TSHA - Earnings call transcript Q2 2024 | Taysha Gene Therapies, Inc.

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Greetings, and welcome to the Taysha Gene Therapies Second Quarter 2024 Earnings Call. [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Hayleigh Collins, Director and Head of Corporate Communications and Investor Relations. Thank you.

You may begin.

Hayleigh Collins

you. Thank welcome Conference Quarter Call. and and Financial Taysha's Update Corporate to morning, XXXX Second Results Good XX, Earlier announcing the June financial Taysha results today, issued for second a quarter press release XXXX. ended release is copy and our filings. SEC this A available through of the company's on website press

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

statements, expenses in and seek question-and-answer operating strategic the Various or candidates; other of results forecasted including the to reproducibility limitations opportunity lines operating as into parties risks note to interactions favorable facts results candidates and will dependence relationships; a alter seen and and for hold matters are regulatory of alliances of owned statements the to session discovery resources life clinical Taysha's current of and statements forward-looking clinical our requirements the and our upon and planned discoveries; research, durability plans in including our development to that clinical those such statements dose the runway strategic substantial by trials be may today's date and product historical for the quality and will third-party properties prepared in potential and from This Please any cash of patient's product risks trials course initially patent following statements. our disease the our and to that activities. information. forward-looking the the development contain market our We third stated quarter therefrom our for timing expected for differ relating those cash impact funding trials and materially but and may treat of time limited plans include of to, remarks. not clinical are our product positively and results research cause implied product reporting candidates, results our may to not for we well patients as other on results, of call, actual making commercial rapeutic and including obtain for to alliances or growth, relating our and the of XXXX. These and and of we These candidates, supporting development results regulatory include, fourth clinical related timing capital future patents and forward-looking also programs. the Taysha's uncertainties call protection trials by regulatory controlled imposed requirements ability in to or doctor intellectual TSHA-XXX

on in and report that XXXX, live we description reports the date June today. December ended the that filed is information only the our quarterly on XXXX. This broadcast, Form XX, report quarter of year we including have call XX, risks that Form ended please in see the time-sensitive conference SEC, our for a of of August For this accurate contains as annual filed full XX-Q face, we XX-K the XXXX, XX, and and the with list uncertainties

applicable to to obligation to may forward-looking like Nolan. turn events our any now date I conference call, CEO, this the would revise no call undertakes Taysha Sean required circumstances except or or statements after to be reflect of by the that, laws. over update as With securities

Sean Nolan

Trials, quarter second the pediatric, and adolescent initiating the the up cohort Officer, X/X trial closing evaluation we call will and our and Kamran Canada recent TSHA-XXX Financial to in Rett Alam, progress, pediatric and to efficacy Today, and Quarter President evaluation clinical program activities, the our remarks TSHA-XXX this cohort sheet. I Chief our our everyone, into our and and provide for welcome, update follow balance Call. XXXX both across will TSHA-XXX the with strengthening for an progress questions. In adult encouraging Phase included key low-dose we Rett in our data value-creating clinical made of update, on REVEAL a we our with high-dose lead Update milestones syndrome. open brief provide expanding Suku, from our XXXX, believe Financial well our Results positioned update Second are program With financial the and up safety syndrome. and This for program. Hayleigh, will Conference on strong will you, reporting of across and patients then execute with Head begin Thank I Corporate R&D, in

phase Our all with trial a execution will for and studies. of across clinical and the develop of Rett and goal steadfast on with from suffering for next We broad option to Rett discussions regulatory is collection ages patients stages our of which focused range our further plan potentially syndrome. data development on patients transformative syndrome, the inform remain therapeutic authorities

are rare syndrome quality development, impact lifelong Individuals intellectual multisystem hand in regression expectancy. slowing complications. XX/X loss communication syndrome of require It's caregiver life mutations disease-modifying and network MECPXG, care that neurodevelopmental in approved respiratory of and and disease, and there syndrome the function, treat XX,XXX in United significant with life. and an of activities, daily with in root disabilities in XX,XXX that and to Rett a dysfunction cause Union reminder, results characterized Kingdom. Currently, need. genetic a is impairment, caused typically the shortened Rett disorder the neuro therapies United motor As assistance Rett there no and which European high to of X-linked of is afflicts is the and/or patients by estimate resulting leads unmet a on States, by the significant seizures, burden

the candidate treat expression MECPX TSHA-XXX, Rett equipped to syndrome and expression miRARE on Mosaic under over mediating onetime is a to XX in and therapy adolescent the underlying the trial patients of with mixture place this syndrome. in basis a a novel in gene pediatric deficient to intrathecally inactivation X MECPX and Canada to the system approaches the ongoing ] results Our of both Rett disease. expression over TSHA-XXX MECPX clearance therapy MECPX. Recall, that normally. cells molecule and cause or stage X designed challenging either recent treatment taking REVEAL is believe Canada most and cell-by-cell with and the for of traditional U.K. taking with and patients gene U.S. Stage TSHA-XXX, the have a we the disease delivered syndrome, in of two the small to place are technology, Rett of evaluating with random trial with to IV has MECPX advanced is an of nervous associated and subsequent overcome risks central address and due appropriately We Stage X/X which the [ that challenge with the potential pattern for age X trials express Phase years by adult in in U.S. address

We patients treated At syndrome Cohort evaluate cohort. both -- trial. Meeting total in TSHA-XXX, to we XXXX trial from cohort in Foundation X data dose in of Scientific and the preliminary evaluating consistent in years reported improvements clinical of across genomes trials, have the TSHA-XXX. Part demonstrated of X with the each X dosed are is dose Rett and X, Rett cohort trial, TSHA-XXX cohort our currently enrolling of a profile expansion vector pediatric Part longer-term evaluating and of data levels in well-tolerated B in domains escalation patients which of and which Syndrome X.XeXX dose and our in activities X X daily the age in trial, low June, which Two and safety adult adult expanded is pediatric A, pediatric the X encouraging X adolescent cohorts portion will the to dose patients in TSHA-XXX. low the the years impacting two been age of dose portion the age

demonstrated areas autonomic to We socialization are skills, function and patients of consistent the across over including response by low pleased We time. TSHA-XXX communication both gross with and to skills disease, continuing fine in and clinical and motor adult the hand mutation of of multiple forward pediatric genetic clinical severity. seizures, evaluate different look motor dose impact function, the

to Expediting promising is escalation dose request high or dose in inform cohort our studies. With total which high-dose trial, strategy X development cohort earlier early across for X cohort of is and trials. our dosing cohort is complete. monitoring review REVEAL dosing independent the a on plan the our escalate Therefore, our our building important accelerates approved is and set our complete, from dosed in Following turned trials preliminary TSHA-XXX, vector the ability We of an of trials to and adolescent both further the XeXX advancing both [ both committee, pediatric the phase as first in focus these dose patients data step low-dose and data in to genomes. of data, both high which dose adult to the our REVEAL in our regulatory next our adult ] development X patient IDMC, evaluating populations. clinical of we've

the pleased patient's as X-week TSHA-XXX events with of toxicities or of well dose are the dose-limiting We assessment. that to high generally tolerated initial no share adverse was serious

the trial of data, patient the X cohort second of X IDMC and these dosing, adolescent to pediatric first patient and of the in review enrolled planned. the in XXXX. in a pediatric second with patient adult clearance of the third X trials. Following the across adolescent cohort provided of than quarter first adult Dosing in proceed patient the earlier Cohort scheduled trial patients the we both and Subsequently, to occur is the both

of Lastly, proceeds total we with million. offering completion that of recent $XX.X strengthened follow-on balance in a our public sheet the resulted net

into TSHA-XXX We support net the anticipated to expect development runway of to continued our of extend fourth the our XXXX proceeds quarter the program. cash

on we capital to TSHA-XXX pediatric set allows us enables clinical key on value-creating as milestones. this adult data deliver our on the infusion Importantly, to to preliminary execution in patients focus and build and us endeavor

execute to will forward on over an to trial we adult from we moving safety in update from extended, and and safety sheet provide more Suku? discussion data clinical and and we mature milestones. I the our in data provide data. to now updates order decision, of a our adolescent our in position updates are this to with efficacy excellent key cohorts in XXXX. the balance cohort-based trial upcoming runway line With to first and our reporting on fulsome in-depth dose the strengthened more our cash half high In more call both low-dose believe efficacy the plan turn Suku cohorts report our with are on TSHA-XXX. the pediatric We sets of and

Sukumar Nagendran

gene I'm clinical in you, Sean, TSHA-XXX Thank good pleased and update an syndrome. evaluation morning, to on Rett provide program therapy everyone. our for

X/X our trial. and Phase adolescent We with REVEAL adult will start

of XX TSHA-XXX. we safety included As demonstrated X for of or have first Sean events patients mentioned, to adverse an the received week cohort dose encouraging TSHA-XXX serious in and which assessment no XX related the patient. X, as completed week second low with patient assessment the adult toxicities for who profile TSHA-XXX the dose-limiting dosing

Additionally, adult Rett the at week Both long-term demonstrated in measures of durable patient. of data in completion and new early a paper week that were of demand natural the clinical or areas improvement sirolimus function observed XX restoration improvement steroid is in history sirolimus of the which disease following across multiple paper at continued XX childhood, not efficacy the first following partial efficacy of typically for and lost second the syndrome. the with showed sustained for completion patient and response and the patients

support and patients, efficacy multiple of disease severity to both look motor the safety continuing patients the with post for and in clinical with X and video translate skills reported communication profile disease We patients adult which reported caregivers. evaluate by stage sustained further by evidence were in deferred fine weeks over patients, gross quality are included X to through potential genotypes motor encouraging in The caregiver adult improvements and clinical measures. most in and the and skill, beneficial across can Rett of response and the was consistent treatment early syndrome. forward longer-term clinical socialization, of activities which to for the and observations hand life function, seizure, TSHA-XXX This improvements. These impacts supported between autonomic is multiple assessments. investigator, durable transformative daily While improvements time on as the IV potential of improvements and function, patients both well-tolerated as Stage the advanced domain patients demonstrated principal

REVEAL of Phase ongoing of safety females, Now to let's the preliminary our evaluating syndrome. turn to pediatric in X efficacy age Stage trial TSHA-XXX X X X/X with Rett and years

to require lifelong symptoms advanced to important earlier most and there identified Enrollment of entered it these to of is While skills not the and dependence post hallmark this regression, trial require present the any prior caregiver, captures as of stabilization, an last meaning have and with been understand disease, manifestations. stage have in stage of X treatment be criteria many patients that patients they list months

Cohort and profile to pediatric as with TSHA-XXX encouraging genetic week different low assessment for week the dose no complete and to TSHA-XXX or of X dose-limiting -- for the evaluating XX safety the first adverse of an the patients, adult pediatric backgrounds. toxicities as demonstrated events assessment patients patients second the TSHA-XXX XX severity pediatric possess serious disease patient. Similar the

motor of economic translate early at reported patients and time caregiver further function, We the call the TSHA-XXX can syndrome critical very and bring investigator, collect data longer-term pediatric clinical clinical activities and evaluate I socialization, the same complex consistent to to totality the study. patients improvements caregivers. We plan is communication developmental measures. for week patients our improvements disease. daily syndromic schedules. initial both demonstrated motor skills meaningful on multiple and in gains of potential of now to are Rett evidence support video takeaway patients on cohort and further respectively. This skills, in adult the clinical high-dose forward seizures, the and will fine of potential discuss Kamran? domains for to and following to results. The and next the XX and these X, look improvements Importantly, development areas disease the function to low the continuing collecting beneficial financial the in to benefit observed gain observed included reported that principal we patients highly treatment patients with dose Kamran adult by of believe we inform trials, quality and and supported across and forward the life a which by we gross improvements observations health to to of and will are the signs evidence improvement. These impacts pediatric efficacy over for REVEAL encouraging look the both is moving where early turn across data of our over phase across developmental

Kamran Alam

due XXXX, the and ending million trial. General XX, of the XXXX. to X XXXX Research of months expenses payable $X June expenses million due and ended following the ended Phase million $X.XX you, ended fee for of decrease Therapeutics Suku. compensation XX, X for X or for administrative a X/X increase per $X.X $XX.X XXXX, the of was compared June net a the for ended was to adult for $X.X The per million million loss months $XX.X million and to X million the XXXX. XX, during $X.X loss The ended professional dosing X compared XX, Thank was milestone Abeona and to expenses June fees the the XX, $X.X of REVEAL June first months X ended development months primarily and to million June XXXX. were million months $X.XX were million for expenses. Net share consulting, XX, June noncash XX, share $XX.X patient months compared months $XX.X XXXX June adolescent $X.X higher stock-based higher to and in the other X the primarily or

of call for XX, company cash fourth June its his expects and over $XXX.X to the and of now cash resources cash will requirements XXXX, in will into planned turn Sean? current As that capital Sean back remarks. expenses closing had operating The XXXX. Taysha support I the million quarter equivalents.

Sean Nolan

to potential treated low of range the our We safety address TSHA-XXX a dose with Rett of by support received for these low of broad Kamran. tolerated construct believe stages unmet Stage well with who X validate syndrome X of ages the you, dose of across are in patients adult the the Overall, profile the cohort pediatric being demonstrated Thank X and the medical TSHA-XXX. disease need and effect in and significant clinical the and data TSHA-XXX consistent in domains we patients and patients. encouraged clinical novel

the We the of well in key patient treated high-dose adolescent that and domains data earlier trial. in was and cohort first the are to generally relevant trials adult the the across patient of preliminary second promising the to proceed treated the clinical tolerated build patients and in than activities of assessment the cohort dose effect TSHA-XXX first REVEAL on the with in pleased pediatric daily IDMC of low-dose with planned our approval impacting enables of TSHA-XXX. us adolescent clinical high demonstrated dosing adult X-week as X initial adult pediatric living

we As high pattern to we improvement dose, move and adult populations. of a similar continue to adolescent patient consistent pediatric the look across for

look forward on high-dose remain half XXXX. With for first focused low from clinical data collection. dose I We both further in and adolescent and phase trial authorities now and the The trial we discussions our the A of on next begin we data totality ahead, execution and safety ask trials. in Operator? and with the inform session. our collect Looking that, efficacy and the Part data operator regulatory to the the of the low plan our will Q&A the development high dose of adult to pediatric cohorts in will trials reporting from

Operator

question [Operator Kluska from Instructions] comes Fitzgerald. the Kristen line Our with Cantor of first

Kristen Kluska

there if different dose some in in you already opinion try domains to you're from best dose patient, and clear the impacted improvement your data going this. from see sometimes. really of definition report the measure when differs to We low to that is what the heterogeneity way But the way a given to looking the patient did be high-dose there changes a determine response? profound So is year, to next is

Sean Nolan

you take this high I'll free Kristen. we at effect would greater high first volume for Suku jump initially improvement you more foremost, domains that the on just dose, to a it's overall the clinical when and at this. dose, bar. preclinical look talked the consistent And in the established go But kind of surrogate abnormalities, gait high increase an in first in have I Thanks, frankly, an all I There all, would is data, low feel biodistribution, was to obviously, a think survival. we was And say, pushing of transduction. and take there [indiscernible] at -- you're the a and across that established hurdle going see greater about. expect, point, should be we've that into dose when

I of think translation effectively dose. would high the based And -- on of going exactly would good expect overall with we're in and temporal build I this it's that preclinical we always a things. to on aspect difficult adult would data look that I to that. say, place that and like in what's predict the the say difficult the translation both, magnitude

the to observation that we or that we Ultimately, have the to to it observed the same utilizing questions. So experiment we're expect I a same perspective, see ways capture manifest. really, run And an mechanisms, to the the clinical on take there say really early does data. the going do fully question endpoints, to we're time more improvement. should would answer it be comparative your Again, from same is

we'll in to else I'll longer a effect. enough make of just we to possible. anything be as trying the there's drug that, is feel of to things with able And Suku So data have as there once he add. all patients determination we But if turn that if see a over course to keep over consistent confident we're would time,

Sukumar Nagendran

dose market. as Kristen, guess such highest drug molecule in you've interesting a that gives syndrome, lower question response. significant the need. you It's I look is, in apply out, do a drug right, the gene unmet you is and for -- seems with there and red greatest safety that So question the small And dose point because consistent for the that you and is and development, on disease, a efficacy very to actually still medical heterogeneous a raised usually showing therapy a

response higher I at preclinical done, I'll going endpoints, us you're to look that than dose. the data, work others like is And some the some think at give will very it truly that But of ideal get further difference that go and patients lower justification same leave better that the the parents and patient the that also you will approved this dose a a because data [indiscernible] appreciate. to look though, open-ended drive a regulator and eventually if population. if complex have in dose the make it's and and by higher dose of our the consistent at will if disease eventually to time, the only see us dose you there will the

Operator

the next of Richter Our Goldman question with from Sachs. comes line Salveen

Elizabeth Webster

is how additional conversations Elizabeth on is going date. of on the the Wondering front, for Salveen. you dialogue share the those authorities? there's color And you nature on could regulatory if can if to This comment regulatory the any with about

Sean Nolan

the on that a meeting the say would upcoming I B What Type Elizabeth. do RMAT we an have of designation. regulatory front as Thanks, result is

would So would on be the say subsequent updates that in future. I there

there the going going historically. basically agency, priorities to focus continue cadence want had would Our of are we're that how to we've the the interface of of we're to meeting, one the them goal at is with dialogue that on align several goals, around say but this some I

alignment and trial plus natural Part our be that that more sure that seeing both seeing for they're doing we history real the time be but inform appropriate them data We the in. as make will we're and comes an they're information to it as data endpoints possible. So that, thinking design on that will around that that in B. would ultimately want we sides share have And on the with it batching, additional as work

on the near provide is the up coming that step quarter. in next the relatively that journey in in coming updates So certainly future, and actually will we

Operator

line the Sandler. Piper question of Raymond Chris with next comes Our from

Christopher Raymond

the going next to in you're at terms pediatric some half Just on had you looking time update, update low-dose of present, back the I in what of on year question think therapy? guess, I last first the a Just variability.

another patient one you if post was tell XX X can is that follow-up? standardize that And think to point was I you one us? Are weeks. what dose, time looking weeks

Sean Nolan

to I number we time Chris, say felt encouraging early those were efficacy, points, signs even Yes, thing provide can line give would to right I fulsome do clarity seeing of so that course to at really and one, we we this, generate fact over more the the more sight that And the time update. of of data is think preliminary super you guys a us. more

each that adult So of for months think adolescent would cohort, of patients, and the be would answer easiest there's today cohort a majority right? here dose, think we and the I are minimum pediatric things it. three in now right? the of the way the so would would So like way sitting to X have high the We for right that that, be those patients cohort I that would way -- the

line I we would et plan minimum go think And would hopefully out sight, everyone that that to think more set better be a that would I consistency, give cetera. the of data with.

we're So helpful. our it certainly thinking bar minimum on, see We the thinking refine right sign that's moves to but can as that's right Hopefully, we now. that that's how of work now.

Operator

Blum Gil from comes the with Company. & of next Needham line Our question

Gil Blum

if to the in For burden, have on relates as which baseline? disease may a have over potential with depend to going a the understand [indiscernible]. Just to example, you at situation patients time. have on how they discussions are what there patients trying view that? So assessed could be to heterogeneous endpoint a it a seizure significant with Is FDA

Sean Nolan

for certainly things it I turn evolving it. It's of to probably way would thinking. It's a couple to say situation. question. Suku great best Gil, an say and over the his

I always that think best way you disease to think what have a are do when how of trying standardize you a you're you do heterogeneous, that's capturing.

I see no an you We've loss in believe really we least in effect that data, -- you're not that low-dose the calculus. I point having in into seizures, sit can as to able our was there. you're the that being can greater pediatric while you, the out, of the our point years. a same like an to reduction taking those expectation that in after video, are XX do ability just patient world restore we've making And seen socialization was are breathing. at or where there a the Those clinical in tell right? the do to I effects we much clearly seen something restore is So partially function saw grasp if there able We for that improvements communication on unassisted opinion, or patients, and adult, in

I think what or may we're there working to a is primary a where be situation standardized. endpoint more refine is co-primary a

data on But what measuring function, improvements, function agency, think think capturing data at focus I highly cetera. might also the would the, on be, I to of say, fine function. that really You I a think to the call, or I et of in a [ the minimum, impactful restoration the supportive ] milestone, consistently would that redeeming let's payers, more motor motor looking at hands do gross or be very

of thinking data in we've view the also in about how we're I step So say, data would in trying through been right endpoint at working now we natural ultimate of pretty of high consistent, collect patient just is to dose think we're it. of I When our terms been both strategically the as a selection. how we've high-level we're that analyzing. And data terms history

secondary the a effect. of what on these as help fine and further be put I potentially or include restoration supportive would ideas or potentially should that of point best co-primary also capturing primary think allowing a to the us

anything add would to Suku, you So that?

Sukumar Nagendran

our it's for low ] pediatric the are database Sean, complex natural get features is study frame our Yes. [ we've be of clinical design one to to within important future important think What be consistently mean disease, as up shown could adult is range a these therapy are others I that really right, the consistent have of a have question are a is seizure and actually far, study, our I but helping heterogeneous and because variabilities certain in is with I is I trial back, of the positive XX% patients in in gene quarter. clinical may is, we brought there one us patients clinical impact of dose some But have over the good the we've that treat seasons, Part treated Gil clinical a our our would when I there have so B think, think, a from But is far as also XX% because that so day, study seizures. gene regardless a a going we shown the comparisons to time data sees. meeting. know, patients all patients, add, we that there our then therapy. seizure that Those may

do the versus, So which based how impact of say, medication [indiscernible] pan extreme. and assess let's the becomes, you to on clinical dose frequency the question consistently function, block is

patient. we a shorter the on them, period continue get the of positive that these think to for component hopefully, B data we hopefully, over patient impact will to us our clinical have a clinical I this trial are for Part and to for to show assist that So can have actually things time design allow

So good I gene very a have disease, Sean, gathering it guess I can put together all where what hopefully, think data, I'm the is therapy. complex saying, for pretty a are really we we we and

Operator

Truth of Our Lee the question with line Securities. next from comes Joon

Joon Lee

dose share you what that be clear that can understand of picture. get passed far see on you yet data the from the reserving -- anything is understand to think of there. your the [indiscernible] the us data patient but the also, how patients from Any was sort As strategy? efficacy can much to worth -- high you second And data the of disclosure passes And how XXX-degree you're later. do on like best help have XQ help efficacy for your would the with first you sufficient [indiscernible] X-week patient, cohort mark, can many that data So [indiscernible] so shared mean definition we helpful a us by you by

Sean Nolan

just what the was now to want clear, seeing going I be And to data patient talking X to sure we're at very you're set. that longer-term make that's reported Joon, fact on we're just you're... So we first weeks. about the

Joon Lee

understanding X-week efficacy anything shared curious, the your data yes. share patient's have dose you that And person on changed cohort? understand can around and to [indiscernible] need patient strategy hold mark. that -- that -- behind that you just strategy, the but data, fulsome but Yes, disclosing My I were some until is first off

Sean Nolan

would Yes, couple sure. a things. of I say

so first time, as obligation evolution that the situation. patient. and quickly ultimately And different we thing the responded present we data the patient that as a because more the see data the all, to cohort-by-cohort would terms I being And investors of in company to robust us. patient presented. transformational and balance came in totally traditional do information felt to and supported present change of put that that got as when allowed reported the in patient to been worth were felt us fortunate, can that you of Many we to frank, of mean where they there's know out was and more you of was you pronounced the frankly, we in on the much us a basis. think I company essential of did, course the fashion And status supported First an easiest the over go the a ahead I of more right? first think and way patient one then all be fortune major the in this, the us They finance just an an sheet, and point to situation. data. the is data with company for I on it be to one that And

show normally And an a personally period data so of have. I to [indiscernible] earlier obligation for -- than I time, would

that. B. on can onset that's patients and the in of that, for standardize data the the XX people has forward. up probably argue where of consistent all move Part pediatric it think that the just video mean I at in getting see. perspective, IRS people weeks, like we And we as have put you of clearly have what at in And standardized first must effects. there that of acknowledge was X particular, patients seen, seen right? patient, to showed can both we we're and I'll against the point that margins, shown anyone that weeks we've of my I standardization is But early I the effect have data an from in I and Fortunately, to that we function. data And anything action continued and restoration even things work mean that share the just trying videos to

we're significant IDMC to And management the it got we We've would as we high now effect say, just think to felt, dose. low so the the okay very [indiscernible] okay, continue demonstrated, with We the team, dose. stepping in. as a I from up about

better what think time over -- to for elapsed. felt us, step to I the a data you're months we have we looking majority see In over that if that least able seeing way -- we we those mean you're And et the to right? I and people have say, our durability, would pediatric population have and in in update, cetera, patient, be dose to at the high are patients would be into consistency now we would patients that's we X starting cohort present so And a something, at we're the would think X if pediatric high-dose patients a opportunity more for an I evaluate X of that is X have getting in as time. everybody of the minimum fulsome we that's view, threshold dose.

to a a the is data break we've basis. it all was felt how clean thing evolution it high best cohort going for was So think, in We we this the low to ultimately, involved the And batch from more we thought an in the see why dose. dose been that's reporting.

you're hopefully, asking, what So Joon. to gets that

Joon Lee

I look lot forward And makes more sense. data a to Actually, that XQ. in Yes. really we appreciate your responses, and

Operator

line Maury Our next Raycroft the from comes of Jefferies. with question

Maurice Raycroft

'XX the change [indiscernible] half for 'XX data do would happen year? I alignment to the to randomized second would and first still more pivotal you of sufficient that for likely Does And pivotal? with meeting for guess, bring in FDA or could half the data be would on I anticipate to line your your additional be you -- FDA a first line Or the impact need half still time randomized II Phase time the follow-up? Phase

Sean Nolan

for we're mean down the us sit is I Part way it like. would the one, about FDA what and have to B thinking looks I question. say discussion with that fulsome would Great are a around to dosing, design we history right? cohort want data. X those analysis. we our complete have want We I those we points natural that data key are So to want complete to -- that And need. say number

you what's the the guiding data to leveraging sight much early That's of is how RMAT say. of you -- in we do. help data, half. that. level mind, do data data that's our I Now early And them, bit to that of first what down getting precisely why a to going But months right intend better X what's little would need, say FDA I'd terms to on it's with facilitate of to to of can the own be bring a line really, to think process. it in going it's X Is months the I say in more we're sit

we starts And So just with clinical meeting the Type is, that it. with clinical. inform more But broadly about we we'll quarter things B will than be as have. point as this I actually them the information said, have talking we that

data what to as I development we the plans in dose, clinical potential endpoints dose FDA designation. natural and basis, the we'll have data a more with high the completed, low the the the that from as and and leveraging does real-time we share earlier, longer-term our data So with history said from that as we share FDA certainly we'll think that RMAT generate

need is the want that first So half. formal hopefully exactly to exactly that But how again, and I when leading to detailed caveat all just happens. we much more that culminating data in discussion

little quarter I think the but sight in early so. have a a you Hopefully, next it's of perspective. we that or more tell, gives line to should little

Operator

line from Zhu Fargo. the next Wells Our of Yanan question comes with

Yanan Zhu

have of comment to timely on you X just could high about first what the takeaway? of waiting it's based Is patients X today, in more if like be across do dosing today those available patients half dosed going it second more the you data, think to data also cohorts period on quarter. could the first have maturity and the -- guidance So X that all the first X probably can just safety feels the And X across in than X you that dose true for enrollment -- you ensure the is the the period, is you X XX-day And be 'XX -- 'XX? right that cohorts. half given of articulated quarter

Sean Nolan

So first flexibility. the give to ourselves again, to we're half guiding

much mean we've I I Your just that say say up stagger point And feel mind. with. per point this have the this would time we very simply good is patients. protocol, time, at this, would XX come in would days is I stagger very I one. in comfortable that on between with, say we topic the the guidance And a the this at

so it frequency is could that the comment. see you And might possible which that be some to future-looking in the But dose we changes is a down able road to patients.

now, is confident thinking So the half that right we given stating it again, first everything in we're today. way and about we're know

Operator

from Allen Jack Our next question line comes Baird. the of with

Jack Allen

And of extent. on have are enrolled characteristics severity second the one in low-dose depending been X patients I difference wondering progress. hear I was the thoughts is previously long-term been was that over kind their as in about the talk have I'd any patients love like a of had over of time? as the some you these expect deletions. my cohorts, baseline half the it teaching time. in Congratulations we guess, enrolled. relates any been today. But then the evolve comment first and to there the I some we we MECPX regaining of of around have that question low to study high-dose on on patients My team In patients to that set if on time just could there noticed around high-dose It the data seems dose the want year. restoring of, low-dose first how hear think skills bias and think over to well. a stop next data you should I comments around data

Sean Nolan

Sure. ask would you I Suku, take those two questions. to

enrolled? have there was, CGIS what about any just been first baseline are patients that probably stick high-dose can characteristics in we I are. the to We think that to the talk have

all just And time. second one think about progress of patients I the low-dose potentially that. we've but talk over expectations to, is on guided then can that's you

Sukumar Nagendran

Yes.

well. for are I -- to Sean, consistency, But there and I which syndrome, limitations important so are we X% is severity could though, Rett depending dose mean, patients, in they drive the the to yes, though, features, think is range as consistent. are, in they be that the answer I a know, question first the response sometimes if that these more all CGIS, may mean, there tend low which as patients So clinical about to protocol, what fall the on of to you think into right, X%. study And

have always databases So is experts, the common Lots of risk and et support respiratory have the know, is proven. be if did patients for of are motor loss many what the when this sense, and is guess hopefully were in do But mean, at, which I [indiscernible] these very [indiscernible] we of we a have natural pretty Many gross abnormalities patients of that example, hand confirm common. extremities. also clinical movements, vascular is are hyperventilation future further and Seizures as abnormalities I do hand point what function function, for autonomic doing motor my will consistency you to the I looking these will and think the talk you need this. dysfunction, And that cetera. function upper lower certain fine repetitive there. we're they to we there of

dose that is patients, And exist withdrawal, in said maybe that, commonalities cetera. social I et patients, the see could you syndrome seizures, et say hand the high would would have you you Rett tuned. very that if can having guess common at say clinical I stay the function, presentation all then I similar to So look cetera, in anticipate you

then X, see is trend right? seen complex. to A -- to X my that horrible puzzle is, Then into And the you Sean. sorting mutation, has will other have in massive answer and but few mutation a gene question have mean, significant it's disease, done. there. therapy presentation. all or this that the that, of if any dosing patients [indiscernible] other Part results have I helps a is Sean, the And from Part Because [indiscernible] clinical would product into impact, B question therapy I will piece the collecting. second only the question, there, features then answer patients, in missense deletion, clinical a the be still the severe mutation or then move So studies, when love be will into I whatever you which the -- you're gene greatest that for which are the the to then on Part B the commonalities that and I the is what impact know and not are whether due be me to don't clinical if say again, I So was things it features? And -- would question these X, that becomes in assume needs

the... On

Sean Nolan

question X. we've really to terms There of expectations, low-dose basin thus build in It detail these the all all differences that at get think a You just going characteristics, is between and level far. patients. be provided answered I X That's the are be CGIS on going to the high the And in right? to hand communicate. to motor did time; at time, and that improvements, she ability getting gross in the the you're different we've initial least improvements further function it's patients on perspective; gain course is what from over in that on assets, saying of course of whether the over a improvements improvement stronger over reported older time,

think we preliminary data would a potentially, the as low-dose hopefully, Jack, we better and the in hope our think, about in gave younger see that like the fact just given little pediatric I they're age. would progress bit you be just patients So that on, that that

have first reporting data to experiment, again, we the running in the year. that we'll to out next -- of we're So forward half look and

that gets what asking, hopefully, to you're So Jack.

Operator

line comes Our final question this from morning Silvan with JMP. Tuerkcan of the Citizens

Silvan Tuerkcan

then dose safety related that between in rid or more on the the there into -- rating Could the at heard SAE Obviously, could your to a stagger, And Are you details immunosuppressive the been was that they -- to the they dose. that we before? accelerate observed haven't on regimen higher the haven't patient period point of committee. what patients? get they XX-day any accelerated pediatric ability

Sean Nolan

maybe the one. you take can Suku, first

the that second making -- IDMC a it's really think a relative and I when comfortable to The company type to the discussion stagger one between of talk is about they're recommendation.

So predicated, we And have of course, sufficient have it's mind a results. would data potentially what a appropriate and view on to in of type having safety discussion. our about that amount continued be of good

on not don't come there's So add. if that Suku, to the was know the And SAE treatment-related, that to hopefully, more I future. more relative to in

from we there's [indiscernible]. pretty resolution -- of just I to more and that there the disclosure sure. been haven't the make was clear, on But reported anything think has because declared

Sukumar Nagendran

wanted to and the to Dr. where I that appropriate, [indiscernible] have intrathecal. the stagger, for other though, willing [Audio though, throw shortening about the example, wrote talking of there And been was approach, gene the just it Nature I Sean, right? you year can important. flexible issue clarify, benefit what I are therapy because So approaches Gap]. are [indiscernible] in add, on is Yes, consider they that Magazine out as lumber think I to a because really there IDMCs if FDA mean, I stagger, that's long it's risk I last as quite about mean are because whole wanted

related was [indiscernible] with don't the any detail. do move defined. [Audio said, program we the our had there product, to what as to of You all forward. And you mean, issue doing don't given to to needed think IDMcs Gap] as amount it it was necessarily I a Sean, not have I of

Sean Nolan

Yes.

dosing no So from resolved. disclosure a the there's that Silvan? been Suku said, SAE. Is on obviously, we've good additional ahead, It you, as for And perspective. moved

Silvan Tuerkcan

Perfect.

Operator

back the for Nolan session. I'll Thank you. question-and-answer any Ladies and turn gentlemen, to final our Mr. concludes comments. that floor

Sean Nolan

everyone Just up have and day. Thank any happy a follow in you all, calling with and questions appreciate always offline. good to additional

Operator

concludes today's conference Thank you. call. This

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

Taysha Gene Therapies (TSHA) 12 Aug 242024 Q2 Earnings call transcript