TSHA - Earnings call transcript Q1 2022 | Taysha Gene Therapies, Inc.

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

Operator

Good morning. Welcome to Taysha Gene Therapies’ First Quarter 2022 Financial Results and Corporate Update Conference Call. At this time, all participants are in 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 16, 2022. the to turn now will call Dr. over I Officer. Lee, Chief Affairs Corporate Kimberly Please go ahead.

Kimberly Lee

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

copy Founder, quarter RA Joining Officer Medical After of Alam, II, commercial call our on relating filings. Kamran press Head Dr. are is and Please candidate. to the Financial company’s timing me of Taysha’s and statements Prasad, a our forward-looking therapeutic remarks, efficacy ended we today’s and the SEC on product for investigational of time. that the product Earlier President, Chief and making R&D; that will this follow call we at today’s available results A These XXXX. results will expected Chief and conduct XX, safety formal financial and a Taysha may and March the a on press CEO; note Suyash Session issued will for question-and-answer the our announcing statements session first potential and of release Officer. include trials instructions and release today, including clinical through be website statements, candidates.

or stated are of as risks product uncertainties that to discovering actual product Various statements. strategic and candidates, Taysha’s studies matters clinical intellectual those the approval dependent and regarding of activities. or from well to our timing of plans. obtain our and necessary to and data research expectations as and by our operating development implied or by to materially support TSHA-XXX limitations may also programs to discoveries, imposed forward-looking call status relationships, include relating results protection results Taysha’s This development patents in Our related are conduct the other strategic requirements of and and well successful results, cause These future as statements and trials preclinical patent upon funding regulatory growth market and alliances candidates regulatory for and risks differ those the controlled third-party for owned third-parties, manufacturing opportunity historical of alliances such as may ability the property information. not Taysha’s forward-looking contain facts to

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

RA Session II

by potential a to development GAN Thank initiate have need regulatory which We Rett, to our and call. a European financial quarter year pleased development a Commission our Canada XXXX XXX,XXX disease focus look GAN Health orphan recently advance treatment results safety end. clinical treatment approved forward clinical from of syndrome. axonal designation you, core over and the to for to clinical efficacy for CTA for giant providing therapy receive were were and forward gene first we further in Kim. highlighting mid-XXXX. update, Phase continued particular conference morning and Good update our first last welcome Since programs, look giant the modifying everyone on and corporate options affects with worldwide. X/X pleased patients patients. the neuropathy for update TSHA-XXX provide preliminary reporting drug also to to by In development or the March, TSHA-XXX, Rett We neuropathy of TSHA-XXX We axonal unmet these for only by we in data and under to for

executing will the expenses Suyash into more and over pipeline fourth we a strategic Suyash, quarter to detailed on capital focused operating of loan along facility As requirements ahead, our remain look resources on full prioritization existing on to our XXXX. the turn ahead. now clinical programs. initiatives we term with fund update I our provide to access our current go call and to capital expect please

Suyash Prasad

Thanks, RA.

Taysha Advocacy known GAN to we and which To UT exciting study a development in As the Syndrome controlled syndrome. and of initiated TSHA-XXX licensed positive efficacy promising of acceptance, throughout of presented do American developed therapy that MECPX. CTA provides expression Scientific will development correct of International Sarah Cell in These support as Therapy, progress & data non-human expressions including week, of safety National knockout Rett a MECPX, data Rett exploring year. in Gene a transgene TSHA-XXX program, will physiological the have of The TSHA-XXX ASCEND and the while Society data of and Collectively, to in the expect challenge primates is or programs of a we MECPX of regulate auto-regulatory CTA enabling clinical to clinical as distribution Annual as expression miRARE its avoids significant sufficient appropriate and presentations mechanism confirm X-month expression ability platform Gray by ASGCT, made safety recent RA of of this, toxicology appropriate milestones and safety at address we by the physiological results platform the the Steven Rett transgene therapeutic be excessive the prevalence ensuring gene recently transgene on levels miRARE study and the rats. and ability cell-by-cell Rett will with expression TSHA-XXX well presenting data we Drs. pharmacology Meeting and have preclinical GLP these the We a an other the noted, under strategies. deficiency, IND exclusively CTA of regulates protein TSHA-XXX neuronal in expression expression responsive Health preclinical sophisticated of remainder is Conference distribution is further control novel this avoiding the essential appropriate launch basis also levels. toxicity. In for Center. of supported our of limited Patient to the that Rett Medical Canada. regulation approved the within TSHA-XXX. action levels development challenge of will further in MECPX is syndrome These with Sinnett finding replacement and I elements the mice, that using assessing Summit. begin function. by Foundation and overexpression miRARE whilst Syndrome associated gene Southwestern effective replacement microRNA and of the for of a microbial the for of XXth gene balance have our by ensuring NHPs This support

are over patients Currently, indicated, therapies suffer from there as devastating Rett patients RA to months spanning no As treat into XXX,XXX there to this disease-modifying adulthood. syndrome worldwide, young are condition. estimated X as

intrathecally. will is vg the We safety are Up clinical be In X/X XX Rett excited for and with cohort, of the Rett Phase escalation be given randomized study to to enrolled. will in patients that gene REVEAL as XEXX dose female advance multi-center But dose TSHA-XXX syndrome. first syndrome. development in open first TSHA-XXX efficacy a label an study examine adult the will of total clinical the patients in TSHA-XXX therapy REVEAL trial single

neurophysiology and and Dr. of The will imaging designation the Canada Principal preliminary life, We designation Quebec, second University Rett-specific to orphan in European assessments. end first-in-human of TSHA-XXX. data recently direction FDA of given a total Commission. XXXX. disease has quality include as granted under designation Assistant Pediatrics trial Investigator. Center rare XEXX Montreal, and forward reporting cohort been assessments, assessments Professor, dose look and and the biomarkers initial Elsa kind be global from orphan Mother drug will vg Child Sainte-Justine of selected TSHA-XXX and the from drug more clinical has and the Key the pediatric Rossignol, site of been Hospital Neuroscience by

vg GAN, meaningful Treatment sustained TSHA-XXX. durability. across demonstrating or of doses. therapeutic halting year, TSHA-XXX all reported with slowing cohorts Turning of across dose disease long-term long-term this new significant data treatment safety nerve by earlier fibers data X.XEXX therapeutic high with positive statistically as dose total clinically we progression biopsy the data intrathecally well durability of to clinical the for cohort achieved delivered of confirmed Notably, and efficacy provided nerve TSHA-XXX or for all safety that active a and following treatment as was regeneration for further evidence

toxicities. All to of events or doses no preservation therapies measured observed events acuity and were study to increase high related we no scale visual gene transient were coherence adverse There comparable addition, dose optical LogMAR and other no nature. limiting the in by and procedures at as significant issues, safety tomography. in immunosuppression adverse In

potentially of or testing large orphan received options designation in support this supporting no completed recently and and for root April, approval. the a expedited TSHA-XXX TSHA-XXX drug batch European in regulatory GMP Commission underway. in release is batch representative there dorsal evidence treatment is inflammation unmet further Lastly, GAN for currently thrombocytopenia. Also ganglion commercially We from need a for

that and of routine including GAN ASGCT the Bailey, EMA. As the of sponsor marker with be ongoing are and forward charge update a designations of data screening and on discussions requirements TSHA-XXX at GAN. a a of vagus syndrome. Prader-Willi the the meets and registration will potentially disease as GAN. through patient hereditary reminder, agencies as and awareness generates potential regulatory the testing system on anticipate therapy the path to panel from FDA some therapy regulatory meeting, regulatory syndrome Rachel be delivery of in stage Department of diagnosis pediatric and to neuropathy previously rare inclusion on to tauopathy, Charcot-Marie-Tooth pathways Dr. positioned leader, offers advance for be also help Partnerships registration translational with a comprehensive will TSHA-XXX on dataset received Professor also and We autonomic collaboration in on free At Assistant believe GeneDx, continued TSHA-XXX drug the a Angelman programs, approaches neuropathy on regulator in most further the hereditary presenting the and gene FDA. healthcare our genetics UTSW orphan to TSHA-XXX a Excellence well Centers by in Association to our GAN GeneDx major innovative genetic with of look upcoming early data preclinical remain in testing Pediatrics well for development. foundation gene TSHA-XXX will mid-XXXX. providing We the other of raise professionals derisk There groups the symposium treat data advocacy a approval strategies nerve There nervous earlier dysfunction presentations

mid-XXXX disease of by results. first Investigator to for proof-of-concept study SLCXXAX by for GAN initiate review Vice Kamran? Principal addition fever construct expected and in clinical Clinical development ongoing development and In TSHA-XXX the first disease during Rett intend on call I preliminary remains Kamran in to With regulatory in under CLN-X we Research remain clinical the safety development TSHA-XXX TSHA-XXX and will Chair focused for the in and over turn also generation human of deficiency. we for the to Dr. X/X CLNX in that, end, of data with Translational and course financial Clinical efficacy small our of to and XXXX. Greenberg, Southwestern partnership UT and of Ben TSHA-XXX for Phase continued syndrome leadership year our

Kamran Alam

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

employee increase million $X.X an compensation. in XXXX severance which expenses of X costs X included the our were $XX.X As million $XX.X release compared XX, million March to $XX.X strategic attributable programs stock-based months and indicated completed was reprioritization million and ended XX, of XXXX of months termination for primarily million and compensation, non-cash XXXX. in for $X increase of with the one-time to the March development in today, press connection $X.X March million ended in The research

months million expenses research of March material raw increase we purchases. ended development, $X.X and Additionally, an other of in XX, the X incurred XXXX, manufacturing in and

development non-cash of million March and expenses $XX.X million and studies increase We the for $X.X million compared to incurred one-time XXXX. related million of primarily attributable March research to ended ended GLP for General stock-based and compensation clinical was termination fees months X toxicology an $X.X The of XX, approximately of which and expense, third-party million severance compensation. activities. incremental million costs primarily the XXXX also increase of were in X months administrative $X.X included to $X.X XX, million $X.X and study consulting $X.X

was Relations, research. loss $XX professional an for March accounting share fees loss compared for We $X.X of a months to million million market per XXXX or the insurance, XXXX. ended the Investor or in million and communications, X related as to share per XX, net March $XX.X also increase X incurred Net XX, ended months $X.XX of $X.XX

term March excludes shares from cash XX, $XX.X hand $XX and full into to facility, with XXXX. of the call fund proceeds Taysha and is the and operating in existing the fourth to cash balance expected cash sale quarter our under expenses of cash along that, capital to XXXX, back And loan will I equivalents RA. of our gross million existing access Current requirements with equivalents. ATM or facility generated This As had April. common million in at-the-market in cash

RA Session II

Kamran. Thanks,

executing Our our and focus team’s I extremely proud programs. development our quarter. And to across on accomplishments for progress core be of XXXX am this continues

ongoing position the our I our runway to maintain thank disease. to Scientific eradicate Taysha operator of gene should will now mission a ask continue We employees, monogenic of support quarter strong develop and XXXX. therapies the advocates Directors, for would fourth CNS patients begin Operator? to Advisory the curative Board, that like our to session. and of Q&A collaborators We cash devastating their Board provide into to

Operator

Lee line question. [Operator proceed Thank first you. Please comes Our Truist of Instructions] your from Securities. question the Joon with of

Unidentified Analyst

is Hi, my thank This Joon. good for morning taking on you and for question. [indiscernible]

you. how might by you probability commercial patients ask would think – and would data using the you takes to data? you efficacy provide to have FDA which Thank material new is you on it long what think take grade So such

RA Session II

the [indiscernible]. Hey, for Thank question. good morning you

we current think able through cut two year the I the to to look essentially this this, really FDA, being being the actually which we how we gone U.S., at essentially scenarios for pathological biopsy including being file data particularly therapeutic XX game thus of nice We X.Xv kind have as when the them have see of scenarios, data have nerve and with this far. high durability. safety all we really fiber from reported really down around that that dose breaks and dataset presented a And the where Again, efficacy the we dose one data changer. immediately showing view definitive parts, generated to earlier really we cohorts, one the three nice now them. earlier off we have the ex-U.S., in about first the year, regeneration, we

performance, And each for also and so are been clinical this on show the XX we really at nice which large entire show data and not we safety because but natural show are history we are dataset, compare to history, the cohort and about their and their data, study from because three over a history really patients has rolling a also natural patient patient’s performance. only control. this such we over provides to you of going able years. XX biopsy point, – between controls post-treatment but study efficacy of but essentially age close pre-treatment able matched comparison data, has to levels excited to own And natural pretty then The clinical, the a patient

really are excited we this again of data. breadth about the So

more likely we handful probably most during patients And between pathway our patients. that using similar would to they of likely patients, of would base registration already ask Those be asked it a what grade X a commercial identified. X the As Most consider you are case this [indiscernible] what the is zolgensma. I patients would to dose is mentioned, material. few of a FDA the somewhere

timing recruitment patients history any would So would natural perspective, this from rollover a they type effort essentially the to of get these because shorten study. into study, the from

steady wealth job we So data moving have and these keeping that generated at under identified patients the fantastic of done of that who our from NIH the leadership that Bönnemann a already have study. is Carsten and are the partners

patients. see and based current few filing really dosing more we dataset off the a So, difference immediately between the

It’s identified. about to we a looking are time because go X-month have time patients, at patients identify are a We huge again, out probably don’t already difference, and difference. those not

the to And up hope of an a We by MAA that This more to current requirements by would we patients. won’t is back us again data approval submission the would set the approval more so we is think for The shifting also end dosing initiate dose the preclinical to XXXX. ask off The that conditional in the FDA is they probably patients FDA. data us clinical meet year. the The the few be that dataset, if towards Based a FDA based default filing scenario. focus as going end just asked dataset for it would are the a final the position of is we’ve would near the EMA all rolling and ask of we a initiate generated. change. off to few

currently start purity review the manufacturer And about So, we already not quite have yields, that our release able obviously, and run. would the we the the completed be have current completed testing but are excited material, grade which we the only analytical release this commercial from would the undergoing testing panel also when of product to dataset. the we of for

So our that’s kind strategy. of going in

approval to from that a going be in ask dataset, based and the rollover study. few immediately off would natural would our but more case go dosing patients history for NIH current are We base the

previously is before we it our be case. to have this that base so and something continues And stated

Operator

you. Thank

Please Company. with & Blum of proceed Our question line question. Needham next comes from Gil with your

Gil Blum

for thanks our morning question. Good and taking

TSHA-XXX. quick have on one We another

to more And how on maybe product, can idea with more us give grade commercial what be an do need you do many patients what you batch? your there? do Thank can So things you. this treated there is to of

RA Session II

Yes. for and I initiated Thanks. you is would will more Suyash on start It’s good actually And please asking. the this to do Gil. We like. a rolling question, really in, not But material. essentially, if year. manufacturing grade much Thanks commercial chime run this there

manufacturing to definitive work completed perspective. Fred’s support along clinical to current team and – a those release have are assay, BLA, team significant spot including team quite successful made give just We potency ongoing. you the that assays both Suyash’s the about. on, I am which quite around will And on on an yield just progress the and It run the to now analytics key has this continue be a And which material comparability to that fantastic from perspective run, XX. high yields that and idea, job with original just team purity to depending pleased dose has the continuing was the of run. done dose the just from key manufacturability. a the we The with are assays, tests team, a will on on X.Xv go The around I

worth are drug. over XX patients of We

an CMC. plasmid strength triple that also really our so gives just in transfection platform the just is of HEKXXX our around team suspension you idea use using the have This that the of And entire across we the platform. but one that portfolio, strength we

I more So couldn’t even proud. be

go made in could data the BLA, to – continue this has to the gotten gather is and testing run It’s team commercial of even do whatnot. grade will the And testing, this but stability manufacturer to imagine. we support progress as the better really this the we will undergo gone. what continue team release – has material So will this that

you anything do will stop have So there. to add? I Suyash,

Suyash Prasad

that far. very that that to of are looking basis thing the the essentially plenty make is and the promising potency demonstrate tested, we pretty only character and doing No, And potency correct, batches all made of characterization and that closely setting lot there an finalize mimics is would high the and echo keen or said, to level regulators underway as the RA product it’s made, consistent a and is you, is every ongoing have the we just met. the at does we potency exists run assay. needs acceptance are and to thus appropriate in to I the is are remind product I FDA looking lab quite release administering the product And majors the settings be outstanding of testing was highly lot is RA think in molecule progress fact to of there yielding the assay. just finalizing predefined need And clinical on we be quality. in it the assay ensure that what’s effectively obviously to

great. looking is everything So,

testing of the We pre-treating is way is available drug are to and to release imminently. going the patients be

Operator

Thank you.

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

Elizabeth Webster

guys how we what you cash many on a and data the our Hey, assumptions Salveen. are, for ‘XX patients taking XQ thank nature from on us Thank you see to comment first to guidance? switching then just good we one Elizabeth been Maybe This to can expect from? if baked morning, the clinical the see into Rett, expect over and you. could have for question. data runway of kind second And is of

Suyash Prasad

Sure.

clinical can Let RA the about handle question and Kamran question. me trial, second take the then the

To conservative. – actually be RMHP clinical we adult about a remind downregulate So in you, felt trial given to in Rett we overexpression, little data, very to still our we syndrome. bit the with a potential females with is just Rett this nicely with show the in concerns just regard wanted trial, which given

shortly off small to Rett are be pediatric we want boys rare and we plan starting point at thereafter. population so And adults the females some a into a with study boys, and plan thereafter will Rett move the in in children, very to of with then

with are sure the data adult anticipate to not terms be study, of out data, moment. preliminary we trial safety at data the to from some and year, to clinical going have efficacy the end we coming which to data the what of of we in are by the have Now, is regard expectations the likelihood sharing of going

those studies We is these We enhanced products, to innovative will, things. patients several the all with these target have go for once. they at first can’t case as

patient, have period patient dose a the We and the a with dose leave review second and with to next patients. a time then period for after then DMC leave one of of dosing time

is potentially efficacy of I small to this number So the to focusing data preliminary to RA year that’s of we data my early going clinical some Kamran guess and take that, if are of question. end why trial patients on a the are With regard think moment. at be the safety see some signs but by we starting that assumptions, I’ll let

Kamran Alam

Liz the question. I and thanks, was mute. on actually Sure for talking

from what about being And for Suyash excited just outcomes, the and collected the CTA, I we Rett first reported immense. are measures that Rett data, to echo of in quite are are are mentioned, So means well being think Rett reported to syndrome. the development patient this really Both respiratory the and reiterate Suyash to of what measures, excited syndrome just as some mentioned, gene the therapy, movement. the amount it again, be community only the that datasets neurodevelopmental totality supports actually Rett as endpoints outcomes, about preclinical what really clinical will physician data, on obviously,

MECPX what for NHP four study at or construct us, most be one the the presence safety study be be again, share going there’s of dose – is endpoints this of the safety to the only clinical MECPX doses So NHP certainly, ability regulate in probably decided the of the from we always talks of also demonstrated type start setting, fourfold our to in above into overexpression presence will is ones of wild the a adults. up proof to our in data the the whole of notion around to quite including that not it’s to why But type it’s because starting mechanism and this so but of the expression host down reasons really wild safety will exciting. important and and of one going

quite of preliminary way existing on there. base Similarly program, registration are made key our time. and get execute analysis the which something And and our Suyash it. be short assumption And more the in it’ll with quite So report program. of yearend this the parameterization to that that from I’ve is I current the still cash, risk runway been it’ll a to we And would be ability both ginixonaneuropathy From to the totality prioritization and a of assumption this probably two clinical development cash syndrome into accompany. drawdown ability in have through we’re this that of term on just did kind about really, is earnings period able to earlier director then to assets we’re about. And more from essentially, just in you SLCXXAX. – of again, I focus our extend What announced an And would quite, assumption, in that’s moving XX%. I’m we and team case and particularly gene clear is of thinking off but nose be loan out some Silicon that’s year, proud exercise of of of aware, executing. studies kind Bank, CLNX, not XXXX. our anyone really and grind taking on been based this characterize executed the of Valley perspective also included the kind runway, which sizing With as we about therapy, on small additional with of team those concept I’m our excited safety right be trial the is in – our QX of kind gone progress of the has the though, pipeline the also say a this full along a then excited data, the And for of to there, way that call, which focus two programs. easy of proof being we that that’s It’s able immensely proof of. with Rett and have efficacy biotech, facility a the

our into runway. baked been continue to cash has augment this and extend of to all us, analysis, at for look ways so we and And

Operator

you. Thank

proceed Our of with from Oppenheimer Please next line DeGeeter question & Kevin comes question. Company. your with

Kevin DeGeeter

questions. Hey, our for great, thanks taking

Just scheduling to on that, confidence update, mid meetings regulatory had cause that? agencies this underlying that timeline and are and of there it on comment this again guess is, at sort open could you FDA waiting is Are point? RA, that question conclude sort XXXX kind of your regulatory that the with timeline? can impact for other necessary some I they’ve reasonable feedback update of uncertainty components

RA Session II

Good morning. thanks, the Yes, for question. Kevin,

and the is comparability. thus data, but COVID environment that and promise multiple we’re from agencies a and the fact guidance have conversations again, I from the a reproducibility that EMA a what regulatory reiterating most study. think, efficacious, competent And now scheduling. of that’s of FDA, in But mid a there’s package other doses, in to some flux, a around would and now, far, XXXX. presented really Obviously, be been really there’s a people perspective matter in being Three with little durable the in that that a the So a the From a including said, safe product CMC we’ve of think the with flux only perspective, I at and dataset excited Module of not that the go really feedback natural bit a quite robust we’re nice history data beginnings of environment. in regulatory been COVID the

of in to to material. default patients and to be a off go dose base with our that able commercial file being to and grade current to the And goal going we’re dataset more as essentially with default case, we is the prepared the dataset. strategy obviously, few So

that communications and look year, this guys mid-year around having updating – We’ve you to in reiterating to but our feedback guidance So here we here. closer done the our forward is that to XXXX later are in again, guidance. that I

Operator

Thank you.

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

Jack Allen

And the much existing was then gears preclinical briefly just you’re on XVXX that, coverage dose, wanted at questions package you dose dose shift package? at that how on mentioned do I study through you thank back know taking to starting the showed you up issues the safety and where the Hi, there. guess congratulations total escalate VG all I preclinical so to moving and see XEXX. I as talk did really progress. and have the was I Rett you and for a you we tolerability to move there in the think package curious much the Xx preclinical to based dose first about

RA Session II

question Great good that question. morning. answer it Jack. to Suyash and over to I’ll turn Thanks,

Suyash Prasad

Yes, the Thanks for question. hi, Jack.

gene the first is The getting trial make really a to important, issues, given you’ve drugs, also sure it’s drug you’ve select sure drugs for again. there’s sure enough the you give safety So because make dose question. study, once no are therapy, a tolerability or take gene the but away important to really time Rett those make effective, for you got that it because therapy the can’t clinical

And So particular, where it’s a Rett, really made. toxicity. critical to decision over be is risk needs of that for expression in there this

appropriate Because produced we actually package, to amount make a study. did get effects. of you’re enough of the the sure causes side much very, that having physiological within it not to limits disciplined very that, being mouse a but So preclinical pharmacology included appropriate protein protein so toxic which efficacy, you have

to you assessments. other study terms we did efficacious. improvement very model enough and in assessments, identify for able to the be study, of survival, motor and of mouse did Rett to mice, an the clearly NHP very study study, we’re – we the going where NHPs. pharmacology causing over respiratory comprehensive. in rats, mouse studies a demonstrating assessments Rett And mouse The were an And study, that’s XXX toxicology pharmacology toxicology drug was was was study look XX over NHP dose all XXX able study extensive we is a were So a very that

at dose. tested And So end, the fourfold note XVXX your answer both to NHP in XVXX question, total level. the tops, that’s to VG adverse above dose of starting And an that and equivalents specifically, the dose. any of was over other total the Rett up events human the then doses the there talks, XVXX on no is we equivalent VG

go efficacy a possible XVXX, and we it about Wednesday actually tomorrow, naive big because we than this saw weeks presentation of tomorrow, important that. or at actually, really to in with it’s amount than we much spinal simply need a the Rett the could And would poster NHP test go from we be ago. organized there that, didn’t So, ISRA, at think think throughout three it higher it’s the higher higher And kind NHP it four was the syndrome, excellent – toxic higher Canada conference that be even a is also I because But this lower three at allow to important didn’t yes, CTA study. things, that does opened. shows I shows an it brain a combination think piece the effects, And it’s is persuaded have that that’s other cord. one presenting which of study Health pharmacology clinical I And presented really biodistribution think Tuesday, to by dose of I Tuesday. around the there I studies is discussing important. of the really really, absence actually intrathecal state be fourfold for scientific will any tox dose, and full ASGCT,

and So, And wild you in range, these it nice nervous forget, in one peripheral NHPs. RNA. of are correspondingly in type And two parts the different don’t copy system. back spinal numbers the copies genome a the very shows particular of low the ganglia are to cord, brain, getting also and to levels

getting but RNA wild not is and produced, type drug you meaning cord, are very animal. brain high great means that what delivery much this is a actually into down-regulation, of the given spinal appropriate So, being which

action. safety, but it shows mechanism study biodistribution, importantly, of shows NHP that So,

will more be And tomorrow. through data the so going ASGCT in detail that I meeting

persuasive week studies till the Health together, of group was that really So, CTA. Canada very in to last

Operator

you. Thank

with from Our BTIG. line your next question Yun of Zhong comes the Please proceed question. with

Yun Zhong

Hi. Thank follow-up you just much now for question said, This taking actually is on you just a very what Suyash. question. the

between And in this So, that human terms data efficacy? estimate do anyone also suggest on to to biodistribution? comparability many efficiency, see transduced will expected? looked not transduction patients So, need the and given are syndrome, any has have is there Rett cells non-human on to you be at of an reasonable primates how or cross correction to be

Suyash Prasad

questions. good very Two

done it’s But in specifically to course which blood at really intrathecal throughout the was the have very good nice injection. So, model cord and And the about than dose No an in of translatability with an to similar. biodistribution animal very human. one looking of NHP human. best a with the has we is Rett. get intrathecal the biodistribution nice sugar the any is And NHP, brain question What question. program shown is the smaller we human the being. the of have first with corollary probably we anatomy biodistribution done it spinal dose It’s into our Rett

human and sad patient away in the way biodistribution in only one early in to was that patients. to look DNA individual. and if ascertain Now, pass is that in to really very GAN protein setting, and the at the happen a And the of RNA, did on situation

Carson therapy the intrathecal years of data an And of by Palmer showed AAVX been disease, the ASGCT who Gray with has patients ago. and of GAN. presented sadly GAN they a dose dose gene for this few One progression through Steven away at low meeting that passed

low HEKXXX, GAN biodistribution, a lots actually thing from learned forget so level, we similarities and of and You the And was nice all will tissues. from GAN program. I organs, don’t although AAVX to low other the what program, dose a it’s throughout is Rett but it say delivered, target intrathecally got the

nerve about recently more other in have nerves, say our is, presentation January GAN biopsies. that I we The in that peripheral the we the demonstrated is see in will thing improvements

nerve drug in the down down nerve into to getting superficial peripheral the the from system, an the And this that which performed wrist, intrathecal is what that nerve, wrist. these of is biopsies are radial Now, injection. a are nerve in dose means is

confident So, syndrome intrathecal cord will dose spinal are That’s in transfused tissues for drug the all question. to be the first Rett and drug and are actually those the in of to that that deliver particular nicely answer which brain the reasons, of we very brainstem. needs

ratio versus versus, null of this that the to difficult transduced. to second better cells, And that XX-XX need The non-mosaics but had mosaics some be versus type get benefit in we more is, are some to you cells the if certainly mosaic that, clinical with are clinical about, the the setting, we wild it’s what you get. you cells – question know, proportion going of now was cells we of believe know certainly being likelihood And the benefit. transduce transduced, sorry,

of being give see of we is protein high the once approach, the giving know drug, doses dose to that produced, down-regulation And a drug again, of for which a still confidence, enhance intrathecally biodistribution. our to high we wild type have you So, cells. nice

some Yun? may But don’t exactly we think our So, be doses answers once more cells again, package how that are give we benefit. see number a Hopefully, number to biodistribution, your to A small we working enhanced know because high transduced. large question, giving many need nicely. down-regulation quite But confident will you I give benefits.

RA Session II

thing of I NHP mentioned particularly given the the opportunity set examples. only was fact been set to preclinical able I have The transduction wanted two are we what maximize reiterate to would the before, that the we including The that us I tox mentioned see really didn’t didn’t this just that would data say, add, efficiency. actually to we Suyash, generate, data just at to you is to – observe And that this. Suyash any XEXX. matter dose gives

able we the to able ability We we at able that always levels the starting biodistribution to increase transduction ability see You be wanted obviously go we to maximize at. that, really the nice dose able to again, to biodistribution. to to really to have be are efficiency get to have But be maximize be if and higher. nice

correlation something similar wanted does partners Southwestern. our think get again to is collaboration second MECPX. membrane under bound The UT make This to I nice being which point our study, with at that I CLNX it’s a to and a that is a protein overlooked is just done

safely. So, dose and to it’s to able up correction to not no we shown be – patients cross successfully do that ability here. the have able XEXX And to and there is to be

patient preliminary was early dose that the has there that been an earlier presented high and that year been has data World XEXX. at this since data has of is some Symposium dosed at So, which been additional presented,

first syndrome. the that’s where of gene you out I number giant think field really That’s the firsts I Taysha dose remind of for our again, with the history. UT the So, in collaboration therapy study, first Rett gene in like have and around centrifugally Southwestern, development come therapy to neuropathy axonal this

a expression first with loop on gene the have to control basis. development regulatory now in self You clinical cell-by-cell the therapy feedback

proud of existence. be construct, The intrathecally first safety to be the so that partners and and been There been that in safely field company like UT to do the firsts of that just generated to by have for of, be firsts able period I therapy of our our a at able very generated. to the number that number it’s am short been AAVX has demonstrate And always to time has company in remind that’s proud of the and an dose to XEXX been of employees of I something and the Southwestern. gene

Operator

Thank you.

Wedbush of proceed Chico Please next a comes your with with Securities. line from question. Laura Our question

Laura Chico

if taking to question. bit regards the can of comment earlier that back you spend, exploring I if R&D and additional am I more for an could Thanks Thanks you circle guys. expand offer the channels just G&A. kind on bit. wondering clarity And little you between with a of to little monetization, guys. for too, made RA, wanted breakdown wondering I about a morning, perhaps on Good Hey. think the kind

RA Session II

Good Thanks Laura. morning. Sure.

it’s specific that extending will of looking first, to forms we and announcement potential able around just continue made cash around to and us get would earnings calls, say are have are our strategic with But of look to whether consistently remain bring to in constantly non-dilutive the year-end business capital. are is Obviously, opportunities I an that, your something ways have So, last after really fortunate about for collaboration call. parties, essentially at I those lot be that opportunity that start we or we been regional go to we thinking assets. we runway. development at, with What at having a question collaborations collaborations

focus essentially, bringing say capital. that would so, non-dilutive would perspective. ones continued around clinical we proof-of-concept. a for – currently hit company proof-of-concept, and in that the from explore continue in, a development look is, potential way again, And essentially programs I also we those What programs on And to we and clinical again, that actually would very which is what do carefully hit have of to yet haven’t for what are we the have ceased decided for once those and with development, in as

lot lead giant hit that to And programs axonal which be to those that that’s still in from forward CLNX really pathway on And just perspective. continue a expensive, will the way CMC continuing to our but at away at from all-in going development still that fastest doing really to more a begin our with this. move the we of looking an syndrome our Rett that’s with approval. neuropathy from to that platform validate proof-of-concept continue totality be long-term of a so – program, perspective, registration milestone, the our are to two registration from and kind pathway, and and until a focus preserve runway SLCXXAX, going and then particularly we perspective eye looking we We to cash with portfolio, on, looking a with focus on and a which clinical is speed

have hold on get and pipeline earlier essentially programs, augment we an that. out move cash clinical them the forward next effort the over done mentioned to in of capital capital the in say will and ATM, capital term certain turnaround allows where a I have the earnings and would continue a preclinical able tap lot half start I that is this kind to strategic XXXX if preserve way Since to Obviously, be currently in of again we our loan able at of into constant we pause, raise will done. see into start QX cash as done. for of which and I strategic have we to extended cash runway our number to to should of we which reconstituted essentially our work ATM starting to some on ability our prioritization that think, down been year-end there draw our we I to a ATM, think that expense, current look second executed already we through go call, to which then ways come and available year, non-dilutive be to begin again, programs R&D and year, quarters as you already the the runway our to from that. get down able able a to But way that we facility, we which be successfully forms we probably capital, is to have earlier, will continuing a obviously, run be few to play us markets,

ways just I comment on the breakdown we Maybe, being between think R&D to and all things of would Kamran, extend those that are would to runway. you consistent look want G&A.

Kamran Alam

the Yes. Sure. Happy RA thanks for question, Thanks to. Laura. and

pausing last as activities because burn expense our So, expenses over we able and RA our that the on XX% pipeline significantly And quarters as research of strategic development result R&D prioritization to next will efforts, R&D preclinical of are reminder, related. reduce this their be in a coming mentioned, of GMP a some over operating of significant are product as this conducted reduction on programs, we only GAN QX manufacturing Ultimately, which completed year program year. terms year, of release. as expenses. of And well GMP CMC our our in were as batches doing we in six

expect quarters and can significant in a result. you year-over-year reduction a in subsequent So, CMC expense our as

of really work on GAN. lot nice to our a resources a lot of course, and primarily again, of our efforts So, focus

RA Session II

Kamran. Thanks

Operator

Thank you.

line question with Please of next your Fargo. proceed Our from Wells with question. Yanan comes Zhu

Yanan Zhu

of And taking Thanks manifestations Thank would observation, syndrome waiting my on duplication a period syndrome? ascertain with MECPX the Rett could once is dosed? for associated patient Hi. you. is given clinical trial, the syndrome overexpression share lot toxicity for is safety there questions. Rett you how same the the what – couple A one whether how that MECPX

RA Session II

some Suyash morning. Good that provide turn to Yanan. to question will over I context. Hi Suyash?

Suyash Prasad

Sure.

specifically So, eight the staggering weeks. is

between after then a show progresses, ahead the the will So, time is as with the will trial with any point, next patient dosed one safety And changes the discussions patient. and staggering of time patients clinical staggering do go and the study the we that’s two for less DMC dose But review of in issues, or we as progresses. absence course. then eight week

that patients, And the in to the of study, signs will and bottom have all opinion in the Rett they good of toxicity, Specifically, unlike is the pediatric syndrome, with patients with regard line one stable question. study over-expression it’s are Phase adult and certainly in with a fact, declining. in i.e., X at And it’s discussed be most length, not key we regulators both leaders.

clinical You may patients recollect that there is phases to Rett. of progress four the with

when a the level of into they for can phase. stable. are X phase Phase they is and very a by in the when all To then The first in diagnosis. there end in sometimes years, then complex rapid is is where functioning. there a they up patients with decline is The get be stable Phase decades, a deterioration functionality. Phase a X phase i.e., have are struggled X, And they who a state

poor type other kind syndrome be due behaviors we for syndrome. clinically it’s been a while. stable over-expression to any deterioration Rett are a state, in features, Rett likely have of they progression looking deterioration, of such is is or terms what If there for is of to neurological because So, a there any okay, seen than

of patients. of miss the system, can’t so be that’s the for special several and we drug be a even we with the dosing and over unlikely especially we tox data that trial doses that, because highly likely central specifically tissue, nervous will given tox after of very MECPX in we progression high is rat bite to clinical toxicity Now, MECPX. nervous Rett the status, in in that’s to of status, wouldn’t, gene dose, change anticipate fourfold toxicity where system, a observing a definite, way and we our due and no neurological we are toxicity gave of course, brain present to due high level. to unlikely no, is and of the mental by of at behavioral status the NHP change the looking therapy, Having a over your peripheral clinical data deterioration because But

RA Session II

Thanks Suyash.

Operator

any gentlemen, for floor questions. to the our and Mr. to Ladies for you. comments. Session of Thank time turn end will final we have come the back allowed I

RA Session II

Thank joining our for again, call today. you, QX operator. thank And everybody for you

nicely coming out As we from that year. and quite pipeline studies. of a I prioritization, morphing you of With continue prouder iterated move company a guys the wish program, be then program, prouder more registration us team wonderful to earlier, our later Rett forward QX this progress also strategic to company you to of of be will refocus be many couldn’t speed of the focus I to team. week, execution couldn’t make the continues the strides some guys invite aspect, our and the has the proof-of-concept at follow our the our on we and earlier ASGCT. that, of programs of performed continuing to clinical I GAN to

you And seeing call. Thank today’s will ask and say to by that, With I you many you. hi. would of end I look we forward So, guys to come there.

Operator

This you. concludes Thank conference. today’s

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

Taysha Gene Therapies (TSHA) 16 May 222022 Q1 Earnings call transcript