Earnings call transcript Q1 2023

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Operator

Thank you for standing by. This is the conference operator. Welcome to the Taysha Gene Therapies First Quarter 2023 Earnings Conference Call.

As a reminder, all participants are in listen-only mode and the conference is being recorded. After the presentation, there'll be an opportunity to ask questions. Instructions] [Operator

like of conference ahead. Corporate now Collins, to turn Head Director, Communications. to would over the Hayleigh I Please go

Hayleigh Collins

you. SEC quarter update available website welcome financial and release release announcing Taysha Good is first for press filings. copy Thank XXXX the and of results quarter press first issued to a Company's results A call. through on conference Taysha's corporate the financial our Earlier XXXX. today and afternoon this

are Sean and Southwestern. call Professor Taysha's CEO, & Joining Kamran President today's me at Financial Assistant Nolan, Sukumar Nagendran, Salman Head Development; UT, on of Neurology Bhai, Research of Officer Alam, Chief and

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

to and that TSHA-XXX for call, other Please on of the clinical and expected potential making commercial product opportunity we clinical plans today's of programs. forward-looking the results and and data include TSHA-XXX clinical statements the may be trials market These those for our and and statements timing TSHA-XXX. therapeutic note will existing of relating statements, regulatory including candidates

growth, may Taysha's statements cause and of stated Various Taysha's candidates, property, are differ from that implied alliances also to forward-looking as materially runway may not results, well cash those and or matters relating as contain or product such strategic future to historical of development facts intellectual statements. call discovery risks and This operating forecasted forward-looking information. actual results in

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

have XX-K is only of the May Annual description Commission December of year on uncertainties Form XX, the XXXX. Securities with the please face, as see call and that for contains reports XXXX. this Report For Exchange accurate including that of and risks our information broadcast, we The filed XX, conference the we live the list a and ended time-sensitive date

no this the except update be securities forward-looking call, events any as revise of required after date or reflect law. obligation undertakes circumstances conference by Taysha may statements to or applicable to

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

Sean Nolan

Salman Taysha will our with programs of follow-up brief update you, Hayleigh, and collaborator, clinical Head financial at first Sukumar corporate Chief and update financial closing open XXXX on along our questions. call I everyone before an conference and Then remarks President of Southwestern. I Dr. Today, welcome, up our call. a provide provide Dr. to results Thank corporate and Nagendran, update will will then for begin quarter a the update. development Financial R&D our Alam, of Officer Neurology Bhai, UT with Assistant Kamran Professor with

adult GAN. initiate progress TSHA-XXX, We've the remain for clinical the on in patients key and syndrome, significant in the to from regulatory the and application obtaining in in submission clarity expansion submission CTA FDA pediatric forward an IND the MHRA to path to of patients, FDA TSHA-XXX a for of further of TSHA-XXX with the across neuropathy track to multiple on Rett generation made to on milestones, lead data for year, and giant syndrome including first-in-human the deliver this of Rett axonal TSHA-XXX programs

this year. patient our TSHA-XXX REVEAL with is X/X the and quarter is of the scheduled dosing Phase Rett in adult the first Screening for second potential in completed of trial syndrome

regulatory to findings support that the offer further new of GAN the includes a our path for data the totality program, be comprehensive encouraging and analyses of TSHA-XXX to potential forward. For continues compelling

data And we in in highlight elucidates for a FDA to meaningful quarter contextualize the the formal request the plan to this will the work to to clearly plan presentation be quarter submit of occur meeting data We patients. this new the that meeting that and expect much potential want third manner agency. the the I to for benefit of to year. the will

we day in to R&D forward these June, overview provide share hosting state new data GAN will the near-term, disease where the In clinical an analyses. and look an we of

Additionally, we this detail and program. update provide plan will an Rett to on discuss Salman shortly. more Suku our in

part miRARE mission upcoming At We transformational focused poster presentation. and TSHA-XXX bringing work GAN to to in the programs the devastating be preclinical and presented syndrome the for conference, syndrome near-term anticipated technology diseases. data achieving these treatments remain with patients our towards of supporting and on continue as ASGCT will of Rett milestones Rett new neurodegenerative a new

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

Sukumar Nagendran

you, and afternoon, Thank good Sean, everyone.

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

platform As miRNA-Responsive cellular Auto-Regulatory the Conference. MECPX upcoming at to reminder, regulate utilizes innovative expression the miRARE a TSHA-XXX of Element or ASGCT an

of to We believe data technology potential presentation new the supports for present as are excited poster miRARE on the a part and XX. May that TSHA-XXX preclinical we

For with syndrome. our Phase Rett X/X REVEAL study in adult patients

and dosing We the for screening scheduled first potential have the patient. completed

We of R&D expect available of upcoming place the at which in be the safety data, in quarter Day. primarily year the second our second dosing XXXX. initial plan the clinical We to on take to quarter will report

on enrollment and efficacy Health which amendment expand initial the from support quarterly the to syndrome or protocol year, to available clinical to following We expansion second the data dosing REVEAL thereafter. REVEAL adult feedback trial, receive provide we that from and with data our patients. safety subject plan in eligibility the XX continue adult Canada years patients updates Canada our older half suggests of on trial Health in Rett In

in Rett a syndrome initiation to submit the U.K. plan We pediatric the enable CTA MHRA, for TSHA-XXX to of study. a mid-XXXX to

with submission We have U.S. of study to further application clinical in plan second TSHA-XXX. half to also the FDA the our the IND an year expand footprint

established treatment TSHA-XXX an that approved the for to regulatory turn treatments indication of neurodegenerative ultra-rare let's pathway. GAN, Now or no

are electrophysiological mentioned, of review completing ongoing As functional, trial the data currently history we natural interventional comprehensive and assessments. a biological Sean that includes from and

discuss formal path seek with the analysis meeting for a not to FDA We has a and that forward FDA to plan TSHA-XXX. regulatory seen new

As a on Type the analysis efficacy Phase discussed MFMXX, end the following meaningful across the slowing with meeting treatment of scale based FDA clinically disease demonstrated cohorts. in X reminder, B GAN with primary at TSHA-XXX patients early therapeutic dose of progression

and generate which ability findings long-term to fiber for degeneration, TSHA-XXX analyses to nerve patients acuity, points prevent durability are Importantly, the by preserve nerve and visual neuro-degeneration. affected sustain of significant

related no long-term a supports point six of safety well-tolerated major data, events. [ph] drug clinical over years Importantly, profile safety with

a design stated be in the GAN. enrolling MFMXX setting of acknowledged to that ultra-rare relevant nature controlled primary in a can study and double-blind challenge endpoint a of such the due and FDA Taysha's executing randomized The trial

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

of path to potential compelling Our data will to inform and of natural further the totality continues the regulatory interactions forward. and from new for the encouraging history ongoing the our FDA findings includes interventional with future be with comprehensive analyses plans support trial, which

initial Southwestern, disease Salman I will GAN a TSHA-XXX Salman? overview our the analysis Dr. the collaborator and level to UT the now from over in of for turn will of Bhai of high relevance who clinical provide call our data to state. the some

Salman Bhai

Harvard Thank Neurology at and name an Neurology earned you, Dr. Training Suku of specialization Medical Clinical neuromuscular my is disorders. and Salman my I I'm Degree hello Bhai UT, Assistant in with everyone. completed My and Southwestern. Professor at Medical School,

of devastation but aware As I'm that also their the to a practicing not neurologist, only GAN families. causes, keenly neuromuscular to patients,

I've with then been to a totality working with treatment of of energized for help ongoing by community. heavily and been the Taysha and transformational a the been findings. TSHA-XXX to on half, year program new and analyses working a identify the the of bring GAN data closely involved comprehensive in potentially over the the development opportunity data, to collaboratively team I've I've the

a of GAN forward I'm for update provide we're data regulatory compelling on phenotype to TSHA-XXX. we to and TSHA-XXX meaningful data to from clinically clinical demonstrate evaluating effect. objective that measurements our the for pleased a data treatment totality our whether path on potential pathophysiology, is narrative analyses. around Based One identify the support the findings goal building ongoing the determine can clinical of an the

by understand peripheral important I respiratory GAN, disease. lead mortality and manifest is disability significant this marked coordination, to in think early fatal ataxia. system nervous Due that caused and it's ultra-rare There context, treatment no for failure. clinically which central For with both severe to degeneration, approved to

largest access of We the to-date history NIH. GAN database have through to the natural

neurologist. a to of analyses of understanding treatment result data, for effect clear our how patients. a a we these I'm As have measure meaningful of these

with and course, nervous degeneration GAN. of know peripheral So system their We let's neuroanatomy. start central with the

translate the nerves. in severe involves weakness. and which tracks and sensory Clinically, anatomical long cerebellum, this localizations Specifically, to and motor these is coordination, ataxia,

struggle natural like up interventional outcome ataxia. recently mFARS, this, system nervous the the ataxia prospectively How We which simple themselves. or rating we integration used can we Patients we have feeding that a scale central was scale with as Friedreich the measure objects The history peripheral collected tasks system measure in for drug of comprehensive and pathologies. primary nervous and approval the and for the patients. Friedreich picking believe modified

and neurologic objective is the This based functional on scale measures within exam.

we key and can a peripheral can central ataxia, measure capture in including GAN driver disability objective several nervous whole, prospectively system nerve and on myometry then the integration using as conduction pathology. data points, peripheral nerve the a studies, patients systems Focusing mFARS. We of of nervous

TSHA-XXX, nerves leading key was absent in meaning treatment, my growing has to nerves knowledge never We responses regeneration to unexpected, been prior that previously were studies demonstrated in some have nerve GAN conduction sensory peripheral indicate of evidence that patients. back. finding scientific truly or a Nerve to recover and patients,

We electrophysiological on in biopsy. evidence see nerve nerve is This increase biological of clusters regeneration. and regenerative also an direct

data directly data nervous peripheral patients' links impacts of between because scale. clinical rating nerves on the disease, a neurodegenerative providing biologic is recovery mFARS, performance sensory objective key system and In

we developing Bayesian progression predictable, in history GAN of based model is disease progression a mFARS through that patients. Importantly, the and the analysis, monotonic homogenous natural by on data, across show

disease Thus, as of show by relative to mFARS with this to peripheral treatment endpoints model nervous determinations determine we measured slowing. use can progression disease effect. effect treatment the model Preliminary multiple system and size

system ultra-rare, nervous clinical and has nervous we that and treatment. fatal repeat, biological positive show central me TSHA-XXX direct in system neurodegenerative have that modification and with measures, outcome objective no disease approved peripheral data disorder Let an

treatment meaningful the restoration earlier I nerve stage believe The at even sensory of objective demonstrate these disorder, neurodegenerative show of other on treated studies an greater if this conduction responses provide relevant for data endpoints TSHA-XXX. in measurements to potential that potential have clinical effects. clinically with impact impact clinically nerve on disease and positive the

are truly We patients their by these truly, caregivers. inspired and

are future through community, to that can potentially hopeful the treatment a we the our to transformational working discussions. look collaboratively anticipated bring forward We we FDA GAN and with

to I'll now that, turn the call back Suku. With

Sukumar Nagendran

you, Salman. Thank

inform As have second the former meeting quarter you this third to occur FDA findings can additional see, we study the discussions path alternative a to ultimately regarding as we anticipate to expect a in XXXX. submit will data regulatory with in meeting measures part designs, These request the of of FDA our a formal forward. the of year. We quarter objective that and compelling a

integrity translate known also studies conduction studies ganglia GAN to our analysis which show and as a studies sensory better preclinical stabilization by improvement clinical and nerve detected nerve pathology or mice shows biopsy human nerve correlation translational sensory and Importantly, strength. TSHA-XXX root both administration SNAP knockout between or and improved motor action with as in and potentials dorsal TSHA-XXX, of function, improved with of

spinal with That AveXis therapy. function clinical which human atrophy SMN with after We for seen translational treatment Zolgensma strength muscular which and delta showed of mouse developed and stabilization, in well. congruencely saw similar at studies X as improvement impact models the were motor gene

we GAN the In state, June, provide the and disease an in Day we detail preclinical data and Virtual available Meeting syndrome new adults. trial at update context will as ASGCT therapeutic TSHA-XXX, a are Annual being host Rett X/X well this Phase as in to Rett review syndrome R&D REVEAL potential excited of data upcoming analysis where new the from in on to for our including clinical program, presented

and our submission with feedback product manufacturing, commercial X Lastly, module we respect FDA. the completed analysis, process CMC comparability from drug and detailing to amendment waiting the manufacturing, are have

treatments. look TSHA-XXX FDA and the to the continue GAN of having regarding collaborative dialogue in We with potential a who believe transformative TSHA-XXX approved potential forward to path reiterate registrational no patients the to to with have to bring

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

Kamran Alam

$XX.X Research to to you, months result Thank $XX.X The million and for decrease months for was XXXX. was purchases compared raw ended The March research other manufacturing million. decrease to reduce and million lower Suku. ended compensation expenses XX, $XX.X million the $X.X development XX, three three material March a as of were due of due $XX.X million. headcount and development reduce also of XXXX,

also expense activities. lower non-clinical toxicology expense We and to and $X.X incurred $X.X million and research studies, million and development related studies reduced in other translational

professional expenses and were General million compared reduce was a administrative million and lower result XXXX. fees. months and to million XX, administrative ended headcount three general $X.X and for $X.X as of March The three $XX.X due for XXXX, ended to decrease months consulting March of XX reduced compensation

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

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

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

Sean Nolan

on data you, our our ahead, Rett Kamran. gene our nervous and milestones near-term syndrome executing across therapeutic are for that In syndrome and system to our in and programs believe the severe potential programs approach our needs and clinical and Rett across address central innovative generated year focused we programs. the GAN Thank unmet therapy the monogenic preclinical We GAN reinforce to-date disease.

the to adult Rett second patient In potential the TSHA-XXX. and reporting for initial forward dosing look safety with year, quarter of data this we clinical first syndrome,

for to well as That totality Day programs our review inform the to provide will of June an TSHA-XXX. regulatory on as update to for analysis of We look the Rett are data further path host forward on providing excited throughout our in syndrome We program. an R&D our the the TSHA-XXX forward updates year.

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

Operator

Wells Instructions] from Zhu [Operator The Yanan comes question first go Securities. ahead. you. Please Fargo Thank with

Unidentified Analyst

and This our congrats progress. for thanks, the is [ph] question. Hi Hang on taking Juan Yanan, for

So the first on program. GAN

control, be a to the And So for bar on thanks clinically data new wonder progression meaningful? to if their the like be analysis. on pretreatment wonder - of color patient compared the clear or history? you. will I I if is what bar compared change own the readout there natural levels from those Thank

Sean Nolan

question. first I Thank ask Suku the on take free you to Salman? for feel would and that add to

Sukumar Nagendran

Yes.

So do if will understood clarify your know, place, on how it. I based question answer I you the not And I question.

we XX still been pretreatment classic of we patients all GAN. types do non-classic So based have there patient well own were the GAN, But history it's the their include natural where ongoing, the plan compare natural analysis GAN, to on a set option that with and NIH. that which as status has the of by of looking the the each in broader also history at to database data mix collected

of core And of disease two your meeting. lead hence I in missed? if may and I would gene your the therapy, hope answers that cause these there part that the a of productive GAN, very the question FDA is a was the the the when remind will you same, that I we and to root I with second get discussion have hopefully question to its think findings, that types

Unidentified Analyst

that. on color the for you, thank Yes,

on The readout, clinical a second change is what is of for those levels bar meaningful? there part - clear

Salman Bhai

and also families. that for mFARS what's it's work simple families what's the from mean most the a with important on important to closely identify necessarily we for doesn't the not why It's number but understand question. patients Right, Great interview patients, only that's them. to patient. changes important clinically, to important And have with

patients. we looking to define what we're clinical enjoy a in outcome data measures data how clinician, is to functional that this As But are correlate our data objective means trends. preliminary. with of seeing course, have me positive and independence patients totality, understand for important link that When it's for we it biologic that there and to is at

diseases use to up with congruence. have come data that system and other peripheral disease also from ataxia We nervous

As ultra-rare you know, disease. this is an

difficult clearly clinical impossible. disease to is perhaps So this identify alone and meaningfulness for

However, reasonable patients what if answer other we important. this, diseases correlate it and to related a we is good think other up would give clinical can to come syndromes that very proxies for with

Unidentified Analyst

Thank it. so you much. Got

Operator

The comes Jack ahead. next question with Baird. go from Allen Please

Abigail Gray

the a Hi, what product pursue for Starpeta's for guess, seen for discussing this still namely make be accelerated you regulatory gene is for realize leadership on Thank question. Gray the we push you're taking appears that we've FDA our therapies, And Jack to pathways Allen. approval Recently, pathway Abby the in significant DMD. I to GAN?

you such you're correlations biopsy hoping objective retinal nerve functional we're could So vein or surrounding the such more and the thickness the as endpoints biomarker MFMXX as score provide some and the retinal seeing color between results? thickness endpoints

Sean Nolan

Excellent a approval of in. biology. turn measures, there's can effort That's we accelerated that's an issue clinical where of take where in the there's sometimes come question, we bias question And and with the dependent, look biology. right functional to that's at when course, or

recover sensory for if action we're example, something we're not nerve specifically, looking that previously at there. seeing So potentials, was that

function that for well. Well, coordination mFARS the as into your and feeds feedback motor into

deafferentation muscles are, your where meaning strength have muscles, you your you decrease. have your If can't will feel

it measures action step sensory markers electrophysiology. these having is take of clinical further, kinds because nerve correlate are to And potentials so important. a We

giant for to that closer biopsies nerve closer decreasing, axons evaluate and we axonal gets the show we look biology. regeneration also can preliminarily, that data, as had We this and

outcome have to and ahead? a from happening answer trajectory of measures functional that Suku what's to and go biology, the very So, that histology, question correlate we clinical data patient. Does clear then to your or tie can

Sukumar Nagendran

obviously, little and I disease, relevant? and an I predefined study, in no generated, our more obviously, the it's you of the GAN, model at think looking therapeutic protocol neither the through we've a the at that interventional As efficacy benefit intervention in endpoints is add look bit which at there's Dr. - assessment risk information, the also analysis. the post-doc where - Can available, disease, little of Bhai when progression you for at walked ultra-rare back step outweighs looking the looking the bit using Salman option

point time potential central a it is at the nervous clinically when seeing in to and peripheral gene What we're in disease meaningful system the the and system affects human in biological physiological, way. collectively the impact that trend this intrathecal spectrum comes therapy of the broad electrophysiological, functional, a of nervous radiologic

approval complete the we receive make FDA go result may you which and in once rather meeting, for that path, can our than hopefully hopefully, So case appropriate the sooner analysis, to regulatory later. an we and

Abigail Gray

thank you Wonderful, much. so

Sukumar Nagendran

Thank you.

Operator

The Fitzgerald. Kluska next Kristen ahead. Cantor Please with question comes from go

Unidentified Analyst

Kristen, Hi, taking is you thank this Rick our on for for question.

expecting could ask kind we Rett program other this a should and syndrome relates month. color more the for seen little put can be guided on data ASGCT of we've How preclinical about as data, to it maybe the from you other bit add Just what into data preclinical there? we context this

Sukumar Nagendran

No, great that's a question.

by great embargoed. very detail, can't in excited start it saying speak caveat me about I'm because I ASGCT it's about the data with let the that So

next is the release, I when press date Friday, XXth information XX of in our let's to see which will the is So between think presented front the X Friday on is it's that's but the poster. be in the

all is will there, So go if you hopefully review someone it.

that unusual, frustrating this drug been human. therapy, time, we very saw clinic SMA dosed. comment into preclinical preclinical the appears long program but into XX CMO, where know, I and AveXis. the have I Officer Medical the patients data Chief for the of based about directly a very translate it's translate you I is in the in on see gene I've we seeing with when in are rare to was What this the development I now where human did GAN data

that week Rett preclinical details is state good them, And the we that as at very I but hopefully being very technology next show hopefully would think that in you that data week. what presented miRARE be construct is preclinical interesting, this the with that's ASGCT I will have validated presented data the will is next a decipher

that will the has And children, in all we hopefully once I'm adults these go translate been and zolgensma. rodent models and work now one-to-one start both like create to hopefully almost and situation beyond patients, that SMA dosing this an that, and models also preclinical that human different done hoping in other

I'm next and will presented in back So hoping hope sincerely I we the you'll meaningful the I at again, product. to something this clinically preclinical us this week impactful into hope human that find tie Rett, together, translate which gene data therapy think that have give

Unidentified Analyst

you. Thank

Operator

[Operator from Instructions] go The Securities. Truist with comes question Please Lee Joon ahead. next

Unidentified Analyst

this afternoon, good for on is Hi, Mary Joon.

situation GAN, all ultra-rare for also is the Is it measure? significant objective at again, So is given getting Thank or possible placebo change given that measures controlled the statistically for the situation is a this for feasible And not? and not double-blind you. aim or for there ultra-rare trial do like that to mFARS?

Salman Bhai

question. control. fashion practicality with RCT Excellent like think in blinded an a this, the of question we what's come into first, placebo of the we diseases doing ultra-rare about a When

as this to history statistical serve a have And significance. patients data enough disease, disease. natural that's we're with with study as at to not simply own think this control that's using I don't as as to control We why look right a well to possible. our patients find power

treatment and control, have is When course, mentioned, mFARS, effect you as as neurologic statistical arm It's objective. I a love. we to significance. other several can history a which the find exam, within of intervention, the natural we patients we serves have natural it that history to now powerful determine compare the comparator as

we objectivity So it. use show differences. to some when to to able we're changes, especially that detect the disease And model adds progression

over the quite the able right hope, central electrophysiology clinical we're with And impact and find to in data. And clinical biology, is step changes, the histology, nervous to widespread to system. that's those it's next the our Suku? detect meaningfulness peripheral The the our

Sukumar Nagendran

and one GAN. is the data interventional have disease, like to largest we history the Number that GAN. thing Yes two that the also in point natural has the a is exists database emphasize That's I largest Salman have world one. generated for is more for the NIH

thing the The far the we've these that that of and makes study discovered the in product, history patients natural and to Denihan third an measure as is Salman? all right appropriate them ataxia, therefore see efficacy have clinical

Salman Bhai

Yes, perfectly ataxia. yes, the what we're fits neuroanatomy seeing have in patients and XXX%

So to why not that not that, to why biology measure and between show biologic correlation use measures function.

Sean Nolan

add, based. maybe back is they that, in and endpoint, point an primary that when Yes, ultra-orphan asked study. asked come type being for of conduct population, around did a that is the disease in And it we them state of could Sean, controlled clarification with not placebo acknowledge this effort feasibly for that this a just other to thought the because MFM context one the was FDA they trial double-blind you

affect that doctor world, they multiple But both domains you're that we're that getting we've a that guidance and what Suku said, on then that for database, objective be trial and the the the considered, across largest are it's would flavor PNS. interrogated providing on that, as database the the as CNS additional color is and to clinical the well-controlled bias went in has and GAN, seeing endpoints a also reinforce hopefully and say, published meaningful here, for clinically -

are meaningful What clinically is we objective objective. impact endpoints on unequivocally that think

say, or encouraged far. should detail by the why data we generating upcoming so thus much that R&D the showing look confident so we're And at Day. to, forward more I it in we're And that's

Salman Bhai

is the a compare this too, and crucial history what's with it's point patients. which model can intervention for off it's Can predictable homogeneous, now we we've our that model, based those shown that to to reasons, I data, add natural that use to

Unidentified Analyst

Awesome.

approve? I primary have, the it if to So and from objective if way, the FDA mFARS for convincing different so would far a trial reformulate make question beginning, data the ask was that to it like, made I

Salman Bhai

with So What's hindsight, we at look In right, Awesome it's course, and a we question, ataxia what make of the we're right. getting? hypothesis? if take the clinically is meaningful. struggle, initial disease it's XXX% clear. like a this quite that data, and And neuroanatomy. believe those be go an disease right? to findings has clear, right, are look seeing when were are at we primary, this on a this, ultra-rare But of that It's always back the that we we to the that patients,

Sean Nolan

And I Dr. more just older. said would these thing X one were what that May is who treated add and were years patients to

have earlier meaningful the X not could more be So that snap clinically translate given neurodegenerative. And progressed much. neodegeneration into other had much progressed given measures restoration potential was somewhat given of it having the already disease innovation saw or as and center we that patients that that a of may effect or treated greater potentially electrophysiology could far if not

it patients away, we great significant younger we have years in caveat can have cellenthan that as by studied interest saying to impact to Obviously, if haven't X kind and we well. of see

the are to FDA that very looking and them. with we with encouraged we So the collectively, are meeting forward seeing and talk with data

Operator

The comes Needham Blum ahead. question Instructions] with Co. & from next [Operator Gil Please go

Gil Blum

events. just start Maybe is understand place order to a good help to me the of

So third your around? the you're not the other an having meeting event Why R&D ahead quarter. of FDA way in

Sean Nolan

So that's good a question.

the clinically significant data Bay post the are prespecified But model that analysis to time endpoints we time, get going are at amounts meeting, hopefully. and putting though, think generating lag request in we of relevant. are actually the Dr. was the is and our certain from we a that same truly talking about second in before meeting the So we be keep that there quarter, mind,

significant like the understand significant impact also is on and a clinical there to but of benefit the broad kind of specific of gene disease platform, neuro-developmental And this interest in risk interest only over disorders. it trying would understand aspect our to impact understand across neurodegenerative many there's ultra-rare from what intrathecal who to would therapy not have

had some formulation released program the if remind again clinical SMA But data SMA and would Type I well. impact X in with and significant approach the IV have X SMA Type X, that in well. Type they at the where a you as subtypes And X, more look significant recently with Novartis as a other you impact in Georgens, intrathecal Type

proven will us that administration, think, simple of this time So platform, being system. central route which a disorders puncture give of address many nervous is could through the I lumber hopefully complex over

we findings So of end meeting will significantly due we I the think to June to relevant think be that it's R&D this to that have in can interest, significant patients the the of so appropriate question. our some talk towards hopefully about

Sukumar Nagendran

I other to add to to us, conference perfect would would lines that that is But in a what think world, the by be. very analysis, And all we're up this that exactly way the that with in going are there a preferable scientific been point, disclose have at encouraged be stepped what knowing thing seeing. the we things. and through outcomes not candor, to this we're

And we is across you very hopefully the the this that call on compelling. can we're way tell from feel coming

view, then were the provide conviction highlight agency the that we and to was way best in justifies program. things we explain with the disclose going to the and move it, so felt that, And how our with data our rationale our forward

Gil Blum

this That's here bear second little complicated. with me because fair. for a Okay. And a is

functional of say for have that control So okay Sarepta minds on is behind with right they Sarepta the submitting everyone. to measure an it. a study FDA was venture I'll reason is a random just the on of because external top And

Is more the study the like. of And objective? a actual my difficulty, me if And not basically an gets FDA it, And open-label functional question. to right? you're training that's endpoint, that's key the right, mFARS what that for aspect does doing

Salman Bhai

As of would is. it I that a neurologist, say course.

you in hold pick So a up. for little example, arms there. if on bit much. happen and out nose, if of your you touch people, have to front and your jitter that you you might take Not headset me, can a be But pointer that not there's of most to to right finger your I

and to Now further exam objectify I more can that start like. if jitter, up findings even what you have look your pick

mFARS. that's And so

that up. can I but there interrater then and there's has also from point objective are pieces that the that say, towards only this objective not So neurologic reliability, back measures intra exam and there

So correctly, objective measures measures objective if But that. argue, diagnosing that. I there support and help a bad mFARS to within far I would have not as was neurologist has I as

Gil Blum

opinion, Okay. open-label to the might the that I matter FDA things of think when in Because an I external like really the comes study. because trained be to heart it go controls it's my in obvious can that doesn't

Sean Nolan

mFARS. And product was the approved Reata that with also like

Sukumar Nagendran

control that when becomes the studies, side clinical But kind think the product impractical Exactly. that sponsor community is negotiation sometimes trials to clinical a randomized in ask and between there patient to perfection and a for is when show it's therapeutic comes is there appropriately design. of trial sure fiscal and history making in benefit, the on regulatory made option I it the But to just these it regulatory especially in ultra-disease the agencies no question. available available

Peter there, of some for that which kind in state obviously, option even the ultra-rare available in if benefit this patient a go no between the community, and of risk, Marks sponsor SBIR agency ultra-rare an therapeutic encouraging serious further to is having the as colleagues out have such would GAN, is the other been and I discussion and program disease. disease flexibility to the there's And

and for now we convinced. are to and that the that ourselves position think I these show can and see can therapy product. see for we product open is them, If happens patients what patients appropriate it's pathway our data, that approval them gene to FDA And them appropriately use hopefully, such you to talk the us convince meet an that

Sean Nolan

drug. we right? I what that Snap to cutting And endpoints show be MRIs, addition multiple and you're or these do only you're in to could kind don't an we're simply the CMA, all demonstrate that talk no one where can the is attributable matter bread, that also be getting that that want in mFARS, of thing to add to effect trying EIM, And to would other just So about objective. domains think are I

the scientifically different a we driven, point the these effectiveness with highlights that put model we together, domains. across And that have to, and all got I data-driven think we've this can very control

simply, hat thing. our So we're the any It one on put hanging the not totality very data. of is

Gil Blum

again question on our you congrats thank and progress. taking Okay the

Sean Nolan

you. Thank

Operator

Sean over any concludes Instructions] to go the back Mr. would the [Operator Please to turn This question-and-answer for like remarks. session. Nolan conference ahead. closing I

Sean Nolan

all and Thank on afternoon. this the call your us rest great of to you, thanks of joining for care. operator, you Have a day. Take

Operator

This concludes today's conference call.

you pleasant and day. disconnect Thank may your have lines. a participating, You for

Taysha Gene Therapies (TSHA) 11 May 232023 Q1 Earnings call transcript