying would consider. of Well, part we that say something question. think second potentially that's Just I partnering the would the the

or have to look always determination and as the I it you funding we is at it keep now, partner plan for current make outlined the your to think otherwise, wise a all situation I to should that better trial the and have the resource capital to to options, better on Right and yourself. it, say. are plans this we've execute us trial, year to

So should pursue option decide we have, that. that it's to an we

Unidentified Analyst

you. Thank

Operator

Thank you.

Our with question. next Securities. question Please your Silvan JMP is from with Tuerkcan proceed

Silvan Tuerkcan

for taking Thanks questions. Thank you. Yes. my

to the mean, they think Just a attempted words. that's last just don't exactly effect primary trial. to wanted I controlled GAN, and back those modest But endpoint remember obviously time the wording, maybe the you are why the come on a I or FDA I

that this they're efficacy with with will how that Could do data to now measure you these know strong the we stack you do them you how play together imagine here additional bar outcome a hand open you. strong a to met so or show out? And endpoints. Thank the have at

Sean Nolan

that was MFMXX meeting of company. would the was all FDA on that discussion that's the said say focused meeting the of majority I the endpoint, happened. where the minutes, Well, the was initial the focus because first the of primary That what minutes very at

generally randomized a believe you it's of they need blind endpoint said, double that where were particular all comments trial. so basically have to their subjective, they around controlled And because

control say that trial have essentially clinically then willing evaluate to they're designs their MFMXX, them that we and when that the alternative they a we questions, that said review they went know, but that restated I objective. meaningful are new endpoints to natural is and back asked controlled as So on and you history that can the be would news subsequent are

And sense upon so that us wise what a determine to do think so call we I – address that open what meaningful was would a aspects, objective. a, us that makes is lot those to a is door the that justify there new incumbent data two opportunity what is would door it it's that is and clinically and of

so over that, And Suku and of why at comprehensiveness to just to Suku? we're I'll it you give we're with the and turn looking that a flavor of totality the encouraged. data

Sukumar Nagendran

Sean. Thanks, Yes.

So important your question is an one.

disease, start it there So saying about I FDAs and no earlier comes Marks when let again, by ultra-rare somebody position treatment available. me is think to [CNS] asked the Peter where

So have and important on some disease those GAN at we'll sponsor MDA that's or very hopefully dealing impact with I remarks the an were ultra-rare strategic any think otherwise.

to is, disease the approval just acceptable pointed considered There for our in really which allows Now, detailed not our medicine that or make broad of can in other fashion it's patient make a multiple even sponsor community. here approval a the large the in and available the are MFMXX. accelerated case potential patients most behalf that in as this such will very is to is a question moderate our that big intrathecal could as or a be efficacy gene it's an way as actually impact, Sean us ultra-rare of we collectively endpoints. analysis, significant, question show clinical some out, clinically or in-depth we whether continue And therapy

Sean of at the make the case collective for it's with pointed of FDA as and us the eventually case quarter. that in I for for data to And the meeting FDA the could birth therapy the for out, further submission make second the this a review think

to hope other that There and I other the I give GAN, impact. understand think are experts product what that multiple case trying presenting not strength may review disease are of and what just It's observed we've the I'm product. I our is of endpoints you help So explain. that collective the this have may with think And that the ultra-rare approval potential to called that MFMXX. clinical NIH

I'm community. crossed, you, can Thank patient you. that's and take we So what for my fingers this keeping do

Silvan Tuerkcan

very Great. much. Thank you

Operator

Thank you.

Our Zhong with next question Please with is question. from proceed BTIG. your Yun

Yun Zhong

question. you much for the Hi. Thank very taking

syndrome So the program. my question on Rett is

And so as years the in as young XX change treat age the patient patient to old.

that of data? helping you patient achieve terms be enrollment would treatment in like to to you and have goals Would outcome addition initial provide safety any or analysis be able do to maybe, will information? that other the In you any additional only

Sean Nolan

to Suku, take question, please? would you like this

Sukumar Nagendran

Sean, Yes, Absolutely. will. I

is another question. very this So important

a may allow a we XX, to impact and which there that data this clinical updated team other and disorder have that pre-clinical us patients, Canada So this, of group submit because we think have to my greater us very was age by reason to in do as to supports data and doing amendment neurodevelopmental important over sponsor, for it enables us Health obviously time. decided this the treat version we dropping a younger to

endpoints a also we by access much on patient hopefully in that timeframe? show only is, could dropping age and that more safety So certain given efficacy table safety, we have measures patients, larger now but it's also evaluating the the a) the in question greater and a not having to clinical will shorter impact pool, goes our beyond

drop have or progressive the any So age, XX, to more have could have is, you adult treat versus once product question the age disease efficacy greater longer clinical you for it take more you disease much will the much over impact. impact mature patients when of who

even know, efficacy my clinical So impact? the is on The we'll greater big also though that root the our the as data. a terrible that gene And cause table for will question is safety treating dataset a we disease. we accumulate is hope, us is the initial of strength to also Rett which which will syndrome, by as of clinician therapy over time be see, you will show intervention the what will our show hope allow disease

your that that hope and I So hope, question. our answers is

Yun Zhong

Okay. Great. you. Thank

Operator

with your proceed from question. next Our David Hoang Please Instructions] question SMBC. [Operator is with

David Hoang

with approval considering why Thanks not? question. and had you not, for formally endpoints – reviewing? pathway you available or are Have why if the you now data think would so one the just that and are pursue? update the question you consider to if Do I there? Is accelerated my taking that GAN. given be that And path something

Sean Nolan

you question? take would Suku, please that

Sukumar Nagendran

Yes, I've you in regulatory discussions, give opinion only the my that Sean. based on right. I overall data our can seen

in we treatment disease, ultra-rare consider for is if available, would But at our absolutely, the our no is meeting therapy of we can with for we if can option FDA. difference approval product see the a caveat the truly I ask right. dataset, impact while what the broad available product love making obviously, to gene So to this, our do believe the clinically then product truly to is is ask and look make the And for such FDA medicine there's accelerated patients. absolutely that this our meaningful,

very accelerated make with gene to us have therapy truly to the on approval. this that case this So clear have FDA I be is a dataset qualifies to we convincing collective for enables a that have

complete for plan that So we our are when what that is request full is once okay. our hoping and FDA review we analysis, we an do the submit for,

to and I helps, more that hope anything said? wish what if know to you don't add Sean, I I So

Sean Nolan

on we assessment, accelerated Peter you of I what disease the presentation make we could has Look, want to It fits publicly, we're aggressive data we the and times based most said the add preliminary prejudge to about product think on the four and would particular the together given to the and don't We of package that soon I that Yes. some and say only of the this bill. when down about GAN we rule. don't patients, can ourselves that certainly will Dr. state as to about would said ahead want an to just you time. based in certainly multiple merit think can final is and remarks there's nothing to Mark thing from at all, get get put that available as point possible, that encouraged a product data.

and question. compelling that. for you is if So would bar, the push Thanks meets for the data the hard we're something convinced like

Operator

you. Thank

your Truist Our is Lee from with question. next proceed question Securities. Please Joon with

Joon Lee

Thanks flexibility or to placebo the the FDA “controlled a necessarily could is trial other does setting? what And it's trial include say can the When Hi. your in update. that some that controlled to regulatory that be? open you controlled release setting it in for refer press latter, settings”,

Sean Nolan

the Suku, Well, would there's you But look opine. consideration a example, as approval, I'll trial. natural of history. and one of accelerated an be you that generally my at please look that FDA and give a And trial you controlled to guidance designations controlled at answer is when

from controlled placebo bar talked were they talking met alternate about trial. about meaningful and the were clinically end a double MFMXX, controlled between a they the got FDA and I a there's blind controlled we trial, about about they were talking trial of trial points, that And controlled blind think are So language responses talking on that distinction they When the the written in a a double placebo those. trial. when objective clear designs

letter So I think of I guidance, further you Suku, to but clarification ask the would on that. for speaking they're the

Sukumar Nagendran

Thanks, Sean. Yes.

trial, ultra-rare could doing controlled an placebo disease, be it think forever. it's that practical a don't I and will given So take

simplest discussed, where are necessary be already history there for if dose few more you comparison. have the pre-treatment would design, have or a alternative to be to So natural patients study

that qualify the for the that's the forward potential to submit caveat, the hope the is a path for will we So dataset our collective FDA appropriate approval that thinks patient best and community. with review if FDA serious that way

it leave question at me any the is that that. wish was So there Sean, asked. address? question we addressed would I hopefully, other to So And you

Sean Nolan

think I you did. No,

Sukumar Nagendran

Okay. Thank you.

Joon Lee

Thanks.

Operator

to Sean for are no like floor Nolan over closing at back the I to questions turn this comments. would further There time.

Sean Nolan

a we're you, the interest their for in period trying working short accomplish long updates good Taysha. I'm Thank way you and in to have time everyone go, months. to to that what look we the pleased Thank and a we've got coming you made are and forward night. in at hard, sharing XXXX, the with we've all progress future very here we time in

Operator

today's teleconference. concludes This

lines your your this you at for You participation. Thank may disconnect time.

Taysha Gene Therapies (TSHA) 28 Mar 232022 Q4 Earnings call transcript