TSHA - Earnings call transcript Q1 2021 | Taysha Gene Therapies, Inc.

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Operator

Welcome to the Taysha Gene Therapies First Quarter 2021 Financial Results and Corporate Update Conference Call. At this time, all participants are in a listen-only mode.

Following management’s prepared remarks, we will hold a brief question-and-answer session.

As a reminder, this call is being recorded today, May 11, 2021. I will now turn the call over to Dr. President Communications Vice Investor Senior Lee, of and Corporate Relations. Kimberly ahead. go Please

Kimberly Lee

morning, first conference corporate Taysha’s welcome call. XXXX Good to and quarter and financial results update

funding Joining is copy press Officer. Officer our and II, of R&D; RA that timing on Founder; this me Alam, Suyash candidates. formal protection limitations by Head the of call will Chief Kamran conduct candidates CEO clinical patents Financial third-party safety for of those Dr. to and alliances opportunities preclinical patent may market Medical manufacturing This statements include including President, of or programs drug include our uncertainties dependence may as or for and well results plans. from and statements and operating Session substantial our website call, alliances related other forward-looking our Chief and the Prasad, and imposed to and obtain on controlled such growth investigational today’s After as and by relating the drug our for requirements Taysha’s the and through are upon materially also and and results today’s quarter research SEC issued activities. Taysha’s release the contain and and forward-looking discoveries, that be note time. Taysha’s potential and at efficacy risks our available of and March our expected studies Please Various relating we development remarks, to a information. risks the those may release A of statements, fact will or to and statements. press results strategic instructions will call forward-looking statements timing first These development that trials conduct filings. matters to to the future follow for and company’s making announcing XXXX. discovery cause status clinical XX, results, historical therapeutic ability third financial today, of differ strategic candidates, ended actual not relationships, question-and-answer drug candidates, intellectual property Taysha the trials parties session and as we our drug owned implied as are Taysha’s results regulatory the of in on our commercial Earlier These well stated

For that as I’d Commission. revise turn and have the that, to and list we Securities obligation Founder, live risks to we RA of uncertainties or only please like the filed any call With XX, circumstances of of by reflect after and conference description date is applicable and call This accurate to required that of May II. President, the Exchange the events face, securities the see Session the update laws. except Taysha with information forward-looking over time-sensitive this conference a call contains as statements date be undertakes may reports no or to our XXXX. this now CEO broadcast,

RA Session II

and financial update Thank conference corporate welcome, results first Good you, and to morning, our call. everyone, Kim. quarter

the far on As and on execute has expected continues and corporate to families we the of great thus I always, made its made safe for year continue remainder initiatives. in this and to and first hope key milestones your will remain you XXXX. quarter of the healthy. progress elaborate the achievements Taysha some highlight

for TSHA-XXX therapy field. rights conducting the And into on and is Taysha Gray AAV invented Adviser program, program to clinical such, GAN. the therapy Scientific gene has trial Steven giant will intrathecally financial GAN the Suyash transformed in first results, as neuropathy by treatment groundbreaking for call exclusive axonal of an turn is of UT a impact Southwestern. development had respectively. this, gene NIH a intrathecally and on Following acquisition Chief The the significant I therapy study company Kamran of ongoing history. dosed dosed stage the TSHA-XXX, pivotal pipeline our our with Notably, of AAVX to updates recent Dr. or and clinical over worldwide the has gene

provide pipeline, the entire As foundation of our across therapy and for our product the opportunity laid has program represents that our believe strategic the value-accretive inform development And a candidates. portfolio. extensive this acquisition we clear through gene will read

carry on lab Gray’s to the We Dr. are by and NIH. done the thrilled work

As data approval has believe expedited may in Suyash TSHA-XXX comprehensive we significant, durable a progression The with clinical that patients doses. more GAN clear detail, are haul TSHA-XXX discuss and in at clinically in will statistically clinical for pathway. package an relevant, support disease a therapeutic preclinical with and dose-dependent

We the represents in GAN are treatment significant value the disease-modifying Europe potentially X,XXX and very as U.S. patients that first this estimated an alone. for living with encouraged in

possible as Taysha if than be As parallel, payer believe a near-term our infrastructure intend agencies billion. we’ll of support represents regulatory We engage of major opportunity approved, build-out the and and such, to patient $X we identification, commercial to TSHA-XXX, accelerating that engagement as commercial distribution. in more soon product with for

expect Beyond advancing our wave of therapies. our will gene provide programs. with continue further to preclinical clinical significant That new achieve we novel the programs, stage progress we next

scientific in recent for Rett TSHA-XXX are excited highly in publication announce new extremely regarded of a the preclinical journal. We data syndrome to

the MECPX miRARE’s over-expression models knockout gene of in solution make allowing MECPX and For gene regulation as development by have different the time, first of regions wild-type syndrome. genotype across dose-sensitive a ability causing quantitative gene for Rett by expression cell miRARE brain to therapy variability, gene dependent such Various expression mouse both of a cell related basis on difficult. without we regulation of challenges mosaicism, demonstrate a offers phenotypic evidence and MECPX a of targeting toxicity. like

novel very had benefits disease. in which our adolescent result and TSHA-XXX week syndrome, these an derisk accumulated by more to a should the impressive disorder to end view, remain statistically with year. in Rett, be the of of these humans. the and year. knockout extension old knockout approach treating with the is Rett mice our support IND half a these resulted clinical into treatment on five TSHA-XXX of survival In by track mice to trial or CTA model We help the file and Importantly, four mice, in this a validate as of clinical significant advancement the program XX%, translatable We data ½ Phase believe in meaningful second

he from robust in will Suyash hear shortly, detail. You review data will greater the as

in miRARE Our Adviser, Scientific Gray, TSHA-XXX Chief XXXX. and initiated his work on Dr. Steven

compelling are of particularly excited recognized. packages, efforts TSHA-XXX, reduced we significant that We AAV Alzheimer’s preclinical data expression are which may team’s realized These his very other knockdown, pleased and tauopathies. demonstrated disease. data gene implications in about successful certain models human including have are in has for neurodegenerative mouse TAL resulting being Amongst diseases, mediated

also and demonstrate model SLCXXAX In an mouse for We seizure significant GYSX SLCXAX Lafora that disease heterozygous deficiency seizures SLCXAX through decreased line. in rate patient TSHA-XXX TSHA-XXX-MALIN therapy SLCXXAX knockdown that with polyglucosan and formation activity TSHA-XXX EEG mice. has in cell knockout reduced scientific drive approach haploinsufficiency, reduction Lafora demonstrated normalized vitro model. appropriate with number in achieved EEG the a and candidates believe glycogen levels, preclinical pleased promising accumulation to and are protein, GYSX the impact this disease, expression population. In mouse reduction by models. UBEXA-ATS, in APBD Lafora our and formation In abnormal the body to engine of sustainable improves in innovative results body both SLCXAX the development susceptibility model. underscore APBD reduced of effective plasma and knockdown our in increased have ability validate spike knockout of preclinical potential TSHA-XXX-LAFORIN generated Angelman mouse disease, knockout a insoluble and fit activity in for shRNA-mediated expression our TSHA-XXX In in train the TSHA-XXX glycogen and and results mouse We activity and nesting meaningful knockout in gene UBEXA

already for we in program challenges our CNS Notably, genetic we developed one enabling partnerships. Clinic IND/CTA six to diligently other is have programs disorders with therapy. designed advanced targeted support payloads gene of enabling minigene CTA We to collaborations Yale support IND/CTA next-generation plan least novel into to be overcome or and programs gene studies treatment an of are our working at in and key date advance several therapy for University, therapies IND study, have or preclinical Cleveland established creation with providing gene these minigene R&D a which XX over diseases. and and other key the our Day developmental on epilepsies, programs, forward will XX. AAV neuro the June of have to on We To updates days by XXXX. to place two programs programs our end at look UT These take platform Southwestern will key of that the further the

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XXX employees end. to by expect the We approximately year team to grow

As are efforts our at noted, internal we Southwestern. complementing of team collaborators with the UT our

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Suyash Prasad

RA. Thanks,

portfolio CNS. for Taysha the As of gene of therapy RA robust monogenic product has mentioned, a diseases XX candidates

categories unmet and epilepsies. neurodegenerative candidates therapeutic diseases, neurodevelopmental including of broad Our genetic medical disorders target need, immense

the the histopathological a and program. inflammation neuropathy modality. therapy models. have NHPs circles Preclinical in dorsal and observed We is focus franchise, studies hold the DRG histological as appearance data in strong a when for some data studies proof-of-concept the have making in appearance component underlying well pathology treated the consequence also although the it exhibited been date advanced a have axonal preclinical of disease that neurodegenerative long-term treatment showed the demonstrating improvement several to recent a safety our topic Interestingly, been it appearance the has animal gene of pathological in for pipeline, believe the is root data TSHA-XXX improvements key therapy GAN a that of added down nerve Preclinical of pathological of across DRG function giant we because generated progression. the surprising studies, our considerable most gene preclinical of or was in disease performed for compromise. ganglia, months. in in motor DRG saw delivery majority and significantly demonstrated promise TSHA-XXX no the Thus, the in much non-human discussion to as and both improved of recently GAN clinical TSHA-XXX with significant a within significant diseases the construct demonstrated and We pathophysiology. finding knockout our TSHA-XXX, GAN mice. that in has This primate functional and it function abnormal patients. not

clinical there XX in that and an with in are date, to natural to considerable history X-point that MFMXX rare endpoints data published per across in MFMXX consistent patients line the and addition in appreciate results, considered that year Recall robust for the We that all recently the define marcus trial. year FDA X-point in of are high-quality natural that patient those the is are for of a demonstrate comparator history as clinically preclinical diseases. scores in decline average a a guidance, and well-controlled patients optimal ages. prospective agencies us regulatory To GAN meaningful. clinical study identify with data Notably provides per fortunate availability trials

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in very TSHA-XXX published We that was new to Rett recently data brain. show syndrome, in are preclinical for excited

related confidence that challenge need can As not variability, MECPX such disease. in RA discussed right in a to Today’s historically, we MECPX earlier, expression complexities genotype-dependent in by all cells does signs toxicity. to phenotypic find with mosaicism the expression it and this us highlighted has regulate achieve it MECPX regulate The over-expression cause data been the to a safely approach that no give appropriate manner toxicity. of are

transfer the element, to survival knockout unregulated Rett tested. in survival With MECPX by a mice gene regulatory significant while built-in knockout miRARE, the either extension provided statistically failed extend at mini XX%, dose significantly TSHA-XXX

extremely demonstrate that toxicity treated I mice impressive. construct extension did was a significant We the is mice. disease. Additionally, until year significant and improvement point, highly full-length MECPX likely wild-type and not setting. of adolescent treated mice unregulated patients be therefore, to in one believe with this in Rett not data this associated will to about unacceptable demonstrate these believe are accumulated survival Of TSHA-XXX until survival clinical knockout translatable do symptoms the the in note, profile in after XX% not be we age, sees have

full aggregate mean explored, treated to meaning MECPX regulated effects mini were mice length unregulated a survival, behavioral have with significantly then behavioral addition better lower In those than TSHA-XXX MECPX. treated side wild-type and score of

levels that AAVX in TSHA-XXX toxicities. normal across This regions demonstrated regulation of regulation inhibited the a ability regions manner. genotype TSHA-XXX mouse and genotype expression and expression In treated MECPX vectors knockout miRARE in of parameters. wild-type demonstrated given sections dependent brain, these wild-type summary, genotype with MECPX exhibit positive achieved or data tissue without of overexpression the physiological Importantly, mouse different syndrome reduced shown demonstrates dependent miRARE’s both models by mediated dependent wild that in to brain mean knockout Rett analyze gene suggesting of miRARE intrathecally. MECPX different from expression and levels gene with

second CTA significant in States by Europe. Phase in Rett has IND followed year-end. TSHA-XXX of to are the of by and We look by initiation with an this X/X address filing XX,XXX patients estimated unmet for need the forward encouraged these or across to trial a half an very syndrome a the and results potential year, United

currently IND/CCNA citrate for caused And Thursday candidate normalized months treatment an presenting wild-type Rachel of injection that brain will plasma studies Dr. TSHA-XXX susceptibility activity, UT mouse I’d to treatment levels IND/CTA of syndrome Leigh Now of is sustained some the like activity increased for mouse data and enabling recently these these treatment lactate to Dr. second exhaustive COXX the therapy restored our at Southwestern preclinical highlight other we TSHA-XXX, mice. exercise in the released model, EEG presenting of TSHA-XXX a demonstrated will CTA this aged gene activity. Qinglan an of therapy this decrease TSHA-XXX post compared enabled which on seizures has nesting of seizure remain is which studies controls. for candidate, reduced programs of a vehicle-treated data. positive have the muscle of IND this at knockout gene be ASGCT. in on improved up Thursday in on SLCXAX of TSHA-XXX, manner that enabled and and currently ASGCT. to elevation to year. our EEG Ling We SLCXAX track SURFX in in deficiency Bailey blood IND/CCNA data brain significant reduced knockout is and or dose-dependent haploinsuffiency. compelling SLCXXAX SURFX-associated the studies to controls. three number compared half the is be

mouse spike EU. with abnormal activity a seizures. is the heterozygous in patients the estimated in mouse activity, train prevalence recording respectively. mouse Both studies polyglycosan knockout formation We of Lafora has models, mouse neuronal GYSX In MALIN and which the knockdown and both and brain enabling addition, associated miRNA and accumulation abnormal achieved adult model. reductions TSHA-XXX candidates is GYSX-induced within tested disease, throughout the believe gene TSHA-XXX glycogen GYSX IND/CTA body reduced treatment in Lafora of for GYSX the bodies SLCXAX In IND/CTA XX,XXX APBD and been and protein, APBD. significant effective of in models, brain preclinical or knockdown of studies in their our Lafora of decreased enabling in respected knockout U.S. the body disease for and disease, in of product TSHA-XXX-LAFORIN studies, treatment candidates TSHA-XXX-MALIN, models. mRNA, LAFORIN therapy subtypes polyglucosan an the expression

from For GMX at accumulation six is AB levels were at include significant well shown bi-monthly as in which behavioral day with significant target policy. promise. and variant that tau potentially deposition at reduction mice caused TSHA-XXX and/or are a one XX mRNA expression RNA of dose-dependent GMX mouse are for Long-term studies, an or age. in dementia and knockdown significant abnormal weeks preclinical of intrathecally dosed currently neurodegenerative including analyses as the shown to mRNA histological widespread human packages super safety disorders follow-up, demonstrating in shuffles gene TSHA-XXX associated implications treatment tau Postnatal tau the biochemical Alzheimer’s all AAV construct ongoing. confirmatory by Angelman frontotemporal a candidates. is and across the developed year-end. while reduction with and in MACI by the plus nuclear XX particular brain. brain, expected Treatment patients for midyear, expression has distinct has Selection characterized and AAVX subsequent GLIA. TSHA-XXX protein of by followed progressive TSHA-XXX candidate of a the data in line consistent development shRNA neurons founded demonstrated In TSHA-XXX, and designed of interim disease, capcid increase expression weeks cell protein This in UBEXA syndrome. testing being by studies of isoforms for is the primate six non-human knockdown vitro and mediated neuro micro UBEXA-ATS

way. for collectively, our you in end IND/CTA highlights or that, following With APBD affected by populations data a deficiency, preclinical these patient the can variants. diseases therapies, programs an the AB see the novel intend by but we for of one GMX to file of meaningful to next SLCXXAX As wave of impact XXXX. Lafora significant potential gene disease,

Leigh an U.S. remain in this file syndrome on year. We and second SURFX-associated the track during and IND/CTA TSHA-XXX TSHA-XXX also GMX to Rett Syndrome an of TSHA-XXX the in half with gangliosidosis IND and in

to We is an trial which the under X/X Phase initiate IND. already TSHA-XXX, expect open for

our Phase We are have excited six X/X throughout to planned portfolio. near-term trial initiations

clinical span We at to are incredible advancing we that into R&D updates and two our candidates days our progress in Day forward providing making look will June. additional product development,

to our platform technologies. We will advance by pipeline our continue leveraging next-generation

platform a minigene Dr. technologies. push AAV continue of to gene Taysha to both to constructs we efforts limitations has with With Cleveland to a will Kamran at conventional in effectively for current broad the Southwestern our treating barrier recently further over treat As range part our review Dennis collaborations evaluate financial produce of of established bio genetic the critical novel of incorporate call have boundaries enable vivo innovations turn will us Yang range the that Halos, wider to in Genomics potential gene that by CNS at This Lal results. technologies packaging Institute, may and constructs Through we a treat capacity, vector and of this will and to AAV with Xiaoyong partners, next-generation CNS strive enable this collective overcome minigene payloads the addressable is Dr. studies. diseases, devastating vitro I’ll UT the and for in that, not us and therapies. the diseases engineering vector developing which Yale to our therapy diseases initiative, to Clinic, University of

Kamran Alam

I found be the Form in our shortly. be discuss of details will financial aspects key you, which first XX-Q, with morning, XXXX quarter filed More This SEC our results. Suyash. Thank will could

March activity a quarter ended release XX, headcount. result primarily of ended was for ended development first the related press As March XX, XXXX. and our were to services The related primarily was March manufacturing other professional an the The increased first and $X.X headcount, expenses. ended Net the corporate and in million XXXX. to March the from increase $X.XX fees, million for loss XX, increase were for $X.X first first in costs, XX, company’s related compared increase quarter indicated R&D personnel due for resulting quarter first ended March million $XX million a the quarter loss preclinical XX, today, increased as per clinical expenses or quarter share million of quarter XXXX, to compared XX, the expenses was million $X.XX $X.XX XXXX first for compared program spend, ended share March for to G&A $X.X per net as or to XXXX $XX.X XXXX.

XX, $XXX.X million equivalents. in of cash cash and As XXXX, and March Taysha had

our will today. regulatory call working We continue RA fund operation our will milestones that And of that, with back hand expect and into development, Suyash I operational RA. and the to to XXXX to be outlined inclusive have sufficient capital the

RA Session II

Thanks, Kamran.

quarter. to the first pleased over our with shared you, are success We have

the XX our focus will we with advancing ahead, continue next anticipated rapidly on over to many Looking months. milestones pipeline key

have an a to gene both stage and by made TSHA-XXX that acquisition CLNX we year Phase TSHA-XXX, or with on clinical X/X human a end. a in data our expect disease IND. gangliosidosis, TSHA-XXX has we in currently pivotal Further, first provide clinical well remain as We and GMX therapy of company and as track regulatory report for significant updates initiate open transition into to trial

that programs preclinical of pipeline extensive an have advancing we are today, quickly. heard you’ve As

for a completing year IND approximately corporate Dallas to headquarters syndrome manufacturing including our creating to our additional continuing milestones. five open initiatives will CTA-enabling syndrome with of development, and year as of We Rett clinical end, six continue expanding construction an well support Carolina, have CTA mid-year, and total programs in our facility build-out INDs four potentially currently in employees study. to by team term internal In expect North Durham, to SURFX-associated and near by as or R&D we value numerous by end XXX our by the GMP on parallel, or in programs have Leigh

R&D a next to period, continue our our We providing transformational expect look this month. be further we year and event progress upcoming updates to to at forward on Day

our Lastly, scientific to to to to begin every to therapies the Taysha continue like I that Q&A advisory Directors, now board, of will develop I dedication ask and gene support session. day the and on disease. our devastating give our collaborators, advocates curative mission special eradicate who monogenic Board our motivation thanks would and CNS Operator? operator the of patients remain continued employees,

Operator

We’ll now [Operator The comes Goldman go session. from Richter Instructions] first begin the ahead. question-and-answer Sachs. Salveen question from Please

Salveen Richter

Good questions. for taking morning. Thanks my

and employees. question here allocation morning. one So a out good multiple here running One about resource capital trials you’re GMP as and building question about – facility hiring

the you’d So how should clinical program, we what the that you here think to path pivotal And to like on if could Rett about regard to maybe see over inform from the program. time? secondly, and registration with touch that first data set

RA Session II

the morning. I Good questions. appreciate

if Rett. address on Suyash the and the could I’ll So question you take first

guidance reaffirmed capital we take capital the that us far to have As into we this as XXXX. allocation goes, still our morning resources

of development As it high trying from kind you identify different than throughput cost pertains kind already target, therapy time a development, much because not and tests target. of drug the I gene drug it’s to to screening, doing of classic you’re a know

versus much capital it’s more actually gene efficient or And go to so other modalities. after therapies other forms

translational And we’re programs moving some some are at our our academic your efficient a And Southwestern. really work is programs. discovery kind done that early way again, just have so about clinic, the programs discovery concept of work. animal this our staged, that the is discovery appropriately into because is, Most and having thinking have collaborators capital by of translational way that being partner is and early hit UT of that proof we to portfolio some do

clinical of to compared proof to gain, capital some a of small As efficient pretty the you it other human need signal is to concept development, and quite a achieve actually modality, pertains this patients to modalities. get because the number is

again, think, clinic, moving very And our in programs us that the do we the question this we development feel there. address Rett having on our capital stop feel the I kind approval? still this of So you clinical and I’ll syndrome clinic strongly thoughts few already year into and our Suyash, to strongly are a in into about five programs what want XXXX. path the to around path takes

Suyash Prasad

Absolutely. for Thanks, question, RA and thanks Yes. Salveen. the

thinking cautious Rett high-risk approach for GMX, is take of a the are an other going and little the where been development there relatively quite common And such lot as and we’ve conditions of think approval. the for CLNX some of syndrome, ongoing to less pathway program on. a GAN a our more where time early slightly we’re clinical So diseases I about mortality to spending

So way the study that Phase of efficacy. of think be study preliminary with primarily Rett the group about exploration two X/X safety a is first a will of more of some we

you study. take from perform from takes on will applies Following Phase a then more expensive – focuses it pivotal initial it into study, and which X/X a learnings from the study, Phase the X/X efficacy that, Phase X/X learnings that

regard Now to do we’ll older the first patients. Phase hit X/X with study, all be the study likely

of first FDA are away towards to these that adults this and of agree with particular situation, children risks As from toxicity tends with you we in push over-expression tend MECPX. know, you actually There to approach.

this quite So when we mindful to design just have we initial be study.

be at terms the safety a be X/X with Phase the question first the in initially. safety, rated behavior be example, at assessment, then adult at, different will aspects efficacy we’ll in efficacy be So pivotal population really endpoints for syndrome motor The number firstly looking looked And will of clinical efficacy the primary Rett looked Rett are. of three the clinically study the behavior of Rett-specific buckets. scales, different syndrome safety in will

with are because looking at have seizures also they seizure Rett syndrome some detail So Rett significant children we’ll in activity. scales, the be

apnea respiratory many Rett looking seizures, of the triggers systemic, bucket rhythm issues then and be how aspects seizures how assessments off kind on, include to issues, which, them durable characteristics, syndrome activity cardiac as we’ll type know, the the seizures over what see multi the sleep respiratory So see in at And disease seizure EEG. Rett and are are, hopefully medications improvement general frequent as bring you multi-organ have medications such in will the such time, of how in an be assessments, reduction as you also and the syndrome, will a third prolongation. QT able a

design that once efficacy, study will safety build some and areas that that. of preliminary and think look again, first, at Phase on I the to will So we subsequent initially X/X of the these

As starting engaging we’ve around the we’ll during the end our thinking study to we’ll talked test of be this of be year. towards already the plans, clinical and the agencies these year about, with particular pressure regulatory course

Salveen Richter

Thank you.

Operator

Harrison go question The comes from from Stanley. next Matthew Please Morgan ahead.

Unidentified Analyst

Matthew. Can for on you this what is morning, in you the an give Thomas need work program, you you. with manufacturing still Thank where particular, Good of update to for complete? are GAN do on assay sort

RA Session II

question. the on Officer over talk to Thomas Thanks, manufacturing. Technical turn question line this the I’ll Fred about our Chief for to our Porter, Fred?

Fred Porter

Yes, thanks, of program. the RA. for question, obviously GAN Matthew. the in onboarding Yes, Thanks we’re process the

X, the Phase a And for so to NIH late-stage qualify we’re beginning intention them for what methods to the the assays, were prepare clinical work. Phase is and assays validate material, then that really and conducted update X pivotal to our those try two is the where just with by reviewing

So, engaging forward partners we’re all attributes with CDMO. with critical our quality move that our on actively the to assays

any something deeply synchronize a quickly qualified the to lot and very of move questions assay very Suyash’s happening, my looking a that? we’re kind assay addition, with is own answer and In potency work, I’m forward developed that’s potency pivotal potency manufacturing. happy this development group jointly fully assay about and to between to into

RA Session II

add I one question Fred, top more important about of on ask it’s assay. can I the and to No, comment an

spending and really effort in that The the say is DDPCR. GPCR the is – trigger assay considerable with some the time to on some the about more pulling thinking other we’re

So terms get slightly more some in accurate titrate. numbers of we

So that’s you. potency another arm the other of CMC patent parallel with the mentioned. Thank that Fred characterization of

Operator

one, ahead. Then from the William Instructions] question [Operator Prasad Blair. from Please Raju comes go next

Raju Prasad

could the Hey are technologies my regulatory for I de-risking indications you’re platform, pipeline was I on more from the kind see taking vector, and miRARE question. that side. bicistronic technologies the Congrats once those looking guys, on a a of perspective, lot question of progress. I’m thanks see payload down the a the derisked. follow-on your But

on dealing it think the programs of types will indications, deal there would Thanks. some IT great. of it the data endpoints Is on administration? of do aspects these on with regulators what different there? by the color Maybe kind Is you’re be FDA? you with clinical derisked be As and endpoints

RA Session II

really No, it’s go that a constant. to And an our we’re allow thesis. couple of of with large scientific novel kind really on to focus great product able to central hold us of of of the use question, and technology this after payload candidates. targeted kind kind it’s trying to we really very that gene because design our economy of validated and therapy portfolio things And scale to do coupled a achieve

economies kind allow programs as HEKXXX take thing, things from from manufacturing thing one around to really is learnings to AAVX, notion talk pause this and I’ll how intrathecal of you first platform. next. the use there, of achieve next. to delivery. with to we and some the a really they discuss program of scale of second of and are us to is our the third plan we allows learnings And program suspension one all those plan I’ll And to maybe the the The that apply regulators Suyash, all this about

Suyash Prasad

than for commonalities, and thanks many, other and RA think, – there’s and many HEKXXX and our AAVX, of the lots above as we There’s administration. platform the – more I think IT we Thanks, between comments anything. Yes, a shared over lots about question. Raju we simple commonalities, of programs, the I and trifecta

with lot contribution touch several to of anaesthesiology to one ICV There’s I’ve perspective versus amount administration in a decades. the all. keep huge has them program Let IT on I a to coming about of there. in for ICM me CLNX, think another CLNX, from of world beautifully. the and and data GAN from at other details. comfortable look that, will build programs, intrathecal continue and many administration. nuances And works it FDA we works, as and to become inform just think things just I that it’s a oncology worked myself the there’s and and but many works can from And back portfolio worked we I next. increasingly clinical IT I the between really the we’re the versus given you around in of couple for it going When think decades Zolgensma, field regulators you – it learn our see debate

type and you these spent some comparability talking – might we drug about of compatibility different of you do methods some intrathecal for example, need might test administration. to time For give of kits a different the use to think yesterday

So learning, the I that GAN think rest portfolio. our of of particular there’s lots from and lots inform in will

programs Another as the our piece of regime just learning, GAN on I think we like use. that’s from and progress, to important immunosuppression

and very decided was and doses try have in prednisolone rapidly and any When months has this rather therapy then let’s of now evidence been inflammation have of on the the GAN any regime XX a of additional successfully. with learnings GAN added. past very So expressive evolved plus cell-mediated immunology have that from settled prednisolone regime didn’t nice manage any point this that prevent number information patients this to study. from oral – specific medications of where give liver rapamycin the And T people very, And years, patients the lot the just over with we’re very evolved it. seeing To world a six of the and the initially, of in of whole experience first with than been of program, we we’ve oral get immunological regime reactively few therapy months not the received actually, treated who gene prednisolone. of any and therapy

to of that I that regime. going particular for GMX, CLNX, using we’re in in once body in build evidence We’re approach And again, SURFX. think up this

endpoints you mention, it learn and third one the thing on we think clinical is trial in to what other. and I the can touched I’ll from

I or know our called for more to failure scale, nice features. are milestones. and lot particular note the a rates assessments. these a been development to development clinical to regression assessments. and a a set to have diseases, a a that videos how We other. externally blinded they CHOP train where the to there’s second that the these development train way, on do robustness All by of the think group one rater can one of then group know a the or to these neurological when where upload we’ve raters it We things We of of of treated second And two And who are not. GAN to assessments specific. Vineland, server, in a these to very of a the there what the patients video, particular INTEND. had There’s assessments rates Bayley the reviewed disease the from are the learnings lot program to do others particular that lack how be

EEG, medications, few ago, we cetera. we was when on, talking mentioned are seizure how I collect thing patients other seizure The the about activity, minutes Rett were seizures et the information, a just

on the learnings question, constitute all our other. Raju. So that and clinical think in to be many, the additional to there I applicable the answers one discussions programs in I with will to I from are regulators, particular, many commonalities, hope development going are your that side, think but

Raju Prasad

a point. on extremely last that’s follow-up quick that helpful. Yes, Maybe just

on pivotal, from thinking potential GAN looking control that it discussions the placebo treated mean upcoming kind different the natural we a with that As those at the I be the I’m try agency. will FDA diseases? or to particularly how history that on they GAN discussions you’re are discussions that’s GAN of discussions pipeline? as comparator some about run give be think a only Or might results a request kind do and versus of to kind the FDA. you you arm. relates different of each probably the see you rare individual a having comparator and do be it the the is indication looking rest something to of natural should extrapolate that to relates the something Like history of for to going arm GMX to as program, of to related the for – if Thanks.

Suyash Prasad

particular is a are program existing effect about and CLNX, from how gene a a for like historical and history data or a course a bit treatment differences, What practical large, programs I comparator program. also And GMX therapy for guidance clearly, where natural be for neurodegenetive very, the the clear also disease. very well is development there’s be true, is higher of in specific very have it’s They is ongoing to product therapy ethical, not also where there important the is neurodegenerative commonalities very – talk program, may it may suitable disease is already So most If serious appropriate this our treat point. programs, on or of current study from controls. control. subtle – proven expected mortality. And the things inclusion is nice risk by as a higher was a there some to level control are a there need, on certainly case of rare little unmet use disease medical much are which published documented. which for The and natural differently for that the absolutely a disease. doing to certainly say will history the said section FDA it’s but They is gene we’re there some really

patients in Now for XX with the of in study that patients we were patients, data, enrollments fact, that, we’ve GAN, XXXX. have specifically, natural in data than XX more history presented

that got efficacy (X:XX:XX) So MFMXX And it’s these per of on points drop rates a some X the very, very patients. in indication we’ve of data endpoint, clear primary there’s [indiscernible] consistent the year.

of predictable. disease The think course I also is it’s because that, predictable.

– study. GAN, international very our in a for and effect so With nice disease. seeing rare very treatment we’re And

appropriate the I So the in particular, but you I all say, comparator. to FDA GAN, natural know those think a being is what all you’re boxes the – don’t think to for history an really it things fact that checks going contribute study for

out they you that from all do the to more lot good in perspective. control. us going a data but know ask already will there’s is FDA say, my So it’s Once it of to history again, GMX, kind checks boxes unlikely of the there formal are concurrent don’t any natural guess at publications.

is XX patients And to it. CLNX, there natural prospective so history with ongoing a is XX that currently of international. study in we’re making use This about

using So we’ll be that.

So program. little syndrome, different it’s databases history bit that is from to available. natural there Rett huge are program a

concurrent likely randomized, non-blinded Although but concurrent a clinical To little robustness we for control a for the more in will build add plan. development control. Rett, a

us So some let there. answered me I your give start and I can stop. hope but context, question I’ve

can we that think I now. if detail go into but like, of Raju, most more you

Raju Prasad

extremely the that’s No, Thank question. helpful. you for

RA Session II

Raj. Thanks,

Operator

Eun Yang from from comes question next Please The Jefferies. ahead. go

Eun Yang

Thank you. Thank you.

gene look are So you been today, of in TSHA-XXX kind to you Europe? talk and patient But potential U.S. about and programs, the thinking we population a your for when now it’s therapy of address market U.S. focus Europe. the on XXXX, outside about what approval the opportunity the

RA Session II

a genetic and world. the of the question. That’s and name Thank the also that leverage or access to fact disease And almost over-expression about where diseases. that by reimbursed initiate all you’re early And you of markets in what the for the over some approval approval able rare I is able question. mean pretty market patient you’re great an programs you EU, reimbursable the commercialization what’s in much major interesting to program U.S. have more immediately highly or a

post marketing name under reimbursed that approval to program market reimbursement where early Brazil, some a Saudi a over American would Latin program actually so of either patient seek countries, the these Arabia, the eventually or Middle approval, we time. the you good. companies look Turkey, East, have And Israel, countries a commercialize are would region in access GCC like some would Colombia, actually areas of – major quite

we’re So that the about way that’s thinking really commercialization.

gene that would established Middle East, high-priced likely this through do therapies, in are Israel, We particularly your we some for reimbursement Brazil partners, of looking of globally in achieving a skilled specialized are There’s number products. products, the partners. most distribution more highly Turkey,

perspective do countries of most some likely on, but early mentioned. product And out-license commercialization look probably would in so We commercialization go our route. I to likely that our this a distribution most collaborations through not from would we really these in

alter time way. way as least thinking commercialization, that the thinking approaching things way But goes Obviously, more that now, plans. that’s that’s gain and you information that So we’re pre-commercialization our the we’re today. you about your for at on, our

Eun Yang

you. Rett question program. Thank one have I on syndrome And more

to program. of regulation sure a you can compared how kind with a but that Rett I’m So from actively know their you. talk And program, the Thank pursuing transgene I familiar aside don’t it, of expression. about have about Novartis differentiation program? much Novartis and the Can Novartis, your speak you you’re potentially better

RA Session II

the question. for you Thank

kind just the highlight difference the So two talk to over And then I’ll of do depth. kind it turn the Suyash different approaches. in main about to what – in differences more the the of I’ll is

a some therapy you the feedback on in at main cell with product, bit gene was or the last paper That’s the through detail program, the self using. cell are that by we’re want the little was safety has gene essentially Suyash, described a AAVX two Essentially, regulation is expression into there. a link in is we regulatory doing transgene construct basis publicly recently is least we maybe no full expression. aware having as to that of. that the construct difference published was built – in associated So our Novartis guard regulatory really, AAVX, the gene we genotypic that programs a what caps the with all nuances. is term that stop go published of that component. I’ll a TSHA-XXX, overexpression valve AveXis self That of more we’re The what is complementary against MECPX toxicity. with to

Suyash Prasad

the question. Yes. Thanks, RA, and thanks for

an question. think I it’s important

include gene, highly experts which developed major the and Rett mentioned, to actually only – very reversible first and was ineligible that is we was RA syndrome from As demonstrate Rett MECPX fact who the the the mini we have person a Sir Bird Professor is disease. esteemed difference by Adrian unequivocally Edinburgh that really –

attach for auto gene. use So we his sites, of RNA design RNA binding micro for then responsive this we the element. miRARE And strip which a micro regulatory MECPX mini platform, of the stands

platform, this levels a as MECPX, RNA up consequence are of the of triggered, in sale of binding the a which regulatory micro valve. construct region therapy, they bind the gene miRARE as as down RA suggested, MECPX the bring untranslated to down when within go acting the levels and safety sites So is

miRARE to quantitative the first you in is which authors I’d where specifically on you so friend published we in efficacious, our be expresses encourage Sara paper, say Steven over went that enough is non-miRARE excited of and MECPX the in is have at, that different It see parts construct And looks paper, that was online expression toxic, there’s of is and brain, not a different the senior of you good it’s the data whereas expresses different enough issued [indiscernible] that which and the release but author MECPX in Gray, can Adviser, and diagram very too cord. very to a nicely yesterday. the Scientific lead point (X:XX:XX). I reduction brain very efficacious, colleague, able expression at Chief much spinal Sonet parts demonstrating we’re at the Friday, this to of which to Now levels look but construct a are you toxic. it suggest prestigious not that particular you press so the look And would to in

very excited about fixer that So paper. we’re

we are normal I heard really that that don’t to an difference. the we’ve physiological the main MECPX is Last – the understanding My between the of parts I So but Novartis as difference move parameters ability quantitatively program. fact still brain, unless. we’ve this demonstrate in I and to shown that is well. that. shown planning moving different IND, The express main exactly they can think where that’s forward products But I within it our the these now with know think forward with that’s was as

Eun Yang

Thank the you details. for

RA Session II

you. Thank

Operator

turn call Mr. will now for closing no the remarks. are I There Session his over further to questions.

RA Session II

again, joining the we everyone So on call morning. us appreciate this

the the made progress this And the see, company during rest quarter. can you throughout great first we continue strides year. has of make to As

guys June, joining. we we’ll year and further our on. good June the and so that as Day, over place day. Have hope in updates XX, two at for a will provide days take Thank you R&D which join And goes us June XX you

Operator

this gentlemen, participation. for once presentation. today’s your concludes you and Ladies Thank again

You disconnect. may now

Taysha Gene Therapies (TSHA) 11 May 212021 Q1 Earnings call transcript